Fragile X SyndromePhase 2NCT05418049
Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge
A Phase 2 study testing Baclofen, Memantine and Roflumilast in people with Fragile X Syndrome, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The aim of this study is to utilize neurophysiologic assessments, behavioral measures and clinical measures to assess how much deficits associated with Fragile X Syndrome from pre-dose to post-dose using pharmacology. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- Baclofen, Memantine and Roflumilast
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Sep 2022to 5 Mar 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 11Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Evaluating the Neurophysiologic and Clinical Effects of Single-Dose Baclofen, Roflumilast, Memantine, and Placebo in Fragile X Syndrome
The aim of this study is to utilize neurophysiologic assessments, behavioral measures and clinical measures to assess how much deficits associated with Fragile X Syndrome from pre-dose to post-dose using pharmacology.
- Conditions
- Fragile X Syndrome
- Being tested
- Baclofen, Memantine, Roflumilast, Placebo
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BaclofenDrug
30mg - Supplied as 10mg and 20mg tablets
MemantineDrug
Also called NAMENDA
two 10 mg tablets
RoflumilastDrug
Also called DAILIRESP
250 mcg capsule
PlaceboDrug
Placebo pill
| Study group | Role | What they receive |
|---|---|---|
| Experimental Study Participants | Experimental | Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses. Receives: Drug: Baclofen; Drug: Memantine; Drug: Roflumilast |
| Control Study Participants | Placebo comparator | Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in EEG Relative Gamma Power
EEG relative gamma power at rest was calculated as the percent of power in the gamma frequencies relative to the sum of power in all frequency bands, averaged across electrodes, and calculated separately at pre-dose and post-dose timepoints. To assess the impact of drug, the pre-dose relative gamma power was subtracted from post-dose relative gamma power. Higher numbers indicate more relative gamma power post-dose; lower numbers indicate more relative gamma power pre-dose.
Measured: Pre-dose, 3-hour post-dose
Secondary outcomes (3)
Clinical Global Impressions-Improvement
The Clinical Global Impressions - Improvement (CGI-I) requires the clinician to assess how much the patient's illness has changed relative to pre-dose, from 1 (very much improved) to 7 (very much worse).
Measured: Pre-dose, 3-hour post-dose
Clinical Global Impressions-Improvement-Caregiver
The CGI-I requires the caregiver to assess how much the patient's illness has changed relative to pre-dose, from 1 (very much improved) to 7 (very much worse).
Measured: Pre-dose, 3-hour post-dose
Visual Analog Scale - Caregiver
The parent or caregiver will be asked to assess how much anxiety the participant is expressing on a line between two endpoints (no anxiety to worsened anxiety).
Measured: Pre-dose, 3-hour post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2022
- Study start8 Sep 2022
- Primary completion28 Oct 2025
- Study completion5 Mar 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- This study was designed as a 4-intervention crossover, with all study participants receiving all possible interventions. These are placebo, baclofen, roflumilast, and memantine.
- Masking
- None (open label)
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Responsible party
- Sponsor
- Investigators
- Craig A. Erickson, M.D., Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05418049
- Sponsor's protocol ID
- 2022-0202
- Other IDs
- U54HD104461
- Status last verified
- Nov 2025
- First submitted
- 31 May 2022
- Last updated
- 23 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating the Neurophysiologic and Clinical Effects of Single-Dose Baclofen, Roflumilast, Memantine, and Placebo in Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT05418049
- Acronym / sponsor ID
- 2022-0202
- Conditions
- Fragile X Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Study start
- 8 Sep 2022 (actual)
- Primary completion
- 28 Oct 2025 (actual)
- Study completion
- 5 Mar 2026 (estimated)
- First posted
- 14 Jun 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to utilize neurophysiologic assessments, behavioral measures and clinical measures to assess how much deficits associated with Fragile X Syndrome from pre-dose to post-dose using pharmacology.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This study was designed as a 4-intervention crossover, with all study participants receiving all possible interventions. These are placebo, baclofen, roflumilast, and memantine.
| Arm | Type | Receives |
|---|---|---|
| Experimental Study Participants | Experimental | Drug: Baclofen, Drug: Memantine, Drug: RoflumilastParticipants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses. |
| Control Study Participants | Placebo comparator | Drug: PlaceboParticipants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Baclofen30mg - Supplied as 10mg and 20mg tablets | Drug | Experimental Study Participants |
| MemantineAlso: NAMENDAtwo 10 mg tablets | Drug | Experimental Study Participants |
| RoflumilastAlso: DAILIRESP250 mcg capsule | Drug | Experimental Study Participants |
| PlaceboPlacebo pill | Drug | Control Study Participants |
Primary outcome
Change in EEG Relative Gamma Power
Time frame: Pre-dose, 3-hour post-dose
EEG relative gamma power at rest was calculated as the percent of power in the gamma frequencies relative to the sum of power in all frequency bands, averaged across electrodes, and calculated separately at pre-dose and post-dose timepoints. To assess the impact of drug, the pre-dose relative gamma power was subtracted from post-dose relative gamma power. Higher numbers indicate more relative gamma power post-dose; lower numbers indicate more relative gamma power pre-dose.
3 secondary outcomes
Clinical Global Impressions-Improvement
Time frame: Pre-dose, 3-hour post-dose
Clinical Global Impressions-Improvement-Caregiver
Time frame: Pre-dose, 3-hour post-dose
Visual Analog Scale - Caregiver
Time frame: Pre-dose, 3-hour post-dose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2025.
Related trials for Fragile X Syndrome
Not yet recruitingPhase 2
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
Aggressive Behavior in Fragile X Syndome
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Not yet recruiting
Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
Neurodevelopmental Disorders, Autism Spectrum Disorder (ASD)
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Not yet recruitingPhase 2/3
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Craig A. Erickson, M.D. principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Baclofen, Memantine and Roflumilast in people with Fragile X Syndrome, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 23 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in EEG Relative Gamma Power (time frame: Pre-dose, 3-hour post-dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Dec 2025.