Trial · NCT02579759
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)
NCT02579759, Phase 3, Completed, Pharnext S.C.A.; results first posted 2020-02-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT). Trials, record NCT02579759. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02579759
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)
- NCT number
- NCT02579759
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 323 participants, actual
- Lead sponsor
- Pharnext S.C.A.
- Conditions
- Charcot-Marie-Tooth Disease Type 1A
- Interventions
- PXT3003 dose 1, PXT3003 dose 2, placebo
- First posted
- 2015-10-20
- Last updated
- 2020-02-27
- Other registries' IDs
- eudract:2015-002378-19
- Results first posted
- 2020-02-27
- Limitations and caveats
Two events occured during the trial due to crytals in Dose 2 formulation: hold of all subjects enrolled in Germany (Jun-17) and discontinuation of Dose 2 arm by the sponsor worldwide due to discovery of crystals in the ICH stability batch in Sep-17.
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Neuropathy Limitation Scale (ONLS) Total Score | From Baseline to Month 15 | The primary efficacy variable used in the main analysis is the mean of the available ONLS values at month 12 and month 15. The ONLS is a disability scale that was derived and improved from the Overall Disability Sum Score (ODSS) to measure limitations in the everyday activities of the upper limbs (rated on 5 points) and the lower limbs (rated on 7 points). The total score is a 12-point scale: 0 (no disability) to 12 (maximum disability). Lower values in the ONLS indicate a better clinical condition. Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin). |
| Secondary | Mean of Ten Meter Walking Test (10MWT) | From Baseline to Month 15 | This outcome measure is the mean of the available 10MWT values at month 12 and month 15. The 10MWT is a simple to administer, standardized, reliable and valid evaluation of functional exercise capacity and gait that has been used to evaluate neurologic disorders and CMT patients. Lower Time to Walk 10 Meters values indicate a better clinical condition. Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin). |
| Secondary | Mean of the CMTNS-v2 Sensory Score | From Baseline to Month 15 | This outcome measure is the mean of the available CMTNS-v2 Sensory Score values at month 12 and month 15. The CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4. The CMTNS-v2 Sensory score is summed of items 1+4+5 of CMTNS-v2 (Sensory symptoms, Pinprick sensibility and Vibration). It is a 12-point score: 0 (no impairment) to 12 (maximum impairment). Lower CMTNS-v2 Sensory Score values indicate a better clinical condition. Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin). |
| Secondary | Mean of the CMTNS-v2 Examination Score (CMTES-v2) | From Baseline to Month 15 | This outcome measure is the mean of the available CMTNS-v2 Examination Score values at month 12 and month 15. The CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4. The CMTES-v2 is summed of item 1 to 7 of the CMTNS-v2 (limited to impairment items and excluding electrophysiological items). It is a 28-point score: 0 (no impairment) to 28 (maximum impairment). Lower CMTES-v2 values indicate a better clinical condition. Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin). |
| Secondary | Mean of the Results at the Nine-Hole Peg Test (9-HPT) | From Baseline to Month 15 | This outcome measure is the mean of the available 9-HPT values at month 12 and month 15. The Nine-Hole Peg Test (9HPT) is a simple timed test of fine motor coordination of extremitied in the upper limbs. It measures the time needed by the patient to insert 9 pegs in nine holes and to remove them (normal required time 18 seconds). Lower 9HPT values indicate a better clinical condition. Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin). |
| Secondary | Number of Subjects With at Least One TEAE | The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months) | Safety selection was to include all randomized patients that have received at least one dose of study treatment. Safety and tolerability of PXT3003 were compared to placebo on the incidence of treatment-emergent adverse events (TEAEs); they were evaluated by type/nature, severity/intensity, seriousness, and relationship to study drug. |
| Secondary | Incidence of AE Leading to Withdrawal of Study Drug | The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months) | Safety and tolerability of PXT3003 were compared to placebo on the incidence of TEAEs leading to withdrawal of study drug. |
| Secondary | Incidence of SAEs | The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months). | Safety and tolerability of PXT3003 were compared to placebo on the incidence of serious adverse events (SAEs). |
| Other | Mean of the CMTNS-v2 Sensory Symptoms | From Baseline to Month 15 | This outcome measure is the mean of the available CMTNS-v2 Sensory Symptoms values at month 12 and month 15. The CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4. The CMTNS-v2 Sensory Symptoms is the first item of the CMTNS-v2. It is a 4-point score: 0 (no impairment) to 4 (maximum impairment). Lower CMTNS-v2 Sensory Symptoms values indicate a better clinical condition. Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin). |
