Zatolmilast for Fragile X Syndrome

Recruiting at23 trial locations

Age: < 65

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Recruiting

Sponsor: Tetra Discovery Partners

Disqualifiers: Cardiovascular, Renal, Hepatic, Psychiatric, others

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

You can continue taking your current medications if they are stable and within the allowed types. You must be on no more than 3 prescribed psychotropic medications, and these must be at a stable dose for at least 4 weeks before the study. Anti-epileptic medications are allowed if they are for seizures and must be stable for 12 weeks before the study.

Research Team

Eligibility Criteria

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.

Inclusion Criteria

My non-drug treatments have been the same for the last 4 weeks.

Willing to practice barrier contraception if sexually active; abstinence accepted

I haven't had a seizure for 3 months, or 2 years if not taking seizure medication.

Exclusion Criteria

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

My kidney function is impaired with high creatinine levels.

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

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part 1: Open-label Pharmacokinetics Assessment

Open-label, single-dose pharmacokinetics assessment of BPN14770 25 mg and 50 mg

1 week

Part 2: Double-blind Treatment

Double-blind, randomized treatment with either BPN14770 or placebo

13 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• BPN14770 (Other)
• Placebo ()
• zatolmilast ()

Trial Overview 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.

Participant Groups

Group I: Study Drug Active Control 1 Intervention

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

Group II: Placebo Arm Placebo Group 1 Intervention

Subjects will receive matching Placebo

Who Is Running the Clinical Trial?

Tetra Discovery Partners

Lead Sponsor

Trials

9

Recruited

1,000+