Fragile X Syndrome (FXS)Phase 2ROCKETNCT03697161
A Study of OV101 in Individuals With Fragile X Syndrome
A Phase 2 study testing OV101 (gaboxadol) in people with Fragile X Syndrome (FXS), run by Ovid Therapeutics Inc. at 6 sites in United States.
Trial summary
The purpose of this study is to assess the safety, tolerability and efficacy of oral OV101 (gaboxadol) in subjects with Fragile X syndrome. Read more about this study
- Who is it for?
- Ages 13 to 22 years, men only
- Conditions the study is for
- Fragile X Syndrome (FXS)
- What is being tested?
- OV101 (gaboxadol)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 6 sitesUnited States
- Study dates
- 17 Sep 2018to 26 Feb 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 13 to 22 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Is male and 13 to 22 years old (inclusive) at the time of informed consent.
- Has a diagnosis of FXS with a confirmed FMR1 full mutation (≥200 CGG repeats).
Who cannot join
- Concomitant disease or condition that are clinically significant and would limit study participation
- Clinically significant lab abnormalities or vital signs at the time of screening
- History of uncontrollable seizure disorder or seizure episodes within 6 months of screening or change in the anticonvulsant pharmacotherapy in the past 3 months.
- Unable or does not have a caregiver able to comply with study requirements.
- Enrolled in any clinical trial within the 30 days before screening.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Ovid Therapeutics Investigative Site
Aurora, Colorado, 80045
Ovid Therapeutics Investigative Site
Baltimore, Maryland, 21205
Ovid Therapeutics Investigative Site
Chicago, Illinois, 60612
Ovid Therapeutics Investigative Site
Cincinnati, Ohio, 45229
Ovid Therapeutics Investigative Site
Nashville, Tennessee, 37212
Ovid Therapeutics Investigative Site
Sacramento, California, 95817
About this study
Official title: A Phase 2, Randomized, Double-Blind, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of OV101 in Fragile X Syndrome
The purpose of this study is to assess the safety, tolerability and efficacy of oral OV101 (gaboxadol) in subjects with Fragile X syndrome.
- Conditions
- Fragile X Syndrome (FXS)
- Being tested
- OV101 (gaboxadol)
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OV101 (gaboxadol)Drug
OV101 (gaboxadol)
| Study group | Role | What they receive |
|---|---|---|
| OV101 (gaboxadol) Regimen 1 | Experimental | Once Daily Receives: Drug: OV101 (gaboxadol) |
| OV101 (gaboxadol) Regimen 2 | Experimental | Twice Daily Receives: Drug: OV101 (gaboxadol) |
| OV101 (gaboxadol) Regimen 3 | Experimental | Three Times Daily Receives: Drug: OV101 (gaboxadol) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Number of Participants with Treatment Emergent Adverse Events
Measured: Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2018
- Study start17 Sep 2018
- Primary completion3 Feb 2020
- Study completion26 Feb 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fragile X Syndrome (FXS)Studies 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
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ovid Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Amit Rakhit, MD, Study director, Ovid Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03697161
- Sponsor's protocol ID
- OV101-17-001
- Acronym
- ROCKET
- Status last verified
- Dec 2023
- First submitted
- 1 Oct 2018
- Last updated
- 23 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Budimirovic DB, Dominick KC, Gabis LV, Adams M, Adera M, Huang L, Ventola P, Tartaglia NR, Berry-Kravis E. Gaboxadol in Fragile X Syndrome: A 12-Week Randomized, Double-Blind, Parallel-Group, Phase 2a Study. Front Pharmacol. 2021 Oct 8;12:757825. doi: 10.3389/fphar.2021.757825. eCollection 2021.
PubMed 34690787
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of OV101 in Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT03697161
- Acronym / sponsor ID
- ROCKET / OV101-17-001
- Conditions
- Fragile X Syndrome (FXS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Ovid Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Sep 2018 (actual)
- Primary completion
- 3 Feb 2020 (actual)
- Study completion
- 26 Feb 2020 (actual)
- First posted
- 5 Oct 2018 (actual)
- Results
- Posted 23 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the safety, tolerability and efficacy of oral OV101 (gaboxadol) in subjects with Fragile X 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 |
|---|---|---|
| OV101 (gaboxadol) Regimen 1 | Experimental | Drug: OV101 (gaboxadol)Once Daily |
| OV101 (gaboxadol) Regimen 2 | Experimental | Drug: OV101 (gaboxadol)Twice Daily |
| OV101 (gaboxadol) Regimen 3 | Experimental | Drug: OV101 (gaboxadol)Three Times Daily |
Who may qualify
- Ages
- 13 to 22 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Is male and 13 to 22 years old (inclusive) at the time of informed consent.
- Has a diagnosis of FXS with a confirmed FMR1 full mutation (≥200 CGG repeats).
Key exclusion criteria (5 listed)
- Concomitant disease or condition that are clinically significant and would limit study participation
- Clinically significant lab abnormalities or vital signs at the time of screening
- History of uncontrollable seizure disorder or seizure episodes within 6 months of screening or change in the anticonvulsant pharmacotherapy in the past 3 months.
- Unable or does not have a caregiver able to comply with study requirements.
- Enrolled in any clinical trial within the 30 days before screening.
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 |
|---|---|---|
| OV101 (gaboxadol)OV101 (gaboxadol) | Drug | OV101 (gaboxadol) Regimen 1, OV101 (gaboxadol) Regimen 2, OV101 (gaboxadol) Regimen 3 |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Time frame: Week 12
Number of Participants with Treatment Emergent Adverse Events
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34690787Budimirovic DB, Dominick KC, Gabis LV, Adams M, Adera M, Huang L, Ventola P, Tartaglia NR, Berry-Kravis E. Gaboxadol in Fragile X Syndrome: A 12-Week Randomized, Double-Blind, Parallel-Group, Phase 2a Study. Front Pharmacol. 2021 Oct 8;12:757825. doi: 10.3389/fphar.2021.757825. eCollection 2021.
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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.
Amit Rakhit, MD study director
Ovid Therapeutics Inc.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing OV101 (gaboxadol) in people with Fragile X Syndrome (FXS), run by Ovid Therapeutics Inc. at 6 sites in United States.
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 2 inclusion and 5 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: finished, as of the sponsor's last update on 23 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. 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. 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?
Yes. Results are posted in the registry (first posted 23 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Treatment Emergent Adverse Events (TEAE) (time frame: Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03697161, which also lists 1 linked publication.
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 Jan 2024.