Fragile X SyndromePhase 3RECONNECTNCT04977986
Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome
A Phase 3 study testing ZYN002 in people with Fragile X Syndrome and Fragile X Syndrome (FXS), run by Harmony Biosciences Management, Inc. at 27 sites in 5 countries.
Trial summary
This 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). Read more about this study
- Who is it for?
- Ages 3 to 29 years
- Conditions the study is for
- Fragile X Syndrome, Fragile X Syndrome (FXS)
- What is being tested?
- ZYN002
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 257 participants
- Where
- 27 sitesin 5 countries
- Study dates
- 13 Sep 2021to 29 Aug 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 29 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female children and adolescents aged 3 to < 30 years, at the time of Screening.
- Patient resides with caregiver who will continue to provide consistent care throughout the study.
- Judged by the Investigator to be in generally good health at Screening based upon the results of medical history, physical exam, 12-lead ECG and clinical laboratory test results. -Laboratory results outside the reference range must be documented as not clinically significant by both the Investigator and Sponsor.
- Participants must have a diagnosis of FXS through molecular documentation of full mutation of the FMR1 gene documented through genetic testing at Screening.
- Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of no more than two anti-seizure medications (ASMs) for the four weeks preceding study Screening; or must be seizure-free for one year if not currently receiving ASMs.
- Patients taking psychoactive medication(s) should be on a stable regimen of not more than three such medications for at least fours weeks preceding Screening and must maintain that regimen throughout the study. Psychoactive medications include (but are not limited to) antipsychotics, antidepressants, anxiolytics, attention-deficit / hyperactivity disorder (ADHD) medications, and medications for sleep.
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- If patients are receiving non-pharmacological, behavioral and/or dietary interventions, they must be stable and have been doing so for three months prior to screening.
- Patients have a body mass index between 12-30 kg/m2 (inclusive) and patients with a body mass index >30 kg/m2 and <40 kg/m2 with normal liver function laboratory values and with no immediate family history of fatty liver disease.
- Females of childbearing potential must have a negative serum pregnancy test at the Screening Visit and a negative serum or urine pregnancy test at all designated visits.
- Patients and parents/caregivers must be adequately informed of the nature and risks of the study and given written informed consent prior to Screening.
- Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures, and in the Investigator's opinion, are reliable and willing and able to comply with all protocol requirements and procedures.
Who cannot join
- Females who are pregnant, nursing or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception as outlined below for the duration of therapy and for three months after the last dose of study medication. Standard acceptable methods of contraception include abstinence (defined as refraining from heterosexual intercourse from screening to three months after the last dose of study medication: abstinence only applicable for females <18 years) or the use of a highly effective method of contraception, including hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, spermicide, vasectomy, or intrauterine device. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not an acceptable method of contraception.
- Patient has transitioned to independent living or living in a residential facility such as a university setting or congregate care.
- History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients.
- Exposure to any investigational drug or device less than or equal to 30 days prior to Screening or at any time during the study.
- Alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin levels greater than or equal to 2 times the upper limit of normal or alkaline phosphatase levels greater than or equal to 3 times the upper limit of normal.
- Use of cannabis or any THC or CBD-containing product within 3 months of Screening Visit or during the study (aside from ZYN002).
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- Patient has a positive drug screen, including ethanol, cocaine, THC, barbiturates, amphetamines (unless prescribed), benzodiazepines (except midazolam or comparable administered for blood draws and ECG collection), and opiates.
- Patient is using the following AEDs (medications for the treatment of seizures and/ or epilepsy): clobazam, phenobarbital, ethosuximide, felbamate, carbamazepine, phenytoin, or vigabatrin.
- Patient is using a strong inhibitor/inducer of CYP3A4 or sensitive substrate of CYP3A4 including but not limited to the following medications: midazolam (except single doses administered for the purposes of obtaining blood samples and ECG's), oral ketoconazole, fluconazole, nefazadone, rifampin, alfentanil, alfuzosin, amiodarone, cyclosporine, dasatinib, docetaxol, eplerenone, ergotamine, everolimus, fentanyl, halofantrine, irinotecan, lapatinib, levomethadyl, lumefantrine, nilotinib, pimozide, quinidine, ranolazine, sirolimus, tacrolimus, temsirolimus, toremifene, tretinioin, vincristine, vinorelbine, St. John's Wort, and grapefruit Juice/products.
- Patients may not be taking any benzodiazepines (except single doses administered for the purposes of obtaining blood samples and ECGs) at screening or throughout the study.
