Duchenne Muscular Dystrophy (DMD)Phase 2NCT07911995
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
A Phase 2 study testing Tegacorat in people with Duchenne Muscular Dystrophy (DMD), run by Grünenthal GmbH.
Trial summary
This Phase 2 clinical trial will evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of tegacorat oral solution in male participants 4 to less than 7 years of age with Duchenne muscular dystrophy (DMD). Read more about this study
- Who is it for?
- Ages 4 to 6 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- Tegacorat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- No sites listed yet
- Study dates
- 9 Nov 2026to 4 Jan 2030
- 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 4 to 6 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male participant aged ≥4 years and <7 years at randomization.
- Clinically and genetically confirmed diagnosis of Duchenne muscular dystrophy.
- Body weight between 13 kg and 35 kg at screening.
- Able to walk independently without assistive devices.
- Able to complete the TTSTAND assessment without assistance in less than 10 seconds.
- Evidence of varicella immunity by serology or vaccination history.
- Parent(s)/legal guardian(s) provide informed consent and participant assent as required.
Who cannot join
- Moderate or severe renal impairment.
- Moderate or severe hepatic impairment.
- Clinically significant abnormal laboratory findings.
- Chronic or latent infection that may increase study risk.
- Acute illness that could affect safety.
- Symptomatic cardiomyopathy.
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- Diabetes mellitus.
- Severe behavioral, cognitive, or psychiatric disorders interfering with study participation.
- Adrenal insufficiency.
- Clinically significant ECG abnormalities.
- Hypersensitivity to study intervention or its components.
- Current or recent use of prohibited treatments or investigational products.
- Prior exposure to delandistrogene moxeparvovec.
- Participation in another clinical trial within the protocol-defined washout period.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Randomized, Open-label Phase 2 Trial Consisting of a 24-Week Core Phase Followed by a 72-Week Extension Phase to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat Oral Solution in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy (DMD)
This Phase 2 clinical trial will evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of tegacorat oral solution in male participants 4 to less than 7 years of age with Duchenne muscular dystrophy (DMD). Participants will be randomized to one of three tegacorat treatment groups during a 24-week Core Phase. Participants who complete the Core Phase will continue into a 72-week Extension Phase. The study will assess muscle function, ambulatory performance, safety outcomes, pharmacokinetics, pharmacodynamic biomarkers, and health-related quality of life over a total treatment period of up to 96 weeks.
Read the detailed description
DMD is a severe, progressive neuromuscular disorder caused by mutations in the dystrophin gene, resulting in muscle degeneration and loss of function. Current standard-of-care glucocorticoid therapy may slow disease progression but is associated with significant long-term adverse effects. Tegacorat is a selective glucocorticoid receptor agonist and modulator (SEGRAM) that has demonstrated anti-inflammatory activity with reduced glucocorticoid receptor-mediated transactivation in clinical and nonclinical studies.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- Tegacorat
- Keywords
- Tegacorat, Duchenne Muscular Dystrophy, SEGRAM, Muscular Dystrophy, Pediatric Neuromuscular Disease, Glucocorticoid Receptor Modulator
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TegacoratDrug
Oral solution
| Study group | Role | What they receive |
|---|---|---|
| Tegacorat Low dose | Experimental | Tegacorat Treatment Group 1 Receives: Drug: Tegacorat |
| Tegacorat Mid Dose | Experimental | Tegacorat Treatment Group 2 Receives: Drug: Tegacorat |
| Tegacorat High dose | Experimental | Tegacorat Treatment Group 3 Receives: Drug: Tegacorat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
Change from baseline to Week 24 in TTSTAND velocity.
Measured: Baseline to Week 24
Extension Phase: Incidence of Adverse Events
Safety and Tolerability.
Measured: First dose through Week 96
Secondary outcomes (17)
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
Change from baseline to Week 24 in SV95C measured using wearable sensor technology.
Measured: Baseline to Week 24
Core Phase: Change From Baseline in 10-Meter Walk/Run (10MWR) velocity
Change From Baseline in Functional Test Performance.
Measured: Baseline to Week 24
Core Phase: Change From Baseline in Time-to-Climb-4-Stairs (TTCLIMB) velocity
Change From Baseline in Functional Test Performance.
Measured: Baseline to Week 24
Core Phase: Incidence of Adverse Events
Safety and Tolerability.
Measured: First dose through Week 24
Extension Phase: Time to Treatment Discontinuation Due to Adverse Events
Time to permanent discontinuation of study treatment due to an adverse event.
Measured: Up to Week 96
Extension Phase: Change From Baseline in Weight and BMI Z-Scores
Change from baseline in weight and body mass index (BMI) Z-scores.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in Cushingoid Features
Change from baseline in Cushingoid features.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in Glucose Metabolism
Change from baseline in fasting glucose and fasting serum insulin.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in PARS-III Score
Change from baseline in Personal Adjustment and Role Skills Scale (PARS-III) score.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in Bone Mineral Density
Change from baseline in bone mineral density assessed by dual-energy X-ray absorptiometry (DXA).
Measured: Baseline to Week 96
Extension Phase: Vertebral Fracture Assessment
Assessment of vertebral fractures by lateral thoracolumbar spine X-ray.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in Height Z-Score
Change from baseline in height Z-score.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in Bone Age
Change from baseline in bone age assessed by left hand X-ray.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in TTSTAND Velocity
Change From Baseline in Functional Test Performance.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in 10MWR Velocity
Change From Baseline in Functional Test Performance.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in TTCLIMB Velocity
Change From Baseline in Functional Test Performance.
