Fragile X SyndromePhase 3NCT05358886
A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome
A Phase 3 study testing BPN14770/ zatolmilast in people with Fragile X Syndrome, run by Tetra Discovery Partners at 17 sites in United States.
Trial summary
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) with Fragile X Syndrome Read more about this study
- Who is it for?
- Ages 18 to 45 years, men only
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- BPN14770/ zatolmilast
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 171 participants
- Where
- 17 sitesUnited States
- Study dates
- 1 Nov 2022to 18 Jul 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 18 to 45 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- Male subject aged 18 to 45 years at screening visit.
- Subject has FXS with a molecular genetic confirmation of the full fragile X mental retardation-1 (FMR1)mutation (≥200 CGG repetitions).
- Subject is able to swallow capsules.
- Current treatment with ≤3 prescribed psychotropic medications. Anti-epileptic medications are permitted and are not counted as psychotropic medications if they are used for the treatment of seizures. Anti-epileptics for other indications, such as the treatment of mood disorders, count towards the limit of permitted medications.
- Permitted concomitant psychotropic medications must be at a stable dose and dosing regimen for at least 4 weeks prior to screening and must remain stable during the period between screening and the commencement of the study treatment.
- Anti-epileptic medications must be at a stable dose and dosing regimen for 12 weeks prior to screening and must remain stable during the period between screening and commencement of the study treatment.
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- Subjects with a history of seizure disorder who are currently receiving treatment with anti-epileptics must have been seizure free for 3 months preceding screening or must be seizure free for 2 years if not currently receiving anti-epileptics.
- Behavioral and other non-pharmacological treatments/interventions must be stable for 4 weeks prior to screening and must remain stable during the period between screening and first dose of study treatment and throughout the study. Minor changes in hours or times of therapy that are not considered clinically significant will not be exclusionary. Changes in therapies provided through a program (eg, due to a vacation) are allowed.
- Subject must be willing to practice barrier methods of contraception while on the study if sexually active. Abstinence is also considered a reasonable form of birth control in this study population.
- Subject has a parent, legal authorized guardian, or consistent caregiver.
- Subject and caregiver are able to attend the clinic regularly and reliably.
- If subject is his own legal guardian, he is able to understand and sign informed consent to participate in the study.
- For subjects who are not their own legal guardian, subject's parent/legally authorized guardian is able to understand and sign an informed consent form for their child to participate in the study.
- If subject is not his own legal guardian, subject must provide assent for participation in the study if he has the cognitive ability to do so.
Who cannot join
- Inability to successfully complete the NIH-TCB picture vocabulary and oral reading assessments at screening and baseline. The ability to complete the NIH-TBC oral reading and picture vocabulary subtest at baseline is defined as the ability to complete both subtests, with (1) confirmation from the clinician administering that the test administrations are valid (noted on the administration form) and (2) generation of valid test scores for each test.
- History of or current cardiovascular, renal, hepatic, respiratory, gastrointestinal, psychiatric, neurologic, cerebrovascular, or other systemic disease that would place the subject at risk or potentially interfere with the interpretation of the safety, tolerability, or efficacy of the study treatment.
- Common conditions such as mild hypertension, etc. are allowed per the principal investigator's judgement as long as they are stable and controlled by medical therapy that is constant for at least 4 weeks before randomization.
- Renal impairment, defined as serum creatinine > 1.25 × ULN at screening
- Hepatic impairment, defined as ALT or AST elevation > 2 × ULN at screening. Note: LFTs may be repeated after 1 week to evaluate return to acceptable limits; if LFTs remain elevated, the subject is ineligible to participate.
- Clinically significant abnormalities, in the investigator's judgement, in safety laboratory tests, vital signs, or ECG, as measured during screening.
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- History of substance abuse within the past year, according to investigator assessment.
- Positive COVID-19 test during screening.
- Significant hearing or visual impairment that may affect the subject's ability to complete the test procedures.
- Concurrent major psychiatric condition (eg, major depressive disorder, schizophrenia, or bipolar disorder) as diagnosed by the investigator. Subjects with the additional diagnosis of autism spectrum disorder or anxiety disorder will be allowed.
- Subject has active diseases that would interfere with participation, such as acquired immunodeficiency disorder, hepatitis C, hepatitis B, or tuberculosis.
