Pitt Hopkins SyndromePhase 1NCT07150026
An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
A Phase 1 study testing Vorinostat (SAHA) in people with Pitt Hopkins Syndrome, run by Unravel Biosciences, Inc. at 1 site in Colombia.
Trial summary
The study is an exploratory evaluation of the safety and efficacy of vorinostat in Pitt Hopkins syndrome. Each patient will be self-controlled in an adapted N-of-1 study design methodology with three treatment arms, including a 4-week placebo phase and two vorinostat dose arms, including every 8 weeks of daily dosing… Read more about this study
- Who is it for?
- Ages 3 to 21 years
- Conditions the study is for
- Pitt Hopkins Syndrome
- What is being tested?
- Vorinostat (SAHA)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 1 siteColombia
- Study dates
- 15 Mar 2026to 15 Mar 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects ≥3 years of age and ≤ 21 years of age at time of screening
- Clinical diagnosis of PTHS with documented pathologic mutation in the TCF4 gene
- At time of screening, is in a 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
- Has had a stable pattern of seizure activity for 4 weeks before screening
- Can swallow medication or can take it by gastrostomy tube
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- 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
Who cannot join
- Has another clinically significant medical condition other than those related to their TCF4 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Colombia1 site, 1 recruiting
- Recruiting
Grupo de Investigación Clínica PECET (GIC-PECET)
Medellín
Coordinadora de estudios clinicos, +57 3023575173, liliana.lopez@pecet-colombia.org
About this study
Official title: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome Using an "N of 1" Study Design
The study is an exploratory evaluation of the safety and efficacy of vorinostat in Pitt Hopkins syndrome. Each patient will be self-controlled in an adapted N-of-1 study design methodology with three treatment arms, including a 4-week placebo phase and two vorinostat dose arms, including every 8 weeks of daily dosing at a low dose of 80mg/m2/day and 8 weeks of a higher dose at 160mg/m2/day.
Key objectives of the study include:
- To confirm the safety and tolerability of oral vorinostat 80mg/m2/day and 160mg/ m2/day dose levels when administered to PTHS 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 PTHS
Read the detailed description
Unravel Biosciences, Inc. ("Unravel") proposes to develop an orally administered, once daily novel treatment for the orphan drug indication of Pitt Hopkins syndrome. Unravel has utilized its proprietary drug discovery platform to identify drugs having potential therapeutic value for neurodevelopmental disorders caused by gene mutations shown to have a cascading effect on other genes. Unravel's platform combines human gene regulatory network-based computational drug prediction with in vivo screening in a population-level diversity, CRISPR-edited, Xenopus laevis tadpole disease model. Through use of Unravel's platform, the drug vorinostat ranked highly in predictive scoring for Pitt Hopkins syndrome. In work done for Rett syndrome, another orphan CNS disease with many molecular and phenotypic similarities to Pitt Hopkins syndrome, vorinostat broadly improved both CNS and non-CNS (e.g., gastrointestinal, respiratory, inflammatory) abnormalities in a pre-clinical mouse model of Rett syndrome. Vorinostat was the first treatment to demonstrate nonclinical efficacy across multiple organ systems when dosed after the onset of symptoms, and network analysis revealed a putative therapeutic mechanism for its cross-organ normalizing effects based on its impact on acetylation metabolism and post-translational modifications of microtubules, leading to the selection of vorinostat as a target candidate for further assessment in both Rett and Pitt Hopkins syndromes. The main hypotheses informing the goals and design of the study are as follows:
- Vorinostat is safe and tolerable when dosed in typical Rett patients at dose levels up to 160mg/m2/day
- At a molecular level, vorinostat mitigates the impact of the underlying TCF4 gene mutation in PTHS patients by restoring downstream mRNA synthesis, as measured by transcriptome data
- Vorinostat provides clinical benefit to Pitt Hopkins patients by reducing frequency and severity of clinical signs/symptoms and improving patient quality of life
The study is designed as an exploratory, proof of concept trial to investigate the study hypotheses as stated above and to achieve the primary goals of the trial. The study design adapts the well-known "n of 1" crossover study methodology (Guyatt, et.al., 1990, Kravitz, et.al., 2014), where each patient serves as their own control during comparative analyses of safety and efficacy. Up to 5 patients will be enrolled in the study to explore the hypothesis that vorinostat is a safe and potentially effective treatment for Pitt Hopkins syndrome. Each patient enrolled in the study will be exposed to a 4-week placebo study phase to generate baseline data that will serve as a control as well as two active drug phases with vorinostat treatment, starting at 80mg/m2/day dosing for 8 weeks, followed by dose escalation to 160mg/m2/day for 8 weeks. The study is designed to be single-blinded, where patients and their caregivers will not be aware of their treatment assignment in an attempt to minimize bias where practically possible, especially given the subjective nature of several of the endpoints being evaluated. Investigator, study staff, and sponsor will not be blinded to study treatment assignment.
