CompletedPhase 2

An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome

Australia17 participantsStarted 2022-07-12

Plain-language summary

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Angelman syndrome

Age range3 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Clinical diagnosis of AS with a documented disease-causing genetic etiology known to impact maternally derived UBE3A expression in brain.
✓. Males or females aged 3-17 years
✓. Body Weight of \>12Kg
✓. Subjects with a Clinical Global Impression - Severity (CGI-S) score of 3 or greater
✓. Not actively undergoing regression or loss of skills, defined as no persistent loss of previously acquired developmental skills for a period within 3 months of the Screening visit
✓. Each subject must be able to swallow the study medication provided as a liquid solution.
✓. Caregiver(s) must have sufficient English language skills.

✕. Mosaicism for disease-causing mutation.
✕. Clinically Significant abnormalities in safety laboratory testing or vital signs at screening
✕. Abnormal QTcF interval or prolongation at Screening.
✕. Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and previous COVID 19 infection with last 12 months that required hospitalization.
✕. Unstable or changes to Psychotropic treatment 2 weeks prior to screening .
✕. Excluded concomitant treatments
✕. Actively undergoing regression or loss of skills.
✕. Unstable seizure profile.

Safety and Tolerability

Timeframe: 13 weeks

Pharmacokinetic - Typical AUC24 of 30kg Child

Timeframe: 13 weeks

Pharmacokinetic - Typical t1/2 in 30 kg Child

Timeframe: 13 weeks

NCT IDNCT05011851

SponsorNeuren Pharmaceuticals Limited

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-05-13

Who can participate

Age range3 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Clinical diagnosis of AS with a documented disease-causing genetic etiology known to impact maternally derived UBE3A expression in brain.
✓. Males or females aged 3-17 years
✓. Body Weight of \>12Kg
✓. Subjects with a Clinical Global Impression - Severity (CGI-S) score of 3 or greater
✓. Not actively undergoing regression or loss of skills, defined as no persistent loss of previously acquired developmental skills for a period within 3 months of the Screening visit
✓. Each subject must be able to swallow the study medication provided as a liquid solution.
✓. Caregiver(s) must have sufficient English language skills.

✕. Mosaicism for disease-causing mutation.
✕. Clinically Significant abnormalities in safety laboratory testing or vital signs at screening
✕. Abnormal QTcF interval or prolongation at Screening.
✕. Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and previous COVID 19 infection with last 12 months that required hospitalization.
✕. Unstable or changes to Psychotropic treatment 2 weeks prior to screening .
✕. Excluded concomitant treatments
✕. Actively undergoing regression or loss of skills.
✕. Unstable seizure profile.