Daily oral vorinostat for girls and young women with Rett syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a single-blinded, placebo-controlled, adapted N-of-1 Phase 1 trial testing once-daily oral vorinostat with each participant using a 4-week placebo baseline and then sequential 8-week dosing periods at 80 mg/m2/day and 160 mg/m2/day. Key outcomes include safety and tolerability, changes in clinical and laboratory measures that could indicate benefit, and transcriptome (mRNA) responses. Participants are females aged 6–21 with typical Rett syndrome and a confirmed pathogenic MECP2 mutation who are clinically stable and post-regression. Results will inform dosing, outcome selection, and statistical planning for future trials.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Female subjects ≥6 years of age and ≤ 21 years of age at time of screening
2. Has typical Rett Syndrome (RTT), based on diagnostic criteria for RTT described in Neul, et.al., 2010
3. Has documented, disease causing mutation in the MeCP2 gene
4. 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
5. 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
6. Has had a stable pattern of seizure activity for 4 weeks before screening
7. Can swallow medication or can take it by gastrostomy tube
8. Can wear actigraphy data logging device on wrist or ankle
9. 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)
10. 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 Criteria:

1. 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)
2. Has major surgery planned during the study period
3. Pregnant or nursing women
4. Has a history of brain injury, stroke, other cerebrovascular disease or hypoxic-ischemic encephalopathy
5. Has clinically significant abnormal vital signs at screening or baseline
6. Has an abnormal ECG at screening, including clinically significant QT prolongation
7. Has a clinically significant abnormal laboratory value at screening
8. Liver disease or transaminase levels \> 1.5 times the upper limit of the normal range as determined during screening
9. Has a history of malignancy of any organ system within the past 5 years before screening
10. Is participating in or has participated in another clinical trial within 30 days prior to the screening visit
11. Has been treated with growth hormone, IGF-1, or insulin within 12 weeks of baseline
12. Is taking anticoagulant therapy or other HDAC inhibitors
13. Has had any change to their medication or non-pharmacological treatment within 4 weeks prior to the baseline visit
14. Life expectancy of less than 12 months.
15. Has a history of alcoholism or drug/chemical abuse within 2 years before screening.
16. In the investigator's opinion, is inappropriate for this study for any reason

Where it's running

• Grupo de Investigación Clínica PECET (GIC-PECET)
  Medellín, Colombia Recruiting

Study contacts

• Study coordinator: Neal I Muni, M.D., MSPH
• Email: neal.muni@unravel.bio
• Phone: +1 857-404-8252

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Auditory EEG and behavioral testing for people with Rett syndrome

  This project will record noninvasive EEG while people with Rett syndrome and matched controls listen to simple sounds to see how their brains process sound.

  Recruiting University of Rochester · Rochester, New York · NCT07569445

• Fenfluramine HCl for Rett Syndrome in a Phase 3 study

  This study tests whether fenfluramine hydrochloride helps people with Rett syndrome when compared to a placebo.

  Recruiting at 40 sites Phase 3 UCB Pharma · Little Rock, Arkansas and 39 other locations · NCT07503444

• Efficacy of Bionetide for treating Rett syndrome in females

  This study is testing if an oral medication called Bionetide can help improve behavior and communication in girls and young women aged 5 to 20 with Rett syndrome.

  Recruiting at 18 sites Phase 3 Biomed Industries, Inc. · Phoenix, Arizona and 17 other locations · NCT06840496

• Assessing cognitive function in Rett syndrome patients receiving trofinetide treatment

  This study is testing how well eye-tracking tasks can measure cognitive abilities in people with Rett syndrome who are starting treatment with trofinetide.

  Recruiting University of Minnesota · Minneapolis, Minnesota · NCT06705816

• Identifying biomarkers in Rett Syndrome

  This study is trying to find specific brain signals in girls with Rett Syndrome to see how they differ from girls without the condition and how these signals relate to the severity of their symptoms over time.

  Recruiting at 6 sites Children's Hospital of Philadelphia · Los Angeles, California and 5 other locations · NCT05932589

• Gene editing to correct common MECP2 mutations in Rett syndrome

  This project will test CRISPR/Cas9 delivered with AAV to correct common MECP2 mutations in females with Rett syndrome using lab and animal work.

  Recruiting University of Siena · Siena, Siena · NCT05740761

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.