Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.

2026 data Public-data reference. official source

Fragile X Syndrome

Open-data reference.

17 US clinical trials · 7 currently recruiting

Active & Recent Trials

RECRUITING NA 376 participants

Physical Activity and Community EmPOWERment Project

University of North Carolina, Chapel Hill

NCT06740162

RECRUITING NA 180 participants

Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders

Children's Hospital Medical Center, Cincinnati

NCT06227780

RECRUITING NA 92 participants

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

Rush University Medical Center

NCT06139172

RECRUITING NA 90 participants

Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG-Preschool Age

Children's Hospital Medical Center, Cincinnati

NCT05957549

RECRUITING NA 60 participants

Speech-in-noise Perception in Autism and Fragile X

Children's Hospital Medical Center, Cincinnati

NCT06088589

RECRUITING Phase 2 60 participants

Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)

Mirum Pharmaceuticals

NCT07209462

RECRUITING NA 30 participants

Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant

Children's Hospital Medical Center, Cincinnati

NCT06560242

ACTIVE NOT RECRUITING Phase 3 314 participants

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

Tetra Discovery Partners

NCT05367960

ACTIVE NOT RECRUITING Phase 2 45 participants

Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

Children's Hospital Medical Center, Cincinnati

NCT05418049

COMPLETED Phase 3 125 participants

Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome

Seaside Therapeutics

NCT01282268

COMPLETED 96 participants

Pediatric Patients With Metabolic or Other Genetic Disorders

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

NCT02769949

COMPLETED NA 66 participants

Trial of Minocycline to Treat Children With Fragile X Syndrome

University of California, Davis

NCT01053156

COMPLETED Early Phase 1 29 participants

Neurophysiological and Acute Pharmacological Studies in FXS Patients

Children's Hospital Medical Center, Cincinnati

NCT02998151

COMPLETED Phase 1 21 participants

Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)

Novartis Pharmaceuticals

NCT01482143

COMPLETED Phase 1 15 participants

An Initial Study of AZD7325 in Adults With Fragile X Syndrome

Children's Hospital Medical Center, Cincinnati

NCT03140813

COMPLETED Phase 2 15 participants

ERG/5-HTP in Fragile X Syndrome (FXS)

Elizabeth Berry-Kravis

NCT05030129

COMPLETED Phase 2 14 participants

Acamprosate in Youth With Fragile X Syndrome

Indiana University

NCT01300923

Phase Distribution

PhaseTrial count
Early Phase 1 3
Phase 2 4
Phase 3 2

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Fragile X Syndrome Trial Landscape

ClinicalTrials.gov lists 17 US studies indexed under Fragile X Syndrome, and 7 of those are currently open to recruitment — roughly 41% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

The phase distribution for Fragile X Syndrome shows 2 late-stage studies (Phase 3 and Phase 4 combined) alongside 7 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

Top sponsor activity for Fragile X Syndrome is led by Children's Hospital Medical Center, Cincinnati with 7 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 17 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Fragile X Syndrome?

PlainTrial tracks 17 US clinical trials for Fragile X Syndrome, of which 7 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Fragile X Syndrome?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

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