Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
NCT ID: NCT03023540
Last Updated: 2024-02-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
187 participants
INTERVENTIONAL
2017-03-07
2024-12-31
Brief Summary
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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).
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Detailed Description
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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
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Study Design
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NON_RANDOMIZED
PARALLEL
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.
TREATMENT
NONE
Study Groups
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PXT3003 dose 1
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
PXT3003
Liquid oral solution, twice 5 mL (Dose 1) bid
PXT3003 dose 2
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)
PXT3003
Liquid oral solution, twice 5 mL (Dose 1) bid
Interventions
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PXT3003
Liquid oral solution, twice 5 mL (Dose 1) bid
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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)
17 Years
67 Years
ALL
No
Sponsors
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Synteract HCR (Syneos Health)
UNKNOWN
Premier Research Group plc
UNKNOWN
Greenphire
UNKNOWN
Theradis
UNKNOWN
Amarex
UNKNOWN
Eurofins Optimed
INDUSTRY
Pharnext S.C.A.
OTHER
Responsible Party
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Principal Investigators
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Shahram Attarian, MD
Role: PRINCIPAL_INVESTIGATOR
CHU la Timone, Marseille, France
Teresa Sevilla, MD
Role: PRINCIPAL_INVESTIGATOR
Hospital Universitario i Politécnico La F, Valencia, Spain
Marianne de Visser, MD
Role: PRINCIPAL_INVESTIGATOR
Academic Medical Center, Amsterdam, Netherlands
Mark Roberts, MD
Role: PRINCIPAL_INVESTIGATOR
Selor Royal NHS Foundation Trust, Manchester, UK
Florian Thomas, MD PhD
Role: PRINCIPAL_INVESTIGATOR
Seton Hall-Hackensack-Meridian School of Medicine, Hackensack, USA
Locations
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Department of Neurology, Cedars-Sinai Medical Center
Los Angeles, California, United States
Department of Neurology, McKnight Brain Institute
Gainesville, Florida, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
University of Michigan Health System
Ann Arbor, Michigan, United States
Department of Neurology, University of Minnesota
Minneapolis, Minnesota, United States
Department of Neurology and Psichiatry, Saint Louis University
St Louis, Missouri, United States
Peripheral Neuropathy Center, Neurological Institue Building, Columbia University Medical Center
New York, New York, United States
Saint Luke's Rehabilitation Institute
Spokane, Washington, United States
Departement of Neurology, UZ Leuven
Leuven, , Belgium
University Hospital of Quebec
Québec, Quebec, Canada
Cntre de Reference des Maladies Neuromusculaires, Hopital Swynghedauwl, CHU Lille
Lille, , France
Centre de Reference des Neuropathies Peripheriques Rare, Hopital Dupuytren, CHU Limoges
Limoges, , France
Service de Neurologie et du Sommeil, CHU Lyon Sud
Lyon, , France
Centre de Reference des Maladie Neuromusculaires, CHU la Timone
Marseille, , France
Centre de Reference des Maladie Neuromusculaires, Hotel Dieu, CHU de Nantes
Nantes, , France
Service de Neurologie, Hopital Kremlin Bicetre
Paris, , France
Departement of Neurology, Academic Medical Center
Amsterdam, , Netherlands
Department of neurology, Hospital Univesitario de Bellvitge
Barcelona, , Spain
Servicio de Neurologia, Hospital Universitario La Paz
Madrid, , Spain
Centro de Diagnostico y Tratamiento, Hospital Universitario Virgen del Rocio
Seville, , Spain
Servicio de Neurologia, Hospital Universitario i Politécnic La Fe
Valencia, , Spain
Department of Neurology, Salford Royal NHS Foundation Trust
Salford, Manchester, United Kingdom
Countries
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Other Identifiers
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2015-002379-81
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CLN-PXT3003-03
Identifier Type: -
Identifier Source: org_study_id
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