DDrare: Database of Drug Development for Rare Diseases

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 1- Deoxygalactonojirimycin; 1-Deoxygalactonojirimycin; 1-deoxygalactonojirimycin hydrochloride; 12S-IT; 15 O Water; 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID; 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN; 2-Hydroxypropyl-Beta-Cyclodextrin; 2-aminoethanethiol; 4D-310; 500 0500 0; 89 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled); 89Zr-SBC-103; A (migalastat); A16AB04; A16AX06; AAV2/8.TBG.hARSB; AAV2CUhCLN2 (3x10^12 particle units); AAV9-CAG-coh-SGSH; AAV9-GLB1; AAVrh.10-hMPSIIIA; AAVrh.10CUCLN2; AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies; AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies; AAVrh.10cuARSA; ABO-101; ABO-102; ACT-434964; ADENO-ASSOCIATED VIRAL (AAV) SEROTYPE 8 (AAV2/8) VECTOR WITH LIVER-SPECIFIC THYROXINE-BINDING GLOBULIN (TBG) PROMOTER, DRIVING THE EXPRESSION OF THE HUMAN ARSB GENE; ADMINISTRATION; AGALSIDASE ALFA; AGALSIDASE BETA; AGALSIDASI BETA; AGT-181; AGT-181 (HIRMAb-IDUA); AGT-182; ALDURAZYME*INFUS 1FL 5ML 500U; ARIMOCLOMOL; ARIMOCLOMOL CITRATE; AT1001; AT1001 150 mg; AT2101; ATALUREN; AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING THE HUMAN SGSH GENE; AVR-RD-01; AVR-RD-02; AX; AX 250; Abatacept; Accupro; Accuretic; Acetylcysteine; Adalimumab; Adalimumab Injection [Humira]; Adeno-associated viral vector serotype 9 containing the human N-acetyl-alpha-glucosaminidase gene; Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector; Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector; Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector; Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA; Adeno-associated virus serotype 2/6 encoding human iduronidase (hIDUA) gene donor vector, SB-A6P-HRL Donor Vector; Adrabetadex; Afegostat; Afegostat tartrate; Agalsidase; Agalsidase Alfa; Agalsidase Alpha; Agalsidase Beta; Agalsidase alfa; Agalsidase alpha; Agalsidase beta; Agalsidase beta (GZ419828); Agalsidase beta (recombinant form); Aldurazyme; Aldurazyme (Recombinant Human Alpha-L-Iduronidase); Aldurazyme (laronidase); Alemtuzumab; Alendronate; Alendronate sodium; Alfa-manosidadas humana recombinante; Alpha-galactosidase A; Ambroxol; Anakinra; Angiotensin; Arimoclomol; Arimoclomol (HPMC capsule); Arimoclomol (hard gelatine capsule); Arimoclomol citrate; Arylsulfatase A; Ataluren; Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene; Autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA; Autologous Plasmablasts; Azathioprine; Azathioprine (Aza); B (migalastat); BHP001; BM110; BMN 110; BMN 110 - Every Other Week; BMN 110 - Weekly; BMN 110 Every Other Week; BMN 110 Weekly; BMN 190; BMN 250; BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1); BUSILVEX; BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI; BUSULFANO; Barium; Behavioral: Neurocognitive testing; Behavioral: PICC line placement; Beta-Mercaptoethylamine; Beta-cyclodextrin; Beta-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride; Biological assays; Biopsy; Blood sampling; Brineura; Busilvex; Busulfan; Busulfan, Cyclophosphamide, ATG; Busulfan, Cyclophosphamide, Antithymocyte Globulin; Busulfano; C (migalastat); CALD HR-D (High-Risk, Regimen C); CALD HR-D (High-Risk, Regimen D); CALD SR-A (Standard-Risk, Regimen A); CALD SR-B (Standard-Risk, Regimen B); CEREDASE™; CEREZYME; CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE; CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HUMAN SGSH GENE; CYSTADROPS; CYSTADROPS 0.55% eye drops, solution; CYSTAGON; CYSTEAMINE; CYSTEAMINE HYDROCHLORIDE; Calcium; Calcium carbonate; Campath; Campath-1H; Capsaicin; Carbonate; Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi; Cerdelga; Ceredase; Cerezyme; Cerezyme (imiglucerase for injection); Cerezyme®; Cerezyme® / Imiglucerase; Cerliponase Alfa; Cerliponase alfa; Chemically modified recombinant human sulfamidase; Cholecalciferol; Cholesterol; Clofarabine; Coversyl; Coversyl PLUS; CsA; Cyclophosphamide; Cyclosporine; Cyclosporine A; Cyclosporine A (CsA); Cystadane anhydrous; Cystadrops; Cystagon; Cystagon 150mg; Cystagon 50mg; Cystagon®; Cystagon® (Cysteamine Bitartrate); Cysteamine; Cysteamine (mercaptamine) viscous solution; Cysteamine Bitartrate; Cysteamine Bitartrate Delayed-release Capsules; Cysteamine Bitartrate Delayed-release Capsules (RP103); Cysteamine bitartrate; Cysteamine bitartrate (INN: mercaptamine bitartrate; Cysteamine bitartrate (INN: mercaptamine bitartrate ); Cysteamine bitartrate (INN: mercaptamine bitartrate); Cysteamine hydrochloride; Cysteine; D (migalastat); DNL310; DNL310 Drug Substance; DRX005B; DUOC-01; Device: Administration Kit; Device: Cyclosporine A; Device: Intraventricular Access Device; Device: Intraventricular access device; Device: MRI with contrast agent injection; Device: MRI without contrast agent injection; Device: Mechanical stimulation with Von Frey filaments; Device: OSOME; Device: Temperature sensitivity measurement with Advanced Thermal Stimulation; Diagnostic Test: Audiology assessmentwith ABR; Diagnostic Test: Bone density scan (DEXA; Diagnostic Test: Echocardiogram; Diagnostic Test: Echocardiography at M24; Diagnostic Test: Echocardiography at T0; Diagnostic Test: Electrocardiogram (EKG); Diagnostic Test: Exercise test; Diagnostic Test: Laboratory tests; Dried blood spot (DBS) sampling; E (migalastat); ELIGLUSTAT TARTRATE; ELX-02; ERT; Elaprase; Elaprase 2mg/ml; Elaprase for intravenous (IV) infusion; Elaprase_0.5mg/kg; Elelyso; Elelyso 60 units/kg; Eliglustat; Eliglustat GZ385660; Eliglustat