Clinical Trials in Fragile X Syndrome
To all who have loved ones impacted by Fragile X syndrome, we sincerely thank you for your interest in clinical trials. These trials are beacons of hope during challenging times, and your curiosity and engagement are instrumental in pushing this important research forward.
As a further resource, we offer an enlightening video discussion featuring Holly Roos and Katie Clapp, both mothers to young adults with Fragile X and key figures in FRAXA. Their personal experiences provide unique and heartfelt perspectives on the journey through Fragile X clinical trials.
We understand that you might have questions about what participating in a clinical trial entails. Each study is supervised by a medical professional specializing in Fragile X syndrome. Participation is free, with no need to inform your insurance provider and the freedom to withdraw at any time. Typically, travel expenses are covered.
It's important to know that the FDA usually mandates two successful large-scale clinical trials before considering the approval of a new treatment, however for rare conditions like Fragile X syndrome, one trial with strong results may be sufficient. Upon successful trials and subsequent FDA approval, new drugs become accessible to others diagnosed with Fragile X syndrome, hopefully improving the quality of life for many individuals and families.
Ongoing Fragile X Studies Accepting Participants
FRAXA Research Foundation Joins COMBINEDBrain Consortium for Fragile X Biomarker Research
Help accelerate research on Fragile X syndrome biomarkers by contributing samples to the COMBINEDBrain Consortium’s project. Contact Katie Clapp at FRAXA Research Foundation to learn how you can participate.
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Recruiting: BRIDGE Study (BRain Indicators of Developmental Growth)
This study from the Wilkinson Lab at Boston Children’s Hospital is investigating how differences in brain activity affect learning, language and behavior in children with Fragile X syndrome, Down syndrome, and Autism Spectrum Disorder. One of the goals is to find brain markers that predict cognitive, language, and behavioral difficulties in these groups. Another goal is to better understand the differences in brain activity between young children with and without Fragile X and Down Syndrome, and whether these differences are similar in children with Autism Spectrum Disorder.
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Recruiting: Clinical Study of Non-Invasive EEG for Children Ages 2-7
Dr. Carol Wilkinson, MD PhD, and Dr. Charles Nelson, PhD, at Boston Children’s Hospital are recruiting children ages 2-7 years with Fragile X syndrome to participate in a study of brain differences using non-invasive EEG.
Read More »Additional Fragile X Clinical Trials
Please visit clinicaltrials.gov for a complete listing of Fragile X syndrome clinical trials.
Completed Fragile X Trials: A Journey Towards Progress
Shionogi’s EXPERIENCE Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome
Shionogi’s EXPERIENCE clinical trials for Fragile X syndrome are nearing completion. Enrollment for the adult trial (EXPERIENCE-301) is now closed, while the adolescent trial (EXPERIENCE-204) is in its final phase. Learn more about the study, FRAXA’s role, and the open-label extension.
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Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home Option
Harmony Biosciences is now recruiting for the RECONNECT Phase 3 clinical trial of ZYN002, a potential treatment for Fragile X syndrome. The trial offers an at-home participation option, making it accessible for patients across the US, Australia, and the UK. Join us in this groundbreaking study and contribute to the future of Fragile X syndrome treatment.
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Pharmacologically Activating mGluR7 as a Novel Therapy for Fragile X Syndrome
Join Dr. Tsai and Dr. Kumar on a journey into novel treatment avenues for Fragile X syndrome. Learn how activating mGluR7 could be a game-changer, opening up uncharted therapeutic territory.
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Fragile X Clinical Trial of New PDE4D Inhibitor from Tetra
With a $200,043 grant from FRAXA Research Foundation, Dr. Elizabeth Berry-Kravis completed a successful Phase 2 clinical trial of a PDE4 inhibitor for adult men with Fragile X syndrome. This trial treated 30 males, 18-45 years of age with a new PDE4D allosteric inhibitor from Tetra Discovery Partners using a crossover design, so that everyone got active drug for part of the time and placebo for part of the time.
