Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT) (PLEO-CMT)
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ClinicalTrials.gov Identifier: NCT02579759 |
Recruitment Status :
Completed
First Posted : October 20, 2015
Results First Posted : February 27, 2020
Last Update Posted : February 27, 2020
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Study Type | Interventional |
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Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Investigator, Outcomes Assessor); Primary Purpose: Treatment |
Condition |
Charcot-Marie-Tooth Disease Type 1A |
Interventions |
Drug: PXT3003 dose 1 Drug: PXT3003 dose 2 Drug: placebo |
Enrollment | 323 |
Recruitment Details | |
Pre-assignment Details |
Arm/Group Title | PXT3003 Dose 1 | PXT3003 Dose 2 | Placebo |
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Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months. PXT3003 dose 1: 3 mg baclofen, 0.35 mg naltrexone and 105 mg sorbitol (twice a day, morning and evening with food). |
Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months. PXT3003 dose 2: 6 mg baclofen, 0.70 mg naltrexone and 210 sorbitol (twice a day, morning and evening with food). |
Liquid oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months. PXT3003 matching placebo had the same presentation, the same aspect and taste in order to be undistinguishable (twice a day, morning and evening with food). |
Period Title: Overall Study | |||
Started | 109 | 113 | 101 |
Completed [1] | 85 | 49 | 80 |
Not Completed | 24 | 64 | 21 |
Reason Not Completed | |||
Protocol Violation | 2 | 0 | 0 |
Other (Sponsor stopped Dose 2) | 0 | 1 | 0 |
BfArM hold | 13 | 12 | 12 |
Sponsor stopped Dose 2 | 0 | 41 | 0 |
Non compliance | 0 | 1 | 0 |
Pregnancy | 0 | 1 | 0 |
Inclusion/exclusion criteria | 0 | 0 | 1 |
Adverse Event | 4 | 3 | 1 |
Withdrawal by Subject | 3 | 3 | 5 |
Lost to Follow-up | 2 | 2 | 2 |
[1]
Completed at 12 months
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Arm/Group Title | PXT3003 Dose 1 | PXT3003 Dose 2 | Placebo | Total | |
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Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months PXT3003 dose 1: Liquid oral solution, 5 ml twice a day, morning and evening with food |
Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months PXT3003 dose 2: Liquid oral solution, 5 ml twice a day, morning and evening with food |
Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months placebo: Liquid oral solution, 5 ml twice a day, morning and evening with food |
Total of all reporting groups | |
Overall Number of Baseline Participants | 109 | 113 | 101 | 323 | |
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[Not Specified]
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 109 participants | 113 participants | 101 participants | 323 participants | |
<=18 years |
5 4.6%
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8 7.1%
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2 2.0%
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15 4.6%
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Between 18 and 65 years |
103 94.5%
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104 92.0%
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97 96.0%
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304 94.1%
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>=65 years |
1 0.9%
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1 0.9%
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2 2.0%
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4 1.2%
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 109 participants | 113 participants | 101 participants | 323 participants | |
41.0 (12.3) | 39.6 (13.9) | 42.1 (13.2) | 40.9 (13.2) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 109 participants | 113 participants | 101 participants | 323 participants | |
Female |
60 55.0%
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68 60.2%
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62 61.4%
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190 58.8%
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Male |
49 45.0%
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45 39.8%
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39 38.6%
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133 41.2%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 109 participants | 113 participants | 101 participants | 323 participants | |
Hispanic or Latino |
0 0.0%
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2 1.8%
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1 1.0%
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3 0.9%
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Not Hispanic or Latino |
108 99.1%
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111 98.2%
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100 99.0%
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319 98.8%
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Unknown or Not Reported |
1 0.9%
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0 0.0%
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0 0.0%
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1 0.3%
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Region of Enrollment
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 109 participants | 113 participants | 101 participants | 323 participants |
Canada | 5 | 4 | 5 | 14 | |
Netherlands | 2 | 3 | 3 | 8 | |
Belgium | 4 | 6 | 5 | 15 | |
United States | 21 | 24 | 18 | 63 | |
United Kingdom | 2 | 0 | 1 | 3 | |
France | 31 | 31 | 29 | 91 | |
Germany | 22 | 23 | 22 | 67 | |
Spain | 22 | 22 | 18 | 62 |
Name/Title: | Susanne Dorn |
Organization: | Pharnext |
Phone: | +33 (0)1 41 09 22 30 |
EMail: | contact@pharnext.com |
Responsible Party: | Pharnext SA |
ClinicalTrials.gov Identifier: | NCT02579759 |
Other Study ID Numbers: |
CLN-PXT3003-02 2015-002378-19 ( EudraCT Number ) |
First Submitted: | September 28, 2015 |
First Posted: | October 20, 2015 |
Results First Submitted: | November 18, 2019 |
Results First Posted: | February 27, 2020 |
Last Update Posted: | February 27, 2020 |