tartrate; Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]; Elosulfase alfa; Enalapril; Enalapril and other angiotensin converting enzyme inhibitors; Enriched Hematopoetic Stem Cell Infusion; Enzyme replacement; Enzyme replacement therapy; Enzyme replacement therapy (Agalsidase alpha (Replagal®)); Enzyme replacement therapy (ERT); FABRAZYME; FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO; FLT190; FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML; FLUDARABINA FOSFATO; Fabrazyme; Fabrazyme (agalsidase beta); Fabrazyme 35 mg; Fabrazyme 5 mg; Fludarabina; Fluorescein; GA-GCB; GA-GCB (velaglucerase alfa); GALSULFASE; GC1111_0.5mg/kg; GC1111_1.0mg/kg; GC1119; GENISTEIN; GENZ-682452; GLA; GLUCOCEREBROSIDASE; GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO; GNR-055; GR181413; GR181413A; GR181413A/AT1001 capsule; GR181413A/AT1001 solution; GSK2788723; GZ/SAR402671; GZ385660; GZ402665; GZ402671; GZ402671 / SAR402671; Gabapentin; Galafold; Galsulfase; Gelatine; Gene Activated Human Glucocerebrosidase; Gene Activated Human glucocerebrosidase; Gene activated human glucocerebrosidase; Gene activated human glucocerebrosidase, velaglucerase alfa; Gene-Activated Human Glucocerebrosidase; Gene-Activated Human Glucocerebrosidase 200U/vial; Gene-Activated Human Glucocerebrosidase 400U/vial; Genetic: GLA gene; Genetic: LYS-GM101; Genistein; Genistein aglycone; Genz-112638; Genz-682452-AA; Genz-682452-AU; Glucocerebrosidase; Growth Hormone; Growth hormone; HGT-1110; HGT-1111; HGT-1410; HGT-2310; HIDS DONOR; Hematopoetic Stem Cell Transplantation; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cell infusion; Hematopoietic stem cell transplantation; Heparán N sulfatasa humana recombinante; Heparán N sulfatasa humana recombinante (rhHNS); HuCNS-SC; Human Glucocerebrosidase (prGCD); Human Placental Derived Stem Cell; Hunterase; Hydroxypropyl-beta-cyclodextrin; Hydroxypropyl-ß-cyclodextrin; Hydroxyurea; I2S; I2S-IT; IB1001; IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR; IDURSULFASA; IDURSULFASE; IGF2; IMD; IMD Preparative Regimen; IMIGLUCERASA; IMIGLUCERASE; INN Not available; INNOZIDE; IRT Laronidase; ISU302; IV migalastat HCl; IVA 336; Iduronate 2-sulfatase; Iduronate-2-sulfatase enzyme replacement therapy; Idursulfasa; Idursulfase; Idursulfase (I2S); Idursulfase (I2S)-IT; Idursulfase IT (1 mg); Idursulfase IT (10 mg); Idursulfase IT (30 mg); Idursulfase beta; Idursulfase(12S)-IT; Idursulfase(I2S)-IT; Idursulfase-IT; Imiglucerasa; Imiglucerase; Imiglucerase GZ437843; Imiglucerasi; Imiglucérase (drug) pharmacokinetics; Inactive Reminder Capsules; Inactive Reminder Product; Innozide; Intrathecal recombinant human alpha iduronidase; Iohexol; Isofagamine tartrate; JR-141; JR-141 or Idursulfase; JR-171; KANUMA; Kanuma; LAMAZYM; LARONIDASE; LENOGRASTIM; LYS; LYS-SAF302; Lamazym; Laronidase; Laronidase ERT; Ledipasvir; Ledipasvir/Sofosbuvir; Lenograstim; Lentivirus Alpha-gal A transduced stem cells; Lentivirus-mediated delivery of ARSA to the CNS.; Leucine; Leucovorin; Lipasa ácida lisosómica, esterasa, colesterol (gen humano LIPA), lipasa ácida lisosómica (gen humano LIPA); Lisinopril; Lithium Carbonate; Lithium carbonate; Lopace; Losartan; Losartan and other angiotensin receptor blockers; Lucerastat; Lysodase; Lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); Lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); MABTHERA - 2 FIALE 100 MG 10 ML; MERCAPTAMINE BITARTRATE; MERCAPTAMINE HYDROCHLORIDE; MERCAPTAMINE bitartrate; MIGALASTAT; MIGALASTAT HYDROCHLORIDE; MIGLUSTAT; MIGLUSTAT (ZAVESCA); MIglustat; MONITOR; MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F; MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML; MabThera; Medication to suppress the immune system; Melphalan; Mepsevii; Mercaptamina; Mercaptamine; Mercaptamine bitartrate; Mercaptamine hydrochloride; Mercaptamine, mercamine, 2-aminoethanethiol; Metazym; Methotrexate; Methylprednisolone; Migalastat; Migalastat HCl; Migalastat HCl 150 mg; Migalastat Hydrochloride; Migalastat hydrochloride; Migalastat hydrocloride; Miglustat; Moss-aGal (recombinant human alpha-galactosidase A produced in moss); Mozobil; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Mycophenylate mofetil; N Acetyl L Leucine; N Acetyl L leucine; N acetyl cysteine; N-Acetyl Cysteine; N-Acetyl-L-Leucine; N-Acetyl-L-leucine; N-acetylcysteine; N-acetylgalactosamine 4-sulfatase; N-acetylgalactosamine-6-sulfatase; Naglazyme; Naglazyme (galsulfase); Naglazyme®; Non applicable; OGT 918; OGT918; OGT923; OLENASUFLIGENE RELDUPARVOVEC; OMNIPAQUE; OTL-200 Dispersion for Infusion; Odiparcil; Olenasufligene relduparvovec; Olipudase alfa; Olipudase alfa (rhASM); Omnipaque; Oral migalastat HCl; Osteopetrosis Haploidentical Only Preparative Regimen; Osteopetrosis Only Preparative Regimen; Other: Brief Pain Inventory questionnaire; Other: Challenge agent: capsaicin; Other: Control; Other: Cysteamine bitartrate; Other: Electroencephalogram (EEG) awake andextended overnight; Other: Fluorescein angiography; Other: General and Neurological examination; Other: Ketogenic Diet; Other: Neurology exam; Other: No IT treatment; Other: Palliative Care; Other: Potable water; Other: Quality of Life questionnaire; Other: Sham; Other: Vital signs; Oxygen; PEGUNIGALSIDASE ALFA; PENTOSAN POLYSULFATE SODIUM; PLX-200; PR001; PROCYSBI; PRX-102; PRX-102 (pegunigalsidase alfa); PRX-112; PRX102; PTC124; Paricalcitol; Pegunigalsidase Alfa; Pegunigalsidase alfa; Pegunigalsidase alpha; Pending; Pentosan Polysulfat SP 54® injection solution; Pentosan Polysulphate SP54; Pentosan Polysulphate SP54®; Pentosan