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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 5 March 2018
Main ID:  EUCTR2015-002379-81-NL
Date of registration: 28/03/2017
Prospective Registration: Yes
Primary sponsor: Pharnext SA
Public title: 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.
Scientific title: 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.
Date of first enrolment: 08/01/2018
Target sample size: 290
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-002379-81
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no
Randomised: no
Open: no
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
Number of treatment arms in the trial: 2
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Belgium Canada France Germany Netherlands Spain United Kingdom United States
Contacts
Name: Anne-Claire Coyne    
Address:  11, rue des Peupliers 92130 Issy-Les-Moulineaux France
Telephone: 33141 09 22 65
Email: accoyne@pharnext.com
Affiliation:  Pharnext SA
Name: Anne-Claire Coyne    
Address:  11, rue des Peupliers 92130 Issy-Les-Moulineaux France
Telephone: 33141 09 22 65
Email: accoyne@pharnext.com
Affiliation:  Pharnext SA
Key inclusion & exclusion criteria
Inclusion criteria:
Male or female patients with all of the following inclusion criteria will be considered for randomization:
1. Patient previously randomized to the initial PLEO-CMT study (protocol CLN-PXT3003-02) under placebo and D1 and having completed 15 months of double-blind treatment in that study, including all procedures required at the Study Termination visit (V6) or patients under D2 prematurely discontinued from CLN-PXT3003-02 (due to sponsor decision on September 18th, 2017) must have performed an early V6
2. Patients who completed the V6 assessments within the4 weeks prior to extension study or patients who have completed a new baseline visit (VB) if the V6 assessments were not completed in the 4 weeks prior to study entry.
3. Patient providing a signed written informed consent to participate in the extension study and willing and able to comply with all study procedures and scheduled visits. All minor patients who were included in the primary study will have reached their majority, so only adult patients will be included in the extension study.
3. Female patients must agree to continue using an approved method of birth control throughout the extension study
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 285
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 5

Exclusion criteria:
Patients with any of the following criteria will be excluded from entry:
1. Presenting any clinically significant change in health status that, in the opinion of the Investigator, would prevent the subject from participating in this study or successfully completing this study.
2. Any unauthorized concomitant treatments, in the 4 weeks preceding study entry, as study CLN-PXT3003-02.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
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]
Intervention(s)

Product Code: PXT3003 Dose 1
Pharmaceutical Form: Oral solution
INN or Proposed INN: BACLOFEN
CAS Number: 1134-47-0
Current Sponsor code: NA
Other descriptive name: NA
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 0.6-
INN or Proposed INN: NALTREXONE
CAS Number: 16676-29-2
Current Sponsor code: NA
Other descriptive name: NALTREXONE HYDROCHLORIDE
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 0.07-
INN or Proposed INN: Sorbitol
CAS Number: 50-70-4
Current Sponsor code: NA
Other descriptive name: D-SORBITOL
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 21-

Product Code: PXT3003 Dose 2 (equivalent to twice Dose 1)
Pharmaceutical Form: Oral solution
INN or Proposed INN: BACLOFEN
CAS Number: 1134-47-0
Current Sponsor code: NA
Other descriptive name: NA
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1.2-
INN or Proposed INN: NALTREXONE
CAS Number: 16676-29-2
Current Sponsor code: NA
Other descriptive name: NALTREXONE HYDROCHLORIDE
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 0.14-
INN or Proposed INN: Sorbitol
CAS Number: 50-70-4
Current Sponsor code: NA
Other descriptive name: D-SORBITOL
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 42-

Primary Outcome(s)
Main Objective: The main objective of the study is to assess the long-term safety and tolerability of PXT3003 up to 2 years, and to increase the exposed population receiving PXT3003 for at least 9 months.
Primary end point(s): To assess the safety and tolerability of PXT3003 in CMT1A patients during a long term period, the primary endpoint will be the incidence of Treatment-Emergent Adverse Events (TEAEs) Related to the Investigational Product during the follow-up, in patients exposed to PXT3003 for up to 24 months or 9 months.
The incidence of TEAEs in the two groups P / D1-9m and P/ D2-9m will be compared to the first 9-month placebo period in the primary study.
The incidence of TEAEs in the two groups D1-24m, D2-24m will be compared to the first 9-month period in the primary study for their corresponding group.
Secondary Objective: To gather up-to-2-year data to estimate the long term effect of PXT3003 on clinical, functional and electrophysiological endpoints;
- To compare the effect of PXT3003 in patients receiving PXT3003 active dose from the start of the primary study to patients assigned to a delayed start (i.e., who received placebo during the primary study) in order to determine if PXT3003 slows the progression of the disease
- To assess the evolution of potential blood biomarkers and molecular changes in skin biopsy on longer term.
Timepoint(s) of evaluation of this end point: The incidence of TEAEs in the two groups P/ D1-9m and P / D2-9m will be compared to the first 9-month placebo period in the primary study.
The incidence of TEAEs in the two groups D1-24m, D2-24m will be compared to the first 9-month period in the primary study for their corresponding group
Secondary Outcome(s)
Secondary end point(s): Secondary safety endpoints will be:
- The incidence of all Treatment-Emergent Adverse Events (TEAEs) and evaluation of their type/nature, severity/intensity, seriousness, duration, relationship to study drug, and outcome;
- The incidence of AEs leading to withdrawal of study drug;
- The changes in laboratory parameters, ECGs, vital signs and physical examinations;
Timepoint(s) of evaluation of this end point: For the patients receiving up to 24 months of active dose along the two successive studies (from groups D1-24m and D2-24m) the clinical response will be assessed by the change from V1/baseline of each endpoint.
For the patients who received placebo during the 15-month primary study and active drug during the 9-month extension study (from groups P/D1-9m and P/D2-9m) the clinical response will be assessed after 9 months of PXT3003 treatment by the change from V6/baseline.
The Percentage of responders to PXT3003 defined as a patients improving on ONLS at end of treatment (V9) will be assessed in the patients receiving the active dose of PXT3003 during up to 24 months (from groups D1-24m, D2-24m).
Secondary ID(s)
122505
2015-002379-81-FR
CLN-PXT3003-03
Source(s) of Monetary Support
Pharnext SA
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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