- Conditions
- Pitt Hopkins Syndrome
- Being tested
- Vorinostat (SAHA), Placebo
- Keywords
- Pitt Hopkins, n of 1, interventional
- Medical subject headings
- Pitt-Hopkins syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vorinostat (SAHA)Drug
Also called suberoylanilide hydroxamic acid
oral suspension
PlaceboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| Low dose interventional arm | Experimental | vorinostat low dose 80mg/m2/day Receives: Drug: Vorinostat (SAHA) |
| High dose interventional arm | Experimental | vorinostat 160mg/m2/day dose Receives: Drug: Vorinostat (SAHA) |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of treatment-related adverse events
Treatment-related adverse events coded by MedDRA will be compared qualitatively between low dose treatment, high dose treatment, and placebo groups
Measured: 20 weeks
Drug tolerability as measured by number of treatment discontinuations
The number of patients discontinuing treatment due to adverse events will be assessed between low dose treatment, high dose treatment, and placebo groups
Measured: 20 weeks
Change in transcriptomic profile from baseline, as measured by RNA-seq (transcriptome biomarker analysis)
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.
Measured: 20 weeks
Secondary outcomes (10)
Change from baseline on PTHS-specific Clinical Global Impression Improvement Scale (CGI-I)
7 point Likert scale with 1=very much improved and 7=very much worse
Measured: 20 weeks
Change from baseline in PTHS-specific Clinical Global Impression Severity Scale (CGI-S)
7 point Likert Scale; 1=normal, 7=extremely ill
Measured: 20 weeks
Change from baseline in the Caregiver Impression of Improvement scale (CareGI-I)
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).
Measured: 20 weeks
Change from baseline in the Revised Motor Behavioral Assessment (R-MBA)
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.
Measured: 20 weeks
Change from baseline in Observer-Reported Communication Ability Measure (ORCA)
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.
Measured: 20 weeks
Change from baseline in the International Cooperative Ataxia Rating Scale (ICARS)
A total of 19 questions with four subscale domains is used for assessment. Total possible score = 100, with lower scores representing lower disease burden.
Measured: 20 weeks
Changes in sleeping habits as measured by modified Child Sleep Habits Questionnaire (CSHQ)
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
Measured: 20 weeks
Change in revised Face Legs Activity Cry and Consolability (FLACC) observational pain tool
The Face Legs Activity Cry and Consolability (FLACC) tool is a behavioral pain assessment scale used for nonverbal patients who are unable to self-report their level of pain. Pain is assessed through observation of 5 categories including face, legs, activity, cry, and consolability. Each category is scored from 0 to 2 to provide a total score ranging from 0 to 10. Higher scores represent greater symptom burden. The revised FLACC (Malvya, et.al., 2006) incorporates pain behaviors more often observed in cognitively impaired adults and children and has been demonstrated to have a reasonable level of reliability and validity for use in assessing pain in nonverbal children with cognitive impairment.
Measured: 20 weeks
Reduction in seizure frequency as measured by seizure diary
Caregiver-recorded seizure frequency, duration, and quality will be evaluated for evidence of improvement based on reduction in seizure burden
Measured: 20 weeks
GI symptom measures relative to baseline using a subset of questions from the Gastrointestinal Symptom Rating Scale (GSRS)
A subset of 7 questions from the GSRS will be used in the study, each question rated on a 4 point ordinal scale, "0" representing no symptoms and "3" representing significant symptom burden. Lower scores reflect lower symptom burden.
Measured: 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2025
- Study start15 Mar 2026
- Primary completion31 Dec 2026 (estimated)
- Study completion15 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pitt Hopkins SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Unravel Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Neal I Muni, M.D., MSPH, Study director, Unravel Biosciences, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07150026
- Sponsor's protocol ID
- RVL-001 Study 003
- Other IDs
- 20251103997
- Status last verified
- Sep 2026
- First submitted
- 14 Aug 2025
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome Using an "N of 1" Study Design
- ClinicalTrials.gov ID
- NCT07150026
- Acronym / sponsor ID
- RVL-001 Study 003
- Conditions
- Pitt Hopkins Syndrome
- Keywords
- Pitt Hopkins, n of 1, interventional
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Unravel Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Mar 2026 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 15 Mar 2027 (estimated)
- First posted
- 2 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is an exploratory evaluation of the safety and efficacy of vorinostat in Pitt Hopkins syndrome. Each patient will be self-controlled in an adapted N-of-1 study design methodology with three treatment arms, including a 4-week placebo phase and two vorinostat dose arms, including every 8 weeks of daily dosing at a low dose of 80mg/m2/day and 8 weeks of a higher dose at 160mg/m2/day.