polysulfate; Perdix; Plant Cell Recombinant Glucocerebrosidase; Plant cell expressed recombinant glucocerebrosidase (prGCD); PrGCD; Prednisone; Prevenar13; Procedure: 12 lead electrocardiogram; Procedure: 2-hour Holter Monitor; Procedure: Allogeneic stem cell transplantation; Procedure: Blood draw; Procedure: Brain MRI/MRS/fMRI; Procedure: Contrast sensitivity measurement; Procedure: DEXA scan; Procedure: Echocardiogram; Procedure: Lumbar puncture; Procedure: Magnetic Resonance Imaging; Procedure: Ophthalmology exam; Procedure: Oxygen flow at the optic nerve head measurement; Procedure: Skeletal survey; Procedure: Skin biopsy; Procedure: Slit Lamp assessment and intra-ocular pressure measurement; Procedure: Speech and modified barium swallow study; Procedure: Stem Cell Transplant; Procedure: Stem Cell Transplantation; Procedure: Surgery to implant human CNS stem cells (HuCNS-SC); Procedure: Total body Irradiation; Procedure: Urine collection; Procedure: Visual field testing; Procedure: Whole body Protein turnover; Procedure: quantitative chemical shift imaging (QCSI); Procysbi; Pyrimethamine; RAAV 2/6 hIDS DONOR; RAAV2/5-hNAGLU; RAAV2/5-hNaGlu; RAAV2/6 Left ZFN Vector; RAAV2/6 Right ZFN Vector; RAAV9.CMV.hNAGLU; RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM); RECOMBINANT HUMAN ARYLSULFATASE A; RECOMBINANT HUMAN BETA GLUCURONIDASE; RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1); REPLAGAL; RHGUS; RHNAGLU-IGF2; RITUXIMAB; RP 103; RP103; RVX000222; Radiation: Total Body Irradiation with Marrow Boosting; Rebisufligene etisparvovec; Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A; Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU); Recombinant Human Arylsulfatase A (rhASA); Recombinant Human Arylsulfatase A (rhASA,; Recombinant Human Glucocerebrosidase (prGCD); Recombinant alpha-galactosidase A; Recombinant human Arylsulfatase A (rhASA); Recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase; Recombinant human N-acetylgalactosamine 4-sulfatase; Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110; Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 drug substance; Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS); Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110; Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance; Recombinant human Nacetylgalactosamine-; Recombinant human alpha-galactosidase; Recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL; Recombinant human alpha-mannosidase; Recombinant human arylsulfatase A; Recombinant human beta-glucuronidase; Recombinant human beta-glucuronidase (rhGUS); Recombinant human betaglucuronidase; Recombinant human derived macrophage-targeted beta-Glucocerebrosidase; Recombinant human derived macrophage-targeted beta-glucocerebrosidase; Recombinant human derived macrophage-targeted ß-Glucocerebrosidase; Recombinant human derived macrophage-targeted ß-glucocerebrosidase; Recombinant human glucerebrosidase; Recombinant human glucocerebrosidase; Recombinant human glucocerebrosidase (prGCD); Recombinant human heparan N-sulfatase; Recombinant human heparan N-sulfatase (rhHNS); Recombinant human heparan N-sulfatase [rhHNS]; Recombinant human heparan Nsulfatase (rhHNS); Recombinant human lysosomal acid lipase; Recombinant human lysosomal acid lipase (rhLAL); Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA); Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA), lysosomal acid lipase (human gene LIPA); Recombinant human lysosomal alpha-mannosidase; Recombinant human tripeptidyl peptidase-1; Reduced Intensity Conditioning; Reminder Product; Reminder capsules; Replagal; Replagal (Agalsidase Alfa); Replagal (agalsidase alfa); Replagal agalsidase alfa; Rh Growth Hormone; RhARSA; RhASA; RhASM; RhHNS-10 mg; RhHNS-45 mg; RhHNS-90 mg; RhIDU (recombinant human-Alpha-L-Iduronidase); RhLAMAN; RhNAGLU; RhNAGLU radiolabelled; Rhlaman; Rimiducid; Rituximab; Rivogenlecleucel; SAF-301; SAR402671, GZ402671 or GZ/SAR402671; SB-318; SB-47171; SB-47898; SB-913; SB-A6P-HRL Donor Vector; SB-FIX; SB-IDUA; SBC-102; SBC-102, recombinant human lysosomal acid lipase; SBC-103; SHP611; SOBI003; ST-920; SUB195712; Saline Solution for Injection; ScAAV9.U1A.SGSH; ScAAV9.U1A.hSGSH; Sebelipasa Alfa; Sebelipasa alfa; Sebelipase Alfa; Sebelipase alfa; Sebelipase alfa (SBC-102); Sebelipase alfa 0.35 mg/kg; Sebelipase alfa 1 mg/kg; Sebelipase alfa 3 mg/kg; Sirolimus; Sofosbuvir; Somatropin; Somatropin (DNA origin); Ss-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride; Stem Cell Transplantation; TALIGLUCERASE ALFA; TBD; Taliglucerase Alfa; Taliglucerase alfa; Taliglucerase alfa - Recombinant human glucocerebrosidase; Thiotepa; Thiotepa--escalated dose; Thiotepa--single daily dose; Thyroxine; Trappsol Cyclo; Treosulfan; Triapin; Triapin& triapin Mite; Tropicamide; UPLYSO; USAN: sebelipasa alfa; USAN: sebelipase alpha; UX003; Unrelated Umbilical Cord Blood Transplant; Uplyso; VELAGLUCERASE ALFA; VELMANASE ALFA; VPRIV; VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO; VPRIV 400 Units powder for solution of infusion; VPRIV ®,; VPRIV®; VTS-270; Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana; Velaglucerase alfa; Velaglucerase alfa (VPRIV); Velaglucerasi alfa; Velmanase Alfa; Velmanase Alfa (e.g. Lamazym); Velmanase alfa; Venglustat; Venglustat (GZ/SAR402671); Venglustat GZ402671; Vimizim® (elosulfase alfa); Vitamin D; Vorinostat; Vpriv; Warfarin; Water; ZAVESCA; ZAVESCA 84CPS 100MG; ZESTORIC; Zavesca; Zavesca (Miglustat); Zestoretic 10,20; Zestril; [045251016]; [14C] AT1001; [A16AB04]; [AT1001]; [HGT-1410]; [OTL-200]; [SHP611]; [rhLAMAN]

