DDrare: Database of Drug Development for Rare Diseases

19. ライソゾーム病
［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］

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	NCT00954460 (ClinicalTrials.gov)	August 2009	5/8/2009	Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease	Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated™ Human Glucocerebrosidase (GA-GCB, Velaglucerase Alfa) ERT in Newly Diagnosed or Previously Treated (With Imiglucerase) Patients With Type 1 Gaucher Disease	Gaucher Disease, Type 1	Drug: velaglucerase alfa	Shire	NULL	Approved for marketing	3 Years	N/A	Both		N/A	United States
2	EUCTR2008-001965-27-ES (EUCTR)	23/12/2008	17/09/2008	Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease	Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease	Enfermedad de Gaucher de tipo IType I Gaucher disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease	Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase	Shire Human Genetic Therapies, Inc.	NULL	Not Recruiting			Female: yes Male: yes	102		Spain;United Kingdom
3	EUCTR2008-001965-27-GB (EUCTR)	29/10/2008	15/07/2008	An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy 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	Type I Gaucher disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease		Shire Human Genetic Therapies, Inc.	NULL	Not Recruiting			Female: yes Male: yes	102	Phase 3	Spain;United Kingdom
4	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
5	EUCTR2006-006304-11-IT (EUCTR)	14/04/2008	04/08/2008	A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - ND	A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - ND	Patients with type 1 Gaucher disease who previously received imiglucerase MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease	Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB	SHIRE HUMAN GENETIC THERAPIES, INC.	NULL	Not Recruiting			Female: yes Male: yes	40		United Kingdom;Spain;Italy
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	EUCTR2007-002840-21-IT (EUCTR)	25/02/2008	15/04/2008	A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - GCB039	A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - GCB039	Patients with Gaucher disease of type 1 MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease	Product Name: Gene Activated Human Glucocerebrosidase Product Code: GA-GCB Product Name: imiglucerasi INN or Proposed INN: Imiglucerase	SHIRE HUMAN GENETIC THERAPIES, INC.	NULL	Not Recruiting			Female: yes Male: yes	32		United Kingdom;Spain;Italy
7	NCT00553631 (ClinicalTrials.gov)	January 2008	1/11/2007	Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease	A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Type I Gaucher Disease	Gaucher Disease, Type 1	Biological: velaglucerase alfa;Biological: imiglucerase	Shire	NULL	Completed	2 Years	N/A	All	34	Phase 3	United States;Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom
8	EUCTR2007-002840-21-GB (EUCTR)	29/11/2007	11/10/2007	A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - HGT-GCB-039	A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - HGT-GCB-039	Type I Gaucher disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease	Product Name: Gene Activated Human glucocerebrosidase Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase Trade Name: Cerezyme Product Name: Cerezyme INN or Proposed INN: IMIGLUCERASE Trade Name: Cerezyme Product Name: Cerezyme INN or Proposed INN: IMIGLUCERASE	Shire Human Genetic Therapies, Inc.	NULL	Not Recruiting			Female: yes Male: yes	32		United Kingdom;Spain;Italy
9	EUCTR2007-002840-21-ES (EUCTR)	30/10/2007	18/07/2007	A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039	A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039	Type I Gaucher diseaseEnfermedad de Gaucher tipo 1 MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease	Product Name: Gene Activated Human glucocerebrosidase Product Code: GA-GCB Other descriptive name: Gene activated human glucocerebrosidase Trade Name: Cerezyme INN or Proposed INN: IMIGLUCERASE	Shire Human Genetic Therapies Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	32		United Kingdom;Spain;Italy
10	EUCTR2006-006304-11-ES (EUCTR)	27/08/2007	04/03/2010	A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034	A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034	Type I Gaucher DiseaseEnfermedad de Gaucher de Tipo I MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease	Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB Other descriptive name: Gene activated human glucocerebrosidase	Shire Human Genetic Therapies Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	40		United Kingdom;Spain;Italy
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	NCT00478647 (ClinicalTrials.gov)	July 25, 2007	23/5/2007	Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase	A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase	Gaucher Disease	Biological: GA-GCB (velaglucerase alfa)	Shire	NULL	Completed	2 Years	N/A	All	40	Phase 2;Phase 3	United States;Israel;Poland;Spain;United Kingdom
12	EUCTR2006-006304-11-GB (EUCTR)	03/07/2007	12/02/2007	A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034	A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034	Type I Gaucher Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease		Shire Human Genetic Therapies Inc	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 2;Phase 3	Spain;Germany;Italy;United Kingdom
13	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
14	NCT00391625 (ClinicalTrials.gov)	February 2005	20/10/2006	Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)	An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy	Gaucher Disease	Drug: GA-GCB	Shire	NULL	Completed	18 Years	N/A	All	10	Phase 1;Phase 2	Israel;Romania;Serbia;Former Serbia and Montenegro
15	EUCTR2006-006304-11-DE (EUCTR)		02/11/2007	A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034	A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034	Type I Gaucher Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease	Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase	Shire Human Genetic Therapies Inc	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 2;Phase 3	Spain;Germany;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
16	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

