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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pharnext SA
ClinicalTrials.gov Identifier:
NCT01401257
First received: July 20, 2011
Last updated: December 23, 2016
Last verified: July 2011
Results First Received: December 23, 2016  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Charcot-Marie-Tooth Disease
Hereditary Neuropathy With Liability to Pressure Palsies
Genetic Disorders
Tooth Disorders
Interventions: Drug: PXT3003 Low dose
Drug: PXT3003 Intermediate Dose
Drug: PXT3003 High Dose
Other: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
PXT3003 Low Dose

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

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

PXT3003 Intermediate Dose

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

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

PXT3003 High Dose

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

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

Placebo

Oral Liquid formulation, bid, 12 months

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


Participant Flow:   Overall Study
    PXT3003 Low Dose   PXT3003 Intermediate Dose   PXT3003 High Dose   Placebo
STARTED   21   21   19   19 
COMPLETED   21   19   16   17 
NOT COMPLETED   0   2   3   2 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
PXT3003 Low Dose

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

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

PXT3003 Intermediate Dose

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

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

PXT3003 High Dose

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

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

Placebo

Oral Liquid formulation, bid, 12 months

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

Total Total of all reporting groups

Baseline Measures
   PXT3003 Low Dose   PXT3003 Intermediate Dose   PXT3003 High Dose   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 21   21   19   19   80 
Age 
[Units: Participants]
Count of 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 
[Units: Years]
Mean (Full Range)
 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) 
Gender 
[Units: Participants]
Count of 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 
[Units: Participants]
         
France   21   21   19   19   80 


  Outcome Measures

1.  Primary:   Safety and Tolerability of PXT3003   [ Time Frame: Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up ]

2.  Secondary:   To Obtain Preliminary Data on the Efficacy of PXT3003 on Clinical Scores and Functional Tests   [ Time Frame: Screening, randomization, 3-, 6-, 9- and 12-months treatment ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

3.  Secondary:   To Assess the Pharmacodynamic Effect of PXT3003 on PMP22 mRNA Levels and Intra-epidermal Axon Density in Cutaneous Biopsy   [ Time Frame: Randomization and 12-month treatment ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

4.  Secondary:   To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters   [ Time Frame: Screening, randomization, 3-, 6-, 9- and 12-month treatment ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

5.  Secondary:   To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers   [ Time Frame: Randomization and 3-month treatment ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

6.  Secondary:   To Assess the Plasma Concentrations of PXT3003   [ Time Frame: Randomization, 1-, 6- and 12-month treatment ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Pr Shahram ATTARIAN
Organization: Hôpital La Timone, Marseille
phone: 04 91 38 65 ext 79
e-mail: shahram.attarian@ap-hm.fr


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Pharnext SA
ClinicalTrials.gov Identifier: NCT01401257     History of Changes
Other Study ID Numbers: CLN-PXT3003-01
Study First Received: July 20, 2011
Results First Received: December 23, 2016
Last Updated: December 23, 2016