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04143958 (ClinicalTrials.gov)	September 2020	28/10/2019	To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease	A Randomized, Open-label, Active Comparator, 2-arm, Prospective Study to Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease	Fabry's Disease	Drug: agalsidase beta (GZ419828);Drug: agalsidase alfa	Sanofi	NULL	Withdrawn	16 Years	45 Years	Male	0	Phase 4	Czechia
2	EUCTR2019-000064-21-GB (EUCTR)	20/12/2019	11/02/2020	To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfa	A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing on agalsidase alfa (Replagal) in male patients with classic Fabry disease. - Fabry study with Fabrazyme and Replagal	Fabry's disease MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA	Sanofi Aventis Groupe (SAG)	NULL	Not Recruiting			Female: no Male: yes	35	Phase 4	France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Norway;Germany;Italy;United Kingdom
3	EUCTR2017-001528-23-IT (EUCTR)	17/07/2018	10/11/2020	Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)	A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme¿ (agalsidase beta) or Replagal¿ (agalsidase alfa) - Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered	Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa	PROTALIX LTD	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	30	Phase 3	United States;Czechia;Taiwan;Spain;Turkey;Austria;Italy;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway
4	EUCTR2017-001528-23-DK (EUCTR)	04/07/2018	10/04/2018	Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)	A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa	Protalix Ltd.	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 3	United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway
5	EUCTR2017-001528-23-CZ (EUCTR)	07/05/2018	06/03/2018	Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)	A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa	Protalix Ltd.	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 3	United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
6	EUCTR2016-001318-11-NL (EUCTR)	27/03/2018	17/10/2017	Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)	An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha	Protalix Ltd.	NULL	Not Recruiting			Female: yes Male: yes	22	Phase 3	Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Germany;Netherlands;United Kingdom
7	EUCTR2016-001318-11-DE (EUCTR)	15/12/2017	10/01/2017	Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)	An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]		Protalix Ltd.	NULL	Not Recruiting			Female: yes Male: yes	22	Phase 3	Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
8	EUCTR2016-001318-11-SI (EUCTR)	13/11/2017	05/10/2017	Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)	An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha	Protalix Ltd.	NULL	Not Recruiting			Female: yes Male: yes	22	Phase 3	Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
9	EUCTR2017-001528-23-GB (EUCTR)	18/10/2017	28/06/2017	Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)	A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa	Protalix Ltd.	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 3	United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;Germany;United Kingdom
10	NCT03180840 (ClinicalTrials.gov)	September 27, 2017	29/5/2017	Study of the Safety, Efficacy, & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients	Phase 3, Open-Label, Switch Over Study to Assess Safety, Efficacy & PK of Pegunigalsidase Alfa 2 mg/kg Administered Every 4 Weeks for 52 Weeks in Fabry Disease Patients Currently Treated With Enzyme Replacement Therapy: Fabrazyme® (Agalsidase Beta) or Replagal™ (Agalsidase Alfa)	Fabry Disease	Biological: Pegunigalsidase alfa	Protalix	NULL	Active, not recruiting	18 Years	60 Years	All	30	Phase 3	United States;Belgium;Canada;Czechia;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
11	EUCTR2017-001528-23-BE (EUCTR)	21/08/2017	28/07/2017	Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)	A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa	Protalix Ltd.	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 3	United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;Germany;United Kingdom
12	NCT02956954 (ClinicalTrials.gov)	March 25, 2017	3/11/2016	Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease	Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®)	Anderson-Fabry Disease	Drug: Enzyme replacement therapy (Agalsidase alpha (Replagal®));Procedure: Magnetic Resonance Imaging	University Hospital, Rouen	NULL	Unknown status	18 Years	N/A	All	25	N/A	France
13	EUCTR2016-001318-11-GB (EUCTR)	24/03/2017	04/10/2016	Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)	An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha	Protalix Ltd.	NULL	Not Recruiting			Female: yes Male: yes	22	Phase 3	Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
14	NCT03018730 (ClinicalTrials.gov)	February 23, 2017	9/1/2017	Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)	An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)	Fabry Disease	Biological: PRX-102 (pegunigalsidase alfa)	Protalix	NULL	Active, not recruiting	18 Years	60 Years	All	22	Phase 3	Australia;Canada;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;Czech Republic
15	EUCTR2016-001318-11-CZ (EUCTR)	19/01/2017	12/12/2016	Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)	An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha	Protalix Ltd.	NULL	Not Recruiting			Female: yes Male: yes	22	Phase 3	Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
16	EUCTR2016-001318-11-ES (EUCTR)	11/01/2017	24/10/2016	Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)	An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)	Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha	Protalix Ltd.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	22	Phase 3	Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom
17	EUCTR2010-022709-16-GB (EUCTR)	23/08/2012	21/05/2012	A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT).	AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE	Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	18	Phase 2A	Belgium;United Kingdom
18	EUCTR2010-022636-37-DE (EUCTR)	09/05/2012	10/01/2012	The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease	A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT	Fabry disease MedDRA version: 15.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	Hungary;Germany;Turkey;Switzerland;France;Italy;Austria;Australia;Brazil;Israel;United Kingdom;Slovakia;Russian Federation;Taiwan;Mexico;Argentina;Belgium;Denmark;Japan;United States;Greece;Poland
19	EUCTR2009-015985-75-SI (EUCTR)	22/03/2012	05/03/2012	Follow on study in adult Fabry Disease patients	An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease	Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA	Shire Human Genetic Therapies (HGT), Inc.	NULL	Not Recruiting			Female: yes Male: yes	43	Phase 3;Phase 4	United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
20	EUCTR2010-022636-37-IT (EUCTR)	16/12/2011	13/03/2012	A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT - AT1001-012	A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT - AT1001-012	Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: migalastat hydrocloride Product Code: AT1001 INN or Proposed INN: migalastat hydrocloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Trade Name: REPLAGAL INN or Proposed INN: Agalsidase alfa Other descriptive name: NA Trade Name: FABRAZYME INN or Proposed INN: Agalsidase beta Other descriptive name: NA	AMICUS THERAPEUTICS, INC	NULL	Not Recruiting			Female: yes Male: yes	50		United States;Taiwan;Greece;Turkey;Austria;Russian Federation;Italy;Switzerland;United Kingdom;Mexico;Argentina;Brazil;Belgium;Denmark;Australia;Germany;Japan
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
21	EUCTR2009-015985-75-FI (EUCTR)	15/11/2011	26/10/2011	Follow on study in adault Fabry Disease patients	An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease	Fabry disease MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA	Shire Human Genetic Therapies (HGT), Inc.	NULL	Not Recruiting			Female: yes Male: yes	43		United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
22	EUCTR2010-022636-37-GR (EUCTR)	02/11/2011	23/09/2011	The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease	A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT	Fabry disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	United States;Taiwan;Slovakia;Greece;Turkey;Austria;Israel;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Argentina;Brazil;Poland;Belgium;Denmark;Australia;Germany;Japan
23	EUCTR2009-015985-75-GB (EUCTR)	18/10/2011	19/05/2011	Follow on study in adault Fabry Disease patients	An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease	Fabry disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA	Shire Human Genetic Therapies (HGT), Inc.	NULL	Not Recruiting			Female: yes Male: yes	43	Phase 3;Phase 4	United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
24	EUCTR2010-022709-16-BE (EUCTR)	12/09/2011	10/08/2011	A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT).	AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE	Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	24	Phase 2	France;Canada;Belgium;Australia;Netherlands;United Kingdom;Switzerland
25	NCT01298141 (ClinicalTrials.gov)	August 10, 2011	15/2/2011	A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease	A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal® (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease	Fabry Disease	Biological: agalsidase alfa	Shire	NULL	Completed	N/A	N/A	All	171	Phase 3	Canada
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
26	EUCTR2010-022636-37-AT (EUCTR)	03/08/2011	27/07/2011	The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease	A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT	Fabry disease MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	68	Phase 3	France;United States;Greece;Brazil;Belgium;Denmark;Australia;Austria;Germany;Japan;Italy;United Kingdom
