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

NCT07150013 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2026-05-01

No results posted yet for this study

Summary

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

Interventions

DRUG

Vorinostat (SAHA)

oral suspension

DRUG

Placebo

placebo

Sponsors & Collaborators

  • Unravel Biosciences, Inc.

    lead INDUSTRY

Principal Investigators

  • Neal I Muni, M.D., MSPH · Unravel Biosciences, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
CROSSOVER

Eligibility

Min Age
6 Years
Max Age
21 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-03-15
Primary Completion
2026-12-31
Completion
2027-03-15

Countries

  • Colombia

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07150013 on ClinicalTrials.gov