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	NCT04429984 (ClinicalTrials.gov)	January 31, 2021	9/6/2020	Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India	A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India	Gaucher Disease	Drug: Velaglucerase alfa (VPRIV)	Shire	NULL	Not yet recruiting	N/A	N/A	All	35		NULL
2	NCT03702361 (ClinicalTrials.gov)	September 4, 2018	6/8/2018	Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease	Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study	Primary Disease	Drug: VPRIV	Shaare Zedek Medical Center	NULL	Unknown status	6 Years	75 Years	All	15	Phase 4	Israel
3	EUCTR2015-001578-17-IT (EUCTR)	18/07/2018	04/11/2020	An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease - SHP-GCB-402	Gaucher Disease MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Trade Name: VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO Product Name: Velaglucerase alfa Product Code: [-] INN or Proposed INN: velaglucerasi alfa Other descriptive name: GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO	SHIRE HUMAN GENETIC THERAPIES, INC	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 4	United States;Spain;Israel;Germany;United Kingdom;Italy
4	EUCTR2015-001578-17-DE (EUCTR)	12/09/2016	22/12/2015	An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease	Gaucher Disease MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Trade Name: VPRIV 400 Units powder for solution of infusion INN or Proposed INN: VELAGLUCERASE ALFA Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa	Shire Human Genetic Therapies, Inc.	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 4	United States;Spain;Turkey;Israel;Germany;Italy;United Kingdom;India
5	NCT02574286 (ClinicalTrials.gov)	June 29, 2016	9/10/2015	Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease	Gaucher Disease	Drug: Velaglucerase alfa;Dietary Supplement: Vitamin D	Shire	NULL	Completed	18 Years	70 Years	All	21	Phase 4	United States;Israel;Spain;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
6	EUCTR2015-001578-17-ES (EUCTR)	25/02/2016	13/01/2016	An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment with Velaglucerase alfa on Bone-related Pathology in Treatment-naïve Patients with Type 1 Gaucher Disease	Gaucher Disease MedDRA version: 18.1;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Trade Name: VPRIV 400 Units powder for solution of infusion INN or Proposed INN: VELAGLUCERASE ALFA Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa	Shire Human Genetic Therapies, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	40	Phase 4	United States;Canada;Spain;Israel;Germany;Italy;United Kingdom
7	EUCTR2015-001578-17-GB (EUCTR)	17/02/2016	21/12/2015	An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease	An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease	Gaucher Disease MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Trade Name: VPRIV 400 Units powder for solution of infusion INN or Proposed INN: VELAGLUCERASE ALFA Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa	Shire Human Genetic Therapies, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	19	Phase 4	United States;Canada;Spain;Israel;Germany;United Kingdom
8	NCT04120506 (ClinicalTrials.gov)	January 10, 2016	10/8/2018	Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)	Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study	Gaucher Disease, Type 1	Drug: VPRIV	Shaare Zedek Medical Center	Shire	Active, not recruiting	6 Years	75 Years	All	15	Phase 4	NULL
9	NCT02528617 (ClinicalTrials.gov)	July 2015	28/7/2015	The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease	The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease	Gaucher Disease Type 1;Gaucher Disease Type 3	Drug: Velaglucerase alfa	Baylor Research Institute	Texas Scottish Rite Hospital for Children	Withdrawn	4 Years	14 Years	All	0	Phase 4	United States
10	NCT00635427 (ClinicalTrials.gov)	May 2008	6/3/2008	An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease	An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease	Gaucher Disease, Type 1	Biological: VPRIV ®	Shire	NULL	Completed	2 Years	N/A	All	95	Phase 3	United States;Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tunisia;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	NCT00430625 (ClinicalTrials.gov)	January 2007	1/2/2007	A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease	A Multicenter, Randomized, Double-Blind, Parallel Group, Two-Dose Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease	Gaucher Disease, Type 1	Biological: VPRIV ®,	Shire	NULL	Completed	2 Years	N/A	All	25	Phase 3	Argentina;Israel;Paraguay;Russian Federation;Tunisia;United States
12	EUCTR2012-003427-38-Outside-EU/EEA (EUCTR)		07/04/2016	Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease	A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease	Type 3 Gaucher disease MedDRA version: 18.1;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Trade Name: VPRIV Product Name: Gene-Activated Human Glucocerebrosidase 400U/vial Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: VELAGLUCERASE ALFA	Shire Human Genetic Therapies	NULL	NA			Female: yes Male: yes	6		Tunisia;Egypt;India

