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	JPRN-UMIN000023517	2016/10/01	01/10/2016	A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C		Niemann-Pick disease type C	2-hydroxypropyl-beta-cyclodextrin (HPBCD) 20mg/kg/dose, every 3-4 weeks, for three years	Nara Medical University	NULL	Pending	4years-old	6years-old	Male and Female	1	Not selected	Japan
2	EUCTR2015-001875-32-Outside-EU/EEA (EUCTR)		12/11/2015	A long-term safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection	A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7	Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: pending Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS	Ultragenyx Pharmaceutical Inc.	NULL	NA			Female: yes Male: yes	12		United States
3	EUCTR2015-000104-26-Outside-EU/EEA (EUCTR)		23/07/2015	A safety and efficacy study in young MPS 7 patients (less than 5 years old) receiving enzyme (UX003) replacement by intravenous injection	An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 years old	Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Recombinant human betaglucuronidase Product Code: UX003 INN or Proposed INN: pending Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS	Ultragenyx Pharmaceutical Inc.	NULL	NA			Female: yes Male: yes	7		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

JPRN-UMIN000023517

2016/10/01

01/10/2016

A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C

Niemann-Pick disease type C

2-hydroxypropyl-beta-cyclodextrin (HPBCD) 20mg/kg/dose, every 3-4 weeks, for three years

Nara Medical University

NULL

Pending

4years-old

6years-old

Male and Female

Not selected

Japan

EUCTR2015-001875-32-Outside-EU/EEA
(EUCTR)

12/11/2015

A long-term safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection

A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7

Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Recombinant human beta-glucuronidase
Product Code: UX003
INN or Proposed INN: pending
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS

Ultragenyx Pharmaceutical Inc.

NULL

Female: yes
Male: yes

United States

EUCTR2015-000104-26-Outside-EU/EEA
(EUCTR)

23/07/2015

A safety and efficacy study in young MPS 7 patients (less than 5 years old) receiving enzyme (UX003) replacement by intravenous injection

An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 years old

Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Recombinant human betaglucuronidase
Product Code: UX003
INN or Proposed INN: pending
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS

Ultragenyx Pharmaceutical Inc.

NULL

Female: yes
Male: yes

United States