- Subject is planning to commence psychotherapy or cognitive behavior therapy during the period of the study or had begun psychotherapy or cognitive behavior therapy within 4 weeks prior to screening.
- Subject is an immediate family member of anyone employed by the sponsor, investigator, or study staff.
- Subject has a body mass index of less than 18 kg/m2 or greater than 36 kg/m2.
- Subject has participated in another clinical trial within the 30 days preceding Screening
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
United States17 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Boston Children's Hospital
Boston, Massachusetts, 02115
Rush University Medical Center
Chicago, Illinois, 60612
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229
Greenwood Genetic Center
Greenville, South Carolina, 29605
Amnova Clinical Research
Irvine, California, 92604
University of Kansas Medical Center
Kansas City, Kansas, 66160
Suburban Research Associates
Media, Pennsylvania, 19063
University of Miami
Miami, Florida, 33136
Icahn School of Medicine at Mount Sinai Hospital
New York, New York, 10029
Thompson Autism & Neurodevelopment Center - CHOC
Orange, California, 92868
UC Davis Health System
Sacramento, California, 95817
University of Utah and Primary Childrens Hospital
Salt Lake City, Utah, 84113
Clinic for Special Children
Strasburg, Pennsylvania, 17579
University of Massachusetts Medical School
Worcester, Massachusetts, 01655
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) with Fragile X Syndrome
- Conditions
- Fragile X Syndrome
- Being tested
- BPN14770/ zatolmilast, Placebo
- Keywords
- FXS
- 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.
BPN14770/ zatolmilastDrug
25mg BID BPN14770
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Study Drug | Active comparator | 25mg BID BPN14770 Receives: Drug: BPN14770/ zatolmilast |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
National Institutes of Health Toolbox Cognitive Battery cognition crystallized composite score (NIH-TCB CCC)
National Institutes of Health Toolbox Cognitive Battery cognition crystallized composite score (NIH-TCB CCC), which is calculated from the Picture Vocabulary and Oral Reading domains.
Measured: 13 Weeks
Secondary outcomes (8)
Numerical rating scale (NRS) scores based on subject-specific behaviors vs baseline
Numerical rating scale (NRS) scores based on subject-specific behaviors within the domains of Daily Function and Language
Measured: 13 Weeks
Caregiver Global Impression of Improvement (CaGI-I)
Caregiver Global Impression of Improvement (CaGI-I) for the general domains of Daily Function and Language
Measured: 13 Weeks
Clinical Global Impression Improvement (CGI-I) for - Investigator
Clinical Global Impression Improvement (CGI-I) for - Investigator rated the general domains of Daily Function and Language
Measured: 13 Weeks
Vineland-3 Adaptive Behavior Scale (Vineland-3)
Vineland-3 Adaptive Behavior Scale (Vineland-3), using the composite score and domain scores from communication, daily living skills, and socialization
Measured: 13 Weeks
Verbal Knowledge test from the Stanford-Binet (ed 5) (SB-5)IQ
Verbal Knowledge test from the Stanford-Binet (ed 5) (SB-5) IQ assessment
Measured: 13 Weeks
NIH-TCB domains of Picture Sequence Memory, Flanker Inhibitory Control and Attention, List Sorting Working Memory, Dimensional Change Card Sort, and Speeded Matching
NIH-TCB domains of Picture Sequence Memory, Flanker Inhibitory Control and Attention, List Sorting Working Memory, Dimensional Change Card Sort, and Speeded Matching
Measured: 13 Weeks
Aberrant Behavior Checklist (ABC) scores
Aberrant Behavior Checklist (ABC) scores
Measured: 13 Weeks
Anxiety, Depression, and Mood Scale (ADAMS) scores
Anxiety, Depression, and Mood Scale (ADAMS) scores
Measured: 13 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2022
- Study start1 Nov 2022
- Primary completion18 Jul 2025
- Study completion18 Jul 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
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 171 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tetra Discovery Partners
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elizabeth Berry-Kravis, MD, Principal investigator, Rush Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05358886
- Sponsor's protocol ID
- BPN14770-CNS-301
- Status last verified
- Mar 2026
- First submitted
- 27 Apr 2022
- Last updated
- 10 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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 10 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT05358886
- Acronym / sponsor ID
- BPN14770-CNS-301
- Conditions
- Fragile X Syndrome
- Keywords
- FXS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 171 participants (actual)
- Lead sponsor
- Tetra Discovery PartnersIndustry (pharma, biotech, devices)
- Study start
- 1 Nov 2022 (actual)
- Primary completion
- 18 Jul 2025 (actual)
- Study completion
- 18 Jul 2025 (actual)
- First posted
- 3 May 2022 (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.