| Other | Plasma Concentrations of Baclofen at Trough and at 90 Min After Drug Intake | At Month 12 and Month 15 | Plasma concentration of PXT3003 components were measured at trough (prior to dose) and 90 minutes after drug intake. The mean plasma values of the baseline correspond to half of the administered dose. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Neuropathy Limitation Scale (ONLS) Total Score | From Baseline to Month 15 | mFAS selection | Scores on the ONLS | MEAN |
| SECONDARY | Mean of Ten Meter Walking Test (10MWT) | From Baseline to Month 15 | mFAS selection | Seconds (s) | MEAN |
| SECONDARY | Mean of the CMTNS-v2 Sensory Score | From Baseline to Month 15 | mFAS selection | Scores on the CMTNS-v2 Sensory Score | MEAN |
| SECONDARY | Mean of the CMTNS-v2 Examination Score (CMTES-v2) | From Baseline to Month 15 | mFAS selection | Scores on the CMTES-v2 | MEAN |
| SECONDARY | Mean of the Results at the Nine-Hole Peg Test (9-HPT) | From Baseline to Month 15 | mFAS selection | Seconds (s) | MEAN |
| SECONDARY | Number of Subjects With at Least One TEAE | The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months) | FAS selection | participants | NUMBER |
| SECONDARY | Incidence of AE Leading to Withdrawal of Study Drug | The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months) | FAS selection | participants | NUMBER |
| SECONDARY | Incidence of SAEs | The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months). | FAS selection | participants | NUMBER |
| OTHER_PRE_SPECIFIED | Mean of the CMTNS-v2 Sensory Symptoms | From Baseline to Month 15 | mFAS selection | Scores on the CMTNS-v2 Sensory Symptoms | MEAN |
| OTHER_PRE_SPECIFIED | Plasma Concentrations of Baclofen at Trough and at 90 Min After Drug Intake | At Month 12 and Month 15 | PP selection LLOQ = 30 pg/mL The placebo arm has not been described for this endpoint. | pg/mL | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group90
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Neuropathy Limitation Scale (ONLS) Total Score | PXT3003 Dose 1 | Base | 3.33 | Standard Deviation 1.05 | Scores on the ONLS |
| Overall Neuropathy Limitation Scale (ONLS) Total Score | PXT3003 Dose 2 | Base | 3.05 | Standard Deviation 1.13 | Scores on the ONLS |
| Overall Neuropathy Limitation Scale (ONLS) Total Score | Placebo | Base | 3.23 | Standard Deviation 1.19 | Scores on the ONLS |
| Overall Neuropathy Limitation Scale (ONLS) Total Score | PXT3003 Dose 1 | Fin | 3.25 | Standard Deviation 1.00 | Scores on the ONLS |
| Overall Neuropathy Limitation Scale (ONLS) Total Score | PXT3003 Dose 2 | Fin | 2.82 | Standard Deviation 1.28 | Scores on the ONLS |
| Overall Neuropathy Limitation Scale (ONLS) Total Score | Placebo | Fin | 3.36 | Standard Deviation 1.16 | Scores on the ONLS |
| Mean of Ten Meter Walking Test (10MWT) | PXT3003 Dose 1 | Base | 6.93 | Standard Deviation 1.77 | Seconds (s) |
| Mean of Ten Meter Walking Test (10MWT) | PXT3003 Dose 2 | Base | 7.14 | Standard Deviation 1.77 | Seconds (s) |
| Mean of Ten Meter Walking Test (10MWT) | Placebo | Base | 7.28 | Standard Deviation 1.91 | Seconds (s) |
| Mean of Ten Meter Walking Test (10MWT) | PXT3003 Dose 1 | Fin | 6.47 | Standard Deviation 1.59 | Seconds (s) |
1 to 10 of 90Source ClinicalTrials.gov
See all 90 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | PXT3003 Dose 1 | 109 | |
| Overall Study | STARTED | PXT3003 Dose 2 | 113 | |
| Overall Study | STARTED | Placebo | 101 | |
| Overall Study | COMPLETED | PXT3003 Dose 1 | 85 | |
| Overall Study | COMPLETED | PXT3003 Dose 2 | 49 | |
| Overall Study | COMPLETED | Placebo | 80 | |
| Overall Study | NOT COMPLETED | PXT3003 Dose 1 | 24 | |
| Overall Study | NOT COMPLETED | PXT3003 Dose 2 | 64 | |
| Overall Study | NOT COMPLETED | Placebo | 21 |
Source ClinicalTrials.gov
Adverse events267
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 | Events |
|---|---|---|---|---|---|---|
| other | Hypotension | Vascular disorders | PXT3003 Dose 1 | 1 | 109 | 1 |
| other | Hypotension | Vascular disorders | PXT3003 Dose 2 | 1 | 113 | 1 |
| other | Hypotension | Vascular disorders | Placebo | 2 | 101 | 2 |
| other | Asthenia | General disorders | PXT3003 Dose 1 | 3 | 109 | 3 |
| other | Asthenia | General disorders | PXT3003 Dose 2 | 2 | 113 | 2 |
| other | Asthenia | General disorders | Placebo | 7 | 101 | 9 |
| other | Fatigue | General disorders | PXT3003 Dose 1 | 11 | 109 | 15 |
| other | Fatigue | General disorders | PXT3003 Dose 2 | 2 | 113 | 2 |
| other | Fatigue | General disorders | Placebo | 6 | 101 | 6 |
| other | Influenza like illness | General disorders | PXT3003 Dose 1 | 3 | 109 | 5 |
1 to 10 of 267Source ClinicalTrials.gov
See all 267 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PXT3003 Dose 1 | 0 | 109 |
| Total, all-cause mortality | PXT3003 Dose 2 | 0 | 113 |
| Total, all-cause mortality | Placebo | 0 | 101 |
| Total, other adverse events | PXT3003 Dose 1 | 89 | 109 |
| Total, other adverse events | PXT3003 Dose 2 | 87 | 113 |
| Total, other adverse events | Placebo | 83 | 101 |
| Total, serious adverse events | PXT3003 Dose 1 | 10 | 109 |
| Total, serious adverse events | PXT3003 Dose 2 | 3 | 113 |
| Total, serious adverse events | Placebo | 5 | 101 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
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.
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
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The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02579759
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