- Patient is expected to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
- Patient has an advanced, severe, or unstable disease that may interfere with the study outcome evaluations.
- Patient has acute or progressive neurological disease, psychosis, schizophrenia or any other psychiatric disorder or severe mental abnormalities (other than FXS) that are likely to require changes in drug therapy or interfere with the study objectives or ability to adhere to protocol requirements.
- Patient has a positive result for the presence of HBsAg, HCV, or HIV antibodies.
- Patient has known history of cardiovascular disease, advanced arteriosclerosis, structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, exercise-related cardiac events including syncope and pre-syncope, risk factors for Torsades de pointes (TdP) (e.g., heart failure, hypokalemia, family history of Long QT Syndrome), or other serious cardiac problems.
- Any clinically significant condition or abnormal findings at the Screening Visit that would, in the opinion of the Investigator, preclude study participation or interfere with the evaluation of the study medication.
- Any skin disease or condition including eczema, psoriasis, melanoma, acne, contact dermatitis, scarring, imperfections, lesions, tattoos, or discoloration that may affect treatment application, application site assessments or absorption of the trial drug.
- History of treatment for, or evidence of, drug abuse within the past year.
- Previous participation in a ZYN002 study (with the exception of patients who were screen failures in Study ZYN2-CL-016 and did not enter Study ZYN2-CL-017).
- Patient responds "yes" to Question 4 or 5 on the C-SSRS (Children) during Screening or at any time on study.
Trial locations and contacts
27 sites in 5 countries.
27 sites in 5 countries
United States18 sites
Rare Disease Research
Atlanta, Georgia, 30318
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Boston Children's Hospital
Boston, Massachusetts, 02115
Rush University Medical Center
Chicago, Illinois, 60612
Science 37
Culver City, California, 90230
Greenwood Genetic Center
Greenville, South Carolina, 29605
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
Amnova Clinical Research, LLC
Irvine, California, 92604
University of Mississippi
Jackson, Mississippi, 39216
University of Miami
Miami, Florida, 33136
University of Minnesota Fragile X Clinic (Voyager Clinic)
Minneapolis, Minnesota, 55454
The Fragile X Spectrum Disorder Clinic at Icahn School of Medicine at Mount Sinai, Division of Medical Genetics
New York, New York, 10029
Thompson Autism Center CHOC
Orange, California, 92868
Phoenix Children's Hospital
Phoenix, Arizona, 85016
UC Davis Health System, MIND Institute
Sacramento, California, 95817
Primary Children's Hospital
Salt Lake City, Utah, 84113
Central States Research
Tulsa, Oklahoma, 74136
Children's National Medical center
Washington D.C., District of Columbia, 20010
United Kingdom4 sites
University of Edinburgh
Edinburgh
Leicester Clinical Research
Leicester
King's College
London
Manchester University NHS Foundation Trust
Manchester
Australia3 sites
Lady Cilento Children's Hospital - South Brisbane
Brisbane, Queensland, 4101
Genetics Clinics Australia
Melbourne, Victoria, 3161
Westmead Children's Hospital
Sydney, New South Wales, 2145
New Zealand1 site
Health New Zealand - Te Whatu Ora - Wellington Hospital
Newtown, Wellington Region, 6021
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children, Adolescents and Young Adults With Fragile X Syndrome - RECONNECT
This 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.
Read the detailed description
The study will be comprised of a Screening visit and a combination of seven visits both onsite (face-to-face) and virtual. The assignment of study drug or placebo will be done by a computer-generated system and neither the study doctor, participant or their caregivers will know which treatment is being given to them. The dose of the treatment will depend on the weight of the participants. If the participants weigh less than or equal to 30 kg, they will receive 2 sachets of the gel per day (1 sachet approximately every 12 hours). If the participant weighs more than 30 kg but less than or equal to 50 kg, they will receive 4 sachets of gel per day (2 sachets approximately every 12 hours). Participants who weigh more than 50 kg will receive 6 sachets of gel per day (3 sachets approximately every 12 hours). Parents/ caregivers will be instructed on proper application of the gel. The gel will be applied to clean, dry, intact skin of the upper arms/ shoulders.
Blood samples will be collected for safety analysis of ZYN002. An independent analytical laboratory will also perform CGG repeat and methylation status analyses. Additionally, the parents/caregivers and study doctor will be asked to complete some questionnaires for efficacy and safety assessment.
