ROCKET: A Study of OV101 in Individuals With Fragile X Syndrome

• Sponsor: Ovid Therapeutics Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03697161
• Collaborator: (none)
• Enrollment: 23
• Locations: 6
• Arms: 3
• Actual Duration (Months): 17.3
• Patients Per Site: 3.8
• Patients Per Site Per Month: 0.2

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Fragile X Syndrome (FXS)	Drug: OV101 (gaboxadol)	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 23 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase 2, Randomized, Double-Blind, Parallel-Group Study Evaluating the Safety, Tolerability, and Efficacy of OV101 in Fragile X Syndrome
• Actual Study Start Date: Sep 17, 2018
• Actual Primary Completion Date: Feb 3, 2020
• Actual Study Completion Date: Feb 26, 2020

Arms and Interventions

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)

Outcome Measures

Primary Outcome Measures

1. Incidence of adverse events [Week 12]

Secondary Outcome Measures

1. Aberrant Behavior Checklist- Community (ABC-C) [Change from baseline to week 12]

Other Outcome Measures

1. Clinical Global Impressions- Improvement (CGI-I) [Week 12]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 13 Years to 22 Years
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Ovid Therapeutics Investigative Site	Sacramento	California	United States	95817
2	Ovid Therapeutics Investigative Site	Aurora	Colorado	United States	80045
3	Ovid Therapeutics Investigative Site	Chicago	Illinois	United States	60612
4	Ovid Therapeutics Investigative Site	Baltimore	Maryland	United States	21205
5	Ovid Therapeutics Investigative Site	Cincinnati	Ohio	United States	45229
6	Ovid Therapeutics Investigative Site	Nashville	Tennessee	United States	37212

Sponsors and Collaborators

• Ovid Therapeutics Inc.

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Ovid Therapeutics Inc.
• ClinicalTrials.gov Identifier: NCT03697161
• Other Study ID Numbers: OV101-17-001
• First Posted: Oct 5, 2018
• Last Update Posted: May 11, 2020
• Last Verified: Sep 1, 2019

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Fragile X Syndrome; Syndrome; Gaboxadol

Study Results

No Results Posted as of May 11, 2020