Actively Recruiting
Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome Using an "N of 1" Study Design
Led by Unravel Biosciences, Inc. · Updated on 2026-05-01
15
Participants Needed
1
Research Sites
10 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and potential effects of the drug vorinostat in treating Rett syndrome, a neurodevelopmental disorder seen in females. This exploratory, placebo-controlled trial uses a unique "N of 1" design where each patient serves as her own control. The study aims to confirm vorinostat's tolerability at two dose levels and to assess changes in clinical symptoms and gene expression that may suggest benefits. The trial is sponsored by Unravel Biosciences and builds on promising preclinical findings in models of Rett syndrome. Participants will receive oral vorinostat in two escalating doses: 80mg/m2/day followed by 160mg/m2/day, each for 8 weeks, after a 4-week placebo baseline period. The study is single-blinded, so patients and caregivers will not know when active drug or placebo is given. Up to 15 female patients aged 6 to 21 years with typical Rett syndrome and specific genetic mutations will be enrolled. The trial involves daily dosing and data collection over about 20 weeks. During the study, participants will be assessed through clinical scales measuring symptom severity, behavior, motor function, sleep, gastrointestinal symptoms, and seizure frequency. Laboratory tests including RNA sequencing will analyze changes in gene expression. Safety will be closely monitored by tracking adverse events and treatment discontinuations. The study duration includes baseline, dosing periods, and follow-up, with data used to guide future research and treatment development for Rett syndrome.
CONDITIONS
Brief 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 defined diagnostic criteria
- Documented disease-causing mutation in the MeCP2 gene
- In post-regression phase with stable ambulation, hand function, speech, and communication for 4 months prior to screening
- On stable medication or non-drug treatment for at least 4 weeks before baseline; if on trofinetide, stable dose for 6 months
- Stable seizure pattern for 4 weeks before screening
- Able to swallow medication or receive it via gastrostomy tube
- Able to wear an actigraphy device on wrist or ankle
- If able to bear children, agrees to use effective contraception during study and 3 months after last dose
- Subject or legally authorized representative can provide informed consent and complete caregiver assessments in study language
You will not qualify if you...
- Presence of other significant medical conditions unrelated to MeCP2 mutation (e.g., diabetes, heart, kidney, lung disease, blood disorders, cancer)
- Planned major surgery during study period
- Pregnant or nursing women
- History of brain injury, stroke, cerebrovascular disease, or hypoxic-ischemic encephalopathy
- Clinically significant abnormal vital signs at screening or baseline
- Abnormal ECG including significant QT prolongation
- Clinically significant abnormal lab values at screening
- Liver disease or elevated liver enzymes above 1.5 times normal
- History of cancer within 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
- Medication or treatment changes within 4 weeks before baseline
- Life expectancy less than 12 months
- History of alcoholism or substance abuse within 2 years
- Considered inappropriate for study participation by investigator
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 4 weeks
Participants receive a placebo treatment daily for 4 weeks to establish baseline data for comparison.
Weekly visits for 4 weeks
Duration - 8 weeks
Participants receive 80mg/m2/day dose of vorinostat orally once daily for 8 weeks.
Weekly visits for 8 weeks
Duration - 8 weeks
Participants receive 160mg/m2/day dose of vorinostat orally once daily for 8 weeks following the low dose phase.
Weekly visits for 8 weeks
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
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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