What side effects are associated with this type of intervention?

Common treatments for Fragile X Syndrome (FXS) include medications such as selective serotonin reuptake inhibitors (SSRIs), antipsychotics, and stimulants. SSRIs, used to manage anxiety and mood disorders, can cause side effects like insomnia, gastrointestinal issues, and sexual dysfunction. Antipsychotics, which help with behavioral problems, may lead to weight gain, drowsiness, and metabolic changes. Stimulants, often prescribed for attention deficit hyperactivity disorder (ADHD) symptoms, can result in decreased appetite, sleep disturbances, and increased heart rate. For PDE4D inhibitors like BPN14770, potential side effects may include gastrointestinal discomfort, headache, and dizziness, although specific data on BPN14770 is still being studied.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Fragile X Syndrome that target Phosphodiesterase-4D (PDE4D) inhibition, such as BPN14770. The FDA has not approved any drugs specifically for Fragile X Syndrome, but treatments often focus on managing symptoms with medications used for related conditions like ADHD, anxiety, and mood disorders. These include stimulants, selective serotonin reuptake inhibitors (SSRIs), and antipsychotics.

What other types of research exist?

Promising novel alternatives to BPN14770 for Fragile X Syndrome patients include: 1) Metformin, which has shown potential in improving cognitive function and behavior in FXS patients. 2) Lovastatin, a statin that has demonstrated benefits in reducing behavioral symptoms. 3) Cannabidiol (CBD), which is being investigated for its neuroprotective and anti-anxiety effects. 4) AFQ056 (mavoglurant), a mGluR5 antagonist that has shown promise in reducing symptoms of FXS. These alternatives are in various stages of research and clinical trials, offering potential new avenues for treatment.

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Zatolmilast for Fragile X Syndrome

Q: What is the mechanism of action of this treatment?

Phosphodiesterase-4D (PDE4D) inhibitors, such as BPN14770, work by preventing the breakdown of cyclic adenosine monophosphate (cAMP) within cells. Elevated cAMP levels enhance signaling pathways that are crucial for cognitive function and synaptic plasticity, which are often impaired in Fragile X Syndrome (FXS) patients. By improving these pathways, PDE4D inhibitors can potentially ameliorate cognitive deficits and behavioral issues associated with FXS, offering a targeted therapeutic approach that addresses the underlying molecular abnormalities of the disorder.

Phase 2 & 3

Recruiting

Research Sponsored by Tetra Discovery Partners

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Able to attend clinic regularly and reliably

Male adolescent aged 9 to < 18 years with FXS and molecular genetic confirmation of the full FMR1 mutation (≥ 200 CGG repetitions)

Must not have

Renal impairment (serum creatinine > 1.25 × ULN)

Weight < 25 kg or BMI > 97th percentile for age

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 13 weeks

Summary

This 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.

Who is the study for?

This trial is for male adolescents aged 12 to <18 with Fragile X Syndrome confirmed by genetic testing. They can be on up to three psychotropic medications (excluding anti-epileptics used for seizures), which must be stable in dose for four weeks prior. Participants should not have significant health issues, substance abuse history, or recent participation in other trials.

What is being tested?

The study tests BPN14770's effects and how it's processed in the body compared to a placebo. Part 1 involves an open-label assessment of single doses, while Part 2 is double-blind and randomized between the drug and placebo groups.

What are the potential side effects?

Potential side effects are not detailed here but may include reactions typical of pharmacological interventions such as gastrointestinal discomfort, headaches, or allergic responses. Specific side effects will depend on the study findings.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can regularly and reliably go to the clinic for appointments.

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I am a male aged 9-17 with Fragile X Syndrome confirmed by genetic testing.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My kidney function is impaired with high creatinine levels.

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I weigh less than 25 kg or my BMI is above the 97th percentile for my age.

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I do not have any major psychiatric conditions or active diseases that could interfere with my participation.

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I have cirrhosis or unstable liver disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 13 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~13 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 13 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

NIH Toolbox Cognitive Battery cognition crystallized composite score

Secondary study objectives

CaGI-I for the general domain of Emotions/Behaviors

CaGI-I for the general domains of Daily Function, Language, and Academic Skills.

Investigator rated (CGI-I) for the general domains of Daily Function, Language, and Academic Skills

+5 more

Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: Study DrugActive Control1 Intervention

Subjects will receive 15 mg BID or 25mg BID dose of BPN14770

Group II: Placebo ArmPlacebo Group1 Intervention

Subjects will receive matching Placebo

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Phosphodiesterase-4D (PDE4D) inhibitors, such as BPN14770, work by preventing the breakdown of cyclic adenosine monophosphate (cAMP) within cells. Elevated cAMP levels enhance signaling pathways that are crucial for cognitive function and synaptic plasticity, which are often impaired in Fragile X Syndrome (FXS) patients. By improving these pathways, PDE4D inhibitors can potentially ameliorate cognitive deficits and behavioral issues associated with FXS, offering a targeted therapeutic approach that addresses the underlying molecular abnormalities of the disorder.

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