A Study of OV101 in Individuals With Fragile X Syndrome (ROCKET)

December 28, 2023 updated by: Ovid Therapeutics Inc.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

36

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Sacramento, California, United States, 95817
        • Ovid Therapeutics Investigative Site
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Ovid Therapeutics Investigative Site
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Ovid Therapeutics Investigative Site
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Ovid Therapeutics Investigative Site
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Ovid Therapeutics Investigative Site
    • Tennessee
      • Nashville, Tennessee, United States, 37212
        • Ovid Therapeutics Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

13 years to 22 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: OV101 (gaboxadol) Regimen 1
Once Daily
Drug: OV101 (gaboxadol)
OV101 (gaboxadol)
Experimental: OV101 (gaboxadol) Regimen 2
Twice Daily
Drug: OV101 (gaboxadol)
OV101 (gaboxadol)
Experimental: OV101 (gaboxadol) Regimen 3
Three Times Daily
Drug: OV101 (gaboxadol)
OV101 (gaboxadol)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Time Frame: Week 12
Number of Participants with Treatment Emergent Adverse Events
Week 12

Secondary Outcome Measures

Outcome Measure
Time Frame
Aberrant Behavior Checklist- Community (ABC-C)
Time Frame: Change from baseline to week 12
Change from baseline to week 12

Other Outcome Measures

Outcome Measure
Time Frame
Clinical Global Impressions- Improvement (CGI-I)
Time Frame: Week 12
Week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ovid Therapeutics Inc.

Investigators

  • Study Director: Amit Rakhit, MD, Ovid Therapeutics Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 17, 2018

Primary Completion (Actual)

February 3, 2020

Study Completion (Actual)

February 26, 2020

Study Registration Dates

First Submitted

October 1, 2018

First Submitted That Met QC Criteria

October 3, 2018

First Posted (Actual)

October 5, 2018

Study Record Updates

Last Update Posted (Actual)

January 23, 2024

Last Update Submitted That Met QC Criteria

December 28, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • OV101-17-001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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