CompletedPhase 2

An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)

United States18 participantsStarted 2022-08-08

Plain-language summary

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.

Who can participate

Age range3 Years – 12 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.

Inclusion criteria

✓. Clinical diagnosis of PMS with a documented disease-causing genetic abnormality of SHANK3.
✓. Males or females aged 3-12 years.
✓. Body weight of 12 kg or higher at Screening.
✓. Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at the Screening visit.
✓. 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.

Exclusion criteria

✕. Body weight \< 12kg at screening
✕. Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
✕. Abnormal QTcF interval or prolongation at Screening.
✕. Any other clinically significant finding on ECG at the Screening visit.
✕. Positive for severe acute respiratory syndrome coronavirus 2 (SARSCoV- 2) and previous COVID 19 infection with last 12 months that required hospitalization
✕. Unstable or changes Psychotropic treatment 2 weeks prior to screening .
✕. Excluded concomitant treatments.
✕. Actively undergoing regression or loss of skills.

What they're measuring

Safety and Tolerability

Timeframe: 13 weeks

Pharmacokinetic - Mean AUC24

Timeframe: Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13.

Pharmacokinetic - t1/2

Timeframe: Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13.

Trial details

NCT IDNCT05025241

SponsorNeuren Pharmaceuticals Limited

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-11-01

Results submitted2024-12-11

View on ClinicalTrials.gov More Phelan-McDermid Syndrome trials