Key objectives of the study include:
- To confirm the safety and tolerability of oral vorinostat 80mg/m2/day and 160mg/ m2/day dose levels when administered to PTHS 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 PTHS
Detailed description
Unravel Biosciences, Inc. ("Unravel") proposes to develop an orally administered, once daily novel treatment for the orphan drug indication of Pitt Hopkins syndrome. Unravel has utilized its proprietary drug discovery platform to identify drugs having potential therapeutic value for neurodevelopmental disorders caused by gene mutations shown to have a cascading effect on other genes. Unravel's platform combines human gene regulatory network-based computational drug prediction with in vivo screening in a population-level diversity, CRISPR-edited, Xenopus laevis tadpole disease model. Through use of Unravel's platform, the drug vorinostat ranked highly in predictive scoring for Pitt Hopkins syndrome. In work done for Rett syndrome, another orphan CNS disease with many molecular and phenotypic similarities to Pitt Hopkins syndrome, vorinostat broadly improved both CNS and non-CNS (e.g., gastrointestinal, respiratory, inflammatory) abnormalities in a pre-clinical mouse model of Rett syndrome. Vorinostat was the first treatment to demonstrate nonclinical efficacy across multiple organ systems when dosed after the onset of symptoms, and network analysis revealed a putative therapeutic mechanism for its cross-organ normalizing effects based on its impact on acetylation metabolism and post-translational modifications of microtubules, leading to the selection of vorinostat as a target candidate for further assessment in both Rett and Pitt Hopkins syndromes. The main hypotheses informing the goals and design of the study are as follows:
- Vorinostat is safe and tolerable when dosed in typical Rett patients at dose levels up to 160mg/m2/day
- At a molecular level, vorinostat mitigates the impact of the underlying TCF4 gene mutation in PTHS patients by restoring downstream mRNA synthesis, as measured by transcriptome data
- Vorinostat provides clinical benefit to Pitt Hopkins patients by reducing frequency and severity of clinical signs/symptoms and improving patient quality of life
The study is designed as an exploratory, proof of concept trial to investigate the study hypotheses as stated above and to achieve the primary goals of the trial. The study design adapts the well-known "n of 1" crossover study methodology (Guyatt, et.al., 1990, Kravitz, et.al., 2014), where each patient serves as their own control during comparative analyses of safety and efficacy. Up to 5 patients will be enrolled in the study to explore the hypothesis that vorinostat is a safe and potentially effective treatment for Pitt Hopkins syndrome. Each patient enrolled in the study will be exposed to a 4-week placebo study phase to generate baseline data that will serve as a control as well as two active drug phases with vorinostat treatment, starting at 80mg/m2/day dosing for 8 weeks, followed by dose escalation to 160mg/m2/day for 8 weeks. The study is designed to be single-blinded, where patients and their caregivers will not be aware of their treatment assignment in an attempt to minimize bias where practically possible, especially given the subjective nature of several of the endpoints being evaluated. Investigator, study staff, and sponsor will not be blinded to study treatment assignment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, care provider)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low dose interventional arm | Experimental | Drug: Vorinostat (SAHA)vorinostat low dose 80mg/m2/day |
| High dose interventional arm | Experimental | Drug: Vorinostat (SAHA)vorinostat 160mg/m2/day dose |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
Who may qualify
- Ages
- 3 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Subjects ≥3 years of age and ≤ 21 years of age at time of screening
- Clinical diagnosis of PTHS with documented pathologic mutation in the TCF4 gene
- At time of screening, is in a 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
- Has had a stable pattern of seizure activity for 4 weeks before screening
Key exclusion criteria (16 listed)
- Has another clinically significant medical condition other than those related to their TCF4 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vorinostat (SAHA)Also: suberoylanilide hydroxamic acidoral suspension | Drug | High dose interventional arm, Low dose interventional arm |
| Placeboplacebo | Drug | Placebo |
Primary outcome
Frequency of treatment-related adverse events
Time frame: 20 weeks
Treatment-related adverse events coded by MedDRA will be compared qualitatively between low dose treatment, high dose treatment, and placebo groups
Drug tolerability as measured by number of treatment discontinuations
Time frame: 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
Change in transcriptomic profile from baseline, as measured by RNA-seq (transcriptome biomarker analysis)
Time frame: 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.
10 secondary outcomes
Change from baseline on PTHS-specific Clinical Global Impression Improvement Scale (CGI-I)
Time frame: 20 weeks
Change from baseline in PTHS-specific Clinical Global Impression Severity Scale (CGI-S)
Time frame: 20 weeks
Change from baseline in the Caregiver Impression of Improvement scale (CareGI-I)
Time frame: 20 weeks
Change from baseline in the Revised Motor Behavioral Assessment (R-MBA)
Time frame: 20 weeks
Change from baseline in Observer-Reported Communication Ability Measure (ORCA)
Time frame: 20 weeks
Change from baseline in the International Cooperative Ataxia Rating Scale (ICARS)
Time frame: 20 weeks
Changes in sleeping habits as measured by modified Child Sleep Habits Questionnaire (CSHQ)
Time frame: 20 weeks
Change in revised Face Legs Activity Cry and Consolability (FLACC) observational pain tool
Time frame: 20 weeks
Reduction in seizure frequency as measured by seizure diary
Time frame: 20 weeks
GI symptom measures relative to baseline using a subset of questions from the Gastrointestinal Symptom Rating Scale (GSRS)
Time frame: 20 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Neal I Muni, M.D., MSPH study director
Unravel Biosciences, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Vorinostat (SAHA) in people with Pitt Hopkins Syndrome, run by Unravel Biosciences, Inc. at 1 site in Colombia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Colombia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Neal I Muni, M.D., MSPH (+1 857-404-8252, neal.muni@unravel.bio), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of treatment-related adverse events (time frame: 20 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2026.