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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

160 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-11-12

Study Completion Date

2027-11-15

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Phelan-McDermid Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Phase 3, randomized, double-blind, parallel-group (2-arm), placebo-controlled, multicenter study

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) This is a double-blind study in which pediatric participants, caregivers, investigators, and the sponsor are blinded to study drug for the duration of study. Dose escalation, dose modification, and treatment discontinuation will be performed in a blinded manner.

Study Groups

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NNZ-2591 Arm

The total duration of this study for each participant will be up to approximately 17 to 19 weeks.

Participants will be randomized in a 1:1 ratio to receive orally administered NNZ-2591 during the 13-week Treatment Period.

Group Type EXPERIMENTAL

NNZ-2591

Intervention Type DRUG

The study drug will be administered twice daily orally.

Placebo Arm

The total duration of this study for each participant will be up to approximately 17 to 19 weeks.

Participants will be randomized in a 1:1 ratio to receive orally administered placebo matching NNZ-2591 during the 13-week Treatment Period.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

The study drug will be administered twice daily orally.

Interventions

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NNZ-2591

The study drug will be administered twice daily orally.

Intervention Type DRUG

Placebo

The study drug will be administered twice daily orally.

Intervention Type DRUG

Eligibility Criteria

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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.
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.

Exclusion Criteria

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.

Minimum Eligible Age

3 Years

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No