Roflumilast for FRAXA Syndrome

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Cincinnati Children's Hospital Medical Center, Cincinnati, OH
FRAXA Syndrome+1 More
Roflumilast - Drug
Eligibility
18 - 65
All Sexes
What conditions do you have?
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Study Summary

This trial is testing a possible medication for Fragile X Syndrome by seeing how it affects brain function, behavior, and clinical measures. Roflumilast is used to treat FRAXA Syndrome and is provided free of charge. This treatment was previously approved by the FDA for another condition.

Eligible Conditions

  • FRAXA Syndrome

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 3 Secondary · Reporting Duration: 3 hour post-dose

3 hour post-dose
Expressive Language Sampling (ELS)
3-hour post-dose
NIH Cognitive Toolbox
Pre-dose
Columbia Suicide Rating Scale (CSSR)
Pre-dose, 3-hour post-dose
Change in EEG Relative Gamma Power
Clinical Global Impressions-Improvement
Clinical Global Impressions-Improvement-Caregiver
Eye-tracking
Probabilistic Reversal Learning (PRL)
Test of Attentional Performance for Children (KiTAP) Test of Alertness
Visual Analog Scale - Caregiver

Trial Safety

Safety Progress

1 of 3

Trial Design

1 Treatment Group

All Study Participants
1 of 1
Experimental Treatment

45 Total Participants · 1 Treatment Group

Primary Treatment: Roflumilast · Has Placebo Group · Phase < 1

All Study ParticipantsExperimental Group · 4 Interventions: Baclofen, Memantine, Roflumilast, Placebo · Intervention Types: Drug, Drug, Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Baclofen
2005
Completed Phase 4
~1320
Memantine
2006
Completed Phase 4
~1070
Roflumilast
2013
Completed Phase 4
~9010
Placebo
1995
Completed Phase 3
~2670

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 3 hour post-dose
Closest Location: Cincinnati Children's Hospital Medical Center · Cincinnati, OH
Photo of Cincinnati 1Photo of Cincinnati 2Photo of Cincinnati 3
2011First Recorded Clinical Trial
0 TrialsResearching FRAXA Syndrome
717 CompletedClinical Trials

Who is running the clinical trial?

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NIH
1,789 Previous Clinical Trials
2,223,504 Total Patients Enrolled
Children's Hospital Medical Center, CincinnatiLead Sponsor
736 Previous Clinical Trials
5,414,812 Total Patients Enrolled
Craig A. Erickson, M.D.Principal InvestigatorChildren's Hospital Medical Center, Cincinnati
3 Previous Clinical Trials
295 Total Patients Enrolled

Eligibility Criteria

Age 18 - 65 · All Participants · 5 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
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.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.