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.
Eligibility
Sex
ALL
Min age
9 Years
Max age
45 Years
Healthy volunteers
No
Inclusion Criteria:
1. Participant is aged 9 to 45 years, inclusive.
2. Participant has confirmed/documented history of PPP2R5D Neurodevelopmental Disorder.
3. Current treatment with no more than 3 prescribed psychotropic medications.
4. Participant has a parent, legal authorized guardian or consistent caregiver.
Exclusion Criteria:
1. Participant has body weight less than 25 kilograms (kg).
2. Clinically significant abnormalities, in the investigator's judgment, in safety laboratory tests, vital signs, or electrocardiogram (ECG), as measured during Screening.
3. 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.
4. Participant is planning to commence psychotherapy or cognitive behavior therapy (CBT) during the period of the study or had begun psychotherapy or CBT within 4 weeks prior to Screening.
Primary outcome measure(s)
Change From Baseline at Week 24 in Numerical Rating Scale Within the Domain of Language/Communication — Baseline, Week 24
Number of Participants with Treatment-emergent Adverse Events (TEAEs) — Day 1 up to Week 96
Discontinuation-emergent Signs and Symptoms (DESS) Questionnaire Score — Week 98
Number of Participants with Seizures — Up to Week 96
Number of Participants at Suicidality Risk — Up to Week 96
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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