Rett SyndromePhase 2NCT05625568
Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients
A Phase 2 study testing VYNT-0126 in people with Rett Syndrome, run by Vyant Bio.
Trial summary
This is an exploratory, Phase 2, multicenter, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, and efficacy of oral treatment with VYNT-0126 in female subjects 18-45 years of age with Rett syndrome. Read more about this study
- Who is it for?
- Ages 18 to 45 years, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- VYNT-0126
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- No sites listed yet
- Study dates
- Mar 2023to Jun 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 to 45 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Females age 18-45 (inclusive)
- Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene
- Severity rating of between 10 and 36 (Rett Syndrome Natural History/Clinical Severity Scale)
- Concomitant medications (including approved medications for treatment of Rett syndrome) must be stable for >4 weeks prior to enrollment
- Able to receive liquid study drug orally or via gastrostomy tube (G-tube)
Who cannot join
- Actively undergoing neurological regression;
- Abnormal QT interval, prolongation or significant cardiovascular history
- Excluded concomitant medications
- Current clinically significant (as determined by the investigator). cardiovascular, endocrine, hepatic, renal, or respiratory disease
- Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication
- History of, or current cerebrovascular disease or brain trauma
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- History of, or current, malignancy
- Clinically significant abnormalities in safety laboratory tests, vital signs, or ECG, as measured at screening or baseline
- Any condition which in the investigator's opinion would affect the ability of the subject to participate in the study
- Allergy to VYNT-0126 or any ingredients of the liquid formulation
About this study
Official title: Phase 2, Double-Blind, Randomized, Placebo-Controlled Clinical Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Female Patients
This is an exploratory, Phase 2, multicenter, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, and efficacy of oral treatment with VYNT-0126 in female subjects 18-45 years of age with Rett syndrome.
- Conditions
- Rett Syndrome
- Being tested
- VYNT-0126, Placebo
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VYNT-0126Drug
Liquid for oral administration once daily
PlaceboDrug
Liquid for oral administration once daily
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| 5 mg VYNT-0126 | Experimental | Not described Receives: Drug: VYNT-0126 |
| 10 mg VYNT-0126 | Experimental | Not described Receives: Drug: VYNT-0126 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the two VYNT-0126 doses and placebo. SAEs and AEs will be examined throughout the study.
Measured: Through study completion, approximately 14 weeks
Secondary outcomes (4)
Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ)
Measured: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Change from Baseline in the 24-Item Motor-Behavioral Assessment (MBA)
Measured: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Change from Baseline in Clinical Global Impression of Severity (CGI-S)
Measured: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Clinical Global Impression of Improvement (CGI-I)
Measured: Obtained at the end of dose titration and end of treatment (approximately 14 weeks).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2022
- Study startMar 2023 (estimated)
- Primary completionMar 2024 (estimated)
- Study completionJun 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rett 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Vyant Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05625568
- Sponsor's protocol ID
- VYNT-0126-001
- Status last verified
- Nov 2022
- First submitted
- 7 Nov 2022
- Last updated
- 23 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2, Double-Blind, Randomized, Placebo-Controlled Clinical Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Female Patients
- ClinicalTrials.gov ID
- NCT05625568
- Acronym / sponsor ID
- VYNT-0126-001
- Conditions
- Rett Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Vyant BioIndustry (pharma, biotech, devices)
- Study start
- Mar 2023 (estimated)
- Primary completion
- Mar 2024 (estimated)
- Study completion
- Jun 2024 (estimated)
- First posted
- 23 Nov 2022 (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.
This is an exploratory, Phase 2, multicenter, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, and efficacy of oral treatment with VYNT-0126 in female subjects 18-45 years of age with Rett syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| 5 mg VYNT-0126 | Experimental | Drug: VYNT-0126 |
| 10 mg VYNT-0126 | Experimental | Drug: VYNT-0126 |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Females age 18-45 (inclusive)
- Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene
- Severity rating of between 10 and 36 (Rett Syndrome Natural History/Clinical Severity Scale)
- Concomitant medications (including approved medications for treatment of Rett syndrome) must be stable for >4 weeks prior to enrollment
- Able to receive liquid study drug orally or via gastrostomy tube (G-tube)
Key exclusion criteria (10 listed)
- Actively undergoing neurological regression;
- Abnormal QT interval, prolongation or significant cardiovascular history
- Excluded concomitant medications
- Current clinically significant (as determined by the investigator). cardiovascular, endocrine, hepatic, renal, or respiratory disease
- Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication
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 |
|---|---|---|
| VYNT-0126Liquid for oral administration once daily | Drug | 10 mg VYNT-0126, 5 mg VYNT-0126 |
| PlaceboLiquid for oral administration once daily | Drug | Placebo |
Primary outcome
Adverse Events
Time frame: Through study completion, approximately 14 weeks
Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the two VYNT-0126 doses and placebo. SAEs and AEs will be examined throughout the study.
4 secondary outcomes
Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ)
Time frame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Change from Baseline in the 24-Item Motor-Behavioral Assessment (MBA)
Time frame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Change from Baseline in Clinical Global Impression of Severity (CGI-S)
Time frame: Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).
Clinical Global Impression of Improvement (CGI-I)
Time frame: Obtained at the end of dose titration and end of treatment (approximately 14 weeks).
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2022.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VYNT-0126 in people with Rett Syndrome, run by Vyant Bio.
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 5 inclusion and 10 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: status not confirmed, as of the sponsor's last update on 23 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: Through study completion, approximately 14 weeks). 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 23 Nov 2022.