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-ganglio ... show all
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	EUCTR2011-004410-42-FR (EUCTR)	26/11/2012	19/12/2012	Intracerebral Gene Therapy for MLD	A phase I/II, open labeled, monocentric study of direct intracranial administration of a replication deficient adeno-associated virus gene transfer vector serotype rh.10 expressing the human ARSA cDNA to children with metachromatic leukodystrophy - Intracerebral Gene Therapy for MLD	Early onset forms of MLD MedDRA version: 14.1;Level: SOC;Classification code 10029205;Term: Nervous system disorders;System Organ Class: 10029205 - Nervous system disorders MedDRA version: 14.1;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: AAVrh.10cuARSA Product Code: Non applicable	Inserm	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes		Phase 1;Phase 2	France

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

EUCTR2011-004410-42-FR
(EUCTR)

26/11/2012

19/12/2012

Intracerebral Gene Therapy for MLD

A phase I/II, open labeled, monocentric study of direct intracranial administration of a replication deficient adeno-associated virus gene transfer vector serotype rh.10 expressing the human ARSA cDNA to children with metachromatic leukodystrophy - Intracerebral Gene Therapy for MLD

Early onset forms of MLD
MedDRA version: 14.1;Level: SOC;Classification code 10029205;Term: Nervous system disorders;System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 14.1;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: AAVrh.10cuARSA
Product Code: Non applicable

Inserm

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

France