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) with Fragile X Syndrome
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Study Drug | Active comparator | Drug: BPN14770/ zatolmilast25mg BID BPN14770 |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (14 listed)
- Male subject aged 18 to 45 years at screening visit.
- Subject has FXS with a molecular genetic confirmation of the full fragile X mental retardation-1 (FMR1)mutation (≥200 CGG repetitions).
- Subject is able to swallow capsules.
- Current treatment with ≤3 prescribed psychotropic medications. Anti-epileptic medications are permitted and are not counted as psychotropic medications if they are used for the treatment of seizures. Anti-epileptics for other indications, such as the treatment of mood disorders, count towards the limit of permitted medications.
- Permitted concomitant psychotropic medications must be at a stable dose and dosing regimen for at least 4 weeks prior to screening and must remain stable during the period between screening and the commencement of the study treatment.
Key exclusion criteria (15 listed)
- Inability to successfully complete the NIH-TCB picture vocabulary and oral reading assessments at screening and baseline. The ability to complete the NIH-TBC oral reading and picture vocabulary subtest at baseline is defined as the ability to complete both subtests, with (1) confirmation from the clinician administering that the test administrations are valid (noted on the administration form) and (2) generation of valid test scores for each test.
- History of or current cardiovascular, renal, hepatic, respiratory, gastrointestinal, psychiatric, neurologic, cerebrovascular, or other systemic disease that would place the subject at risk or potentially interfere with the interpretation of the safety, tolerability, or efficacy of the study treatment.
- Renal impairment, defined as serum creatinine > 1.25 × ULN at screening
- Hepatic impairment, defined as ALT or AST elevation > 2 × ULN at screening. Note: LFTs may be repeated after 1 week to evaluate return to acceptable limits; if LFTs remain elevated, the subject is ineligible to participate.
- Clinically significant abnormalities, in the investigator's judgement, in safety laboratory tests, vital signs, or ECG, as measured during screening.
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 |
|---|---|---|
| BPN14770/ zatolmilast25mg BID BPN14770 | Drug | Study Drug |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
National Institutes of Health Toolbox Cognitive Battery cognition crystallized composite score (NIH-TCB CCC)
Time frame: 13 Weeks
National Institutes of Health Toolbox Cognitive Battery cognition crystallized composite score (NIH-TCB CCC), which is calculated from the Picture Vocabulary and Oral Reading domains.
8 secondary outcomes
Numerical rating scale (NRS) scores based on subject-specific behaviors vs baseline
Time frame: 13 Weeks
Caregiver Global Impression of Improvement (CaGI-I)
Time frame: 13 Weeks
Clinical Global Impression Improvement (CGI-I) for - Investigator
Time frame: 13 Weeks
Vineland-3 Adaptive Behavior Scale (Vineland-3)
Time frame: 13 Weeks
Verbal Knowledge test from the Stanford-Binet (ed 5) (SB-5)IQ
Time frame: 13 Weeks
NIH-TCB domains of Picture Sequence Memory, Flanker Inhibitory Control and Attention, List Sorting Working Memory, Dimensional Change Card Sort, and Speeded Matching
Time frame: 13 Weeks
Aberrant Behavior Checklist (ABC) scores
Time frame: 13 Weeks
Anxiety, Depression, and Mood Scale (ADAMS) scores
Time frame: 13 Weeks
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 10 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for Fragile X Syndrome
Not yet recruitingPhase 2
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
Aggressive Behavior in Fragile X Syndome
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Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
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RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Not yet recruitingPhase 2/3
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)
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.
Elizabeth Berry-Kravis, MD principal investigator
Rush Medical Center
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing BPN14770/ zatolmilast in people with Fragile X Syndrome, run by Tetra Discovery Partners at 17 sites in United States.
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 14 inclusion and 15 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 10 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States. 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?
National Institutes of Health Toolbox Cognitive Battery cognition crystallized composite score (NIH-TCB CCC) (time frame: 13 Weeks). 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 10 Jul 2026.