Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
ConditionDuchenne Muscular Dystrophy
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexMale
Age9+
SponsorKeros Therapeutics, Inc.
About this trial
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.
Eligibility criteria
Qualifiers
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, defined as able to walk independently without assistive devices.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- KER-065
Treatment groups
36 Participants
are divided into 3 treatment groupsLocations
Sponsors and collaborators
Source ClinicalTrials.gov