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 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.

Study Overview

• Status: Not yet recruiting
• Conditions: Duchenne Muscular Dystrophy (DMD)
• Intervention / Treatment: Drug: Tegacorat

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 Type: Interventional
• Enrollment (Estimated): 36
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Grünenthal Clinical Trial Helpdesk; Phone Number: +49 241 569 3223; Email: clinical-trials@grunenthal.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 6 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

INCLUSION CRITERIA:

• 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.

EXCLUSION CRITERIA:

• 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.
• 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tegacorat Low dose; Tegacorat Treatment Group 1	Drug: Tegacorat; Oral solution
Experimental: Tegacorat Mid Dose; Tegacorat Treatment Group 2	Drug: Tegacorat; Oral solution
Experimental: Tegacorat High dose; Tegacorat Treatment Group 3	Drug: Tegacorat; Oral solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity	Change from baseline to Week 24 in TTSTAND velocity.	Baseline to Week 24
Extension Phase: Incidence of Adverse Events	Safety and Tolerability.	First dose through Week 96

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)	Change from baseline to Week 24 in SV95C measured using wearable sensor technology.	Baseline to Week 24
Core Phase: Change From Baseline in 10-Meter Walk/Run (10MWR) velocity	Change From Baseline in Functional Test Performance.	Baseline to Week 24
Core Phase: Change From Baseline in Time-to-Climb-4-Stairs (TTCLIMB) velocity	Change From Baseline in Functional Test Performance.	Baseline to Week 24
Core Phase: Incidence of Adverse Events	Safety and Tolerability.	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.	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.	Baseline to Week 96
Extension Phase: Change From Baseline in Cushingoid Features	Change from baseline in Cushingoid features.	Baseline to Week 96
Extension Phase: Change From Baseline in Glucose Metabolism	Change from baseline in fasting glucose and fasting serum insulin.	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.	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).	Baseline to Week 96
Extension Phase: Vertebral Fracture Assessment	Assessment of vertebral fractures by lateral thoracolumbar spine X-ray.	Baseline to Week 96
Extension Phase: Change From Baseline in Height Z-Score	Change from baseline in height Z-score.	Baseline to Week 96
Extension Phase: Change From Baseline in Bone Age	Change from baseline in bone age assessed by left hand X-ray.	Baseline to Week 96
Extension Phase: Change From Baseline in TTSTAND Velocity	Change From Baseline in Functional Test Performance.	Baseline to Week 96
Extension Phase: Change From Baseline in 10MWR Velocity	Change From Baseline in Functional Test Performance.	Baseline to Week 96
Extension Phase: Change From Baseline in TTCLIMB Velocity	Change From Baseline in Functional Test Performance.	Baseline to Week 96
Extension Phase: Change From Baseline in SV95C	Change from baseline in SV95C measured using wearable sensor technology.	Baseline to Week 96

Collaborators and Investigators

• Sponsor: Grünenthal GmbH

Study record dates

Study Major Dates

• Study Start (Estimated): November 9, 2026
• Primary Completion (Estimated): January 4, 2030
• Study Completion (Estimated): January 4, 2030

Study Registration Dates

• First Submitted: October 27, 2026
• First Submitted That Met QC Criteria: October 27, 2026
• First Posted (Actual): November 3, 2026

Study Record Updates

• Last Update Posted (Actual): November 3, 2026
• Last Update Submitted That Met QC Criteria: October 27, 2026
• Last Verified: October 1, 2026

More Information

Terms related to this study

• Keywords: Duchenne Muscular Dystrophy; Muscular Dystrophy; Tegacorat; SEGRAM; Pediatric Neuromuscular Disease; Glucocorticoid Receptor Modulator
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: KF6019-02; 2026-526326-40-00 (Ctis); U1111-1339-2419 (Other Identifier: Universal Trial Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

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

• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No