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17 Fragile X Syndrome Trials
Power is an online platform that helps thousands of Fragile X Syndrome patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Cannabidiol Gel for Fragile X Syndrome
Tulsa, OklahomaZYN002 is a pharmaceutically manufactured Cannabidiol that is developed as a clear gel that can be applied to the skin (called transdermal delivery).
The gel will be applied to clean, dry, intact skin of the shoulders and/or upper arms.
Participants from the ZYN2-CL-016 and ZYN2-CL-033 studies who meet the inclusion criteria and none of the exclusion criteria for study ZYN2-CL-017 are eligible.
Parents/caregivers will apply the study gel twice daily for the 52-week treatment period.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2, 3
Age:3 - 24
Sex:All
550 Participants Needed
Cannabidiol Gel for Fragile X Syndrome
Tulsa, OklahomaThis is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with Fragile X Syndrome (FXS). Eligible participants will participate in up to an 18-week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to \< 30 years will be eligible to participate.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:3 - 29
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Drug Abuse, Cardiovascular Disease, Others
Must Be Taking:Antiseizure, Psychoactive
250 Participants Needed
BPN14770 for Fragile X Syndrome
Kansas City, KansasThis trial is testing a medication called BPN14770 in men aged 18 to 45 who have Fragile X Syndrome. The medication aims to improve brain function by balancing chemicals that help with thinking and memory.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18 - 45
Sex:Male
Key Eligibility Criteria
Disqualifiers:Renal Impairment, Hepatic Impairment, Substance Abuse, Others
150 Participants Needed
Zatolmilast for Fragile X Syndrome
Kansas City, KansasThis trial is testing a medication called BPN14770 in boys with fragile X syndrome. The study aims to see how the medication moves through their bodies and if it helps with their symptoms.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:9 - 18
Sex:Male
Key Eligibility Criteria
Disqualifiers:Cardiovascular, Renal, Hepatic, Psychiatric, Others
150 Participants Needed
BPN14770 for Fragile X Syndrome
Kansas City, KansasThis trial is testing a drug called BPN14770 to see if it is safe for extended use in males with fragile X syndrome. The study includes those who have already participated in earlier trials of the drug. BPN14770 aims to help by improving brain function related to the condition. It has been previously studied for its potential to improve cognitive function and behavioral outcomes in patients with fragile X syndrome.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 3
Age:9 - 45
Sex:Male
300 Participants Needed
Physical Activity Program for Intellectual Disability
Fayetteville, ArkansasPurpose: Conduct a wait-list randomized controlled trial (RCT) of an inclusive physical activity program called PACE for adults with intellectual disability (ID) who are not yet showing signs of Alzheimer's Disease (AD)/age-related dementias (ARD).
Participants: Participants include 120 adults with ID, their caregivers, and their coaches (up to 360 individual participants, grouped as triads), recruited through the University of North Carolina at Chapel Hill and the University of Arkansas. Participants also include 16 exercise professionals.
Procedures (methods): Each cohort will include 20 triads who are randomly assigned to the PACE program or the waitlist control group.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Alzheimer's, Dementia, Others
376 Participants Needed
Methylphenidate for Intellectual Disability and ADHD
Chicago, IllinoisThis study is a randomized, double-blind, placebo-controlled, crossover trial of extended-release liquid methylphenidate (XRMPH) to evaluate the sensitivity of the NIH Toolbox Cognition Battery (NIHTB-CB) to changes in cognition in children and adolescents ages 6 to 17 with intellectual disability (D) and comorbid Attention Deficit Hyperactivity Disorder (ADHD). The sample will include 68 males or females (expected male: female ratio of 1.8:1 with ID and ADHD as determined by structured diagnostic interview and Conners 3 scores. Additional inclusion criteria will include Full Scale IQ above 50 and mental age greater than or equal to 3 years. In addition, participants must be able to complete NIHTB-CB testing and provide valid scores at baseline. After baseline testing, participants will then be randomized to drug or placebo in a 1:1 ratio (N=34 per group) at the end of the baseline visit. XRMPH in oral suspension supplied as Quillivant XR in 5 mg/ml (Tris Pharma, Monmouth Junction, NJ) will be the active treatment. The XRMPH or matching placebo will be started at a dose of 0.3 mg/kg/day and individually titrated over two weeks. Phone calls at the end of weeks 1, 2, and 3 will be used to collect adverse event and response data. If there is no evidence of side effects and ongoing symptoms of ADHD, the dose will be increased to 0.5 mg/kg/day at one week and 0.7 mg/kg/day at 2 weeks (maximum dose of 60 mg per day consistent with FDA labeled use in youth). The Clinical Global Impression (CGI) will be used as a guide to define optimal dose. If side effects occur the dose will be reduced to the dose level at which there were no side effects. Final optimal dose will be established by the end of week 3 and this will be maintained for 2 weeks until 5 weeks post randomization, at which time the follow-up parent and teacher Conners scales, NIHTB-CB, Go/No-Go, and PedsQL will be completed. Participants will have a washout period of 1 week, will then complete re-assessment at the second baseline, and then will cross over to the other treatment (Quillivant to placebo; placebo to Quillivant), also in a double-blind fashion. In the second treatment arm, patients will have the same titration, monitoring and treatment periods as in the first arm, again followed by repeated assessments at the conclusion of 5 weeks. The accrual of participants and number of visits is shown in the Timeline per 6-month period.
