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Can a single dose ease fragile x? new trial tests three drugs

NCT ID NCT05418049

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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

18 to 45 years

Sex

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

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.