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GABA-B Agonist

Single Dose Drug Challenge for Fragile X Syndrome

Phase 2
Recruiting
Led By Craig A. Erickson, M.D.
Research Sponsored by Children's Hospital Medical Center, Cincinnati
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Subjects ages 18-45, with FXS who completed the study entitled "Mechanisms and brain circuits underlying fragile X syndrome" (IRB # 2015-8425) or appropriate baseline measures through Biorepository (2013-7327)
FXS is defined as full FMR1 mutations (>200 CGG repeats) confirmed by genetic testing
Must not have
Potential subjects with a creatinine clearance < 50 mL/min will be excluded
Current use of: Amifampridine, Butalbital, Codeine, Doxylamine, Ethanol, Hydrocodone, Isocarboxazid, Kava, Metoclopramide, Midazolam, Oxybate, Phenelzine, Promethazine, Thalidomide, Tranylcypromine, Trimethobenzamide, Erythromycin, Ketoconazole, Fluvoxamine, Enoxacin, and Cimetidine
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 hour post-dose
Awards & highlights
All Individual Drugs Already Approved
Approved for 5 Other Conditions

Summary

This trial is testing a possible medication for Fragile X Syndrome by seeing how it affects brain function, behavior, and clinical measures.

Who is the study for?
This trial is for adults aged 18-45 with Fragile X Syndrome, confirmed by genetic testing, who are generally healthy and have a Stanford Binet IQ under 85. Participants must not be pregnant or breastfeeding, should have been on stable psychotropic drugs for at least four weeks, and cannot have certain medical conditions or history of drug intolerance.
What is being tested?
The study tests the effects of Baclofen, Memantine, Roflumilast versus a placebo in improving neurophysiologic functions and clinical symptoms associated with Fragile X Syndrome. It measures changes from before to after dosing using various assessments.
What are the potential side effects?
Possible side effects include those commonly associated with Baclofen (muscle weakness, dizziness), Memantine (confusion, headache), and Roflumilast (nausea, weight loss). The exact side effects will vary depending on which medication participants receive.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am aged 18-45 with fragile X syndrome and participated in a specific study or provided baseline measures.
Select...
My genetic test shows I have Fragile X syndrome.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My kidney function, measured by creatinine clearance, is 50 mL/min or higher.
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I am currently taking medications like painkillers, sedatives, or specific antibiotics and antifungals.
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I have had a seizure or uncontrolled epilepsy in the last 6 months.
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I am currently taking medications for seizures or anxiety.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3-hour post-dose
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3-hour 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
Change in EEG Relative Gamma Power
Secondary study objectives
Clinical Global Impressions-Improvement
Clinical Global Impressions-Improvement-Caregiver
Visual Analog Scale - Caregiver
Other study objectives
Expressive Language Sampling (ELS)
Eye-tracking
NIH Cognitive Toolbox
+2 more

Side effects data

From 2015 Phase 4 trial • 12 Patients • NCT03073798
8%
Pulmonary Exacerbation
100%
80%
60%
40%
20%
0%
Study treatment Arm
All Participants

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Experimental Study ParticipantsExperimental Treatment3 Interventions
Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses.
Group II: Control Study ParticipantsPlacebo Group1 Intervention
Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Baclofen
FDA approved
Memantine
FDA approved
Roflumilast
FDA approved

Find a Location

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiLead Sponsor
835 Previous Clinical Trials
6,565,122 Total Patients Enrolled
7 Trials studying Fragile X Syndrome
450 Patients Enrolled for Fragile X Syndrome
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NIH
2,053 Previous Clinical Trials
2,731,509 Total Patients Enrolled
7 Trials studying Fragile X Syndrome
35,436 Patients Enrolled for Fragile X Syndrome
Craig A. Erickson, M.D.Principal InvestigatorChildren's Hospital Medical Center, Cincinnati
3 Previous Clinical Trials
295 Total Patients Enrolled
1 Trials studying Fragile X Syndrome
14 Patients Enrolled for Fragile X Syndrome

Media Library

Baclofen (GABA-B Agonist) Clinical Trial Eligibility Overview. Trial Name: NCT05418049 — Phase 2
Fragile X Syndrome Research Study Groups: Experimental Study Participants, Control Study Participants
Fragile X Syndrome Clinical Trial 2023: Baclofen Highlights & Side Effects. Trial Name: NCT05418049 — Phase 2
Baclofen (GABA-B Agonist) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05418049 — Phase 2
~10 spots leftby Jun 2025
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