Trial · NCT07150026
An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
NCT07150026, Phase 1, Recruiting, Unravel Biosciences, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome. Trials, record NCT07150026. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07150026
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
- NCT number
- NCT07150026
- Status
- Recruiting
- Phases
- Phase 1
- Enrollment
- 5 participants, estimated
- Lead sponsor
- Unravel Biosciences, Inc.
- Conditions
- Pitt Hopkins Syndrome
- Interventions
- Vorinostat (SAHA), Placebo
- First posted
- 2025-09-02
- Last updated
- 2026-09-17
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Frequency of treatment-related adverse events | 20 weeks | Treatment-related adverse events coded by MedDRA will be compared qualitatively between low dose treatment, high dose treatment, and placebo groups |
| Primary | Drug tolerability as measured by number of treatment discontinuations | 20 weeks | The number of patients discontinuing treatment due to adverse events will be assessed between low dose treatment, high dose treatment, and placebo groups |
| Primary | Change in transcriptomic profile from baseline, as measured by RNA-seq (transcriptome biomarker analysis) | 20 weeks | Transcriptome biomarker analysis is widely used to assess dynamic changes in biological systems in response to disease processes and applied stimuli, which cannot be captured with genomic sequencing of DNA mutations and variations. Unravel Biosciences has created a proprietary algorithm for analyzing transcriptome data, identifying underlying signatures of disease progression or recovery. The machine learned gene network approach predicted the use of vorinostat based on restoring a transcriptome signature of Rett syndrome toward a healthy state, which was confirmed preclinically and motivated this clinical trial. The same algorithm will be used to assess time-dependent changes in the transcriptome network signature, to evaluate its ability to capture clinical response data and to measure the extent of restoration of a disease signature back to a healthy state in a human relative to pre-clinical models. |
| Secondary | Change from baseline on PTHS-specific Clinical Global Impression Improvement Scale (CGI-I) | 20 weeks | 7 point Likert scale with 1=very much improved and 7=very much worse |
| Secondary | Change from baseline in PTHS-specific Clinical Global Impression Severity Scale (CGI-S) | 20 weeks | 7 point Likert Scale; 1=normal, 7=extremely ill |
| Secondary | Change from baseline in the Caregiver Impression of Improvement scale (CareGI-I) | 20 weeks | A 5-point Likert scale is used with the following response options: Much Improved (1); Improved (2); Unchanged (3); Worse (4); or Much Worse (5). |
| Secondary | Change from baseline in the Revised Motor Behavioral Assessment (R-MBA) | 20 weeks | Each of the 24 assessment items are scored based on observed or recently reported severity/frequency and scored on a five-point Likert scale between 0 and 4, with higher numbers denoting higher levels of severity/frequency. |
| Secondary | Change from baseline in Observer-Reported Communication Ability Measure (ORCA) | 20 weeks | The ORCA measure consists of 84 total questions with 70 behavioral items within 22 concepts/functions (e.g., refuse object, make choices) that cover expressive, receptive and pragmatic areas of communication, alongside a set of 14 descriptive items that capture important information about the individual's unique ways of communicating. The questionnaire is designed to be completed by the primary caregiver who is most familiar with the individual and the ways they communicate. ORCA measure scoring is done by dedicated statistical algorithm provided by Duke University and response items are not measured using a simple numerical rating scale. |
| Secondary | Change from baseline in the International Cooperative Ataxia Rating Scale (ICARS) | 20 weeks | A total of 19 questions with four subscale domains is used for assessment. Total possible score = 100, with lower scores representing lower disease burden. |
| Secondary | Changes in sleeping habits as measured by modified Child Sleep Habits Questionnaire (CSHQ) | 20 weeks | A modified version of the 45-item caregiver questionnaire will be used. Items are rated on a three-point scale: "usually" if the sleep behavior occurred five to seven times/week; "sometimes" for two to four times/week; and "rarely" for zero to one time/week. Lower scores are suggestive of lower disease burden on sleep behavior |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT07150026
GET https://conductscience.org/api/trials/v1/NCT07150026/registered