Gaboxadol for Fragile X Syndrome

Phase 2

Recruiting

Led By Cragi A Erickson, MD

Research Sponsored by Craig Erickson

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up pre-dose, 60 minutes post-dose

Summary

This trial will involve male participants aged 18 to 40 with Fragile X Syndrome. They will have four visits, two at home and two in clinic, each 14 days apart. At each

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not specified here but generally could include drowsiness, headache, nausea or other reactions common with new medications taken orally.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ pre-dose and post-dose

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~pre-dose and post-dose

This trial's timeline: 3 weeks for screening, Varies for treatment, and pre-dose and post-dose for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

To evaluate target engagement of gaboxadol treatment on high density EEG recordings

Secondary study objectives

To determine whether FMRP levels predict treatment response

To investigate the effect of gaboxadol treatment on clinician-rated measures

To investigate the effect of gaboxadol treatment on eye tracking assessments

+4 more

Trial Design

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).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Gaboxadol

2019

Completed Phase 3

~110

Placebo

1995

Completed Phase 3

~2670