Recruiting

Phase 3

NNZ-2591An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

Sponsor:

Neuren Pharmaceuticals Limited

Code:

NCT07593391

Conditions

Phelan-McDermid Syndrome

Eligibility Criteria

Sex: All

Age: 3 - 12

Healthy Volunteers: Not accepted

Interventions

NNZ-2591

Study Details

Brief summary:

This Phase 3, open-label extension, multicenter study will evaluate long-term safety, tolerability and efficacy of NNZ-2591 in pediatric participants with Phelan- McDermid Syndrome.

Conditions

Phelan-McDermid Syndrome

Study ID

NCT07593391

Start date

Jun 8, 2026

Status verified date

Sep, 2026

Completion date

Nov 12, 2028

Anticipated

Primary completion date

Oct 29, 2028

Anticipated

Eligibility Criteria

Sex: All

Age: 3 - 12

Healthy Volunteers: Not accepted

Inclusion Criteria:

1. Male or female pediatric participants with Phelan-McDermid syndrome ages 3 to 12 years (inclusive) at the time of signing the informed consent for the antecedent study.
2. Participant must have completed all applicable study visits for the antecedent study in which they participated.
3. Body weight ≥ 10 kg at Screening/Baseline.
4. Participants with a PMSA-S overall score ≥ 3 at the Screening and Baseline visits.
5. Not actively undergoing regression or loss of skills.

Exclusion Criteria:

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

Study Design

Enrollment

180 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: NNZ-2591 Arm

The total duration of this study for each participant will be up to up to 56 weeks.

Interventions

NNZ-2591

The study drug will be administered twice daily orally.

Primary outcome measure

Long-term safety and tolerability of NNZ-2591 as assessed by the incidence of adverse events across participants [ Time Frame: Baseline through Safety Follow-Up (Month 12) ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [ Time Frame: Baseline through Month 12 ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [ Time Frame: Baseline through Safety Follow-Up (Month 12) ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [ Time Frame: Baseline through Safety Follow-Up (Month 12) ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [ Time Frame: Baseline through Safety Follow-Up (Month 12) ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [ Time Frame: Baseline through Safety Follow-Up (Month 12) ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [ Time Frame: Baseline through Safety Follow-Up (Month 12) ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. [ Time Frame: Baseline through Month 12 ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. [ Time Frame: Baseline through Month 12 ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. [ Time Frame: Baseline through Month 12 ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. [ Time Frame: Baseline through Month 12 ]
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. [ Time Frame: Baseline through Month 12 ]
Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant clinical laboratory parameters events across participants. [ Time Frame: Baseline through Month 12 ]
Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant physical examination findings across participants. [ Time Frame: Baseline through Month 12 ]

Central Contacts and Locations

Central contacts

Medical Information Lead

231-203-8050 medicalinformation@neurenpharma.com

Locations

Neuren PMS-302 Site #116

Recruiting

Birmingham, Alabama, United States, 35233

Contacts