27	EUCTR2010-022636-37-DK (EUCTR)	03/05/2011	06/04/2011	The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease	A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT	Fabry disease MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	68	Phase 3	United States;France;Greece;Belgium;Brazil;Austria;Australia;Denmark;Germany;United Kingdom;Japan;Italy
28	NCT01363492 (ClinicalTrials.gov)	May 2011	31/3/2011	Safety Study of Replagal® Therapy in Children With Fabry Disease	An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy	Fabry Disease	Biological: Replagal (agalsidase alfa)	Shire	NULL	Completed	7 Years	17 Years	All	15	Phase 2	United States
29	EUCTR2010-022636-37-GB (EUCTR)	15/04/2011	22/12/2010	The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease	A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT	Fabry disease MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	68	Phase 3	United States;France;Greece;Belgium;Brazil;Denmark;Austria;Australia;Germany;Japan;Italy;United Kingdom
30	EUCTR2007-005543-22-SI (EUCTR)	18/03/2011	24/02/2011	An investigation of 3 dosages of Replagal in adult patients with Fabry disease	A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in Adult Patients with Fabry Disease	Fabry Disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Trade Name: Replagal Product Name: Replagal INN or Proposed INN: agalsidase alpha Other descriptive name: Replagal	Shire Human Genetic Therapies, Inc. (Shire HGT)	NULL	Not Recruiting			Female: yes Male: yes	43		United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
31	NCT01304277 (ClinicalTrials.gov)	March 2011	15/2/2011	This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.	A Phase II Comparability Study Between Replagal® Produced From Agalsidase Alfa Manufactured by 2 Different Processes in Adult Male Patients With Fabry Disease	Fabry Disease	Biological: agalsidase alfa	Shire	NULL	Completed	18 Years	65 Years	Male	17	Phase 2	Canada
32	EUCTR2010-022636-37-BE (EUCTR)	18/01/2011	16/12/2010	The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease	A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT	Fabry disease MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA	Amicus Therapeutics, Inc.	NULL	Not Recruiting			Female: yes Male: yes	68	Phase 3	United States;France;Greece;Brazil;Belgium;Denmark;Austria;Australia;Germany;United Kingdom;Japan;Italy
33	EUCTR2007-005543-22-FI (EUCTR)	18/05/2010	23/03/2010	An investigation of 3 dosages of Replagal in adult patients with Fabry disease	A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in Adult Patients with Fabry Disease	Fabry Disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Trade Name: Replagal Product Name: Replagal INN or Proposed INN: agalsidase alpha Other descriptive name: Replagal	Shire Human Genetic Therapies, Inc. (Shire HGT)	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	43		Finland;United Kingdom;Czech Republic;Paraguay;Slovenia;United States;Australia
34	EUCTR2007-005543-22-GB (EUCTR)	06/05/2010	17/08/2009	An investigation of 3 dosages of Replagal in adult patients with Fabry disease	A Multi-center, Open-Label, Randomized Study Evaluating the Safety and Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement Therapy in Adult Patients with Fabry Disease	Fabry Disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]		Shire Human Genetic Therapies, Inc. (Shire HGT)	NULL	Not Recruiting			Female: yes Male: yes	43	Phase 3;Phase 4	United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom
35	NCT01124643 (ClinicalTrials.gov)	April 13, 2010	23/4/2010	Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease	An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease	Fabry Disease	Biological: Replagal	Shire	NULL	Completed	18 Years	N/A	All	35	Phase 3	United States;Australia;Czechia;Finland;Poland;Slovenia;United Kingdom;Czech Republic;Paraguay
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
36	EUCTR2009-015985-75-CZ (EUCTR)	08/03/2010	24/02/2010	Follow on study in adult Fabry Disease patients	An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease	Fabry disease MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA	Shire Human Genetic Therapies (HGT), Inc.	NULL	Not Recruiting			Female: yes Male: yes	43		United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
37	EUCTR2009-015985-75-PL (EUCTR)	03/02/2010	30/11/2009	Follow on study in adault Fabry Disease patients	An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease	Fabry disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA	Shire Human Genetic Therapies (HGT), Inc.	NULL	Not Recruiting			Female: yes Male: yes	43		United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom
38	NCT01031173 (ClinicalTrials.gov)	September 2009	8/12/2009	Treatment Protocol of Replagal for Patients With Fabry Disease	An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.	Fabry Disease	Biological: agalsidase alfa	Shire	NULL	No longer available	N/A	N/A	Both		N/A	United States
39	NCT00864851 (ClinicalTrials.gov)	December 29, 2008	18/3/2009	Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease	A Multi-Center, Open-Label, Randomized Study Evaluating the Safety and Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement Therapy in Adult Patients With Fabry Disease	Fabry Disease	Biological: Replagal	Shire	NULL	Completed	18 Years	N/A	All	44	Phase 3	United States;Australia;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;Czech Republic
40	EUCTR2007-005543-22-CZ (EUCTR)	19/06/2008	02/01/2008	An investigation of 3 dosages of Replagal in adult patients with Fabry disease	A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in Adult Patients with Fabry Disease	Fabry Disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Trade Name: Replagal Product Name: Replagal INN or Proposed INN: agalsidase alpha Other descriptive name: Replagal	Shire Human Genetic Therapies, Inc. (Shire HGT)	NULL	Not Recruiting			Female: yes Male: yes	43		United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
41	NCT00097890 (ClinicalTrials.gov)	November 2004	30/11/2004	Replagal Enzyme Replacement Therapy for Adults With Fabry Disease	An Open Label Six-Month Maintenance Clinical Trial of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease Who Have Completed TKT027	Fabry Disease	Drug: Replagal (Agalsidase Alfa);Drug: Replagal	National Institute of Neurological Disorders and Stroke (NINDS)	NULL	Completed	N/A	N/A	Male	25	Phase 4	United States
42	EUCTR2004-000772-14-CZ (EUCTR)	23/07/2004	28/06/2004	A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027) - TKT027	A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027) - TKT027	Fabry Disease MedDRA version: 6.1;Level: PT;Classification code 10016016	Trade Name: Replagal Product Name: Replagal INN or Proposed INN: agalsidase alfa	TKT Inc	NULL	Not Recruiting			Female: no Male: yes	20	Phase 1;Phase 2	Czech Republic
43	NCT00084084 (ClinicalTrials.gov)	June 2004	5/6/2004	Replagal Enzyme Replacement Therapy for Children With Fabry Disease	An Open Label Clinical Trial of Replagal Enzyme Replacement Therapy In Children With Fabry Disease Who Have Completed Study TKT023 or Who Are Naive to Enzyme Replacement Therapy	Fabry Disease	Drug: Agalsidase alfa	Shire	NULL	Completed	7 Years	17 Years	All	17	Phase 2	United States;Canada
44	NCT00075244 (ClinicalTrials.gov)	January 2004	6/1/2004	Alternative Dosing and Regimen of Replagal to Treat Fabry Disease	A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients With Fabry Disease	Fabry Disease	Drug: Replagal	National Institute of Neurological Disorders and Stroke (NINDS)	NULL	Completed	N/A	N/A	Male	25	Phase 2	United States
45	NCT00357786 (ClinicalTrials.gov)	October 2003	26/7/2006	An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease	An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal® (Registered Trademark) in Patients With Fabry Disease	Fabry Disease	Drug: Replagal agalsidase alfa;Drug: Replagal	National Institute of Neurological Disorders and Stroke (NINDS)	NULL	Completed	39 Years	45 Years	Male	3	Phase 1	United States
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
46	NCT00071877 (ClinicalTrials.gov)	October 2003	3/11/2003	An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease	A Clinical Trial of Replagal Enzyme Replacement Therapy in Children Ages 7 - 17 Years With Fabry Disease	Fabry Disease	Drug: Replagal	National Institute of Neurological Disorders and Stroke (NINDS)	NULL	Completed	N/A	N/A	Both	25	Phase 2	United States
47	NCT00068107 (ClinicalTrials.gov)	September 2003	6/9/2003	Dosing Study of Replagal in Patients With Fabry Disease	Study of Weekly Dosing Regimens of Replagal in Patients With Fabry Disease With Incomplete Clinical Response to Long-Term Therapy	Fabry Disease	Drug: Replagal	Baylor Research Institute	National Institute of Neurological Disorders and Stroke (NINDS)	Completed	N/A	N/A	Male	13	Phase 2	United States
48	NCT00048906 (ClinicalTrials.gov)	November 2002	8/11/2002	Alpha-Galactosidase A Replacement Therapy for Fabry Disease	A Safety and Pharmacokinetic Study of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease	Fabry Disease	Drug: DRX005B	National Institute of Neurological Disorders and Stroke (NINDS)	NULL	Completed	N/A	N/A	Both	3	Phase 2	United States
49	EUCTR2019-000064-21-CZ (EUCTR)		29/08/2019	To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfa	A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme) versus continuing on agalsidase alfa (Replagal) in male patients with classic Fabry disease	Fabry's disease MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA	Sanofi Aventis Groupe (SAG)	NULL	Not Recruiting			Female: no Male: yes	35	Phase 4	France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Norway;Germany;Italy;United Kingdom
50	EUCTR2019-000064-21-NO (EUCTR)		15/08/2019	To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfa	A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical benefits of agalsidase beta (Fabrazyme®) in male patients with classic Fabry disease switching from agalsidase alfa (Replagal®)	Fabry's disease MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA	Sanofi Aventis Groupe (SAG)	NULL	Not Recruiting			Female: no Male: yes	35	Phase 4	France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Germany;Norway;Italy;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT04143958
(ClinicalTrials.gov)

