Charcot-Marie-Tooth Disease, Type IAPhase 3PLEO-CMT-FUNCT03023540
Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
A Phase 3 study testing PXT3003 in people with Charcot-Marie-Tooth Disease, Type IA, run by Pharnext S.C.A. at 23 sites in 7 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 17 to 67 years
- Conditions the study is for
- Charcot-Marie-Tooth Disease, Type IA
- What is being tested?
- PXT3003
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 187 participants
- Where
- 23 sitesin 7 countries
- Study dates
- 7 Mar 2017to 31 Dec 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 17 to 67 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients previously randomized to study CLN-PXT3003-02 under placebo and dose 1 and having completed 15 months of double-blind treatment in that study, including all procedures required at the Study Termination visit (V6) or
- Patients previously randomized to the initial study CLN-PXT3003-02 under dose 2, prematurely discontinued following sponsor decision, and having performed all procedures required at the Study Termination visit (V6)
- Patients whose V6 was performed within 4 weeks before entering the extension study or if not done must have a new baseline visit (VB)
- Female patients must agree to continue using an approved method of birth control throughout the extension study
- Patients must sign a written informed consent, specific to the extension study, in order to participate in this study. In case of minor children aged 16 to 18 years, both parent' and children's consents should be collected
- Patients must have completed 15 months of double-blind treatment in the primary study CLN-PXT3003-02, including all procedures required at the Study Termination visit (V6)
- Female patients must agree to continue using an approved method of birth control throughout the extension study
- Patients must sign a written informed consent, specific to the extension study, in order to participate in this study. In case of minor children aged 16 to 18 years, both parent' and children's consents should be collected
Who cannot join
- Any clinically significant change in health status that, in the opinion of the Investigator, would prevent the subject from participating in this study or successfully completing this study
- Any unauthorized concomitant treatments, as study CLN-PXT3003-02 (e.g. including but not limited to baclofen, naltrexone,sorbitol (pharmaceutical form), opioids, levothyroxin, and potentially neurotoxic drugs such as amiodarone, chloroquine, cancer drugs susceptible to induce peripheral neuropathy)
Trial locations and contacts
23 sites in 7 countries.
23 sites in 7 countries
United States9 sites
University of Michigan Health System
Ann Arbor, Michigan, 48109-5322
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Department of Neurology, McKnight Brain Institute
Gainesville, Florida, 32610
University of Kansas Medical Center
Kansas City, Kansas, 66160
Department of Neurology, Cedars-Sinai Medical Center
Los Angeles, California, 90048
Department of Neurology, University of Minnesota
Minneapolis, Minnesota, 55455
Peripheral Neuropathy Center, Neurological Institue Building, Columbia University Medical Center
New York, New York, 10032
Saint Luke's Rehabilitation Institute
Spokane, Washington, 99202-1330
Department of Neurology and Psichiatry, Saint Louis University
St Louis, Missouri, 63104-1027
France6 sites
Cntre de Reference des Maladies Neuromusculaires, Hopital Swynghedauwl, CHU Lille
Lille
Centre de Reference des Neuropathies Peripheriques Rare, Hopital Dupuytren, CHU Limoges
Limoges
Service de Neurologie et du Sommeil, CHU Lyon Sud
Lyon
Centre de Reference des Maladie Neuromusculaires, CHU la Timone
Marseille
Centre de Reference des Maladie Neuromusculaires, Hotel Dieu, CHU de Nantes
Nantes
Service de Neurologie, Hopital Kremlin Bicetre
Paris
Spain4 sites
Department of neurology, Hospital Univesitario de Bellvitge
Barcelona
Servicio de Neurologia, Hospital Universitario La Paz
Madrid
Centro de Diagnostico y Tratamiento, Hospital Universitario Virgen del Rocio
Seville
Servicio de Neurologia, Hospital Universitario i Politécnic La Fe
Valencia
Netherlands1 site
Departement of Neurology, Academic Medical Center
Amsterdam
United Kingdom1 site
Department of Neurology, Salford Royal NHS Foundation Trust
Salford, Manchester, M6 8HD
About this study
Official title: International, Multi-center, Open Label, Follow-up Extension Study Assessing the Long-term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
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).
