Status:
NOT_YET_RECRUITING
Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome
Lead Sponsor:
Unravel Biosciences, Inc.
Conditions:
Rett Syndrome
Eligibility:
FEMALE
6-21 years
Phase:
PHASE1
Brief 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 t...
Detailed Description
Unravel Biosciences, Inc. ("Unravel") proposes to develop an orally administered, once daily novel treatment for the orphan drug indication of Rett syndrome. Unravel has utilized its proprietary drug ...
Eligibility Criteria
Inclusion
- Female subjects ≥6 years of age and ≤ 21 years of age at time of screening
- Has typical Rett Syndrome (RTT), based on diagnostic criteria for RTT described in Neul, et.al., 2010
- Has documented, disease causing mutation in the MeCP2 gene
- At time of screening, is in the post-regression phase with no degradation of ambulation, hand function, speech or communication skills in the 4 months prior to screening
- Has been on a stable regimen of medication or non-pharmacological treatment for at least 4 weeks prior to the baseline visit; if currently taking trofinetide (Daybue), currently on stable dose for the previous 6 months before screening visit
- Has had a stable pattern of seizure activity for 4 weeks before screening
- Can swallow medication or can take it by gastrostomy tube
- Can wear actigraphy data logging device on wrist or ankle
- If of childbearing potential, must agree to use a highly effective method of contraception during the study and for 3 months after the last study drug administration (i.e., abstinence from sexual activity, hormonal contraceptives associated with inhibition of ovulation, intrauterine device, intrauterine hormone-releasing system)
- Subjects or their legally authorized representative must be able to provide an informed consent and have sufficient language skill to complete caregiver assessments in the language in which the study assessments are provided
Exclusion
- Has another clinically significant medical condition other than those related to MeCP2 mutation (e.g. diabetes mellitus, cardiovascular disease, renal disease, respiratory disease, hematological abnormalities, malignancy)
- Has major surgery planned during the study period
- Pregnant or nursing women
- Has a history of brain injury, stroke, other cerebrovascular disease or hypoxic-ischemic encephalopathy
- Has clinically significant abnormal vital signs at screening or baseline
- Has an abnormal ECG at screening, including clinically significant QT prolongation
- Has a clinically significant abnormal laboratory value at screening
- Liver disease or transaminase levels \> 1.5 times the upper limit of the normal range as determined during screening
- Has a history of malignancy of any organ system within the past 5 years before screening
- Is participating in or has participated in another clinical trial within 30 days prior to the screening visit
- Has been treated with growth hormone, IGF-1, or insulin within 12 weeks of baseline
- Is taking anticoagulant therapy or other HDAC inhibitors
- Has had any change to their medication or non-pharmacological treatment within 4 weeks prior to the baseline visit
- Life expectancy of less than 12 months.
- Has a history of alcoholism or drug/chemical abuse within 2 years before screening.
- In the investigator's opinion, is inappropriate for this study for any reason
Key Trial Info
Start Date :
October 15 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 15 2026
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT07150013
Start Date
October 15 2025
End Date
October 15 2026
Last Update
September 2 2025
Active Locations (1)
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1
Grupo de Investigación Clínica PECET (GIC-PECET)
Medellín, Colombia