DDrare: Database of Drug Development for Rare Diseases

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_ gender	Target_ size	Phase	Countries
1	EUCTR2015-002379-81-NL (EUCTR)	08/01/2018	28/03/2017	An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.	International, multi-center, open-label 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.	Charcot-Marie-Tooth Disease type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Code: PXT3003 Dose 1 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL Product Code: PXT3003 Dose 2 (equivalent to twice Dose 1) INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL	Pharnext SA	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	290	Phase 3	France;United States;Canada;Belgium;Spain;Germany;Netherlands;United Kingdom
2	EUCTR2015-002379-81-DE (EUCTR)	15/08/2017	19/01/2017	An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.	International, multi-center, open-label 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.	Charcot-Marie-Tooth Disease type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Code: PXT3003 Dose 2 (equivalent to twice Dose 1) INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL Product Code: PXT3003 Dose 2 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL	Pharnext	NULL	Not Recruiting	Female: yes Male: yes	290	Phase 3	France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom
3	EUCTR2015-002379-81-GB (EUCTR)	28/07/2017	12/06/2017	An International, multi-center, FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.	International, multi-center, open-label FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.	Charcot-Marie-Tooth Disease type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Code: PXT3003 Dose 1 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL Product Code: PXT3003 Dose 2 (equivalent to twice Dose 1) INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL	Pharnext SA	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	187	Phase 3	United States;France;Canada;Spain;Belgium;Germany;Netherlands;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

EUCTR2015-002379-81-NL
(EUCTR)

08/01/2018

28/03/2017

An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.

International, multi-center, open-label 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.

Charcot-Marie-Tooth Disease type 1A
MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Code: PXT3003 Dose 1
INN or Proposed INN: BACLOFEN
Other descriptive name: NA
INN or Proposed INN: NALTREXONE
Other descriptive name: NALTREXONE HYDROCHLORIDE
INN or Proposed INN: Sorbitol
Other descriptive name: D-SORBITOL
Product Code: PXT3003 Dose 2 (equivalent to twice Dose 1)
INN or Proposed INN: BACLOFEN
Other descriptive name: NA
INN or Proposed INN: NALTREXONE
Other descriptive name: NALTREXONE HYDROCHLORIDE
INN or Proposed INN: Sorbitol
Other descriptive name: D-SORBITOL

Pharnext SA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

290

Phase 3

France;United States;Canada;Belgium;Spain;Germany;Netherlands;United Kingdom

EUCTR2015-002379-81-DE
(EUCTR)

15/08/2017

19/01/2017

An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.

International, multi-center, open-label 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.

Product Code: PXT3003 Dose 2 (equivalent to twice Dose 1)
INN or Proposed INN: BACLOFEN
Other descriptive name: NA
INN or Proposed INN: NALTREXONE
Other descriptive name: NALTREXONE HYDROCHLORIDE
INN or Proposed INN: Sorbitol
Other descriptive name: D-SORBITOL
Product Code: PXT3003 Dose 2
INN or Proposed INN: BACLOFEN
Other descriptive name: NA
INN or Proposed INN: NALTREXONE
Other descriptive name: NALTREXONE HYDROCHLORIDE
INN or Proposed INN: Sorbitol
Other descriptive name: D-SORBITOL

Pharnext

NULL

Not Recruiting

Female: yes
Male: yes

290

Phase 3

France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom

EUCTR2015-002379-81-GB
(EUCTR)

28/07/2017

12/06/2017

An International, multi-center, FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.

International, multi-center, open-label FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.

Pharnext SA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

187

Phase 3

United States;France;Canada;Spain;Belgium;Germany;Netherlands;United Kingdom