Can a single dose ease fragile x? new trial tests three drugs
NCT ID NCT05418049
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether a single dose of baclofen, roflumilast, or memantine can improve brain activity and symptoms in adults with Fragile X syndrome. Forty-five participants aged 18 to 45 will receive each drug or a placebo in separate sessions. The main goal is to measure changes in brain waves using EEG, along with clinician and caregiver ratings of improvement.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- baclofen, roflumilast, memantine
- What this could lead to
- If successful, this could point toward a short-term treatment to ease certain symptoms of Fragile X syndrome.
- What could go wrong
- This is an early phase 2 trial with only 45 participants, testing single doses. It may not show clear benefits, and results may not apply to long-term use.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
About 45 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Sep 2022
- Expected to finish
-
Mar 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 45 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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. * General good health as determined by physical exam, medical history and laboratory work up. * Stanford Binet IQ \<85 * Stable dosing of psychotropic drugs for at least 4 weeks. Exclusion Criteria: * Subjects with a history of intolerance to baclofen, roflumilast, or memantine will be excluded. * Subjects will also be excluded if they have taken any investigational drug within 3 months, have a history of substance abuse or dependence within 6 months, or significant psychiatric or CNS neurological disease unrelated to FXS. * Uncontrolled seizures or history of epilepsy with a seizure in the past 6 months * Auditory or visual impairments that cannot be corrected based on visual and auditory screener benchmarks. * Moderate to severe renal or hepatic impairment and determined by a study physician incorporating data from exam, medical history and laboratory value evaluation among other data points. * Use of barbiturates, benzodiazepines, antiepileptics, or other GABAergic or glutamatergic modulators * 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. * Those taking other psychiatric medications must be on stable doses for 4 weeks before the baseline visit. * Pregnancy or breast-feeding. For female subjects of child bearing potential, a urine pregnancy test will be performed. * Potential subjects with a creatinine clearance \< 50 mL/min will be excluded. * Identified medical issues, inability to tolerate study procedures or study drug per the discretion of the Principal Investigator.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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