KER-065 for Duchenne Muscular Dystrophy: safety and potential benefit in boys and men

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 2, multicenter, open-label trial evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and potential efficacy of KER-065 in adult and pediatric males with Duchenne muscular dystrophy on stable background therapy. Participants are enrolled into one of three cohorts based on walking ability: two late ambulatory cohorts and one early nonambulatory cohort, and will go through screening, a 48-week treatment period, and a 4-week safety follow-up. The study will measure functional and biomarker outcomes to determine if KER-065 can provide benefit beyond current therapies. Key eligibility includes confirmed DMD with ongoing corticosteroid treatment and weight criteria, with specific functional criteria for ambulatory and nonambulatory groups.

Why it matters

Who can join: the full rules

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.

Where it's running

• Optimal Clinical Trials
  Auckland, North Island, New Zealand Recruiting
• NZCR-Christchurch
  Christchurch, South Island, New Zealand Recruiting
• LTD TIM - Tbilisi Institute of Medicine
  Tbilisi, Georgia Not yet recruiting
• Institute of Neurology and Neuropsychology LTD
  Tbilisi, Georgia Not yet recruiting

Study contacts

• Study coordinator: Gina Weaver
• Email: gweaver@kerostx.com
• Phone: 267.799.3345

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• VM100: a nutritional supplement to see if it improves quality of life for boys with Duchenne

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• ELEVIDYS safety in Duchenne patients treated after market approval

  This study tests the safety of ELEVIDYS, with sirolimus as a protective treatment, in male children and young adults with Duchenne Muscular Dystrophy who have previously received ELEVIDYS or are eligible to receive it commercially.

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• SGT-003 gene therapy for ambulant boys and young men with Duchenne muscular dystrophy (IMPACT DUCHENNE)

  This trial will test whether a single intravenous infusion of SGT-003 can help ambulatory boys and young men with Duchenne muscular dystrophy maintain or improve walking and motor function.

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• Evaluating Givinostat for Young Children with Duchenne Muscular Dystrophy

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• Evaluating respiratory functions and exercise capacity in children with neuromuscular diseases

  This study is trying to see how well children with Duchenne Muscular Dystrophy and Spinal Muscular Atrophy can breathe and exercise compared to healthy kids.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.