Trial · NCT01401257

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

NCT01401257, Phase 2, Completed, Pharnext S.C.A.; results first posted 2017-02-16

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A. Trials, record NCT01401257. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01401257

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
NCT number: NCT01401257
Status: Completed
Phases: Phase 2
Enrollment: 80 participants, actual
Lead sponsor: Pharnext S.C.A.
Conditions: Charcot-Marie-Tooth Disease, Hereditary Neuropathy With Liability to Pressure Palsies, Genetic Disorders
Interventions: PXT3003 Low dose, PXT3003 Intermediate Dose, PXT3003 High Dose, Placebo
First posted: 2011-07-25
Last updated: 2017-11-22
Results first posted: 2017-02-16

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Safety and Tolerability of PXT3003	Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up	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.
Secondary	To Obtain Preliminary Data on the Efficacy of PXT3003 on Clinical Scores and Functional Tests	Screening, randomization, 3-, 6-, 9- and 12-months treatment	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.
Secondary	To Assess the Pharmacodynamic Effect of PXT3003 on PMP22 mRNA Levels and Intra-epidermal Axon Density in Cutaneous Biopsy	Randomization and 12-month treatment	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.
Secondary	To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters	Screening, randomization, 3-, 6-, 9- and 12-month treatment	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.
Secondary	To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers	Randomization and 3-month treatment	Dosages of biochemical biomarkers in plasma. Change from baseline after 3-month of treatment.
Secondary	To Assess the Plasma Concentrations of PXT3003	Randomization, 1-, 6- and 12-month treatment	PXT3003 plasmatic concentrations after one administration (randomization) and after 1-,6-and 12-months of treatment.

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Safety and Tolerability of PXT3003	Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	To Obtain Preliminary Data on the Efficacy of PXT3003 on Clinical Scores and Functional Tests	Screening, randomization, 3-, 6-, 9- and 12-months treatment
SECONDARY	To Assess the Pharmacodynamic Effect of PXT3003 on PMP22 mRNA Levels and Intra-epidermal Axon Density in Cutaneous Biopsy	Randomization and 12-month treatment
SECONDARY	To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters	Screening, randomization, 3-, 6-, 9- and 12-month treatment
SECONDARY	To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers	Randomization and 3-month treatment
SECONDARY	To Assess the Plasma Concentrations of PXT3003	Randomization, 1-, 6- and 12-month treatment

Source ClinicalTrials.gov

Results by group4

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Units
Safety and Tolerability of PXT3003	PXT3003 Low Dose	21	Participants
Safety and Tolerability of PXT3003	PXT3003 Intermediate Dose	21	Participants
Safety and Tolerability of PXT3003	PXT3003 High Dose	19	Participants
Safety and Tolerability of PXT3003	Placebo	19	Participants

Source ClinicalTrials.gov

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	PXT3003 Low Dose	21
Overall Study	STARTED	PXT3003 Intermediate Dose	21
Overall Study	STARTED	PXT3003 High Dose	19
Overall Study	STARTED	Placebo	19
Overall Study	COMPLETED	PXT3003 Low Dose	21
Overall Study	COMPLETED	PXT3003 Intermediate Dose	19
Overall Study	COMPLETED	PXT3003 High Dose	16
Overall Study	COMPLETED	Placebo	17
Overall Study	NOT COMPLETED	PXT3003 Low Dose	0
Overall Study	NOT COMPLETED	PXT3003 Intermediate Dose	2

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events24

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Infections and infestations	Infections and infestations	PXT3003 Low Dose	11	21
other	Infections and infestations	Infections and infestations	PXT3003 Intermediate Dose	10	21
other	Infections and infestations	Infections and infestations	PXT3003 High Dose	11	19
other	Infections and infestations	Infections and infestations	Placebo	8	19
other	Musculoskeletal and connective tissue disorders	Musculoskeletal and connective tissue disorders	PXT3003 Low Dose	12	21
other	Musculoskeletal and connective tissue disorders	Musculoskeletal and connective tissue disorders	PXT3003 Intermediate Dose	8	21
other	Musculoskeletal and connective tissue disorders	Musculoskeletal and connective tissue disorders	PXT3003 High Dose	13	19
other	Musculoskeletal and connective tissue disorders	Musculoskeletal and connective tissue disorders	Placebo	7	19
other	Nervous system disorders	Nervous system disorders	PXT3003 Low Dose	8	21
other	Nervous system disorders	Nervous system disorders	PXT3003 Intermediate Dose	8	21

1 to 10 of 24Source ClinicalTrials.gov

See all 24 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	PXT3003 Low Dose
Total, all-cause mortality	PXT3003 Intermediate Dose
Total, all-cause mortality	PXT3003 High Dose
Total, all-cause mortality	Placebo
Total, other adverse events	PXT3003 Low Dose	12	21
Total, other adverse events	PXT3003 Intermediate Dose	10	21
Total, other adverse events	PXT3003 High Dose	13	19
Total, other adverse events	Placebo	11	19
Total, serious adverse events	PXT3003 Low Dose	2	21
Total, serious adverse events	PXT3003 Intermediate Dose	0	21

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
25519680	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01401257

GET https://conductscience.org/api/trials/v1/NCT01401257/events?page=2

API reference For AI assistants