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

Ovid Therapeutics

Status and phase: Completed; Phase 2

Conditions: Fragile X Syndrome (FXS)

Treatments: Drug: OV101 (gaboxadol)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03697161

OV101-17-001

Details and patient eligibility

About

The purpose of this study is to assess the safety, tolerability and efficacy of oral OV101 (gaboxadol) in subjects with Fragile X syndrome.

Enrollment: 36 patients

Sex: Male

Ages: 13 to 22 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

36 participants in 3 patient groups

OV101 (gaboxadol) Regimen 1

Experimental group

Description:

Once Daily

Treatment:

Drug: OV101 (gaboxadol)

OV101 (gaboxadol) Regimen 2

Experimental group

Description:

Twice Daily

Treatment:

Drug: OV101 (gaboxadol)

OV101 (gaboxadol) Regimen 3

Experimental group

Description:

Three Times Daily

Treatment:

Drug: OV101 (gaboxadol)