Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
Public ClinicalTrials.gov record NCT03023540. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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).
Study identification
- NCT ID
- NCT03023540
- Recruitment status
- Not listed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 187 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 17 Years to 67 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 7, 2017 (Actual)
- Primary completion
- Dec 31, 2024 (Estimated)
- Completion
- Dec 31, 2024 (Estimated)
- Last update posted
- Feb 20, 2024
2017 – 2024
United States locations
- U.S. sites
- 9
- U.S. states
- 9
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Department of Neurology, Cedars-Sinai Medical Center | Los Angeles | California | 90048 | — |
| Department of Neurology, McKnight Brain Institute | Gainesville | Florida | 32610 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| Brigham and Women's Hospital | Boston | Massachusetts | 02115 | — |
| University of Michigan Health System | Ann Arbor | Michigan | 48109-5322 | — |
| Department of Neurology, University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Department of Neurology and Psichiatry, Saint Louis University | St Louis | Missouri | 63104-1027 | — |
| Peripheral Neuropathy Center, Neurological Institue Building, Columbia University Medical Center | New York | New York | 10032 | — |
| Saint Luke's Rehabilitation Institute | Spokane | Washington | 99202-1330 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 14 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03023540, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 20, 2024 · Synced Oct 28, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03023540 live on ClinicalTrials.gov.