Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome

Recruiting Phase 1 Interventional Study
Rett Syndrome
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At a Glance
Age
6 – 21
Sex
Female
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
15 (estimated)
Sponsor
Unravel Biosciences, Inc. · Industry
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About This Trial
The RETT REVOLUTION trial is a placebo-controlled, single-blinded, exploratory study with patients serving as their own control ("N of 1" trial design) where the safety and efficacy of vorinostat in the treatment of Rett syndrome will be evaluated. Each patient will be self-controlled in an adapted N-of-1 study design methodology by using a 4-week placebo baseline. Vorinostat dose escalation will …
Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Female subjects ≥6 years of age and ≤ 21 years of age at time of screening 2. Has typical Rett Syndrome (RTT), based on diagnostic criteria for RTT described in Neul, et.al., 2010 3. Has documented, disease causing mutation in the MeCP2 gene 4. At time of screening, is in the…
Contacts

Neal I Muni, M.D., MSPH

+1 857-404-8252

neal.muni@unravel.bio

CONTACT