What side effects are associated with this type of intervention?

Common treatments for Fragile X Syndrome 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 PDE4 inhibitors like BPN14770, potential side effects may include gastrointestinal discomfort, headache, and dizziness, although specific data on PDE4D inhibitors in Fragile X Syndrome is limited.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Fragile X Syndrome. BPN14770, a Phosphodiesterase-4D (PDE4D) inhibitor, is currently being studied for its potential benefits in treating this condition. Other treatments for Fragile X Syndrome are generally symptomatic and may include medications for associated symptoms such as ADHD, anxiety, and mood disorders, but these are not specifically approved for Fragile X Syndrome.

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 reducing behavioral issues in FXS. 2) Ganaxolone, a neurosteroid that modulates GABA receptors and has shown efficacy in reducing anxiety and improving behavior in FXS. 3) Lovastatin, which has been studied for its potential to improve cognitive function and reduce behavioral symptoms in FXS. These alternatives are based on recent clinical trials and ongoing research in the field of neurodevelopmental disorders.

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Phosphodiesterase-4D (PDE4D) Inhibitor

BPN14770 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 AMP (cAMP) within cells. This leads to increased levels of cAMP, which can enhance synaptic plasticity and improve cognitive function. For patients with Fragile X Syndrome, this is particularly important because the disorder is characterized by intellectual disability and cognitive deficits. By improving synaptic function, PDE4D inhibitors have the potential to alleviate some of the cognitive symptoms associated with Fragile X Syndrome, offering a targeted approach to treatment.

Phase 3

Waitlist Available

Led By Elizabeth Berry-Kravis, MD

Research Sponsored by Tetra Discovery Partners

Eligibility Criteria Checklist

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

Must have

Be younger than 65 years old

Must not have

Cirrhosis, unstable chronic liver disease or acute liver disease

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This 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.

Who is the study for?

This trial is for males with Fragile X Syndrome who completed a previous BPN14770 study. They must be able to consent or provide assent, use contraception if sexually active, have a caregiver, and attend regular clinic visits. Excluded are those with substance abuse, renal/hepatic impairment, significant lab/ECG abnormalities from the parent study, major psychiatric conditions (except autism or anxiety), certain diseases like AIDS or hepatitis, or participation in other trials within 30 days.

What is being tested?

The trial tests Zatolmilast/BPN14770 as an ongoing treatment for subjects who've finished prior studies on it. It's an open-label extension meaning everyone gets the drug and there's no placebo group; participants know what they're taking.

What are the potential side effects?

While specific side effects of Zatolmilast/BPN14770 aren't listed here, common ones may include gastrointestinal issues like nausea or diarrhea, headaches, dizziness and potential allergic reactions. Participants' health will be monitored closely throughout.

Eligibility Criteria

Exclusion Criteria

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

Select...

I have cirrhosis or unstable liver disease.

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I do not have any major health issues that could affect my participation in the study.

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My latest kidney test shows my creatinine levels are high.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Safety and tolerability of BPN14770

Secondary study objectives

Aberrant Behavior Checklist (ABC) scores

Anxiety, Depression, and Mood Scale (ADAMS) scores

Caregiver Global Impression of Improvement (CaGI-I) assessments

+5 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Study Drug (BPN14770)Experimental Treatment1 Intervention

25mg BID Study Drug BPN14770 (Adults) or 15mg BID Study Drug BPN14770 (Adolescents with body weight \<43 kg)

0 / 3Eligibility 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 AMP (cAMP) within cells. This leads to increased levels of cAMP, which can enhance synaptic plasticity and improve cognitive function. For patients with Fragile X Syndrome, this is particularly important because the disorder is characterized by intellectual disability and cognitive deficits. By improving synaptic function, PDE4D inhibitors have the potential to alleviate some of the cognitive symptoms associated with Fragile X Syndrome, offering a targeted approach to treatment.

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