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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:	19 March 2012
Main ID:	EUCTR2010-023097-40-FR
Date of registration:	04/10/2010
Prospective Registration:	Yes
Primary sponsor:	Pharnext
Public title:	A Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX3003 in Patients with Charcot-Marie-Tooth Disease Type 1A - non applicable
Scientific title:	A Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX3003 in Patients with Charcot-Marie-Tooth Disease Type 1A - non applicable
Date of first enrolment:	29/11/2010
Target sample size:	80
Recruitment status:	Authorised-recruitment may be ongoing or finished
URL:	https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-023097-40
Study type:	Interventional clinical trial of medicinal product
Study design:	Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no
Phase:

Countries of recruitment
France

Contacts

Name:
Address:
Telephone:
Email:
Affiliation:

Name:
Address:
Telephone:
Email:
Affiliation:

Key inclusion & exclusion criteria

Inclusion criteria:
1. DNA proven CMT1A
2. Muscle weakness in at least foot dorsiflexion (clinical assess-ment)
3. Age between 18 and 65 years
4. Male or non pregnant, non breastfeeding female
5. CMT neuropathy score at screening = 18
6. Agrees to perform neurophysiological studies and two cutaneous biopsies (at baseline and end of treatment) for the determination of PMP22 mRNA expression and histology
7. Providing signed written informed consent to participate in the study and willing and able to comply with all study procedures and scheduled visits.
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
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Patients with another neurological disease
2. Patients using unauthorized concomitant treatments including but not limited to ascorbic acid, opioids, potentially neurotoxic drugs (list provided in appendix 1). Patients who can/agree to stop these medications 4 weeks before randomization can be in-cluded.
3. Patients who have participated in another trial of investigational drug within the past 30 days
4. Concomitant major systemic disease
5. Clinically significant history of unstable medical illness over the last 30 days (unstable angina…)
6. History of significant hematologic, kidney, liver disease, or insu-lin-dependent diabetes
7. Clinically significant abnormalities on the pre-study laboratory evaluation, physical evaluation, ECG
8. ASAT/ALAT and serum creatinin levels above the upper limit of normal (ULN)
9. Limited mental capacity or psychiatric disease rendering the subject unable to provide written informed consent or comply with evaluation procedures
10. History of recent alcohol or drug abuse or non-adherence with treatment or other experimental protocols
11. Female of childbearing potential (apart of patients using ade-quate contraceptive measures), pregnant or breast feeding
12. Suspected inability to complete the study follow-up (foreign workers, transient visitors, tourists or any others for whom fol-low-up evaluation is not assured)
13. Limb surgery within six months before randomization or planned before completion of the trial
14. Known allergy to any of the individual components of PXT3003
15. Porphyria (a contra indication to baclofen)

Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Charcot-Marie-Tooth disease (type 1A).
MedDRA version: 12.1 Level: LLT Classification code 10008414 Term: Charcot-Marie-Tooth disease

Intervention(s)

Product Name: non applicable
Product Code: PXT3003
Pharmaceutical Form: Oral solution
INN or Proposed INN: Baclofen
CAS Number: 1134-47-0
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.3-
INN or Proposed INN: Naltrexone
CAS Number: 16676-29-2
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.035-
INN or Proposed INN: Sorbitol
CAS Number: 50-70-4
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10.5-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use

Product Name: non applicable
Product Code: PXT3003
Pharmaceutical Form: Oral solution
INN or Proposed INN: Baclofen
CAS Number: 1134-47-0
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.6-
INN or Proposed INN: Naltrexone
CAS Number: 16676-29-2
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.07-
INN or Proposed INN: Sorbitol
CAS Number: 50-70-4
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 21-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use

Product Name: non applicable
Product Code: PXT3003
Pharmaceutical Form: Oral solution
INN or Proposed INN: Baclofen
CAS Number: 1134-47-0
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 3-
INN or Proposed INN: Naltrexone
CAS Number: 16676-29-2
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.35-
INN or Proposed INN: Sorbitol
CAS Number: 50-70-4
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 105-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: To assess the clinical and laboratory safety and tolerability of 3 doses of PXT3003 administered orally for 12 months to CMT1A patients

Secondary Objective: • To obtain preliminary data on the efficacy of PXT3003 on clini-cal scores and functional tests
• To assess the pharmacodynamic effect of PXT3003 in cutaneous biopsy: intra-epidermal axon density and mRNA expression of PMP22
• To assess the pharmacodynamic effect of PXT3003 on selected neurophysiological parameters
• To assess the pharmacodynamics of PXT3003 on a series of bio-chemical biomarkers
• To assess the plasma concentrations (peak and trough) of ba-clofen and naltrexone, two components of PXT3003, in CMT1A patients
• To correlate efficacy and safety results with PXT3003 doses

Primary end point(s): Incidence of related Adverse Events (including possibly and probably related AE) with moderate or severe intensity

Secondary Outcome(s)

Secondary ID(s)

non applicable

CLN-PXT3003-01

Source(s) of Monetary Support

Secondary Sponsor(s)

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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