30 Participants Needed

Zatolmilast for PPP2R5D Syndrome

Recruiting at 2 trial locations
SC
Overseen ByShionogi Clinical Trials Administrator Clinical Support Help Line
Age: < 65
Sex: Any
Trial Phase: Phase 2
Sponsor: Shionogi
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you can be on up to 3 prescribed psychotropic medications.

How does the drug BPN14770 differ from other treatments for PPP2R5D Syndrome?

BPN14770, also known as Zatolmilast, is unique because it targets the underlying molecular mechanisms of PPP2R5D Syndrome by modulating the activity of protein phosphatase 2A (PP2A), which is involved in key neuronal processes. This approach is novel as there are currently no standard treatments specifically addressing the genetic and molecular basis of this condition.12345

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.

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.
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-blind Treatment

Participants receive either BPN14770 or placebo BID for 24 weeks

24 weeks

Open-label Extension

Participants receive a weight adjusted dose of BPN14770 BID for 24 weeks

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • BPN14770
Trial Overview The 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.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Shionogi

Lead Sponsor

Trials
122
Recruited
42,100+

Dr. Isao Teshirogi

Shionogi

Chief Executive Officer since 2008

PhD in Pharmaceutical Sciences from the University of Tokyo

Dr. Takuko Sawada

Shionogi

Chief Medical Officer since 2022

MD from a recognized institution (specific details not found)

References

Clinical features of PPP2 syndrome type R5D (Jordan's syndrome) to support standardization of care. [2023]
Clinical, neuroimaging and molecular characteristics of PPP2R5D-related neurodevelopmental disorders: an expanded series with functional characterisation and genotype-phenotype analysis. [2023]
De novo missense variants in PPP2R5D are associated with intellectual disability, macrocephaly, hypotonia, and autism. [2018]
PPP2R1A neurodevelopmental disorder is associated with congenital heart defects. [2023]
Clinical and molecular characteristics of a novel rare de novo variant in PPP2CA in a patient with a developmental disorder, autism, and epilepsy. [2022]
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