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 1
Age:6 - 24
Sex:All
68 Participants Needed
Virtual Therapy for Developmental Disabilities
Chicago, IllinoisThe purpose of this study is to evaluate the effectiveness of an adapted, telehealth functional behavioral therapy (FBTsIDD) specifically focused on promoting appropriate communication and behavioral strategies in individuals with syndromic intellectual and developmental disorders.
Participants will be asked to complete virtual study assessments at intake and then on a monthly basis for the duration of 3-6 months. In addition, participants will attend weekly or biweekly virtual intervention visits with a study therapist.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:2 - 12
Sex:All
Key Eligibility Criteria
Disqualifiers:High Aggression, Medical Instability, Others
92 Participants Needed
Speech Discrimination for Fragile X Syndrome
Cincinnati, OhioIndividuals with Fragile X Syndrome show differences in how they understand and learn language from infancy. They frequently have lifelong delays in speech and language as well. In addition, they experience other auditory symptoms, including being very sensitive to certain sounds as well as being more sensitive than others to loud sounds. The underlying brain activity for sound perception and speech learning in Fragile X is not well understood, especially in the infant and toddler years. This study uses behavioral assessment of speech and language abilities, neuroimaging, and hearing tests to understand how speech and hearing are different in children with Fragile X Syndrome.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:6 - 26
Sex:All
Key Eligibility Criteria
Disqualifiers:Seizures, Premature Birth, Others
30 Participants Needed
Alpha Auditory Entrainment for Fragile X Syndrome
Cincinnati, OhioFragile X Syndrome (FXS) is a complex neurodevelopmental disorder caused by a mutation on the X chromosome. Scientists have investigated FXS extensively in both humans and animals. Thus far, phenotypic rescue in animal models has not resulted in treatment breakthroughs in humans, though some important discoveries have been made. Research has shown that individuals with FXS process sounds differently than those in the typical population, and they also show baseline differences in brain activity, including high gamma activity, increased theta activity, and decreased alpha activity. The investigators' central hypothesis is that these alterations in brain activity (specifically alpha and gamma activity) impair the brain's ability to process new information, thereby impeding cognitive functioning and increasing sensory sensitivity. The investigators propose that auditory entrainment, a technique that involves playing special sounds through headphones, will normalize brain activity in individuals with FXS and lead to increased cognitive function and decreased sensory hypersensitivity.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:5 - 10
Sex:All
Key Eligibility Criteria
Disqualifiers:Auditory Impairments, Visual Impairments, Substance Abuse, Others
180 Participants Needed
Speech Perception for Autism and Fragile X Syndrome
Cincinnati, OhioThe goal of this study is to identify which brain regions are active during speech-in-noise perception, as well as how those regions interact. The investigators are studying brain activation during speech-in-noise in autism and controls as well as individuals with Fragile X Syndrome. The main question\[s\] it aims to answer are: 1) How does the brain's response to background noise affect a person's ability to understand speech? 2) Can visual cues improve hearing in background noise?
