Overview
This Phase 3, randomized, double-blind, parallel-group (2-arm), placebo-controlled, multicenter study will evaluate the efficacy and safety of NNZ-2591 compared to placebo in pediatric participants with Phelan- McDermid Syndrome.
Description
After providing informed consent/assent, pediatric participants with Phelan-McDermid syndrome ages 3-12 years of age will enter the 4-week Screening Period and undergo assessments for eligibility, baseline characteristics and symptom severity.
Once eligibility is confirmed, participants will be randomized in a 1:1 ratio to receive either orally administered NNZ-2591 or matching placebo during the 13-week Treatment Period. Subsequently, a 2-week safety follow-up period will occur immediately after the completion of the Treatment Period.
Eligibility
Inclusion Criteria:
- Male or female pediatric participants with Phelan-McDermid syndrome ages 3 to 12 years (inclusive) at the time of signing the informed consent.
- Clinical diagnosis of Phelan-McDermid syndrome with a documented disease-causing genetic abnormality of SHANK3.
- Body weight ≥ 10 kg at Screening.
- Participants with a PMSA-S overall score ≥ 3 at the Screening and Baseline visits.
- Not actively undergoing regression or loss of skills.
Exclusion Criteria:
- Use of exclusionary medication or unstable treatment regimens of acceptable concomitant medications as required by the protocol.
- Current treatment with more than 3 allowable psychotropic medications.
- Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications).
- Psychotropic medications or any other medication used for a chronic illness (not including antibiotics, pain relievers, anti-diarrheals, and laxatives) with doses and dosing regimen that have not been stable for at least 4 weeks before Screening. If the treatment was discontinued, the discontinuation must have occurred no fewer than 2 weeks before the start of Screening.
- Any intercurrent seizures in the past 6 months and /or more than 1 seizure in the past 12 months. •A single febrile seizure in the 6 months prior to screening is allowable if no rescue medication was required.
- Abnormal liver function laboratory results during the Screening period, as defined by the protocol
- Abnormal QT interval on Screening ECG as defined by the protocol.