Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

NCT ID: NCT05418049

Last Updated: 2025-12-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-09-08

Study Completion Date

2026-03-05

Brief Summary

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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.

Detailed Description

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Conditions

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Fragile X Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

This study was designed as a 4-intervention crossover, with all study participants receiving all possible interventions. These are placebo, baclofen, roflumilast, and memantine.
Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Quadruple masking (participant, care provider, investigator and outcomes assessor)

Study Groups

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Experimental Study Participants

Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses.

Group Type EXPERIMENTAL

Baclofen

Intervention Type DRUG

30mg - Supplied as 10mg and 20mg tablets

Memantine

Intervention Type DRUG

two 10 mg tablets

Roflumilast

Intervention Type DRUG

250 mcg capsule

Control Study Participants

Participants received, in random order, a single dose of placebo, baclofen, roflumilast or memantine with a two-week washout period between doses.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo pill

Interventions

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Baclofen

30mg - Supplied as 10mg and 20mg tablets

Intervention Type DRUG

Memantine

two 10 mg tablets

Intervention Type DRUG

Roflumilast

250 mcg capsule

Intervention Type DRUG

Placebo

Placebo pill

Intervention Type DRUG

Other Intervention Names

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NAMENDA DAILIRESP

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

45 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

Sponsor Role collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Craig A. Erickson, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Locations

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Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Countries

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United States

Other Identifiers

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U54HD104461

Identifier Type: NIH

Identifier Source: secondary_id

View Link

2022-0202

Identifier Type: -

Identifier Source: org_study_id