Participants will complete the following:
* hearing tests
* cognitive and behavioral measures
* questionnaires about their symptoms
* both passive and active hearing tasks while brain activity is recorded with a neuroimaging cap Results will be compared between individuals with autism with and without Fragile X Syndrome as well as individuals without autism.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:15 - 35
Sex:All
Key Eligibility Criteria
Disqualifiers:Hearing Loss, Uncorrected Vision, Others
Must Not Be Taking:EEG-affecting Medications
60 Participants Needed
Single Dose Drug Challenge for Fragile X Syndrome
Cincinnati, OhioThe aim of this study is to utilize neurophysiologic assessments, behavioral measures and clinical measures to assess how much deficits associated with Fragile X Syndrome from pre-dose to post-dose using pharmacology.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18 - 45
Sex:All
Key Eligibility Criteria
Disqualifiers:Substance Abuse, Epilepsy, Renal Impairment, Others
Must Be Taking:Psychotropic Drugs
45 Participants Needed
Speech Discrimination Assessments for Fragile X Syndrome
Cincinnati, OhioIndividuals with Fragile X Syndrome show differences in how they understand and learn language from infancy. They frequently have lifelong delays in speech and language as well. In addition, they experience other auditory symptoms, including being very sensitive to certain sounds as well as being more sensitive than others to loud sounds. The underlying brain activity for sound perception and speech learning in Fragile X is not well understood, especially in the infant, toddler, and preschool years. This study uses behavioral assessment of speech and language abilities, neuroimaging, and hearing tests to understand how speech and hearing are different in children with Fragile X Syndrome.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:24 - 4
Sex:All
Key Eligibility Criteria
Disqualifiers:Seizures, Premature Birth, Others
90 Participants Needed
SPG601 for Fragile X Syndrome
Cincinnati, OhioThis Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18 - 45
Sex:Male
10 Participants Needed
Sertraline for Anxiety in Neurodevelopmental Disorders
London, OntarioThere are currently no approved medications for the treatment of anxiety in children and youth with neurodevelopmental disorders (NDDs), both common and rare. Sertraline, a selective serotonin reuptake inhibitor, has extensive evidence to support its use in children's and youth with anxiety but not within NDDs. More research is needed to confirm whether or not sertraline could help improve anxiety in children and youth with common and rare neurodevelopmental conditions. This is a pilot study, in which we plan to estimate the effect size of reduction in anxiety of sertraline vs. placebo. across rare and common neurodevelopmental disorders, and determine the best measure(s) to be used as a primary transdiagnostic outcome measure of anxiety, as well as diagnosis specific measures in future, larger-scale clinical trials of anxiety in NDDs.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:8 - 17
Sex:All
Key Eligibility Criteria
Disqualifiers:Diabetes, HIV, Hepatitis, Others
Must Not Be Taking:SSRIs, MAOIs, Pimozide, Others
130 Participants Needed
PIXI for Neurogenetic Disorders
Research Triangle Park, North CarolinaThis trial tests a program called PIXI, which helps parents of infants with genetic disorders. The program educates parents about their baby's condition, coaches them on how to interact with their baby, and provides family support. The goal is to improve the baby's development and family well-being.
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Unphased
Age:< 99
Sex:All
120 Participants Needed
Metformin for Fragile X Syndrome
Edmonton, AlbertaThis trial tests metformin in people with fragile X syndrome aged 6 to 35 years. Metformin, a diabetes medication, may help improve their behavior, language skills, and control their appetite by balancing brain signals. The study will assess its safety and effectiveness over several months. Metformin, commonly used for diabetes, has shown promise in improving cognitive and behavioral issues in fragile X syndrome based on preliminary studies.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:6 - 35
Sex:All
Key Eligibility Criteria
Disqualifiers:Non-cooperative, Major Illness, Metabolic Acidosis, Others
Must Not Be Taking:Metformin
120 Participants Needed
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Frequently Asked Questions
How much do Fragile X Syndrome clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Fragile X Syndrome clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Fragile X Syndrome trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Fragile X Syndrome is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Fragile X Syndrome medical study ?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Fragile X Syndrome clinical trials ?
Most recently, we added Physical Activity Program for Intellectual Disability, Sertraline for Anxiety in Neurodevelopmental Disorders and SPG601 for Fragile X Syndrome to the Power online platform.