Condition

Fragile X Syndrome clinical trials and research

• 10Clinical trials
• 53NIH research projects
• 5Countries

Newest Fragile X Syndrome clinical trials

All recruiting trials (10)

• CTH120 in Fragile X syndrome: safety and early effectiveness in adult males

  This study tests CTH120 versus placebo to see if it is safe and helps adults with Fragile X syndrome.

  Recruiting at 2 sites Phase 2 Connecta Therapeutics, S.L. · Barcelona, Barcelona and 1 other locations · NCT07654114

• MRM-3379 for males with Fragile X Syndrome (BLOOM)

  This trial will test three doses of the oral drug MRM-3379 in males with Fragile X Syndrome aged 16–45 (placebo-controlled), with a parallel open-label group for ages 13–15.

  Recruiting at 17 sites Phase 2 Mirum Pharmaceuticals, Inc. · Phoenix, Arizona and 17 other locations · NCT07209462

• Inclusive physical activity program for adults with intellectual disabilities

  This study is testing a new fitness program for adults with intellectual disabilities to see if it helps them be more active and feel better over 16 weeks.

  Recruiting University of North Carolina, Chapel Hill · Fayetteville, Arkansas and 1 other locations · NCT06740162

• Understanding sound perception in infants with Fragile X Syndrome

  This study tests how infants and toddlers with Fragile X Syndrome hear and understand sounds compared to other children to help identify early signs of language delays.

  Recruiting Children's Hospital Medical Center, Cincinnati · Cincinnati, Ohio · NCT06560242

• Cannabidiol for Anxiety and GABA Function in Fragile X Syndrome

  This study tests a cannabidiol oral solution to see if it helps anxiety and brain signaling in people with Fragile X syndrome.

  Recruiting Phase 2 Université de Sherbrooke · Sherbrooke, Quebec · NCT06261502

• Using sound to improve cognitive function and sensory sensitivity in youth with developmental disorders

  This study is testing if listening to special sounds can help improve thinking skills and reduce sensitivity to noise in children with Fragile X Syndrome and Autism Spectrum Disorder.

  Recruiting Children's Hospital Medical Center, Cincinnati · Cincinnati, Ohio · NCT06227780

• Non-invasive prenatal diagnosis for single gene disorders

  This study is testing a new way to safely diagnose single-gene disorders in unborn babies using a simple blood test from the mother, starting as early as 9 weeks into the pregnancy.

  Recruiting Assistance Publique - Hôpitaux de Paris · Paris · NCT06147414

• Comparing Sertraline and Placebo for Treating Anxiety in Children with Neurodevelopmental Disorders

  This study is testing if the medication sertraline can help reduce anxiety in children and teens aged 8-17 with neurodevelopmental disorders like autism and Fragile X syndrome compared to a placebo.

  Recruiting at 6 sites Phase 2 Holland Bloorview Kids Rehabilitation Hospital · Calgary, Alberta and 6 other locations · NCT06081348

• Understanding sound perception in preschoolers with Fragile X Syndrome

  This study is trying to see how preschoolers with Fragile X Syndrome hear and understand sounds and language compared to other kids, to help identify early signs of speech and language challenges.

  Recruiting Children's Hospital Medical Center, Cincinnati · Cincinnati, Ohio · NCT05957549

• Investigating psychiatric and cognitive traits in specific genetic syndromes

  This study is testing how certain medications can help improve mental and thinking skills in people with Velocardiofacial Syndrome, Williams Syndrome, and Fragile X Syndrome.

  Recruiting Phase 4 The Chaim Sheba Medical Center · Petah Tikva · NCT00768820

NIH-funded research (newest 40 of 53)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Understanding Fragile X Syndrome: How it progresses and affects daily life

  A long-term study using existing Fragile X data to map how the condition changes over time in children and young adults, including how autism, race, and location influence outcomes.

  NIH research Rush University Medical Center · Chicago, United States · NIH-11623438

• Sleep and mood links in Fragile X Syndrome

  A short, patient-friendly study to understand how sleep problems relate to mood and depressive symptoms in people with Fragile X–linked autism.

  NIH research Wake Forest University Health Sciences · Winston-Salem, United States · NIH-11456853

• Connecting 3D genome misfolding to gene silencing in Fragile X syndrome

  A study in people with Fragile X syndrome focuses on how 3D genome folding errors may turn off important genes, contributing to the condition’s features.

  NIH research Washington University · Saint Louis, United States · NIH-11445523

• Cortical circuit problems in Fragile X mice and people

  Researchers are studying how brain circuits go awry in Fragile X syndrome by comparing mice and people to understand why certain brain rhythms and responses are altered.

  NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11419234

• Brain biology in fragile X syndrome: how brain signals unfold and vary in patients

  This grant supports research to understand brain signals and why symptoms vary in people with fragile X syndrome, aiming to translate findings into better care.

  NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11419206

• Brain research on Fragile X Syndrome to improve therapies

  A multi-year program studies brain function and differences in Fragile X Syndrome to help develop better treatments for people with FXS.

  NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11419164

• Understanding anxiety in Fragile X syndrome across childhood

  A study follows children and young people with Fragile X syndrome to understand how anxiety starts, stays, and what predicts it over time.

  NIH research University of South Carolina at Columbia · Columbia, United States · NIH-11412542

• Family genetics linking autism and fragile X syndrome

  The team looks at whether changes in the FMR1 gene are linked to social language difficulties in people with autism, fragile X syndrome, and their family members.

  NIH research Northwestern University · Chicago, United States · NIH-11403972

• Therapeutic potential of rescuing mis-splicing in Fragile X Syndrome

  A patient-centered look at using antisense therapy to fix mis-spliced FMR1 RNA in Fragile X Syndrome.

  NIH research Univ of Massachusetts Med Sch Worcester · Worcester, United States · NIH-11369805

• Fragile X syndrome in the human brain

  A study using post-mortem human brain tissue to better understand Fragile X syndrome and autism at the cellular level, aiming to uncover clues for future therapies.

  NIH research University of Colorado Denver · Aurora, UNITED STATES · NIH-11369399

• How brain support cells (astroglia) contribute to Fragile X syndrome

  Researchers are looking at how changes in brain support cells and small RNA molecules may drive symptoms in people with Fragile X syndrome.

  NIH research Tufts University Boston · Boston, United States · NIH-11358362

• Adults with Fragile X Syndrome: Health and Life Course Across Adulthood

  A long-term study following adults with Fragile X Syndrome to understand how their health and daily lives change from early adulthood through older age.

  NIH research University of Wisconsin-Madison · Madison, United States · NIH-11345941

• Therapeutic targeting of protein balance in Fragile X Syndrome

  A research project at Boston Children's Hospital investigates how imbalances in protein production and degradation in brain cells contribute to Fragile X Syndrome and whether adjusting this balance could reduce symptoms.

  NIH research Boston Children's Hospital · Boston, United States · NIH-11344169

• Brain circuit causes of social touch sensitivity in Fragile X

  This work looks at how problems in brain wiring lead to strong dislike or fear of social touch in people with Fragile X syndrome and related autism.

  NIH research University of California Los Angeles · Los Angeles, United States · NIH-11325052

• Predicting Fragile X Syndrome severity with new long-read sequencing markers

  A small R21 study will explore genetic and molecular markers from long-read sequencing to help forecast how severely Fragile X affects a patient.

  NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11312781

• R-loop target to reactivate FMR1 in Fragile X syndrome

  A patient-friendly approach aims to reactivate the FMR1 gene in people with Fragile X syndrome using a novel genetic strategy.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11295216

• NMDA receptor control of rapid network changes in fragile X-related autism

  This project explores how NMDA receptors shape the fast changes in spinal networks that may contribute to autism spectrum disorder, using fragile X as a model.

  NIH research Emory University · Atlanta, United States · NIH-11288244

• Problems in specific brain cells linked to Fragile X symptoms

  This research looks at whether problems with certain brain cells called parvalbumin interneurons lead to sensory sensitivity in people with Fragile X Syndrome.

  NIH research University of California Los Angeles · Los Angeles, United States · NIH-11284007

• Early short‑lived brain connections in Fragile X syndrome

  This project looks at how early‑life brain wiring shapes later development in Fragile X syndrome and whether changing that wiring could help children with FXS.

  NIH research Cold Spring Harbor Laboratory · Cold Spring Harbor, United States · NIH-11262944

• Reelin protein in Fragile X syndrome

  The team is testing whether boosting a brain protein called Reelin can improve brain connections and help people with Fragile X syndrome.

  NIH research University of South Florida · Tampa, United States · NIH-11253286

• Understanding brain function in Fragile X Syndrome

  This study is looking at how Fragile X Syndrome affects the brain in both mice and people to find out what causes the challenges faced by those with the condition, with the goal of creating better treatments and understanding for everyone affected by FXS.

  NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11179534

• Understanding how sensory issues affect social touch in Fragile X Syndrome

  This study is looking at how people with Fragile X Syndrome feel and react to different kinds of social touch, like hugs or handshakes, to help us understand their social interactions better and find ways to support them.

  NIH research University of California Los Angeles · Los Angeles, United States · NIH-11144607

• Health and Life Journeys for Adults with Fragile X Syndrome

  This project looks at the health and daily lives of adults with Fragile X Syndrome to understand how their condition changes over time.

  NIH research University of Wisconsin-Madison · Madison, United States · NIH-11128632

• Understanding brain development in fragile X syndrome

  This study is looking at how the brain connections of young children with fragile X syndrome, a common cause of autism, develop and how their sensory experiences can help improve treatments, with the hope of finding better ways to support these kids as they grow.

  NIH research Cold Spring Harbor Laboratory · Cold Spring Harbor, United States · NIH-11126842

• Understanding Fragile X Syndrome as Children and Young Adults Grow

  This project collects detailed information about how Fragile X Syndrome affects children and young adults over time to help create better support and treatments.