September 2020

28/10/2019

To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease

A Randomized, Open-label, Active Comparator, 2-arm, Prospective Study to Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease

Fabry's Disease

Drug: agalsidase beta (GZ419828);Drug: agalsidase alfa

Sanofi

NULL

Withdrawn

16 Years

45 Years

Male

Phase 4

Czechia

EUCTR2019-000064-21-GB
(EUCTR)

20/12/2019

11/02/2020

To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfa

A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing on agalsidase alfa (Replagal) in male patients with classic Fabry disease. - Fabry study with Fabrazyme and Replagal

Fabry's disease
MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trade Name: Fabrazyme
Product Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA

Sanofi Aventis Groupe (SAG)

NULL

Not Recruiting

Female: no
Male: yes

Phase 4

France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Norway;Germany;Italy;United Kingdom

EUCTR2017-001528-23-IT
(EUCTR)

17/07/2018

10/11/2020

Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)

A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme¿ (agalsidase beta) or Replagal¿ (agalsidase alfa) - Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered

Fabry disease (a-galactosidase A deficiency)
MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alfa

PROTALIX LTD

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;Czechia;Taiwan;Spain;Turkey;Austria;Italy;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway

EUCTR2017-001528-23-DK
(EUCTR)

04/07/2018

10/04/2018

Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)

A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa)

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alfa

Protalix Ltd.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway

EUCTR2017-001528-23-CZ
(EUCTR)

07/05/2018

06/03/2018

Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)

A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa)

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alfa

Protalix Ltd.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2016-001318-11-NL
(EUCTR)

27/03/2018

17/10/2017

Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)

An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alpha

Protalix Ltd.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Germany;Netherlands;United Kingdom

EUCTR2016-001318-11-DE
(EUCTR)

15/12/2017

10/01/2017

Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)

An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)

Fabry disease (a-galactosidase A deficiency)
MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Protalix Ltd.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom

EUCTR2016-001318-11-SI
(EUCTR)

13/11/2017

05/10/2017

Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)

An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alpha

Protalix Ltd.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom

EUCTR2017-001528-23-GB
(EUCTR)

18/10/2017

28/06/2017

Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)

A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa)

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alfa

Protalix Ltd.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;Germany;United Kingdom

NCT03180840
(ClinicalTrials.gov)

September 27, 2017

29/5/2017

Study of the Safety, Efficacy, & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients

Phase 3, Open-Label, Switch Over Study to Assess Safety, Efficacy & PK of Pegunigalsidase Alfa 2 mg/kg Administered Every 4 Weeks for 52 Weeks in Fabry Disease Patients Currently Treated With Enzyme Replacement Therapy: Fabrazyme® (Agalsidase Beta) or Replagal™ (Agalsidase Alfa)