NCT00954460
(ClinicalTrials.gov)

August 2009

5/8/2009

Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated™ Human Glucocerebrosidase (GA-GCB, Velaglucerase Alfa) ERT in Newly Diagnosed or Previously Treated (With Imiglucerase) Patients With Type 1 Gaucher Disease

Gaucher Disease, Type 1

Drug: velaglucerase alfa

Shire

NULL

Approved for marketing

3 Years

N/A

Both

N/A

United States

EUCTR2008-001965-27-ES
(EUCTR)

23/12/2008

17/09/2008

Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease

Enfermedad de Gaucher de tipo IType I Gaucher disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease

Product Name: Gene-Activated Human Glucocerebrosidase
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: Gene activated human glucocerebrosidase

Shire Human Genetic Therapies, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

102

Spain;United Kingdom

EUCTR2008-001965-27-GB
(EUCTR)

29/10/2008

15/07/2008

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

Type I Gaucher disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease

Shire Human Genetic Therapies, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

102

Phase 3

Spain;United Kingdom

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

EUCTR2006-006304-11-IT
(EUCTR)

14/04/2008

04/08/2008

A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - ND

Patients with type 1 Gaucher disease who previously received imiglucerase
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease

Product Name: Gene-Activated Human Glucocerebrosidase
Product Code: GA-GCB

SHIRE HUMAN GENETIC THERAPIES, INC.

NULL

Not Recruiting

Female: yes
Male: yes

United Kingdom;Spain;Italy

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

EUCTR2007-002840-21-IT
(EUCTR)

25/02/2008

15/04/2008

A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - GCB039

Patients with Gaucher disease of type 1
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease

Product Name: Gene Activated Human Glucocerebrosidase
Product Code: GA-GCB
Product Name: imiglucerasi
INN or Proposed INN: Imiglucerase

SHIRE HUMAN GENETIC THERAPIES, INC.

NULL

Not Recruiting

Female: yes
Male: yes

United Kingdom;Spain;Italy

NCT00553631
(ClinicalTrials.gov)

January 2008

1/11/2007

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

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

Gaucher Disease, Type 1

Biological: velaglucerase alfa;Biological: imiglucerase

Shire

NULL

Completed

2 Years

N/A

All

Phase 3

United States;Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom

EUCTR2007-002840-21-GB
(EUCTR)

29/11/2007

11/10/2007

Type I Gaucher disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease

Product Name: Gene Activated Human glucocerebrosidase
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: Gene activated human glucocerebrosidase
Trade Name: Cerezyme
Product Name: Cerezyme
INN or Proposed INN: IMIGLUCERASE
Trade Name: Cerezyme
Product Name: Cerezyme
INN or Proposed INN: IMIGLUCERASE

Shire Human Genetic Therapies, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

United Kingdom;Spain;Italy

EUCTR2007-002840-21-ES
(EUCTR)

30/10/2007

18/07/2007

A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039

Type I Gaucher diseaseEnfermedad de Gaucher tipo 1
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease

Product Name: Gene Activated Human glucocerebrosidase
Product Code: GA-GCB
Other descriptive name: Gene activated human glucocerebrosidase
Trade Name: Cerezyme
INN or Proposed INN: IMIGLUCERASE

Shire Human Genetic Therapies Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

United Kingdom;Spain;Italy

EUCTR2006-006304-11-ES
(EUCTR)

27/08/2007

04/03/2010

A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034

Type I Gaucher DiseaseEnfermedad de Gaucher de Tipo I
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease

Product Name: Gene-Activated Human Glucocerebrosidase
Product Code: GA-GCB
Other descriptive name: Gene activated human glucocerebrosidase

Shire Human Genetic Therapies Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

United Kingdom;Spain;Italy

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

NCT00478647
(ClinicalTrials.gov)

July 25, 2007

23/5/2007

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase

Gaucher Disease

Biological: GA-GCB (velaglucerase alfa)

Shire

NULL

Completed

2 Years

N/A

All

Phase 2;Phase 3

United States;Israel;Poland;Spain;United Kingdom

EUCTR2006-006304-11-GB
(EUCTR)

03/07/2007

12/02/2007

A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034

Type I Gaucher Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease

Shire Human Genetic Therapies Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2;Phase 3

Spain;Germany;Italy;United Kingdom

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

NCT00391625
(ClinicalTrials.gov)

February 2005

20/10/2006

Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy

Gaucher Disease

Drug: GA-GCB

Shire

NULL

Completed

18 Years

N/A

All

Phase 1;Phase 2

Israel;Romania;Serbia;Former Serbia and Montenegro

EUCTR2006-006304-11-DE
(EUCTR)

02/11/2007

A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034

Type I Gaucher Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease

Product Name: Gene-Activated Human Glucocerebrosidase
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: Gene activated human glucocerebrosidase

Shire Human Genetic Therapies Inc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2;Phase 3

Spain;Germany;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

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