DDrare: Database of Drug Development for Rare Diseases

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.

Drug = ACE-083; ACIDO ASCORBICO; ACIDO ASCORBICO DC.IT; Acetate; Ascorbic acid; Ascorbic acid (Vitamin C); Ascorbic acid vit C; BACLOFEN; Baclofen; Biotin; CEBION 500*20CPR MAST 500MG; CEBION 500MG 20CPR MAST.ARAN; Coenzyme Q10; D-BIOTIN; D-SORBITOL; ElleOne; FLX-787-ODT (orally disintegrating tablet); MD1003; Mexiletine; NALTREXONE; NALTREXONE HYDROCHLORIDE; Naltrexone; Non applicable; PXT3003; PXT3003 Dose 1; PXT3003 Dose 2; PXT3003 Dose 2 (equivalent to twice Dose 1); PXT3003 High Dose; PXT3003 Intermediate Dose; PXT3003 Low dose; PXT3003 dose 1; PXT3003 dose 2; RS-BACLOFEN; SODIO ASCORBATO DC.IT; Sorbitol; ULIPRISTAL ACETATE; Ulipristal; Vitamin C

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	NCT02967679 (ClinicalTrials.gov)	December 5, 2016	8/11/2016	SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study	SERENDEM Study: MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study	Chronic Inflammatory Demyelinating Polyneuropathy;Peripheral Neuropathy;Charcot-Marie-Tooth Disease;Charcot-Marie-Tooth Disease Type 1A;Charcot-Marie-Tooth Disease, Type 1B;Anti-MAG Neuropathy	Drug: MD1003	MedDay Pharmaceuticals SA	NULL	Completed	20 Years	85 Years	All	15	Phase 2	France
2	EUCTR2015-001150-15-FR (EUCTR)	14/03/2016	02/03/2016	SERENDEM study: high dose biotin in patients suffering from disability related to peripheral nerve diseases	SERENDEM study: MD1003 in patients suffering from demyelinating neuropathies, an open label pilot study	chronic inflammatory demyelinating polyradiculoneuropathy, demyelinating neuropathy associated with immunoglobulin M (IgM) monoclonal gammopathy and antibodies against myelin-associated glycoprotein (MAG), Charcot Marie Tooth Ia (CMT Ia) neuropathy MedDRA version: 18.1;Level: LLT;Classification code 10066137;Term: Anti-MAG neuropathy;System Organ Class: 10029205 - Nervous system disorders MedDRA version: 18.1;Level: PT;Classification code 10057645;Term: Chronic inflammatory demyelinating polyradiculoneuropathy;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: biotin Product Code: MD1003 INN or Proposed INN: D-BIOTIN	MEDDAY PHARMACEUTICALS	NULL	Not Recruiting			Female: yes Male: yes	15	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

NCT02967679
(ClinicalTrials.gov)

December 5, 2016

8/11/2016

SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study

SERENDEM Study: MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study

Chronic Inflammatory Demyelinating Polyneuropathy;Peripheral Neuropathy;Charcot-Marie-Tooth Disease;Charcot-Marie-Tooth Disease Type 1A;Charcot-Marie-Tooth Disease, Type 1B;Anti-MAG Neuropathy

Drug: MD1003

MedDay Pharmaceuticals SA

NULL

Completed

20 Years

85 Years

All

Phase 2

France

EUCTR2015-001150-15-FR
(EUCTR)

14/03/2016

02/03/2016

SERENDEM study: high dose biotin in patients suffering from disability related to peripheral nerve diseases

SERENDEM study: MD1003 in patients suffering from demyelinating neuropathies, an open label pilot study

chronic inflammatory demyelinating polyradiculoneuropathy, demyelinating neuropathy associated with immunoglobulin M (IgM) monoclonal gammopathy and antibodies against myelin-associated glycoprotein (MAG), Charcot Marie Tooth Ia (CMT Ia) neuropathy
MedDRA version: 18.1;Level: LLT;Classification code 10066137;Term: Anti-MAG neuropathy;System Organ Class: 10029205 - Nervous system disorders
MedDRA version: 18.1;Level: PT;Classification code 10057645;Term: Chronic inflammatory demyelinating polyradiculoneuropathy;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: biotin
Product Code: MD1003
INN or Proposed INN: D-BIOTIN

MEDDAY PHARMACEUTICALS

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

France