Gaboxadol for Fragile X Syndrome

Recruiting in Palo Alto (17 mi)

Overseen byCragi A Erickson, MD

Age: 18 - 65

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Craig Erickson

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This is a single dose, placebo-controlled study. Male subjects aged 18 to 40 years (inclusive) with a diagnosis of FXS. Eligible subjects may enroll in this study comprised of two in home and two in clinic visits each 14 days apart, for a total of four visits. Subjects will be given single dose gaboxadol (10 mg) or matched placebo at each of these visits to take orally. Thus, all enrolled subjects will receive placebo at home and in clinic and receive gaboxadol at home and in clinic in a blinded fashion.

Eligibility Criteria

This trial is for adult males aged 18 to 40 with Fragile X Syndrome. Participants will have a series of four visits, two at home and two in clinic, each spaced out by two weeks. They must be able to take oral medication.

Inclusion Criteria

Subject consents to participate, or if they are not their own legal guardian, offers assent supported by legally authorized representative consent

Subject and caregiver are both able to understand the spoken national language clearly and caregiver can read and write to complete study assessments

I am a man aged between 18 and 40.

+5 more

Exclusion Criteria

Significant changes in any educational, behavioral, and/or dietary interventions the month prior to Screening

Has a clinically significant heart rate or BP at Screening as judged by the Investigator

Has abnormal baseline laboratory assessments including, but not limited to, ALT or AST or total bilirubin >1.5 × the ULN, serum creatinine >1.5 x ULN or other clinically relevant laboratory abnormality

+8 more

Participant Groups

The study tests the effects of a single dose of Gaboxadol (10 mg) compared to a placebo. Each participant will receive both the actual drug and placebo in different visits without knowing which one they are taking.

1Treatment groups

Experimental Treatment

Group I: All Study ParticipantsExperimental Treatment2 Interventions

Participants received, in random order, a single dose of placebo or Gaboxadol with a two-week washout period between doses. The gaboxadol dosage selected for this study is 10 mg or Placebo as a single dose on each study visit (two at home and two in clinic).