Fabry Disease

Biological: Pegunigalsidase alfa

Protalix

NULL

Active, not recruiting

18 Years

60 Years

All

Phase 3

United States;Belgium;Canada;Czechia;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2017-001528-23-BE
(EUCTR)

21/08/2017

28/07/2017

Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)

A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa)

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alfa

Protalix Ltd.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;Germany;United Kingdom

NCT02956954
(ClinicalTrials.gov)

March 25, 2017

3/11/2016

Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease

Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®)

Anderson-Fabry Disease

Drug: Enzyme replacement therapy (Agalsidase alpha (Replagal®));Procedure: Magnetic Resonance Imaging

University Hospital, Rouen

NULL

Unknown status

18 Years

N/A

All

N/A

France

EUCTR2016-001318-11-GB
(EUCTR)

24/03/2017

04/10/2016

Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)

An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)

Fabry disease (a-galactosidase A deficiency)
MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alpha

Protalix Ltd.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom

NCT03018730
(ClinicalTrials.gov)

February 23, 2017

9/1/2017

Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)

An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)

Fabry Disease

Biological: PRX-102 (pegunigalsidase alfa)

Protalix

NULL

Active, not recruiting

18 Years

60 Years

All

Phase 3

Australia;Canada;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;Czech Republic

EUCTR2016-001318-11-CZ
(EUCTR)

19/01/2017

12/12/2016

Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)

An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alpha

Protalix Ltd.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2016-001318-11-ES
(EUCTR)

11/01/2017

24/10/2016

Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)

An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
INN or Proposed INN: Pegunigalsidase alpha

Protalix Ltd.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom

EUCTR2010-022709-16-GB
(EUCTR)

23/08/2012

21/05/2012

A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT).

AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE

Fabry disease
MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

Phase 2A

Belgium;United Kingdom

EUCTR2010-022636-37-DE
(EUCTR)

09/05/2012

10/01/2012

The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease

A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT

Fabry disease
MedDRA version: 15.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Hungary;Germany;Turkey;Switzerland;France;Italy;Austria;Australia;Brazil;Israel;United Kingdom;Slovakia;Russian Federation;Taiwan;Mexico;Argentina;Belgium;Denmark;Japan;United States;Greece;Poland

EUCTR2009-015985-75-SI
(EUCTR)

22/03/2012

05/03/2012

Follow on study in adult Fabry Disease patients

An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease

Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA

Shire Human Genetic Therapies (HGT), Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3;Phase 4

United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom

EUCTR2010-022636-37-IT
(EUCTR)

16/12/2011

13/03/2012

A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT - AT1001-012

Product Name: migalastat hydrocloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrocloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Trade Name: REPLAGAL
INN or Proposed INN: Agalsidase alfa
Other descriptive name: NA
Trade Name: FABRAZYME
INN or Proposed INN: Agalsidase beta
Other descriptive name: NA

AMICUS THERAPEUTICS, INC

NULL

Not Recruiting

Female: yes
Male: yes

United States;Taiwan;Greece;Turkey;Austria;Russian Federation;Italy;Switzerland;United Kingdom;Mexico;Argentina;Brazil;Belgium;Denmark;Australia;Germany;Japan

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2009-015985-75-FI
(EUCTR)

15/11/2011

26/10/2011

Follow on study in adault Fabry Disease patients

An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease

Fabry disease
MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA

Shire Human Genetic Therapies (HGT), Inc.

NULL

Not Recruiting

Female: yes
Male: yes

United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom

EUCTR2010-022636-37-GR
(EUCTR)

02/11/2011

23/09/2011

Fabry disease
MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;Taiwan;Slovakia;Greece;Turkey;Austria;Israel;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Argentina;Brazil;Poland;Belgium;Denmark;Australia;Germany;Japan

EUCTR2009-015985-75-GB
(EUCTR)

18/10/2011

19/05/2011

Follow on study in adault Fabry Disease patients

An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease

Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA

Shire Human Genetic Therapies (HGT), Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3;Phase 4

United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom

EUCTR2010-022709-16-BE
(EUCTR)

12/09/2011

10/08/2011

A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT).

AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

Phase 2

France;Canada;Belgium;Australia;Netherlands;United Kingdom;Switzerland

NCT01298141
(ClinicalTrials.gov)

August 10, 2011

15/2/2011

A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease

A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal® (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease

Fabry Disease

Biological: agalsidase alfa

Shire

NULL

Completed

N/A

All

171

Phase 3

Canada

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2010-022636-37-AT
(EUCTR)

03/08/2011

27/07/2011

Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;United States;Greece;Brazil;Belgium;Denmark;Australia;Austria;Germany;Japan;Italy;United Kingdom

EUCTR2010-022636-37-DK
(EUCTR)

03/05/2011

06/04/2011

Fabry disease
MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;France;Greece;Belgium;Brazil;Austria;Australia;Denmark;Germany;United Kingdom;Japan;Italy

NCT01363492
(ClinicalTrials.gov)

May 2011

31/3/2011

Safety Study of Replagal® Therapy in Children With Fabry Disease

An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy

Fabry Disease

Biological: Replagal (agalsidase alfa)

Shire

NULL

Completed

7 Years

17 Years

All

Phase 2

United States

EUCTR2010-022636-37-GB
(EUCTR)

15/04/2011

22/12/2010

Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;France;Greece;Belgium;Brazil;Denmark;Austria;Australia;Germany;Japan;Italy;United Kingdom

EUCTR2007-005543-22-SI
(EUCTR)

18/03/2011

24/02/2011

An investigation of 3 dosages of Replagal in adult patients with Fabry disease

A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in Adult Patients with Fabry Disease

Fabry Disease
MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: agalsidase alpha
Other descriptive name: Replagal

Shire Human Genetic Therapies, Inc. (Shire HGT)

NULL

Not Recruiting

Female: yes
Male: yes

United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT01304277
(ClinicalTrials.gov)

March 2011

15/2/2011

This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.

