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.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
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.
Type d'étude
Inscription (Estimé)
Phase
- Phase 2
Contacts et emplacements
Coordonnées de l'étude
- Nom: Grünenthal Clinical Trial Helpdesk
- Numéro de téléphone: +49 241 569 3223
- E-mail: clinical-trials@grunenthal.com
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La 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.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: Randomisé
- Modèle interventionnel: Affectation parallèle
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: Tegacorat Low dose
Tegacorat Treatment Group 1 |
Oral solution |
|
Expérimental: Tegacorat Mid Dose
Tegacorat Treatment Group 2 |
Oral solution |
|
Expérimental: Tegacorat High dose
Tegacorat Treatment Group 3 |
Oral solution |
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
Délai: Baseline to Week 24
|
Change from baseline to Week 24 in TTSTAND velocity. |
Baseline to Week 24
|
|
Extension Phase: Incidence of Adverse Events
Délai: First dose through Week 96
|
Safety and Tolerability. |
First dose through Week 96
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
Délai: Baseline to Week 24
|
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
Délai: Baseline to Week 24
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 24
|
|
Core Phase: Change From Baseline in Time-to-Climb-4-Stairs (TTCLIMB) velocity
Délai: Baseline to Week 24
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 24
|
|
Core Phase: Incidence of Adverse Events
Délai: First dose through Week 24
|
Safety and Tolerability. |
First dose through Week 24
|
|
Extension Phase: Time to Treatment Discontinuation Due to Adverse Events
Délai: Up to Week 96
|
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
Délai: Baseline to Week 96
|
Change from baseline in weight and body mass index (BMI) Z-scores. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Cushingoid Features
Délai: Baseline to Week 96
|
Change from baseline in Cushingoid features. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Glucose Metabolism
Délai: Baseline to Week 96
|
Change from baseline in fasting glucose and fasting serum insulin. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in PARS-III Score
Délai: Baseline to Week 96
|
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
Délai: Baseline to Week 96
|
Change from baseline in bone mineral density assessed by dual-energy X-ray absorptiometry (DXA). |
Baseline to Week 96
|
|
Extension Phase: Vertebral Fracture Assessment
Délai: Baseline to Week 96
|
Assessment of vertebral fractures by lateral thoracolumbar spine X-ray. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Height Z-Score
Délai: Baseline to Week 96
|
Change from baseline in height Z-score. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Bone Age
Délai: Baseline to Week 96
|
Change from baseline in bone age assessed by left hand X-ray. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in TTSTAND Velocity
Délai: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in 10MWR Velocity
Délai: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in TTCLIMB Velocity
Délai: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in SV95C
Délai: Baseline to Week 96
|
Change from baseline in SV95C measured using wearable sensor technology. |
Baseline to Week 96
|
Collaborateurs et enquêteurs
Parrainer
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Estimé)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
- Maladies musculo-squelettiques
- Maladies du système nerveux
- Maladies musculaires
- Maladies neuromusculaires
- Maladies génétiques, innées
- Maladies génétiques liées à l'X
- Troubles musculaires atrophiques
- Maladies et anomalies congénitales, héréditaires et néonatales
- Dystrophies musculaires
- Dystrophie Musculaire, Duchenne
Autres numéros d'identification d'étude
- KF6019-02
- 2026-526326-40-00 (Ctis)
- U1111-1339-2419 (Autre identifiant: Universal Trial Number)
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Description du régime IPD
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
Type d'informations de prise en charge du partage d'IPD
- PROTOCOLE D'ÉTUDE
- CIF
- RSE
Informations sur les médicaments et les dispositifs, documents d'étude
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