- Conditions
- Fragile X Syndrome, Fragile X Syndrome (FXS)
- Being tested
- ZYN002, Placebo
- Keywords
- Human Genetics and Inherited Disorders, Other human genetics and inherited disorders
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZYN002Drug
Also called cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery)
Pharmaceutically manufactured cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery)
PlaceboDrug
Also called Placebo Comparator, Matching Placebo
Placebo formulated as a clear gel that can be applied to the skin (transdermal delivery)
Other Names:
Placebo Comparator Matching Placebo
| Study group | Role | What they receive |
|---|---|---|
| ZYN002 | Experimental | Pharmaceutically manufactured cannabidiol, which is formulated as a clear gel for transdermal delivery. Receives: Drug: ZYN002 |
| Placebo | Placebo comparator | Placebo is formulated as a clear gel for transdermal delivery. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 1 score in patients with complete methylation of the FMR1 gene.
The ABC-C FXS is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials
Measured: Change from Baseline to Week 18
Secondary outcomes (11)
Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 2 in patients with complete methylation of the FMR1 gene.
The ABC-C FXS is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials
Measured: Change from Baseline to Week 18
Change on the Caregiver Global Impression of Change (CaGI-C) for Pre-specified parameter among patients with complete methylation of the FMR1 gene.
The CaGI-C global impression of change is a 7-point Likert scale designed to measure behavioral symptomatic change at a specific time compared to baseline.
Measured: Change from Baseline to Week 18
Clinical Global Impression- Improvement (CGI-I) scale among patients with complete methylation of the FMR1 gene.
The CGI-I global improvement item is a 7-point Likert scale to rate the behavioral change in a child at a specified time compared to baseline.
Measured: Change from Baseline to Week 18
Change in ABC-C FXS pre-specified Subscale 1 among all randomized patients (complete and partial methylation of the FMR1 gene).
The ABC-C FXS is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials
Measured: Change from Baseline to Week 18
Number of patients with adverse events
Collection of adverse events
Measured: Day 1, Week 2, Week 4, Week 6, Week 10, Week 14, Week 18 and through 4-week post-dose telephone follow-up
Number of participants with abnormal physical and neurological exams
Physical and neurological exams
Measured: Screening, Day 1 and Week 18
Number of participants with abnormal clinical laboratory results
Laboratory tests and urinalysis
Measured: Screening, Week 10 and Week 18
Number of participants with abnormal vital sign results
Vital sign measurements (blood pressure, heart rate, respiratory rate and temperature)
Measured: Screening, Day 1, Week 2 and Week 18
Number of participants with abnormal ECG
12-lead ECG
Measured: Screening and Week 18
Withdrawal characteristics of ZYN002 using the Penn Physician Withdrawal Checklist
Penn Physician Withdrawal Checklist
Measured: Week 18 and 4-week post last dose
Skin tolerability as assessed using daily skin diary
Daily skin irritation diary
Measured: Daily from Day 1 through Week 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2021
- Study start13 Sep 2021
- Primary completion29 Aug 2025
- Study completion29 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, double-blind, placebo-controlled, multiple center study
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 257 participants (actual)
Sponsor and investigators
- Lead sponsor
- Harmony Biosciences Management, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04977986
- Sponsor's protocol ID
- ZYN2-CL-033
- Acronym
- RECONNECT
- Status last verified
- Aug 2026
- First submitted
- 20 Jul 2021
- Last updated
- 1 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children, Adolescents and Young Adults With Fragile X Syndrome - RECONNECT
- ClinicalTrials.gov ID
- NCT04977986
- Acronym / sponsor ID
- RECONNECT / ZYN2-CL-033
- Conditions
- Fragile X Syndrome, Fragile X Syndrome (FXS)
- Keywords
- Human Genetics and Inherited Disorders, Other human genetics and inherited disorders
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 257 participants (actual)
- Lead sponsor
- Harmony Biosciences Management, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Sep 2021 (actual)
- Primary completion
- 29 Aug 2025 (actual)
- Study completion
- 29 Aug 2025 (actual)
- First posted
- 27 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This 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.
Detailed description
The study will be comprised of a Screening visit and a combination of seven visits both onsite (face-to-face) and virtual. The assignment of study drug or placebo will be done by a computer-generated system and neither the study doctor, participant or their caregivers will know which treatment is being given to them. The dose of the treatment will depend on the weight of the participants. If the participants weigh less than or equal to 30 kg, they will receive 2 sachets of the gel per day (1 sachet approximately every 12 hours). If the participant weighs more than 30 kg but less than or equal to 50 kg, they will receive 4 sachets of gel per day (2 sachets approximately every 12 hours). Participants who weigh more than 50 kg will receive 6 sachets of gel per day (3 sachets approximately every 12 hours). Parents/ caregivers will be instructed on proper application of the gel. The gel will be applied to clean, dry, intact skin of the upper arms/ shoulders.