Read the detailed description
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).
- Conditions
- Charcot-Marie-Tooth Disease, Type IA
- Being tested
- PXT3003
- Keywords
- Charcot Marie Tooth Type 1A, Peripheral neuropathy, PXT3003
- Medical subject headings
- Charcot-Marie-Tooth Disease, Peripheral Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PXT3003Drug
Liquid oral solution, twice 5 mL (Dose 1) bid
| Study group | Role | What they receive |
|---|---|---|
| PXT3003 dose 1 | Active comparator | 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 Receives: Drug: PXT3003 |
| PXT3003 dose 2 | Active comparator | 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) Receives: Drug: PXT3003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 9 or 24 months
Secondary outcomes (12)
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
Measured: 9 or 24 months
Incidence of adverse events leading to withdrawal of study drug
Incidence of adverse events leading to withdrawal of study drug
Measured: 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
Measured: 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
Measured: 9 or 24 months
Nine-hole Peg Test (9-HPT)
Nine-hole Peg Test (9-HPT)
Measured: 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)
Measured: 9 or 24 months
Time to walk 10 meters
Time to walk 10 meters
Measured: 9 or 24 months
Compound Muscle Action Potential (CMAP) on ulnar nerve
Compound Muscle Action Potential (CMAP) on ulnar nerve
Measured: 9 or 24 months
Sensory Nerve Action Potential (SNAP) on radial nerve
Sensory Nerve Action Potential (SNAP) on radial nerve
Measured: 9 or 24 months
Nerve conduction velocity (NCV)
Nerve conduction velocity (NCV)
Measured: 9 or 24 months
Quality of Life (EQ-5D)
Quality of Life (EQ-5D)
Measured: 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)
Measured: 9 or 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2017 (estimated)
- Study start7 Mar 2017
- Primary completion31 Dec 2024 (estimated)
- Study completion31 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Charcot-Marie-Tooth Disease, Type IAStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Patients who completed the pivotal phase III study CLN-PXT3003-02 are allowed to continue in this open-label study. In Period 1, patients who were randomly assigned to placebo or low-dose PXT3003 in pivotal phase III study were assigned to receive low-dose PXT3003 (5 mL), whereas patients assigned to high-dose PXT3003 continued on that dose (receiving the high-dose PXT3003 (5 mL) or twice the low-dose for each administration (i.e. 10 mL)). The primary objective was to assess safety and tolerability for prolonged exposure to PXT3003, and to evaluate the effect on disability in patients with mild to moderate CMT1A. In Period 2, all patients are allowed to continue in an open-label fashion to receive high-dose PXT3003 (receiving twice the low-dose for each administration (i.e. 10 mL). The objective is to mainly assess the safety and tolerability in the aforementioned patient population.
- Masking
- None (open label)
- Enrolment
- 187 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pharnext S.C.A.
- Sponsor type
- University, hospital or other
- Collaborators
- Synteract HCR (Syneos Health), Premier Research, Greenphire, Theradis, Amarex, Eurofins Optimed
- Responsible party
- Sponsor
- Investigators
- Shahram Attarian, MD, Principal investigator, CHU la Timone, Marseille, FranceTeresa Sevilla, MD, Principal investigator, Hospital Universitario i Politécnico La F, Valencia, SpainMarianne de Visser, MD, Principal investigator, Academic Medical Center, Amsterdam, NetherlandsMark Roberts, MD, Principal investigator, Selor Royal NHS Foundation Trust, Manchester, UKFlorian Thomas, MD PhD, Principal investigator, Seton Hall-Hackensack-Meridian School of Medicine, Hackensack, USA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03023540
- Sponsor's protocol ID
- CLN-PXT3003-03
- Other IDs
- 2015-002379-81, 2015
- Acronym
- PLEO-CMT-FU
- Status last verified
- Feb 2024
- First submitted
- 26 Dec 2016
- Last updated
- 20 Feb 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- International, Multi-center, Open Label, Follow-up Extension Study Assessing the Long-term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
- ClinicalTrials.gov ID
- NCT03023540
- Acronym / sponsor ID
- PLEO-CMT-FU / CLN-PXT3003-03
- Conditions
- Charcot-Marie-Tooth Disease, Type IA
- Keywords
- Charcot Marie Tooth Type 1A, Peripheral neuropathy, PXT3003
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 187 participants (actual)
- Lead sponsor
- Pharnext S.C.A.University, hospital or other
