Study of Zatolmilast (BPN14770) in Participants With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome [JS])

Launched by SHIONOGI · Dec 2, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a medication called Zatolmilast (also known as BPN14770) to see how safe it is and how well people can tolerate it. The trial is specifically for individuals aged 9 to 45 who have been diagnosed with Jordan's Syndrome, which is also known as PPP2R5D Neurodevelopmental Disorder. The researchers want to understand how this medication affects participants with this condition, which can influence how they learn and develop.

To participate in the study, individuals must be between 9 and 45 years old and have a confirmed diagnosis of the disorder. They should not be taking more than three prescribed medications for mental health. It’s important to note that participants will need a parent, guardian, or caregiver involved in the study. However, some people may not qualify, such as those who weigh less than 25 kilograms or have serious health issues that the researchers believe could affect the study. Participants will be monitored throughout the trial to ensure their safety and help the researchers gather important information about the medication.

Gender

ALL

Eligibility criteria

• 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.