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

10. august 2026 oppdatert 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.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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.

Studietype

Intervensjonell

Registrering (Antatt)

180

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn

Tar imot friske frivillige

Nei

Beskrivelse

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: NNZ-2591 Arm
The total duration of this study for each participant will be up to up to 56 weeks.
Legemiddel: NNZ-2591
Studiemedikamentet vil bli administrert to ganger daglig oralt.

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Long-term safety and tolerability of NNZ-2591 as assessed by the incidence of adverse events across participants
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: Baseline through Month 12
Incidence of abnormal, clinically significant physical examination findings
Baseline through Month 12

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Neuren Pharmaceuticals Limited

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

8. juni 2026

Primær fullføring (Antatt)

29. oktober 2028

Studiet fullført (Antatt)

12. november 2028

Datoer for studieregistrering

Først innsendt

24. mars 2026

Først innsendt som oppfylte QC-kriteriene

13. mai 2026

Først lagt ut (Faktiske)

18. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

12. august 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

10. august 2026

Sist bekreftet

1. august 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • NEU-2591-PMS-302

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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