  NIH research Rush University Medical Center · Chicago, United States · NIH-11126511

• Hearing and sound‑localization problems in Fragile X syndrome

  This work looks at whether reduced myelin (nerve insulation) causes trouble hearing and locating sounds in people with Fragile X syndrome.

  NIH research Oklahoma State University Stillwater · Stillwater, United States · NIH-11125625

• Understanding social and spatial thinking challenges in Fragile X syndrome using rodent models

  This study is looking at how Fragile X syndrome impacts social skills and memory by examining the brains of mice, hoping to uncover what causes these challenges so we can better understand the condition.

  NIH research University of Texas at Austin · Austin, United States · NIH-11098452

• New Treatment for Fragile X Syndrome by Correcting a Gene Mistake

  This research explores a new way to treat Fragile X Syndrome by fixing a specific error in the FMR1 gene using special genetic tools.

  NIH research Univ of Massachusetts Med Sch Worcester · Worcester, United States · NIH-11093322

• Understanding how 3D genome misfolding affects gene expression in fragile X syndrome

  This study is looking into how changes in the structure of our genes might cause issues in people with fragile X syndrome, which can lead to challenges like social anxiety and learning difficulties, and it aims to find new ways to help improve their lives.

  NIH research University of Pennsylvania · Philadelphia, United States · NIH-11092845

• How anxiety starts and what predicts it in Fragile X syndrome

  This project looks at how and when anxiety begins and changes over time in children and adolescents with Fragile X syndrome to find early warning signs.

  NIH research University of South Carolina at Columbia · Columbia, United States · NIH-11090419

• Understanding how gene silencing affects fragile X syndrome

  This study is looking into how changes in a specific gene related to fragile X syndrome might affect the way genes work, which could help us understand more about the condition and its symptoms.

  NIH research University of Pennsylvania · Philadelphia, United States · NIH-11081615

• Investigating the role of Reelin in Fragile X syndrome

  This study is looking at how a protein called Reelin might help improve symptoms of Fragile X syndrome, which affects thinking and behavior, and it hopes to find new ways to treat this condition through gene therapy.

  NIH research University of South Florida · Tampa, United States · NIH-11073783

• Understanding brain cell dysfunction in Fragile X Syndrome

  This study is looking at how certain brain cells work differently in people with Fragile X Syndrome, which might help us understand why they have trouble processing sensory information, and it could lead to new ways to help improve these challenges.

  NIH research University of California Los Angeles · Los Angeles, United States · NIH-11046639

• Understanding how glial cells affect hearing in a mouse model of Fragile X syndrome

  This study looks at how certain brain cells in mice with Fragile X syndrome affect hearing and sound processing, hoping to find out why some people with this condition have trouble with sensory experiences.

  NIH research Washington University · Saint Louis, United States · NIH-11045062

• Understanding how mothers and infants with Fragile X Syndrome interact

  This study looks at how mothers and their babies with Fragile X Syndrome connect and interact, comparing these moments to those between mothers and babies who develop typically, to help understand the unique challenges these families face and improve support for them.

  NIH research University of South Carolina at Columbia · Columbia, United States · NIH-11039921

• Investigating how SETD2 affects brain function and its link to Fragile X Syndrome

  This study is looking at how a gene called SETD2 affects brain function and behavior in conditions like autism and Fragile X Syndrome, using mice to learn more about how changes in this gene might help us understand these disorders better and improve care for patients.

  NIH research Ut Southwestern Medical Center · Dallas, United States · NIH-11025896

• Understanding the development and challenges of Fragile X Syndrome in children and young adults

  This study is looking to learn more about how children and young adults with Fragile X Syndrome grow and develop, while also understanding how it affects their families, so we can improve support and services for everyone involved.

  NIH research Rush University Medical Center · Chicago, United States · NIH-11013778

• Understanding how microRNA changes affect Fragile X Syndrome

  This study is looking at how tiny molecules called microRNAs are involved in Fragile X Syndrome, a condition that affects learning and behavior, to help find new ways to improve the lives of those who have it.

  NIH research Emory University · Atlanta, United States · NIH-10934352

• Investigating how certain brain receptors affect brain function in Fragile X Syndrome

  This study is looking at how certain brain receptors might affect people with Fragile X Syndrome and autism, especially regarding their sensitivity to sensory experiences, and it aims to find new ways to help improve brain function through testing different treatments.

  NIH research Ut Southwestern Medical Center · Dallas, United States · NIH-10926838

• Understanding how RNA affects gene regulation in Fragile X syndrome

  This study is looking at how RNA affects genes linked to Fragile X syndrome, which can cause developmental challenges, to help us understand the condition better and find new ways to improve treatments for those affected.

  NIH research University of Pennsylvania · Philadelphia, United States · NIH-10912731

Related topics

Quick answers

How many Fragile X Syndrome clinical trials are recruiting right now?

10, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 5 countries. The most sites are in United States (5 trials), Canada (2 trials), Spain (2 trials), Israel (1 trial), France (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 4, Phase 4: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

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