Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: KER-065.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy. Basic parameters: from 9 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Multicenter, Phase 2, Open-Label Study Evaluating the Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Overview
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.
Detailed description
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)
Interventions
- Drug KER-065
KER-065 will be administered subcutaneously (SC)
Primary outcome measures
- Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs) [Time frame: Up to approximately 3 years]
Secondary outcome measures (9)
- KER-065 serum concentration by visit, as appropriate [Time frame: Up to Week 100]
- Number and proportion of participants with treatment-emergent ADA (antidrug antibody) by visit [Time frame: 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) [Time frame: Up to Week 96]
- Change from baseline by visit in muscle volume and intramuscular fat by skeletal muscle MRI [Time frame: Up to Week 96]
- Ambulatory: Change from baseline by visit in North Star Ambulatory Assessment (NSAA) total score [Time frame: Up to Week 96]
- Ambulatory: Change from baseline by visit in 4-stair climb (4SC) [Time frame: Up to Week 96]
- Ambulatory: Change from baseline by visit in 10-meter walk/run (10MWR) test [Time frame: Up to Week 96]
- Ambulatory: Change from baseline by visit in TTR (time to rise) [Time frame: Up to Week 96]
- Nonambulatory: Change from baseline by visit in PUL (Performance of Upper Limb) v2.0 score [Time frame: Up to Week 96]
Eligibility criteria
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.
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07704099 · KER-065-B201