RecruitingPhelan-McDermid Syndrome

A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

Eligible age

3–12 yrs

Accepts

All genders

Locations

10 states

Healthy volunteers

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About this study

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.

Sponsor: Neuren Pharmaceuticals Limited

You may qualify if…

✓ 1. Male or female pediatric participants with Phelan-McDermid syndrome ages 3 to 12 years (inclusive) at the time of signing the informed consent.
✓ 2. Clinical diagnosis of Phelan-McDermid syndrome with a documented disease-causing genetic abnormality of SHANK3.
✓ 3. Body weight ≥ 10 kg at Screening.
✓ 4. Participants with a PMSA-S overall score ≥ 3 at the Screening and Baseline visits.
✓ 5. Not actively undergoing regression or loss of skills.

You may not qualify if…

✕ 1. Use of exclusionary medication or unstable treatment regimens of acceptable concomitant medications as required by the protocol.
✕ 2. Current treatment with more than 3 allowable psychotropic medications.
✕ 3. Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications).
✕ 4. 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.
✕ 5. 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.
✕ 6. Abnormal liver function laboratory results during the Screening period, as defined by the protocol
✕ 7. Abnormal QT interval on Screening ECG as defined by the protocol.