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Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy

9. juli 2026 opdateret af: Keros Therapeutics, Inc.

A Multicenter, Phase 2, Open-Label Study Evaluating the Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with Duchenne Muscular Dystrophy (DMD) on stable background therapy.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

This is a Phase 2, multicenter, open-label study of KER-065.

The study will consist of 3 periods:

  • Screening Period (up to 6 weeks)
  • Treatment Period (96 weeks)
  • Safety Follow-up Period (4 weeks)

Participants will be enrolled in parallel into 1 of 3 treatment cohorts:

  • Cohort A1 (Late Ambulatory)
  • Cohort A2 (Late Ambulatory)
  • Cohort N1 (Nonambulatory)

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

36

Fase

  • Fase 2

Kontakter og lokationer

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

Studiekontakt

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
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

Key Inclusion Criteria:

  • Diagnosis of DMD, defined as the presence of phenotypic features at screening consistent with DMD AND documented mutation in the dystrophin gene consistent with the diagnosis of DMD using a clinically validated genetic test.
  • Receiving a stable regimen of systemic CS (including, but not limited to, prednisone, prednisolone, deflazacort, or vamorolone) for at least 90 days before screening.
  • Body weight of ≥ 25.0 kg.

Ambulatory Participants Only (Cohort A1 and A2):

  • Ambulatory, defined as able to walk independently without assistive devices.
  • Able to TTR in < 10 seconds.
  • Has a NSAA score ≥ 15 points.
  • Cohort 2 only: Documentation of a stable dose of an approved exon-skipping therapy.

Nonambulatory Participants Only (Cohort N1):

  • Nonambulatory, characterized as being unable to ambulate for a minimum of 3 months before first dose with onset of nonambulatory status AND a NSAA walk score of 0 and inability to perform the 10MWR.
  • PUL v2.0 entry item score of 3 to 5, inclusive.

Key Exclusion Criteria:

  • Clinical symptoms or signs of cardiomyopathy or heart failure.
  • Exposure to any approved or investigational dystrophin restoration gene therapy product.
  • Exposure to any approved or investigational dystrophin restoration product other than gene therapy (Except for exon-skipping therapy for Cohort A2).
  • Exposure to any approved or investigational histone deacetylase inhibitor, antimyostatin therapy, therapy targeting transforming growth factor-beta ligands, or cell-based therapy.
  • Use of any other pharmacological treatment, except for CS
  • Treatment with immunosuppressant therapy (other than CS)
  • History of fracture of the upper limb

Nonambulatory Participants Only (Cohort N1):

  • Elbow-flexion contractures > 30° in both upper extremities.
  • Forced vital capacity (FVC) of < 50% or requirement for daytime or nocturnal ventilation, except for nocturnal non-invasive ventilation AND inability to perform consistent FVC measurements within ± 15% during paired testing.

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: Ikke-randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Cohort A1 (Late Ambulatory)
Participants will receive stable corticosteroid (CS) along with KER-065.
Medicin: KER-065
KER-065 will be administered subcutaneously (SC)
Eksperimentel: Cohort A2 (Late Ambulatory)
Participants will receive stable CS, exon skipper along with KER-065.
Medicin: KER-065
KER-065 will be administered subcutaneously (SC)
Eksperimentel: Cohort N1 (Nonambulatory)
Participants will receive stable CS along with KER-065.
Medicin: KER-065
KER-065 will be administered subcutaneously (SC)

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs)
Tidsramme: Up to approximately 3 years
To evaluate the safety and tolerability of KER-065 in ambulatory and nonambulatory participants with DMD
Up to approximately 3 years

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
KER-065 serum concentration by visit, as appropriate
Tidsramme: Up to Week 100
To assess the pharmacokinetics (PK) of KER-065 in ambulatory and nonambulatory participants with DMD
Up to Week 100
Number and proportion of participants with treatment-emergent ADA (antidrug antibody) by visit
Tidsramme: Up to Week 100
To assess the immunogenicity of KER-065 in ambulatory and nonambulatory participants with DMD
Up to Week 100
Change from baseline by visit in bone mineral density (BMD), fat mass, and lean body mass, as measured by dual- energy X-ray absorptiometry (DXA)
Tidsramme: Up to Week 96
To assess the effect of KER-065 on body composition in ambulatory and nonambulatory participants with DMD
Up to Week 96
Change from baseline by visit in muscle volume and intramuscular fat by skeletal muscle MRI
Tidsramme: Up to Week 96
To assess the effect of KER-065 on skeletal muscle in ambulatory and nonambulatory participants with DMD
Up to Week 96
Ambulatory: Change from baseline by visit in North Star Ambulatory Assessment (NSAA) total score
Tidsramme: Up to Week 96
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Up to Week 96
Ambulatory: Change from baseline by visit in 4-stair climb (4SC)
Tidsramme: Up to Week 96
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Up to Week 96
Ambulatory: Change from baseline by visit in 10-meter walk/run (10MWR) test
Tidsramme: Up to Week 96
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Up to Week 96
Ambulatory: Change from baseline by visit in TTR (time to rise)
Tidsramme: Up to Week 96
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Up to Week 96
Nonambulatory: Change from baseline by visit in PUL (Performance of Upper Limb) v2.0 score
Tidsramme: Up to Week 96
To assess the effect of KER-065 on motor function in nonambulatory participants with DMD
Up to Week 96

Samarbejdspartnere og efterforskere

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

Sponsor

Keros Therapeutics, Inc.

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)

14. september 2026

Primær færdiggørelse (Anslået)

17. juli 2029

Studieafslutning (Anslået)

14. august 2029

Datoer for studieregistrering

Først indsendt

9. juli 2026

Først indsendt, der opfyldte QC-kriterier

9. juli 2026

Først opslået (Faktiske)

15. juli 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

15. juli 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

9. juli 2026

Sidst verificeret

1. juli 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • KER-065-B201

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

produkt fremstillet i og eksporteret fra U.S.A.

Ja

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 Duchennes muskeldystrofi

Kliniske forsøg med KER-065

Søg i lignende forsøg

Sponsorer og samarbejdspartnere

Medicinske tilstande

Narkotikainterventioner

CROs by country

CROs in Mauritius

Betingelser

Sjældne sygdomme

Narkotikainterventioner

Kosttilskud

Sponsor / samarbejdspartnere

Placeringer

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