CLN-PXT3003-06

Summary

The purpose of this study is to evaluate the effectiveness of an investigational drug called PXT3003 for the treatment of Charcot-Marie-Tooth type 1A (CMT1A disease). Researchers will compare PXT3003 to placebo (inactive substance) on the disease progression and symptoms of CMT1A, as well as determine whether the drug is tolerable and safe for patients with CMT1A. CMT1A is caused by inherited mutations (changes) in genes that cause the peripheral nerves to become damaged, which leads to muscle weakness, numbness, and sensory loss. Participants will be randomly assigned to one of two treatment groups. Group 1 will receive PXT3003 twice daily, and Group 2 will receive a placebo (twice daily).


Inclusion Criteria

  • Double-blind Treatment Period
  • Male and non-pregnant female subjects, aged 16 to 65 years with a genetically proven diagnosis of CMT1A. Notes: a) A report of a genetic test confirming PMP22 duplication and therefore a diagnosis of CMT1A must be available in the subject's record at the clinical site. b) In the absence of a report of a genetic test confirming PMP22 duplication in the subject's medical record, a confirmatory genetic test must be conducted via the central laboratory as part of Screening. c) In the exceptional case wherein subject was randomized into the study without meeting(a) or (b), an unscheduled confirmatory genetic test will be performed. In the event of a negative genetic test result, the subject will be withdrawn from the study.
  • Able to provide written informed consent/assent and comply with study procedures.
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Study Location(s)

Beverly

More about this Clinical Trial

Full Title

A Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A

Details
Disease Type/Condition

Charcot-Marie-Tooth (CMT)

Principal Investigator

Lewis, Richard

Age Group

Adult

Phase

III

IRB Number

STUDY00001460

ClinicalTrials.gov ID

NCT04762758

Key Eligibility
ClinicalTrials.gov

Contact
Phone
310-423-6252
Email
clinicaltrials@cshs.org
Want to join the study or
learn more?

Details
Disease Type/Condition

Charcot-Marie-Tooth (CMT)

Principal Investigator

Lewis, Richard

Age Group

Adult

Phase

III

IRB Number

CLN-PXT3003-06

ClinicalTrials.gov ID

NCT04762758

Key Eligibility
ClinicalTrials.gov

Contact
Phone
310-423-6252
Email
clinicaltrials@cshs.org
Want to join the study or
learn more?