Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01401257
Recruitment Status : Completed
First Posted : July 25, 2011
Results First Posted : February 16, 2017
Last Update Posted : November 22, 2017
Sponsor:
Information provided by (Responsible Party):
Pharnext SA

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Charcot-Marie-Tooth Disease
Hereditary Neuropathy With Liability to Pressure Palsies
Genetic Disorders
Interventions Drug: PXT3003 Low dose
Drug: PXT3003 Intermediate Dose
Drug: PXT3003 High Dose
Other: Placebo
Enrollment 80
Recruitment Details  
Pre-assignment Details  
Arm/Group Title PXT3003 Low Dose PXT3003 Intermediate Dose PXT3003 High Dose Placebo
Hide Arm/Group Description

Oral Liquid formulation, 1/100, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, 1/50, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, 1/10, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, bid, 12 months

Placebo: Liquid,5 ml, twice a day, 12-month treatment

Period Title: Overall Study
Started 21 21 19 19
Completed 21 19 16 17
Not Completed 0 2 3 2
Arm/Group Title PXT3003 Low Dose PXT3003 Intermediate Dose PXT3003 High Dose Placebo Total
Hide Arm/Group Description

Oral Liquid formulation, 1/100, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, 1/50, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, 1/10, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, bid, 12 months

Placebo: Liquid,5 ml, twice a day, 12-month treatment

Total of all reporting groups
Overall Number of Baseline Participants 21 21 19 19 80
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 21 participants 19 participants 19 participants 80 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
21
 100.0%
21
 100.0%
19
 100.0%
19
 100.0%
80
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 21 participants 21 participants 19 participants 19 participants 80 participants
47.9
(18 to 63)
44.3
(23 to 64)
44.6
(24 to 63)
43.2
(21 to 65)
45.1
(18 to 65)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 21 participants 19 participants 19 participants 80 participants
Female
14
  66.7%
13
  61.9%
10
  52.6%
11
  57.9%
48
  60.0%
Male
7
  33.3%
8
  38.1%
9
  47.4%
8
  42.1%
32
  40.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
France Number Analyzed 21 participants 21 participants 19 participants 19 participants 80 participants
21 21 19 19 80
1.Primary Outcome
Title Safety and Tolerability of PXT3003
Hide Description

The Primary Objective is to assess the clinical and laboratory safety and tolerability of three doses of PXT3003 administered orally for 12 months to CMT1A patients versus placebo.

Number of participants with adverse events in each arm.

Time Frame Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title PXT3003 Low Dose PXT3003 Intermediate Dose PXT3003 High Dose Placebo
Hide Arm/Group Description:

Oral Liquid formulation, 1/100, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, 1/50, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, 1/10, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, bid, 12 months

Placebo: Liquid,5 ml, twice a day, 12-month treatment

Overall Number of Participants Analyzed 21 21 19 19
Measure Type: Count of Participants
Unit of Measure: Participants
21
 100.0%
21
 100.0%
19
 100.0%
19
 100.0%
2.Secondary Outcome
Title To Obtain Preliminary Data on the Efficacy of PXT3003 on Clinical Scores and Functional Tests
Hide Description

Efficacy scores and functional tests will be assessed CMTNS/CMTES:ONLS, VAS, fatigue, pain, six minute walk test (6MWT), nine-hole peg test, quantified muscular testing (QMT; hand grip and foot dorsiflexion), CGI.

For each test or score, change from baseline after 3-,6-, 9- and 12-months of treatment.

Time Frame Screening, randomization, 3-, 6-, 9- and 12-months treatment
Outcome Measure Data Not Reported
3.Secondary Outcome
Title To Assess the Pharmacodynamic Effect of PXT3003 on PMP22 mRNA Levels and Intra-epidermal Axon Density in Cutaneous Biopsy
Hide Description

A cutaneous biopsy (consisting in 2 small punch biopsies) will be performed to assess PMP22 mRNA expression and intra-epidermal axon density.

Change from baseline after 12-month of treatment.

