Duchenne Muscular DystrophyPhase 2NCT07704099
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
A Phase 2 study testing KER-065 in people with Duchenne Muscular Dystrophy, run by Keros Therapeutics, Inc. at 4 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 9 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- KER-065
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 25 Sep 2026to 23 Sep 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 9 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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)
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- 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.
Trial locations and contacts
4 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 2 countries
Georgia2 sites, 2 recruiting
- Not yet recruiting
LTD TIM - Tbilisi Institute of Medicine
Tbilisi, 0160
- Not yet recruiting
Institute of Neurology and Neuropsychology LTD
Tbilisi, 0186
New Zealand2 sites, 2 recruiting
- Recruiting
Optimal Clinical Trials
Auckland, North Island, 1010
- Recruiting
NZCR-Christchurch
Christchurch, South Island, 8011
About this study
Official title: 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.
Read the 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 (48 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 (Early Nonambulatory)
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- KER-065
- Keywords
- Recombinant fusion protein, Muscle-wasting disease, Ambulatory function, Pharmacokinetics, Pharmacodynamics
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KER-065Drug
Also called Rinvatercept
KER-065 will be administered subcutaneously (SC)
| Study group | Role | What they receive |
|---|---|---|
| Cohort A1 (Late Ambulatory) | Experimental | Participants will receive stable corticosteroid (CS) along with KER-065. Receives: Drug: KER-065 |
| Cohort A2 (Late Ambulatory) | Experimental | Participants will receive stable CS, exon skipper along with KER-065. Receives: Drug: KER-065 |
| Cohort N1 (Early Nonambulatory) | Experimental | Participants will receive stable CS along with KER-065. Receives: Drug: KER-065 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to approximately 14 months
Secondary outcomes (9)
KER-065 serum concentration by visit, as appropriate
To assess the pharmacokinetics (PK) of KER-065 in ambulatory and nonambulatory participants with DMD
Measured: Up to Week 52
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
Measured: Up to Week 52
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
Measured: Week 12, Week 24 and Week 48
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
Measured: Week 24 and Week 48
Ambulatory: Change from baseline by visit in North Star Ambulatory Assessment (NSAA) total score
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Measured: Week 24 and Week 48
Ambulatory: Change from baseline by visit in 4-stair climb (4SC)
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Measured: Week 24 and Week 48
Ambulatory: Change from baseline by visit in 10-meter walk/run (10MWR) test
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Measured: Week 24 and Week 48
Ambulatory: Change from baseline by visit in time to rise (TTR)
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Measured: Week 24 and Week 48
Nonambulatory: Change from baseline by visit in Performance of Upper Limb (PUL) v2.0 score
To assess the effect of KER-065 on motor function in nonambulatory participants with DMD
Measured: Week 24 and Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jul 2026
- Study start25 Sep 2026
- Primary completion26 Aug 2028 (estimated)
- Study completion23 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Keros Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07704099
- Sponsor's protocol ID
- KER-065-B201
- Status last verified
- Oct 2026
- First submitted
- 9 Jul 2026
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Phase 2, Open-Label Study Evaluating the Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT07704099
- Acronym / sponsor ID
- KER-065-B201
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Recombinant fusion protein, Muscle-wasting disease, Ambulatory function, Pharmacokinetics, Pharmacodynamics
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Keros Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 25 Sep 2026 (actual)
- Primary completion
- 26 Aug 2028 (estimated)
- Study completion
- 23 Sep 2028 (estimated)
- First posted
- 15 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 (48 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 (Early Nonambulatory)
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort A1 (Late Ambulatory) | Experimental | Drug: KER-065Participants will receive stable corticosteroid (CS) along with KER-065. |
| Cohort A2 (Late Ambulatory) | Experimental | Drug: KER-065Participants will receive stable CS, exon skipper along with KER-065. |
| Cohort N1 (Early Nonambulatory) | Experimental | Drug: KER-065Participants will receive stable CS along with KER-065. |
Who may qualify
- Ages
- 9 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
- Able to TTR in < 10 seconds.
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KER-065Also: RinvaterceptKER-065 will be administered subcutaneously (SC) | Drug | Cohort A1 (Late Ambulatory), Cohort A2 (Late Ambulatory), Cohort N1 (Early Nonambulatory) |
Primary outcome
Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs)
Time frame: Up to approximately 14 months
To evaluate the safety and tolerability of KER-065 in ambulatory and nonambulatory participants with DMD
9 secondary outcomes
KER-065 serum concentration by visit, as appropriate
Time frame: Up to Week 52
Number and proportion of participants with treatment-emergent ADA (antidrug antibody) by visit
Time frame: Up to Week 52
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: Week 12, Week 24 and Week 48
Change from baseline by visit in muscle volume and intramuscular fat by skeletal muscle Magnetic Resonance Imaging (MRI)
Time frame: Week 24 and Week 48
Ambulatory: Change from baseline by visit in North Star Ambulatory Assessment (NSAA) total score
Time frame: Week 24 and Week 48
Ambulatory: Change from baseline by visit in 4-stair climb (4SC)
Time frame: Week 24 and Week 48
Ambulatory: Change from baseline by visit in 10-meter walk/run (10MWR) test
Time frame: Week 24 and Week 48
Ambulatory: Change from baseline by visit in time to rise (TTR)
Time frame: Week 24 and Week 48
Nonambulatory: Change from baseline by visit in Performance of Upper Limb (PUL) v2.0 score
Time frame: Week 24 and Week 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing KER-065 in people with Duchenne Muscular Dystrophy, run by Keros Therapeutics, Inc. at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Georgia, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Gina Weaver (267.799.3345, gweaver@kerostx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs) (time frame: Up to approximately 14 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2026.