Blood samples will be collected for safety analysis of ZYN002. An independent analytical laboratory will also perform CGG repeat and methylation status analyses. Additionally, the parents/caregivers and study doctor will be asked to complete some questionnaires for efficacy and safety assessment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Randomized, double-blind, placebo-controlled, multiple center study
| Arm | Type | Receives |
|---|---|---|
| ZYN002 | Experimental | Drug: ZYN002Pharmaceutically manufactured cannabidiol, which is formulated as a clear gel for transdermal delivery. |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo is formulated as a clear gel for transdermal delivery. |
Who may qualify
- Ages
- 3 to 29 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Male or female children and adolescents aged 3 to < 30 years, at the time of Screening.
- Patient resides with caregiver who will continue to provide consistent care throughout the study.
- Judged by the Investigator to be in generally good health at Screening based upon the results of medical history, physical exam, 12-lead ECG and clinical laboratory test results. -Laboratory results outside the reference range must be documented as not clinically significant by both the Investigator and Sponsor.
- Participants must have a diagnosis of FXS through molecular documentation of full mutation of the FMR1 gene documented through genetic testing at Screening.
- Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of no more than two anti-seizure medications (ASMs) for the four weeks preceding study Screening; or must be seizure-free for one year if not currently receiving ASMs.
Key exclusion criteria (20 listed)
- Females who are pregnant, nursing or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception as outlined below for the duration of therapy and for three months after the last dose of study medication. Standard acceptable methods of contraception include abstinence (defined as refraining from heterosexual intercourse from screening to three months after the last dose of study medication: abstinence only applicable for females <18 years) or the use of a highly effective method of contraception, including hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, spermicide, vasectomy, or intrauterine device. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not an acceptable method of contraception.
- Patient has transitioned to independent living or living in a residential facility such as a university setting or congregate care.
- History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients.
- Exposure to any investigational drug or device less than or equal to 30 days prior to Screening or at any time during the study.
- Alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin levels greater than or equal to 2 times the upper limit of normal or alkaline phosphatase levels greater than or equal to 3 times the upper limit of normal.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZYN002Also: cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery)Pharmaceutically manufactured cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery) | Drug | ZYN002 |
| PlaceboAlso: Placebo Comparator, Matching PlaceboPlacebo formulated as a clear gel that can be applied to the skin (transdermal delivery) Other Names: Placebo Comparator Matching Placebo | Drug | Placebo |
Primary outcome
Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 1 score in patients with complete methylation of the FMR1 gene.
Time frame: Change from Baseline to Week 18
The ABC-C FXS is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials
11 secondary outcomes
Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 2 in patients with complete methylation of the FMR1 gene.
Time frame: Change from Baseline to Week 18
Change on the Caregiver Global Impression of Change (CaGI-C) for Pre-specified parameter among patients with complete methylation of the FMR1 gene.
Time frame: Change from Baseline to Week 18
Clinical Global Impression- Improvement (CGI-I) scale among patients with complete methylation of the FMR1 gene.
Time frame: Change from Baseline to Week 18
Change in ABC-C FXS pre-specified Subscale 1 among all randomized patients (complete and partial methylation of the FMR1 gene).
Time frame: Change from Baseline to Week 18
Number of patients with adverse events
Time frame: Day 1, Week 2, Week 4, Week 6, Week 10, Week 14, Week 18 and through 4-week post-dose telephone follow-up
Number of participants with abnormal physical and neurological exams
Time frame: Screening, Day 1 and Week 18
Number of participants with abnormal clinical laboratory results
Time frame: Screening, Week 10 and Week 18
Number of participants with abnormal vital sign results
Time frame: Screening, Day 1, Week 2 and Week 18
Number of participants with abnormal ECG
Time frame: Screening and Week 18
Withdrawal characteristics of ZYN002 using the Penn Physician Withdrawal Checklist
Time frame: Week 18 and 4-week post last dose
Skin tolerability as assessed using daily skin diary
Time frame: Daily from Day 1 through Week 18
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Fragile X Syndrome
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
27 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ZYN002 in people with Fragile X Syndrome and Fragile X Syndrome (FXS), run by Harmony Biosciences Management, Inc. at 27 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, United Kingdom, Australia, Ireland, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 1 score in patients with complete methylation of the FMR1 gene. (time frame: Change from Baseline to Week 18). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2026.