Back to resultsA Study of OV101 in Individuals With Fragile X Syndrome (ROCKET)
Primary Purpose
Fragile X Syndrome (FXS)
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
OV101 (gaboxadol)
Sponsored by
About this trial
This is an interventional treatment trial for Fragile X Syndrome (FXS)
Eligibility Criteria
Inclusion Criteria:
- 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).
Exclusion Criteria:
- 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.
Sites / Locations
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
OV101 (gaboxadol) Regimen 1
OV101 (gaboxadol) Regimen 2
OV101 (gaboxadol) Regimen 3
Arm Description
Once Daily
Twice Daily
Three Times Daily
Outcomes
Primary Outcome Measures
Incidence of adverse events
Secondary Outcome Measures
Aberrant Behavior Checklist- Community (ABC-C)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03697161
Brief Title
A Study of OV101 in Individuals With Fragile X Syndrome
Acronym
ROCKET
Official Title
A Phase 2, Randomized, Double-Blind, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of OV101 in Fragile X Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
September 2019
Overall Recruitment Status
Completed
Study Start Date
September 17, 2018 (Actual)
Primary Completion Date
February 3, 2020 (Actual)
Study Completion Date
February 26, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ovid Therapeutics Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to assess the safety, tolerability and efficacy of oral OV101 (gaboxadol) in subjects with Fragile X syndrome.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fragile X Syndrome (FXS)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
23 (Actual)
8. Arms, Groups, and Interventions
Arm Title
OV101 (gaboxadol) Regimen 1
Arm Type
Experimental
Arm Description
Once Daily
Arm Title
OV101 (gaboxadol) Regimen 2
Arm Type
Experimental
Arm Description
Twice Daily
Arm Title
OV101 (gaboxadol) Regimen 3
Arm Type
Experimental
Arm Description
Three Times Daily
Intervention Type
Drug
Intervention Name(s)
OV101 (gaboxadol)
Intervention Description
OV101 (gaboxadol)
Primary Outcome Measure Information:
Title
Incidence of adverse events
Time Frame
Week 12
Secondary Outcome Measure Information:
Title
Aberrant Behavior Checklist- Community (ABC-C)
Time Frame
Change from baseline to week 12
Other Pre-specified Outcome Measures:
Title
Clinical Global Impressions- Improvement (CGI-I)
Time Frame
Week 12
10. Eligibility
Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
13 Years
Maximum Age & Unit of Time
22 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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).
Exclusion Criteria:
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Amit Rakhit, MD
Organizational Affiliation
Ovid Therapeutics Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Ovid Therapeutics Investigative Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
34690787
Citation
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.
Results Reference
derived
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A Study of OV101 in Individuals With Fragile X Syndrome
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