A Phase 2 interventional study of BPN14770 and Placebo in Jordan's Syndrome and PPP2R5D Neurodevelopmental Disorder, sponsored by Shionogi. Active, not recruiting at 3 sites in United States. Open to participants aged 9 Years to 45 Years. Per ClinicalTrials.gov, last updated 2026-06-09.
Sponsored by Shionogi · Phase 2, Interventional, and Treatment
The primary objective of this study is to assess the efficacy, safety, and tolerability of BPN14770 in participants aged 9 to 45 years with JS.
Shionogi is the lead sponsor of 104 studies on the registry; 10 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 21 (54%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Participants will receive a weight adjusted dose of BPN14770 twice daily (BID) during the 24-week double-blind period. Participants who complete the double-blind period will have the opportunity to continue in the open-label extension (OLE) period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week OLE period. Eligible participants who complete the double-blind period and OLE period may be offered the opportunity to participate in a 48-week extended open-label extension (Ext OLE) period. Adult participants (≥18 years of age at the start of Ext OLE) will receive an age-adjusted dose of BPN14770 BID during the Ext OLE period, while participants younger than 18 years at the start of Ext OLE will receive a weight-adjusted dose of BPN14770 BID.
Drug: BPN14770
Participants will receive BPN14770-matching placebo BID during the 24-week double-blind period. Participants who complete the double-blind period will have the opportunity to continue in the OLE period. Participants will receive a weight adjusted dose of BPN14770 BID during the 24-week OLE period. Eligible participants who complete the double-blind period and OLE period may be offered the opportunity to participate in a 48-week Ext OLE period. Adult participants (≥18 years of age at the start of Ext OLE) will receive an age-adjusted dose of BPN14770 BID during the Ext OLE period, while participants younger than 18 years at the start of Ext OLE will receive a weight-adjusted dose of BPN14770 BID.
Drug: BPN14770 · Drug: Placebo
Capsules for oral administration
Also known as: Zatolmilast
Capsules for oral administration
Change From Baseline at Week 24 in Numerical Rating Scale Within the Domain of Language/Communication
Time frame: Baseline, Week 24
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Time frame: Day 1 up to Week 96
Discontinuation-emergent Signs and Symptoms (DESS) Questionnaire Score
Time frame: Week 98
Number of Participants with Seizures
Time frame: Up to Week 96
Number of Participants at Suicidality Risk
Time frame: Up to Week 96
Plan to share: No
No publications or documents are linked to this record.
This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Shionogi