A Phase 3 interventional study of PXT3003 in Charcot-Marie-Tooth Disease, Type IA, sponsored by Pharnext S.C.A.. Active, not recruiting at 23 sites in 7 countries. Open to participants aged 17 Years to 67 Years. Per ClinicalTrials.gov, last updated 2024-02-20.
Sponsored by Pharnext S.C.A. · Phase 3, Interventional, and Treatment
All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary study CLN-PXT3003-02, i.e. treatment with PXT3003 or placebo, are eligible to continue in the extension study CLN-PXT3003-03.
Period 1: Patients randomised to PXT3003 dose 1 or placebo in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL). Patients randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL).
Period 2: All patients continue on twice dose 1 (2X5mL).
PXT3003 is a rational design, fixed combination of low-dose (RS) baclofen, naltrexone hydrochloride and D-sorbitol. The use of PXT3003 in a multicenter, randomised, placebo controlled phase II study (CLN-PXT3003-01) was well-tolerated and safe in patients with CMT1A for the three dose-levels investigated (Attarian et al., 2014). The intermediate and high dose of PXT3003 demonstrated an improvement of disability in this patient population.
Subsequently a multicenter, randomised, placebo controlled phase III study (CLN-PXT3003-02) to assess the efficacy and safety of PXT3003 in the treatment of patients with CMT1A was initiated in December 2015. In March 2017 the first patients completed the 15-month treatment with PXT3003 and rolled over into the extension study CLN-PXT3003-03.
During Period 1 (9 months), patients that were randomised to PXT3003 dose 1 or placebo in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL). Patients randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued in the extension study on on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL). During Period 2, all patients continue on twice dose 1 (2X5mL).
113 studies on the registry are indexed under Charcot-Marie-Tooth Disease; 38 are open to participants now.
This study's enrollment of 187 is above the median of 41 across 62 interventional studies indexed under Charcot-Marie-Tooth Disease.
Browse Charcot-Marie-Tooth Disease studies →Pharnext S.C.A. is the lead sponsor of 6 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria after September 18th 2017:
Inclusion Criteria until September 18th 2017:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Period 1, PXT3003 : Liquid oral solution (0.6 mg/mL baclofen, 0.07 mg/mL naltrexone HCl and 210 mg/mL D-sorbitol), 5 mL bid (taken morning and evening with food) for 9 consecutive months
Drug: PXT3003
Period 1, PXT3003: Liquid oral solution (1.2 mg/mL baclofen, 0.14 mg/mL naltrexone HCl and 420 mg/mL D-sorbitol), 5 mL bid (taken morning and evening with food) for 9 consecutive months Period 2, PXT3003: Liquid oral solution (0.6 mg/mL baclofen, 0.07 mg/mL naltrexone HCl and 210 mg/mL D-sorbitol), 10 mL bid (taken morning and evening with food)
Drug: PXT3003
Liquid oral solution, twice 5 mL (Dose 1) bid
Incidence of treatment-emergent adverse events (TEAEs) related to PXT3003 during the follow-up in patients with CMT1A
Incidence of treatment-emergent adverse events (TEAEs) related to PXT3003 during the follow-up in patients with CMT1A
Time frame: 9 or 24 months
Incidence of all TEAEs and their evaluation of type/nature, severity/intensity, seriousness, duration, relationship to study drug, and outcome
Incidence of all TEAEs and their evaluation of type/nature, severity/intensity, seriousness, duration, relationship to study drug, and outcome
Time frame: 9 or 24 months
Incidence of adverse events leading to withdrawal of study drug
Incidence of adverse events leading to withdrawal of study drug
Time frame: 9 or 24 months
Overall Neuropathy Limitation Scale (ONLS) score, and its arm and leg sub-items
Overall Neuropathy Limitation Scale (ONLS) score, and its arm and leg sub-items
Time frame: 9 or 24 months
Charcot-Marie-Tooth Neuropathy Score - version 2 (CMTNS-V2), and its sub-items
Charcot-Marie-Tooth Neuropathy Score - version 2 (CMTNS-V2), and its sub-items
Time frame: 9 or 24 months
Nine-hole Peg Test (9-HPT)
Nine-hole Peg Test (9-HPT)
Time frame: 9 or 24 months
Quantified Muscular Testing (QMT) by hand grip and foot dorsiflexion dynamometry (mean of both sides)
Quantified Muscular Testing (QMT) by hand grip and foot dorsiflexion dynamometry (mean of both sides)
Time frame: 9 or 24 months
Time to walk 10 meters
Time to walk 10 meters
Time frame: 9 or 24 months
Compound Muscle Action Potential (CMAP) on ulnar nerve
Compound Muscle Action Potential (CMAP) on ulnar nerve
Time frame: 9 or 24 months
Sensory Nerve Action Potential (SNAP) on radial nerve
Sensory Nerve Action Potential (SNAP) on radial nerve
Time frame: 9 or 24 months
Nerve conduction velocity (NCV)
Nerve conduction velocity (NCV)
Time frame: 9 or 24 months
Quality of Life (EQ-5D)
Quality of Life (EQ-5D)
Time frame: 9 or 24 months
Visual analog scale on self-assessment of individualized main impairment in daily activities (defined at baseline with the patient)
Visual analog scale on self-assessment of individualized main impairment in daily activities (defined at baseline with the patient)
Time frame: 9 or 24 months
Through plasma concentration of PXT3003
Measurement of baclofen, naltrexone and 6-beta-naltrexone plasma concentration at the through
Time frame: at month 6 and 9
Peak plasma concentration of PXT3003
Measurement of baclofen, naltrexone and 6-beta-naltrexone plasma concentration at the through
Time frame: at month 6 and 9
Plan to share: No
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Studies of the drug tested here: PXT3003
Pharnext S.C.A.