Time Frame Randomization and 12-month treatment
Outcome Measure Data Not Reported
4.Secondary Outcome
Title To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters
Hide Description

Electrophysiological examination will be performed to assess sensory and motor responses of the median and ulnar nerves (non-dominant side) including: NCV, compound muscle action potential (CMAP) and SNAP.

Change from baseline after 3-,6-, 9- and 12-months of treatment.

Time Frame Screening, randomization, 3-, 6-, 9- and 12-month treatment
Outcome Measure Data Not Reported
5.Secondary Outcome
Title To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers
Hide Description

Dosages of biochemical biomarkers in plasma.

Change from baseline after 3-month of treatment.

Time Frame Randomization and 3-month treatment
Outcome Measure Data Not Reported
6.Secondary Outcome
Title To Assess the Plasma Concentrations of PXT3003
Hide Description PXT3003 plasmatic concentrations after one administration (randomization) and after 1-,6-and 12-months of treatment.
Time Frame Randomization, 1-, 6- and 12-month treatment
Outcome Measure Data Not Reported
Time Frame 13 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title PXT3003 Low Dose PXT3003 Intermediate Dose PXT3003 High Dose Placebo
Hide Arm/Group Description

Oral Liquid formulation, 1/100, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, 1/50, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, 1/10, bid, 12 months

PXT3003: Liquid,5 ml, twice a day, 12-month treatment

Oral Liquid formulation, bid, 12 months

Placebo: Liquid,5 ml, twice a day, 12-month treatment

All-Cause Mortality
PXT3003 Low Dose PXT3003 Intermediate Dose PXT3003 High Dose Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
PXT3003 Low Dose PXT3003 Intermediate Dose PXT3003 High Dose Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/21 (9.52%)   0/21 (0.00%)   0/19 (0.00%)   0/19 (0.00%) 
Gastrointestinal disorders         
Hemorrhoids  1 [1]  1/21 (4.76%)  0/21 (0.00%)  0/19 (0.00%)  0/19 (0.00%) 
Musculoskeletal and connective tissue disorders         
osteoarthritis  1 [2]  1/21 (4.76%)  0/21 (0.00%)  0/19 (0.00%)  0/19 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.1
[1]
Unrelated to study medication.
[2]
Unrelated to study medication
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
PXT3003 Low Dose PXT3003 Intermediate Dose PXT3003 High Dose Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   12/21 (57.14%)   10/21 (47.62%)   13/19 (68.42%)   11/19 (57.89%) 
Gastrointestinal disorders         
Gastrointestinal disorders  1  7/21 (33.33%)  5/21 (23.81%)  10/19 (52.63%)  11/19 (57.89%) 
Infections and infestations         
Infections and infestations  1  11/21 (52.38%)  10/21 (47.62%)  11/19 (57.89%)  8/19 (42.11%) 
Musculoskeletal and connective tissue disorders         
Musculoskeletal and connective tissue disorders  1  12/21 (57.14%)  8/21 (38.10%)  13/19 (68.42%)  7/19 (36.84%) 
Nervous system disorders         
Nervous system disorders  1  8/21 (38.10%)  8/21 (38.10%)  11/19 (57.89%)  9/19 (47.37%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.1
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

No individual publication (paper, abstract, poster, oral presentation) is allowed until results of the complete study have been published.

The Investigator agrees that any proposed publication, presentation or use of results arising from the Investigation will be submitted to Pharnext for review and written agreement prior to submission.

Results Point of Contact
Name/Title: Pr Shahram ATTARIAN
Organization: Hôpital La Timone, Marseille
Phone: 04 91 38 65 ext 79
Responsible Party: Pharnext SA
ClinicalTrials.gov Identifier: NCT01401257     History of Changes
Other Study ID Numbers: CLN-PXT3003-01
First Submitted: July 20, 2011
First Posted: July 25, 2011
Results First Submitted: December 23, 2016
Results First Posted: February 16, 2017
Last Update Posted: November 22, 2017