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

Sponsor
Unravel Biosciences, Inc.
Study ID
NCT07150013
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
FEMALE
Age
6 Years - 21 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

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 occur every 8 weeks of daily dosing: placebo, 80mg/m2/day, 160mg/m2/day. Key study objectives will include: * To confirm the safety and tolerability of oral vorinostat 80mg/m2/day and 160mg/ m2/day dose levels when administered to typical Rett patients * To identify the nature and magnitude of treatment response to vorinostat, as measured by changes in clinical and laboratory parameters indicative of trend towards benefit, as well as changes in mRNA expression (transcriptome response) * Provide a data-driven justification for future study design and statistical analysis plan for subsequent clinical studies assessing safety and efficacy of vorinostat in Rett syndrome

Key Dates

Start date
Mar 15, 2026
Status verified
Apr 2026
Primary completion
Dec 31, 2026
Completion
Mar 15, 2027

Study Design

Enrollment
15 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Experimental: Low dose interventional arm
    80mg/m2/day dose vorinostat
  • Experimental: High dose interventional arm
    160mg/m2/day dose vorinostat
  • Placebo Comparator: Placebo
    placebo

Primary Outcome Measure

Frequency of treatment-related adverse events [ Time Frame: 20 weeks ]

Central Contacts

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