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

ConditionDuchenne Muscular Dystrophy
Trial statusRecruiting
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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • KER-065

    Drug

    KER-065 will be administered subcutaneously (SC)

Treatment groups

36 Participants
are divided into 3 treatment groups
Group A: Cohort A1 (Late Ambulatory)Experimental treatment 1 intervention
Group B: Cohort A2 (Late Ambulatory)Experimental treatment 1 intervention
Group C: Cohort N1 (Early Nonambulatory)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs)

To evaluate the safety and tolerability of KER-065 in ambulatory and nonambulatory participants with DMD

Time frame
Up to approximately 14 months

Secondary outcomes

1

KER-065 serum concentration by visit, as appropriate

To assess the pharmacokinetics (PK) of KER-065 in ambulatory and nonambulatory participants with DMD

Time frame
Up to Week 52
2

Number and proportion of participants with treatment-emergent ADA (antidrug antibody) by visit

To assess the immunogenicity of KER-065 in ambulatory and nonambulatory participants with DMD

Time frame
Up to Week 52
3

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)

To assess the effect of KER-065 on body composition in ambulatory and nonambulatory participants with DMD

Time frame
Week 12, Week 24 and Week 48
4

Change from baseline by visit in muscle volume and intramuscular fat by skeletal muscle Magnetic Resonance Imaging (MRI)

To assess the effect of KER-065 on skeletal muscle in ambulatory and nonambulatory participants with DMD

Time frame
Week 24 and Week 48

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Keros Therapeutics, Inc.

Lead sponsor

Source ClinicalTrials.gov
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