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

NCT04429984
(ClinicalTrials.gov)

January 31, 2021

9/6/2020

Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India

A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India

Gaucher Disease

Drug: Velaglucerase alfa (VPRIV)

Shire

NULL

Not yet recruiting

N/A

All

NULL

NCT03702361
(ClinicalTrials.gov)

September 4, 2018

6/8/2018

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study

Primary Disease

Drug: VPRIV

Shaare Zedek Medical Center

NULL

Unknown status

6 Years

75 Years

All

Phase 4

Israel

EUCTR2015-001578-17-IT
(EUCTR)

18/07/2018

04/11/2020

An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease - SHP-GCB-402

Gaucher Disease
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trade Name: VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO
Product Name: Velaglucerase alfa
Product Code: [-]
INN or Proposed INN: velaglucerasi alfa
Other descriptive name: GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO

SHIRE HUMAN GENETIC THERAPIES, INC

NULL

Not Recruiting

Female: yes
Male: yes

Phase 4

United States;Spain;Israel;Germany;United Kingdom;Italy

EUCTR2015-001578-17-DE
(EUCTR)

12/09/2016

22/12/2015

An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease

Trade Name: VPRIV 400 Units powder for solution of infusion
INN or Proposed INN: VELAGLUCERASE ALFA
Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa

Shire Human Genetic Therapies, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 4

United States;Spain;Turkey;Israel;Germany;Italy;United Kingdom;India

NCT02574286
(ClinicalTrials.gov)

June 29, 2016

9/10/2015

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease

Gaucher Disease

Drug: Velaglucerase alfa;Dietary Supplement: Vitamin D

Shire

NULL

Completed

18 Years

70 Years

All

Phase 4

United States;Israel;Spain;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

EUCTR2015-001578-17-ES
(EUCTR)

25/02/2016

13/01/2016

An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment with Velaglucerase alfa on Bone-related Pathology in Treatment-naïve Patients with Type 1 Gaucher Disease

Gaucher Disease
MedDRA version: 18.1;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trade Name: VPRIV 400 Units powder for solution of infusion
INN or Proposed INN: VELAGLUCERASE ALFA
Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa

Shire Human Genetic Therapies, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 4

United States;Canada;Spain;Israel;Germany;Italy;United Kingdom

EUCTR2015-001578-17-GB
(EUCTR)

17/02/2016

21/12/2015

An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease

Trade Name: VPRIV 400 Units powder for solution of infusion
INN or Proposed INN: VELAGLUCERASE ALFA
Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa

Shire Human Genetic Therapies, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 4

United States;Canada;Spain;Israel;Germany;United Kingdom

NCT04120506
(ClinicalTrials.gov)

January 10, 2016

10/8/2018

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study

Gaucher Disease, Type 1

Drug: VPRIV

Shaare Zedek Medical Center

Shire

Active, not recruiting

6 Years

75 Years

All

Phase 4

NULL

NCT02528617
(ClinicalTrials.gov)

July 2015

28/7/2015

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

Gaucher Disease Type 1;Gaucher Disease Type 3

Drug: Velaglucerase alfa

Baylor Research Institute

Texas Scottish Rite Hospital for Children

Withdrawn

4 Years

14 Years

All

Phase 4

United States

NCT00635427
(ClinicalTrials.gov)

May 2008

6/3/2008

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease

Gaucher Disease, Type 1

Biological: VPRIV ®

Shire

NULL

Completed

2 Years

N/A

All

Phase 3

United States;Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tunisia;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

NCT00430625
(ClinicalTrials.gov)

January 2007

1/2/2007

A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

A Multicenter, Randomized, Double-Blind, Parallel Group, Two-Dose Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease

Gaucher Disease, Type 1

Biological: VPRIV ®,

Shire

NULL

Completed

2 Years

N/A

All

Phase 3

Argentina;Israel;Paraguay;Russian Federation;Tunisia;United States

EUCTR2012-003427-38-Outside-EU/EEA
(EUCTR)

07/04/2016

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease

Type 3 Gaucher disease
MedDRA version: 18.1;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Trade Name: VPRIV
Product Name: Gene-Activated Human Glucocerebrosidase 400U/vial
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: VELAGLUCERASE ALFA

Shire Human Genetic Therapies

NULL

Female: yes
Male: yes

Tunisia;Egypt;India