Clinical trials: Charcot-Marie-Tooth Type 1A
CMT1A
Ulipristal Acetate In Disease Charcot-Marie-Tooth Type of 1A
The disease Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited peripheral neuropathy, for which no treatment has proved its effectiveness.
- Trial name
- UPACOMT (NCT02600286)
- Testing
- EllaOne
- Where
- 5 sites in France
- Volunteers
- 23 people
- Sponsor
- University Hospital, Strasbourg, France
Charcot-Marie-Tooth Neuropathy Type 1A
Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A
This clinical trial is an open-label one-time injection dose study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into muscles in both legs in CMT1A subjects with PMP22 gene duplication.
- Trial name
- NCT03520751
- Testing
- scAAV1.tMCK.NTF3
- Where
- United States
- Sponsor
- Nationwide Children's Hospital
Charcot-Marie-Tooth Disease, Type IA
Assessing 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.
- Trial name
- PLEO-CMT-FU (NCT03023540)
- Testing
- PXT3003
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 187 people
- Sponsor
- Pharnext S.C.A.