Zatolmilast for PPP2R5D Syndrome

Recruiting in Palo Alto (17 mi)

+2 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Shionogi

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The primary objective of this study is to assess the safety and tolerability of BPN14770 in participants aged 9 to 45 years with JS.

Research Team

Eligibility Criteria

This trial is for individuals aged 9 to 45 with Jordan's Syndrome (JS), a neurodevelopmental disorder. It includes those with related conditions like Klinefelter and Triple X Syndromes, but the full eligibility criteria are not provided.

Inclusion Criteria

Participant has a parent, legal authorized guardian or consistent caregiver

I have been diagnosed with PPP2R5D Neurodevelopmental Disorder.

I am currently taking 3 or fewer medications for mental health.

See 1 more

Exclusion Criteria

Clinically significant abnormalities, in the investigator's judgment, in safety laboratory tests, vital signs, or electrocardiogram (ECG), as measured during Screening

Concurrent major psychiatric condition (e.g., Major Depressive Disorder, Schizophrenia or Bipolar Disorder) as diagnosed by the investigator. Participants with additional diagnosis of Autism Spectrum Disorder or Anxiety Disorder will be allowed

I am starting or have recently started psychotherapy or CBT.

See 1 more

Treatment Details

Interventions

BPN14770 (Phosphodiesterase Inhibitor)

Trial OverviewThe study tests Zatolmilast (BPN14770) against a placebo to see if it's safe and tolerable for people with JS. Participants will be randomly assigned to receive either the drug or an inactive substance.

Participant Groups

2Treatment groups

Experimental Treatment

Placebo Group

Group I: BPN14770Experimental Treatment1 Intervention

Participants will receive a weight adjusted dose of BPN14770 twice daily (BID) during the 24-week Double-blind Period. Participants in the BPN14770 arm who complete the Double-blind Period will have the opportunity to continue in the Open-label Extension Period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week Open-label Extension Period.

Group II: PlaceboPlacebo Group2 Interventions

Participants will receive BPN14770 matching placebo BID during the 24-week Double-blind Period. Participants in the placebo arm who complete the Double-blind Period will have the opportunity to continue in the Open-label Extension Period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week Open-label Extension Period.