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Recruiting: Clinical Study of Non-Invasive EEG for Children Ages 2-7
Dr. Carol Wilkinson, MD PhD, and Dr. Charles Nelson, PhD, at Boston Children’s Hospital are recruiting children ages 2-7 years with Fragile X syndrome to participate in a study of brain differences using non-invasive EEG.
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Lovamix: Clinical Trial of Combined Treatment of Minocycline and Lovastatin in Fragile X Syndrome
With a $66,714 grant from the FRAXA Research Foundation awarded over 2015-2017, Dr. Francois Corbin at the Universite of Sherbrooke will test the safety and synergistic effects of lovastatin and minocycline in patients with Fragile X syndrome.
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Positive Results Reported in Phase II Fragile X Clinical Trial of PDE4D Inhibitor Zatolmilast from Tetra Therapeutics
Today, Tetra Therapeutics announces the first unequivocally positive phase 2 clinical trial in Fragile X syndrome, press release below. The results do not depend on carving out a subset of patients or post hoc analysis.
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Fragile X Clinical Trial of AZD7325 in Adults
With a $51,000 grant from FRAXA Research Foundation, Dr. Craig Erickson conducting a double-blind, placebo-controlled clinical trial of AZD7325 in adults ages 18-50 with Fragile X syndrome at Cincinnati Children’s Hospital. The compound being studied is an investigational new drug from AstraZeneca that targets GABA (A) receptors.
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Clinical Trial of Ganaxolone in Patients with Fragile X Syndrome
With a $90,000 grant from FRAXA Research Foundation funded during 2014-2015, Dr. Frank Kooy and colleagues at the University of Antwerp are conducting a double blind crossover trial of ganaxolone in patients with Fragile X syndrome. Results of this study were mixed (see Marinus: Results from Phase 2 Exploratory Clinical Study Support Continued Development of Ganaxolone in Fragile X Syndrome.)
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Neuren’s Tofinetide Successful in Phase 2 Clinical Trial in Fragile X
We are pleased to share great news adapted from Neuren’s press release: Neuren’s phase 2 trial has successfully established proof of concept and provides a strong rationale for Neuren to move forward with developing trofinetide for Fragile X syndrome. In this initial small trial with a relatively short treatment period, trofinetide was very well tolerated, with the high dose (70 mg/kg twice daily) demonstrating a consistent pattern of clinical improvement, observed in both clinician and caregiver assessments.
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Phase 1 Clinical Trial of Mega Green Tea Extract in Fragile X Syndrome
With a $124,000 grant from the FRAXA Research Foundation from 2012-2014, Dr. Mara Dierssen and Dr. Rafael de la Torre conducted preclinical studies in Fragile X knockout mice and a clinical trial in Fragile X patients using Mega Green Tea Extract, which contains 45% by weight epigallocatechin gallate (EGCG).
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Pilot Clinical Trial of Lithium in Fragile X Shows Promising Results
With a $65,000 grant from FRAXA Research Foundation in 2005, Dr. Berry-Kravis at the Rush University Medical Center conducted a pilot clinical trial of lithium in 15 patients with Fragile X syndrome. Results published.
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Encouraging Results from First Trial of Minocycline in Fragile X
With a $40,000 grant from FRAXA, Dr. Carlo Paribello and his team at the Surrey Place Centre Fragile X clinic in Toronto, Ontario, ran an open label trial to see if minocycline can improve learning and reduce anxiety and behavioral problems in people with Fragile X. Twenty participants between the ages of 13 and 35 years took minocycline for two months.
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Clinical Trial of Aripiprazol in Fragile X Syndrome
With a FRAXA Research Foundation grant of $30,000 in 2006, Dr. Erickson conducted a pilot clinical trial of an available medicine, aripiprazole (brand-name Abilify). This was an open-label 12-week trial in 12 people ages 6–25 years with Fragile X. Results were promising, and published: 10 of the 12 participants showed behavioral improvements.
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Melatonin Clinical Trial in Fragile X
With a $60,000 grant from FRAXA Research Foundation from 1998-1999, Dr. Randi Hagerman and her team at the University of California studied the effects of different compounds on individuals with Fragile X syndrome, focusing specifically on melatonin. Results published.
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