A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
ConditionPhelan-McDermid Syndrome
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age3-12
SponsorNeuren Pharmaceuticals Limited
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
NNZ-2591
DrugThe study drug will be administered twice daily orally.
Placebo
DrugThe study drug will be administered twice daily orally.
Treatment groups
160 Participants
are divided into 2 treatment groupsGroup A: NNZ-2591 ArmExperimental treatment 1 intervention
Group B: Placebo ArmPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score.
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score. The PMSA-C scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.
Time frame
Week 132
Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in the Vineland Adaptive Behavior Scales-3, Interview version (Vineland-3) receptive communication subdomain raw score.
Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in the Vineland Adaptive Behavior Scales-3, Interview version (Vineland-3) receptive communication subdomain raw score. A higher raw score for the receptive communication subdomain indicates better adaptive behavior.
Time frame
Week 13Secondary outcomes
1
Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) overall score.
Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) overall score. The CIC scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.
Time frame
Week 132
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores.
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores. The PMSA-C domain scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.
Time frame
Week 133
Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) domain scores.
Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) domain scores. The CIC scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.
Time frame
Week 134
Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) domain scores.
Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) domain scores. The PMSA-S scores range from 1 to 7 with 1 indicating typical for age, not at all impaired and 7 among the most severely impaired.
Time frame
Week 13Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov