Actively Recruiting
Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome
Led by Unravel Biosciences, Inc. · Updated on 2026-05-01
15
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Female subjects aged 6 to 21 years at screening
- Diagnosed with typical Rett Syndrome based on established criteria
- Documented disease-causing mutation in the MeCP2 gene
- In post-regression phase with no decline in movement, hand use, speech, or communication in the 4 months before screening
- On a stable medication or non-drug treatment regimen for at least 4 weeks before baseline; if on trofinetide, stable dose for 6 months before screening
- Stable seizure activity pattern for 4 weeks before screening
- Able to swallow medication or use gastrostomy tube
- Able to wear an actigraphy device on wrist or ankle
- If able to bear children, agrees to use highly effective contraception during the study and 3 months after last dose
- Participant or legal representative can provide informed consent and complete caregiver assessments in the study language
You will not qualify if you...
- Having other significant medical conditions not related to MeCP2 mutation (e.g., diabetes, heart, kidney, lung diseases, blood disorders, cancer)
- Planned major surgery during study period
- Pregnant or nursing
- History of brain injury, stroke, cerebrovascular disease, or hypoxic-ischemic encephalopathy
- Abnormal vital signs or ECG at screening, including significant QT prolongation
- Clinically significant abnormal lab values at screening
- Liver disease or elevated liver enzymes above 1.5 times normal
- History of any malignancy in the past 5 years
- Participation in another clinical trial within 30 days before screening
- Treatment with growth hormone, IGF-1, or insulin within 12 weeks before baseline
- Taking anticoagulants or other HDAC inhibitors
- Any medication or treatment changes within 4 weeks before baseline
- Life expectancy under 12 months
- History of alcoholism or drug abuse in past 2 years
- Considered inappropriate for the study by the investigator for any reason
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Grupo de Investigación Clínica PECET (GIC-PECET)
Medellín, Colombia
Actively Recruiting
Research Team
N
Neal I Muni, M.D., MSPH
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
NON_RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
3
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