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Non applicable    (DrugBank: -)

2 diseases
告示番号疾患名（ページ内リンク）臨床試験数
10シャルコー・マリー・トゥース病1
19ライソゾーム病1

10. シャルコー・マリー・トゥース病　［臨床試験数：31，薬物数：38（DrugBank：9），標的遺伝子数：11，標的パスウェイ数：14］
Searched query = "Charcot-Marie-Tooth disease", "CMT"
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.
1 / 31 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2010-023097-40-FR
(EUCTR)		29/11/201004/10/2010A Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX3003 in Patients with Charcot-Marie-Tooth Disease Type 1A - non applicableA Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX3003 in Patients with Charcot-Marie-Tooth Disease Type 1A - non applicable Charcot-Marie-Tooth disease (type 1A).
MedDRA version: 12.1;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease		Product Name: non applicable
Product Code: PXT3003
INN or Proposed INN: Baclofen
INN or Proposed INN: Naltrexone
INN or Proposed INN: Sorbitol
Product Name: non applicable
Product Code: PXT3003
INN or Proposed INN: Baclofen
INN or Proposed INN: Naltrexone
INN or Proposed INN: Sorbitol
Product Name: non applicable
Product Code: PXT3003
INN or Proposed INN: Baclofen
INN or Proposed INN: Naltrexone
INN or Proposed INN: Sorbitol		PharnextNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		80Phase 2France

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.
1 / 784 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2011-004410-42-FR
(EUCTR)		26/11/201219/12/2012Intracerebral Gene Therapy for MLDA 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		InsermNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		Phase 1;Phase 2France