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Home › Conditions › Fragile X Syndrome

Fragile X Syndrome

Part of X-Linked Intellectual Disability

2 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Fragile X Syndrome

18 trials tracked for Fragile X Syndrome: 3 in phase 3 or 4 and 3 with published results. The most-cited published study has 66 citations.

18Trials tracked
3Phase 3 & 4
0Recruiting
3With published results
Phase distribution
Phase 4 2 Phase 3 1 Phase 2 12 Phase 1 1 Other / NA 2
  1. Phase 3 Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) Completed · 66 cited
  2. Phase 4 Riluzole in Fragile X Syndrome Completed · 65 cited
  3. Phase 4 Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome Completed · 56 cited
  4. Phase 2 AFQ056 for Language Learning in Children With FXS Completed
  5. Phase 2 Ganaxolone Treatment in Children With Fragile X Syndrome Completed
  6. Phase 2 Trial of Sertraline to Treat Children With Fragile X Syndrome Completed
Show 12 more trials
  1. Phase 2 Acamprosate in Youth With Fragile X Syndrome Completed
  2. Phase 2 A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome Completed
  3. Phase 2 Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome Completed
  4. Phase 2 A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome Completed
  5. Phase 2 Randomized Controlled Study of Donepezil in Fragile X Syndrome Completed
  6. Phase 2 ERG/5-HTP in Fragile X Syndrome (FXS) Completed
  7. Phase 2 Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome Completed
  8. Phase 2 Study of SPG601 in Adult Men With Fragile X Syndrome Completed
  9. Phase 2 Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome Completed
  10. Phase 1 Neurophysiological and Acute Pharmacological Studies in FXS Patients Completed
  11. N/A Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome Completed
  12. N/A Trial of Minocycline to Treat Children With Fragile X Syndrome Completed

Showing the 18 most-cited and recently-updated of 18 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Fragile X Syndrome: what the trials found

New clinical data for Fragile X Syndrome includes the evaluation of SPG601 in a Phase 2 trial 1.

Several pharmacological interventions have been evaluated in established trials. Riluzole was assessed in a Phase 4 study 2, and Acamprosate was evaluated in both Phase 1 and Phase 2 trials, with the latter showing changes in neuropsychological status scores at 4 hours post-dose [10, 16].

The intervention AFQ056 demonstrated significant results in behavioral symptoms; specifically, patients in Stratum I receiving 100 mg BID showed a change of 8.6 and -9.4 in ABC-CFX scores, while various doses in Stratum II also showed reductions in total scores 13. However, other trials for AFQ056 did not show statistically significant improvements in intentional communication or developmental quotients 7.

Lovastatin was evaluated in a Phase 4 trial regarding expressive language scores 3. Additionally, ZYN002 (Cannabidiol Transdermal Gel) showed a statistically significant reduction in the Social Avoidance Subscale during an ad hoc analysis (p=0.02), though it did not reach significance in the full analysis set for social avoidance or irritability 4.

Recent results — preliminary, needs further review

  • SPG601 is currently being evaluated in a Phase 2 trial 1.
  • BPN14770 (not yet corroborated) showed mixed results in crystallized cognition scores 5.
  • 5-Hydroxytryptomans (not yet corroborated) was assessed for clinical global impression and cognitive battery scores 6.
  • Ganaxolone (not yet corroborated) was evaluated regarding CGI-I and anxiety scales 8.
  • Sertraline (not yet corroborated) was evaluated in a Phase 2 trial for expressive language raw and standard T scores 9.

For the clinician treating this condition

  • Acamprosate has been established in Phase 1 and 2 trials as a studied intervention for neuropsychological status changes [10, 16].
  • AFQ056 showed evidence of reducing behavioral symptoms (ABC-CFX scores) in specific patient strata, though it did not significantly impact communication or developmental quotients in other cohorts [7, 13].
  • ZYN002 (Cannabidiol Transdermal Gel) demonstrated a statistically significant reduction in social avoidance in an ad hoc analysis, but results were not significant in the full analysis set for social avoidance or irritability 4.

AI synthesis of 10 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Females with Fragile X Syndrome exhibit high prevalence of anxiety, depression, and ADHD
Psychiatry Meta-analysis
Females with Fragile X Syndrome exhibit high prevalence of anxiety, depression, and ADHD High Rates of Anxiety and Depression in Fragile X Females
This systematic review and meta-analysis of 37 studies identifies significant psychiatric morbidity in females with Fragile X syndrome. Find…
Females with Fragile X syndrome show high rates of anxiety, depression, and ADHD, highlighting the need for specific support for this group.
Frontiers Jul 9, 2026
Arbaclofen Phase 3 Trials Establish Clinically Meaningful Change Thresholds in Fragile X Syndrome
Drug Pipeline Phase III
Arbaclofen Phase 3 Trials Establish Clinically Meaningful Change Thresholds in Fragile X Syndrome New Rules for Measuring Behavior Change in Fragile X
A review of two Phase 3 trials in 278 individuals with Fragile X Syndrome (aged 5-50 years) established clinically meaningful change thresho…
Imagine a parent watching their child struggle with daily routines.
Apr 24, 2026