- Collaborators
- Synteract HCR (Syneos Health), Premier Research, Greenphire, Theradis, Amarex, Eurofins Optimed
- Study start
- 7 Mar 2017 (actual)
- Primary completion
- 31 Dec 2024 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 18 Jan 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Patients who completed the pivotal phase III study CLN-PXT3003-02 are allowed to continue in this open-label study. In Period 1, patients who were randomly assigned to placebo or low-dose PXT3003 in pivotal phase III study were assigned to receive low-dose PXT3003 (5 mL), whereas patients assigned to high-dose PXT3003 continued on that dose (receiving the high-dose PXT3003 (5 mL) or twice the low-dose for each administration (i.e. 10 mL)). The primary objective was to assess safety and tolerability for prolonged exposure to PXT3003, and to evaluate the effect on disability in patients with mild to moderate CMT1A. In Period 2, all patients are allowed to continue in an open-label fashion to receive high-dose PXT3003 (receiving twice the low-dose for each administration (i.e. 10 mL). The objective is to mainly assess the safety and tolerability in the aforementioned patient population.
| Arm | Type | Receives |
|---|---|---|
| PXT3003 dose 1 | Active comparator | Drug: PXT3003Period 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 |
| PXT3003 dose 2 | Active comparator | Drug: PXT3003Period 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) |
Who may qualify
- Ages
- 17 to 67 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Patients previously randomized to study CLN-PXT3003-02 under placebo and dose 1 and having completed 15 months of double-blind treatment in that study, including all procedures required at the Study Termination visit (V6) or
- Patients previously randomized to the initial study CLN-PXT3003-02 under dose 2, prematurely discontinued following sponsor decision, and having performed all procedures required at the Study Termination visit (V6)
- Patients whose V6 was performed within 4 weeks before entering the extension study or if not done must have a new baseline visit (VB)
- Female patients must agree to continue using an approved method of birth control throughout the extension study
- Patients must sign a written informed consent, specific to the extension study, in order to participate in this study. In case of minor children aged 16 to 18 years, both parent' and children's consents should be collected
Key exclusion criteria (2 listed)
- Any clinically significant change in health status that, in the opinion of the Investigator, would prevent the subject from participating in this study or successfully completing this study
- Any unauthorized concomitant treatments, as study CLN-PXT3003-02 (e.g. including but not limited to baclofen, naltrexone,sorbitol (pharmaceutical form), opioids, levothyroxin, and potentially neurotoxic drugs such as amiodarone, chloroquine, cancer drugs susceptible to induce peripheral neuropathy)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PXT3003Liquid oral solution, twice 5 mL (Dose 1) bid | Drug | PXT3003 dose 1, PXT3003 dose 2 |
Primary outcome
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 treatment-emergent adverse events (TEAEs) related to PXT3003 during the follow-up in patients with CMT1A
12 secondary outcomes
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
Time frame: 9 or 24 months
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
Time frame: 9 or 24 months
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)
Time frame: 9 or 24 months
Time to walk 10 meters
Time frame: 9 or 24 months
Compound Muscle Action Potential (CMAP) on ulnar nerve
Time frame: 9 or 24 months
Sensory Nerve Action Potential (SNAP) on radial nerve
Time frame: 9 or 24 months
Nerve conduction velocity (NCV)
Time frame: 9 or 24 months
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)
Time frame: 9 or 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shahram Attarian, MD principal investigator
CHU la Timone, Marseille, France
Teresa Sevilla, MD principal investigator
Hospital Universitario i Politécnico La F, Valencia, Spain
Marianne de Visser, MD principal investigator
Academic Medical Center, Amsterdam, Netherlands
23 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing PXT3003 in people with Charcot-Marie-Tooth Disease, Type IA, run by Pharnext S.C.A. at 23 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, France, Spain, Belgium, Canada and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs) related to PXT3003 during the follow-up in patients with CMT1A (time frame: 9 or 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2024.