Measured: Baseline to Week 96
Extension Phase: Change From Baseline in SV95C
Change from baseline in SV95C measured using wearable sensor technology.
Measured: Baseline to Week 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2026
- Study start9 Nov 2026 (estimated)
- Primary completion4 Jan 2030 (estimated)
- Study completion4 Jan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Grünenthal GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07911995
- Sponsor's protocol ID
- KF6019-02
- Other IDs
- 2026-526326-40-00, U1111-1339-2419
- Status last verified
- Oct 2026
- First submitted
- 27 Oct 2026
- Last updated
- 3 Nov 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Information available on the Grünenthal Group Web Site (https://www.grunenthal.com/science/clinical-trial-transparency/data-sharing) and according to the European Federation of Pharmaceutical Industries and Associations (EFPIA) Data Sharing Principles
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-label Phase 2 Trial Consisting of a 24-Week Core Phase Followed by a 72-Week Extension Phase to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat Oral Solution in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT07911995
- Acronym / sponsor ID
- KF6019-02
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Tegacorat, Duchenne Muscular Dystrophy, SEGRAM, Muscular Dystrophy, Pediatric Neuromuscular Disease, Glucocorticoid Receptor Modulator
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Grünenthal GmbHIndustry (pharma, biotech, devices)
- Study start
- 9 Nov 2026 (estimated)
- Primary completion
- 4 Jan 2030 (estimated)
- Study completion
- 4 Jan 2030 (estimated)
- First posted
- 3 Nov 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.
This Phase 2 clinical trial will evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of tegacorat oral solution in male participants 4 to less than 7 years of age with Duchenne muscular dystrophy (DMD). Participants will be randomized to one of three tegacorat treatment groups during a 24-week Core Phase. Participants who complete the Core Phase will continue into a 72-week Extension Phase. The study will assess muscle function, ambulatory performance, safety outcomes, pharmacokinetics, pharmacodynamic biomarkers, and health-related quality of life over a total treatment period of up to 96 weeks.
Detailed description
DMD is a severe, progressive neuromuscular disorder caused by mutations in the dystrophin gene, resulting in muscle degeneration and loss of function. Current standard-of-care glucocorticoid therapy may slow disease progression but is associated with significant long-term adverse effects. Tegacorat is a selective glucocorticoid receptor agonist and modulator (SEGRAM) that has demonstrated anti-inflammatory activity with reduced glucocorticoid receptor-mediated transactivation in clinical and nonclinical studies.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Tegacorat Low dose | Experimental | Drug: TegacoratTegacorat Treatment Group 1 |
| Tegacorat Mid Dose | Experimental | Drug: TegacoratTegacorat Treatment Group 2 |
| Tegacorat High dose | Experimental | Drug: TegacoratTegacorat Treatment Group 3 |
Who may qualify
- Ages
- 4 to 6 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Male participant aged ≥4 years and <7 years at randomization.
- Clinically and genetically confirmed diagnosis of Duchenne muscular dystrophy.
- Body weight between 13 kg and 35 kg at screening.
- Able to walk independently without assistive devices.
- Able to complete the TTSTAND assessment without assistance in less than 10 seconds.
Key exclusion criteria (14 listed)
- Moderate or severe renal impairment.
- Moderate or severe hepatic impairment.
- Clinically significant abnormal laboratory findings.
- Chronic or latent infection that may increase study risk.
- Acute illness that could affect safety.
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 |
|---|---|---|
| TegacoratOral solution | Drug | Tegacorat High dose, Tegacorat Low dose, Tegacorat Mid Dose |
Primary outcome
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
Time frame: Baseline to Week 24
Change from baseline to Week 24 in TTSTAND velocity.
Extension Phase: Incidence of Adverse Events
Time frame: First dose through Week 96
Safety and Tolerability.
17 secondary outcomes
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
Time frame: Baseline to Week 24
Core Phase: Change From Baseline in 10-Meter Walk/Run (10MWR) velocity
Time frame: Baseline to Week 24
Core Phase: Change From Baseline in Time-to-Climb-4-Stairs (TTCLIMB) velocity
Time frame: Baseline to Week 24
Core Phase: Incidence of Adverse Events
Time frame: First dose through Week 24
Extension Phase: Time to Treatment Discontinuation Due to Adverse Events
Time frame: Up to Week 96
Extension Phase: Change From Baseline in Weight and BMI Z-Scores
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in Cushingoid Features
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in Glucose Metabolism
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in PARS-III Score
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in Bone Mineral Density
Time frame: Baseline to Week 96
Extension Phase: Vertebral Fracture Assessment
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in Height Z-Score
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in Bone Age
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in TTSTAND Velocity
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in 10MWR Velocity
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in TTCLIMB Velocity
Time frame: Baseline to Week 96
Extension Phase: Change From Baseline in SV95C
Time frame: Baseline to Week 96
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 3 Nov 2026.
- Individual participant dataSharing planned: Yes. Information available on the Grünenthal Group Web Site (https://www.grunenthal.com/science/clinical-trial-transparency/data-sharing) and according to the European Federation of Pharmaceutical Industries and Associations (EFPIA) Data Sharing Principles
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tegacorat in people with Duchenne Muscular Dystrophy (DMD), run by Grünenthal GmbH.
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 7 inclusion and 14 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: opening soon, as of the sponsor's last update on 3 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
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 Grünenthal Clinical Trial Helpdesk (+49 241 569 3223, clinical-trials@grunenthal.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity (time frame: Baseline to Week 24). The study lists 2 primary outcomes in all. 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 3 Nov 2026.