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En studie av NNZ-2591 hos barn som deltar med Phelan-McDermid syndrom

25 september 2026 uppdaterad av: Neuren Pharmaceuticals Limited

En fas 3-randomiserad, dubbelblind, placebokontrollerad studie för att undersöka effektiviteten och säkerheten hos oralt administrerat NNZ-2591 jämfört med placebo hos pediatriska deltagare med Phelan-McDermid syndrom

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.

Studieöversikt

Status

Rekrytering

Betingelser

Intervention / Behandling

Detaljerad beskrivning

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.

Studietyp

Interventionell

Inskrivning (Beräknad)

160

Fas

  • Fas 3

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studieorter

  • Förenta staterna
    • Alabama
      • Birmingham, Alabama, Förenta staterna, 35233
    • California
      • Glendale, California, Förenta staterna, 91203
      • Palo Alto, California, Förenta staterna, 94305
      • San Diego, California, Förenta staterna, 92123
      • San Rafael, California, Förenta staterna, 94903
    • District of Columbia
      • Washington D.C., District of Columbia, Förenta staterna, 20010
    • Georgia
      • Atlanta, Georgia, Förenta staterna, 30322
    • Illinois
      • Chicago, Illinois, Förenta staterna, 60612
    • Maryland
      • Chevy Chase, Maryland, Förenta staterna, 20815
    • Massachusetts
      • Brookline, Massachusetts, Förenta staterna, 02445
      • Lexington, Massachusetts, Förenta staterna, 02421
    • Michigan
      • Ann Arbor, Michigan, Förenta staterna, 48109
    • New York
      • New York, New York, Förenta staterna, 10029
    • Ohio
      • Cincinnati, Ohio, Förenta staterna, 45229
      • Columbus, Ohio, Förenta staterna, 43205
    • Texas
      • Houston, Texas, Förenta staterna, 77030
    • Virginia
      • Charlottesville, Virginia, Förenta staterna, 22903
  • Kanada
    • Ontario
      • Toronto, Ontario, Kanada, M4G 1R8

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

3 år till 12 år (Barn)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Allt

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: Randomiserad
  • Interventionsmodell: Parallellt uppdrag
  • Maskning: Fyrdubbla

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: 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.
Läkemedel: NNZ-2591

The study drug will be administered twice daily orally.
Placebo-jämförare: 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.
Läkemedel: Placebo

The study drug will be administered twice daily orally.

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score.
Tidsram: Week 13

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.
Week 13
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.
Tidsram: Week 13

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.
Week 13

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) overall score.
Tidsram: Week 13

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.
Week 13
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores.
Tidsram: Week 13

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.
Week 13
Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) domain scores.
Tidsram: Week 13

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.
Week 13
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.
Tidsram: Week 13

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.
Week 13
Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMSA-S overall score.
Tidsram: Week 13

Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMSA-S overall score. 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.
Week 13
Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMS Clinician Domain Specific Rating Scale (PMS-DSRS) scores.
Tidsram: Week 13

Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMS Clinician Domain Specific Rating Scale (PMS-DSRS) scores. The PMS-DSRS scores range from 0 to 4 with 0 indicating Symptom Not Present and 4 indicating Very Severe.
Week 13

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Neuren Pharmaceuticals Limited

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

12 november 2025

Primärt slutförande (Beräknad)

31 oktober 2027

Avslutad studie (Beräknad)

15 november 2027

Studieregistreringsdatum

Först inskickad

13 november 2025

Först inskickad som uppfyllde QC-kriterierna

8 december 2025

Första postat (Faktisk)

15 december 2025

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

28 september 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

25 september 2026

Senast verifierad

1 september 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • NEU-2591-PMS-301

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

NEJ

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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