Denna sida har översatts automatiskt och översättningens korrekthet kan inte garanteras. Vänligen se engelsk version för en källtext.

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

10 augusti 2026 uppdaterad av: Neuren Pharmaceuticals Limited

A Phase 3 Open-label Extension Study to Investigate the Long-term Safety and Efficacy of Orally Administered NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

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.

Studieöversikt

Status

Rekrytering

Betingelser

Intervention / Behandling

Detaljerad beskrivning

After providing informed consent/assent, pediatric participants with Phelan-McDermid syndrome who participated in previous studies (NEU-2591-PMS-301 and NEU-2591-PMS-001) will undergo assessments for eligibility, baseline characteristics and symptom severity. Once eligibility is confirmed, participants will receive orally administered NNZ-2591 during the 52-week Treatment Period. A 2-week safety follow-up period will occur immediately after the completion of the Treatment Period.

Studietyp

Interventionell

Inskrivning (Beräknad)

180

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
    • California
      • Glendale, California, Förenta staterna, 91203
      • San Rafael, California, Förenta staterna, 94903
    • Maryland
      • Chevy Chase, Maryland, Förenta staterna, 20815
    • Massachusetts
      • Lexington, Massachusetts, Förenta staterna, 02421
    • Ohio
      • Cincinnati, Ohio, Förenta staterna, 02421
    • Texas
      • Houston, Texas, Förenta staterna, 77030

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

  • Barn

Tar emot friska volontärer

Nej

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 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.

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: N/A
  • Interventionsmodell: Enskild gruppuppgift
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: NNZ-2591 Arm
The total duration of this study for each participant will be up to up to 56 weeks.
Läkemedel: NNZ-2591
Studieläkemedlet kommer att administreras två gånger dagligen oralt.

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Long-term safety and tolerability of NNZ-2591 as assessed by the incidence of adverse events across participants
Tidsram: Baseline through Safety Follow-Up (Month 12)
Incidence of TEAEs, AESI and SAEs across participants
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.
Tidsram: Baseline through Month 12
Change from Baseline in ECG Heart Rate (bpm)
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.
Tidsram: Baseline through Safety Follow-Up (Month 12)
Change from Baseline PR Interval (ms QRS interval (ms)
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.
Tidsram: Baseline through Safety Follow-Up (Month 12)
Change from Baseline in QT interval (ms)
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.
Tidsram: Baseline through Safety Follow-Up (Month 12)
Change from Baseline in QTcB interval (ms)
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.
Tidsram: Baseline through Safety Follow-Up (Month 12)
Change from Baseline in QTcF interval (ms)
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.
Tidsram: Baseline through Safety Follow-Up (Month 12)
Change from Baseline in RR interval (ms)
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.
Tidsram: Baseline through Month 12
Change from Baseline for heart rate (bpm)
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.
Tidsram: Baseline through Month 12
Change from Baseline for respiration rate (breaths per minute)
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.
Tidsram: Baseline through Month 12
Change from Baseline for Temperature (Celsius)
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.
Tidsram: Baseline through Month 12
Change from Baseline for Diastolic Blood Pressure (mm Hg)
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.
Tidsram: Baseline through Month 12
Change from Baseline for Systolic Blood Pressure (mm Hg)
Baseline through Month 12
Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant clinical laboratory parameters events across participants.
Tidsram: Baseline through Month 12
Incidence of abnormal and clinically significant laboratory parameters
Baseline through Month 12
Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant physical examination findings across participants.
Tidsram: Baseline through Month 12
Incidence of abnormal, clinically significant physical examination findings
Baseline through Month 12

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score
Tidsram: Months 3 and 12
Efficacy of NNZ-2591 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.
Months 3 and 12
Efficacy of NNZ-2591 as measured by the change from baseline in the Vineland Adaptive Behavior Scales-3, Interview version (Vineland-3) receptive communication subdomain raw score.
Tidsram: Months 3 and 12
Efficacy of NNZ-2591 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.
Months 3 and 12
Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores.
Tidsram: Months 3 and 12
Efficacy of NNZ-2591 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.
Months 3 and 12
Efficacy of NNZ-2591 as measured by the Caregiver Impression of Change (CIC) domain scores.
Tidsram: Months 3 and 12
Efficacy of NNZ-2591 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.
Months 3 and 12
Efficacy of NNZ-2591 as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) domain scores.
Tidsram: Months 3 and 12
Efficacy of NNZ-2591 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.
Months 3 and 12
Efficacy of NNZ-2591 as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) overall score.
Tidsram: Months 3 and 12
Efficacy of NNZ-2591 as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (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.
Months 3 and 12
Efficacy of NNZ-2591 as measured by the change from baseline in PMS Clinician Domain Specific Rating Scale (PMS-DSRS) scores.
Tidsram: Months 3 and 12
Efficacy of NNZ-2591 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.
Months 3 and 12

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)

8 juni 2026

Primärt slutförande (Beräknad)

29 oktober 2028

Avslutad studie (Beräknad)

12 november 2028

Studieregistreringsdatum

Först inskickad

24 mars 2026

Först inskickad som uppfyllde QC-kriterierna

13 maj 2026

Första postat (Faktisk)

18 maj 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

12 augusti 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

10 augusti 2026

Senast verifierad

1 augusti 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • NEU-2591-PMS-302

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

Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .

Prenumerera