A Phase II Comparability Study Between Replagal® Produced From Agalsidase Alfa Manufactured by 2 Different Processes in Adult Male Patients With Fabry Disease

Fabry Disease

Biological: agalsidase alfa

Shire

NULL

Completed

18 Years

65 Years

Male

Phase 2

Canada

EUCTR2010-022636-37-BE
(EUCTR)

18/01/2011

16/12/2010

Product Name: migalastat hydrochloride
Product Code: AT1001
INN or Proposed INN: migalastat hydrochloride
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Product Name: Inactive Reminder Capsules
Product Code: Inactive Reminder Capsules
INN or Proposed INN: Not applicable
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA
Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA

Amicus Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;France;Greece;Brazil;Belgium;Denmark;Austria;Australia;Germany;United Kingdom;Japan;Italy

EUCTR2007-005543-22-FI
(EUCTR)

18/05/2010

23/03/2010

An investigation of 3 dosages of Replagal in adult patients with Fabry disease

A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in Adult Patients with Fabry Disease

Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: agalsidase alpha
Other descriptive name: Replagal

Shire Human Genetic Therapies, Inc. (Shire HGT)

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Finland;United Kingdom;Czech Republic;Paraguay;Slovenia;United States;Australia

EUCTR2007-005543-22-GB
(EUCTR)

06/05/2010

17/08/2009

An investigation of 3 dosages of Replagal in adult patients with Fabry disease

A Multi-center, Open-Label, Randomized Study Evaluating the Safety and Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement Therapy in Adult Patients with Fabry Disease

Fabry Disease
MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Shire Human Genetic Therapies, Inc. (Shire HGT)

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3;Phase 4

United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom

NCT01124643
(ClinicalTrials.gov)

April 13, 2010

23/4/2010

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease

Fabry Disease

Biological: Replagal

Shire

NULL

Completed

18 Years

N/A

All

Phase 3

United States;Australia;Czechia;Finland;Poland;Slovenia;United Kingdom;Czech Republic;Paraguay

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2009-015985-75-CZ
(EUCTR)

08/03/2010

24/02/2010

Follow on study in adult Fabry Disease patients

An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease

Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA

Shire Human Genetic Therapies (HGT), Inc.

NULL

Not Recruiting

Female: yes
Male: yes

United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom

EUCTR2009-015985-75-PL
(EUCTR)

03/02/2010

30/11/2009

Follow on study in adault Fabry Disease patients

An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease

Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA

Shire Human Genetic Therapies (HGT), Inc.

NULL

Not Recruiting

Female: yes
Male: yes

United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom

NCT01031173
(ClinicalTrials.gov)

September 2009

8/12/2009

Treatment Protocol of Replagal for Patients With Fabry Disease

An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.

Fabry Disease

Biological: agalsidase alfa

Shire

NULL

No longer available

N/A

Both

N/A

United States

NCT00864851
(ClinicalTrials.gov)

December 29, 2008

18/3/2009

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

A Multi-Center, Open-Label, Randomized Study Evaluating the Safety and Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement Therapy in Adult Patients With Fabry Disease

Fabry Disease

Biological: Replagal

Shire

NULL

Completed

18 Years

N/A

All

Phase 3

United States;Australia;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;Czech Republic

EUCTR2007-005543-22-CZ
(EUCTR)

19/06/2008

02/01/2008

An investigation of 3 dosages of Replagal in adult patients with Fabry disease

A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in Adult Patients with Fabry Disease

Fabry Disease
MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: agalsidase alpha
Other descriptive name: Replagal

Shire Human Genetic Therapies, Inc. (Shire HGT)

NULL

Not Recruiting

Female: yes
Male: yes

United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT00097890
(ClinicalTrials.gov)

November 2004

30/11/2004

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

An Open Label Six-Month Maintenance Clinical Trial of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease Who Have Completed TKT027

Fabry Disease

Drug: Replagal (Agalsidase Alfa);Drug: Replagal

National Institute of Neurological Disorders and Stroke (NINDS)

NULL

Completed

N/A

Male

Phase 4

United States

EUCTR2004-000772-14-CZ
(EUCTR)

23/07/2004

28/06/2004

A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027) - TKT027

Fabry Disease
MedDRA version: 6.1;Level: PT;Classification code 10016016

Trade Name: Replagal
Product Name: Replagal
INN or Proposed INN: agalsidase alfa

TKT Inc

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2

Czech Republic

NCT00084084
(ClinicalTrials.gov)

June 2004

5/6/2004

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

An Open Label Clinical Trial of Replagal Enzyme Replacement Therapy In Children With Fabry Disease Who Have Completed Study TKT023 or Who Are Naive to Enzyme Replacement Therapy

Fabry Disease

Drug: Agalsidase alfa

Shire

NULL

Completed

7 Years

17 Years

All

Phase 2

United States;Canada

NCT00075244
(ClinicalTrials.gov)

January 2004

6/1/2004

Alternative Dosing and Regimen of Replagal to Treat Fabry Disease

A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients With Fabry Disease

Fabry Disease

Drug: Replagal

National Institute of Neurological Disorders and Stroke (NINDS)

NULL

Completed

N/A

Male

Phase 2

United States

NCT00357786
(ClinicalTrials.gov)

October 2003

26/7/2006

An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease

An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal® (Registered Trademark) in Patients With Fabry Disease

Fabry Disease

Drug: Replagal agalsidase alfa;Drug: Replagal

National Institute of Neurological Disorders and Stroke (NINDS)

NULL

Completed

39 Years

45 Years

Male

Phase 1

United States

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT00071877
(ClinicalTrials.gov)

October 2003

3/11/2003

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

A Clinical Trial of Replagal Enzyme Replacement Therapy in Children Ages 7 - 17 Years With Fabry Disease

Fabry Disease

Drug: Replagal

National Institute of Neurological Disorders and Stroke (NINDS)

NULL

Completed

N/A

Both

Phase 2

United States

NCT00068107
(ClinicalTrials.gov)

September 2003

6/9/2003

Dosing Study of Replagal in Patients With Fabry Disease

Study of Weekly Dosing Regimens of Replagal in Patients With Fabry Disease With Incomplete Clinical Response to Long-Term Therapy

Fabry Disease

Drug: Replagal

Baylor Research Institute

National Institute of Neurological Disorders and Stroke (NINDS)

Completed

N/A

Male

Phase 2

United States

NCT00048906
(ClinicalTrials.gov)

November 2002

8/11/2002

Alpha-Galactosidase A Replacement Therapy for Fabry Disease

A Safety and Pharmacokinetic Study of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease

Fabry Disease

Drug: DRX005B

National Institute of Neurological Disorders and Stroke (NINDS)

NULL

Completed

N/A

Both

Phase 2

United States

EUCTR2019-000064-21-CZ
(EUCTR)

29/08/2019

To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfa

A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme) versus continuing on agalsidase alfa (Replagal) in male patients with classic Fabry disease

Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA

Sanofi Aventis Groupe (SAG)

NULL

Not Recruiting

Female: no
Male: yes

Phase 4

France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Norway;Germany;Italy;United Kingdom

EUCTR2019-000064-21-NO
(EUCTR)

15/08/2019

To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfa

A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical benefits of agalsidase beta (Fabrazyme®) in male patients with classic Fabry disease switching from agalsidase alfa (Replagal®)

Trade Name: Fabrazyme
INN or Proposed INN: AGALSIDASE BETA
Trade Name: Replagal
INN or Proposed INN: AGALSIDASE ALFA

Sanofi Aventis Groupe (SAG)

NULL

Not Recruiting

Female: no
Male: yes

Phase 4

France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Germany;Norway;Italy;United Kingdom