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

20. maj 2026 opdateret af: 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.

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Detaljeret 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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

180

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn

Tager imod sunde frivillige

Ingen

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

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: NNZ-2591 Arm
The total duration of this study for each participant will be up to up to 56 weeks.
Medicin: NNZ-2591
Studiemedicinen vil blive administreret to gange dagligt oralt.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Neuren Pharmaceuticals Limited

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

20. maj 2026

Primær færdiggørelse (Anslået)

29. oktober 2028

Studieafslutning (Anslået)

12. november 2028

Datoer for studieregistrering

Først indsendt

24. marts 2026

Først indsendt, der opfyldte QC-kriterier

13. maj 2026

Først opslået (Faktiske)

18. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

22. maj 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

20. maj 2026

Sidst verificeret

1. maj 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • NEU-2591-PMS-302

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med Phelan-McDermid syndrom

Kliniske forsøg med NNZ-2591

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Sponsor / samarbejdspartnere

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