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.
Studienübersicht
Status
Bedingungen
Intervention / Behandlung
Detaillierte Beschreibung
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.
Studientyp
Einschreibung (Geschätzt)
Phase
- Phase 2
Kontakte und Standorte
Studienkontakt
- Name: Grünenthal Clinical Trial Helpdesk
- Telefonnummer: +49 241 569 3223
- E-Mail: clinical-trials@grunenthal.com
Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
Akzeptiert gesunde Freiwillige
Studienberechtigte Geschlechter
Beschreibung
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.
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: Zufällig
- Interventionsmodell: Parallele Zuordnung
- Maskierung: Keine (Offenes Etikett)
Waffen und Interventionen
Teilnehmergruppe / Arm |
Intervention / Behandlung |
|---|---|
|
Experimental: Tegacorat Low dose
Tegacorat Treatment Group 1 |
Oral solution |
|
Experimental: Tegacorat Mid Dose
Tegacorat Treatment Group 2 |
Oral solution |
|
Experimental: Tegacorat High dose
Tegacorat Treatment Group 3 |
Oral solution |
Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
|
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
Zeitfenster: Baseline to Week 24
|
Change from baseline to Week 24 in TTSTAND velocity. |
Baseline to Week 24
|
|
Extension Phase: Incidence of Adverse Events
Zeitfenster: First dose through Week 96
|
Safety and Tolerability. |
First dose through Week 96
|
Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
|
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: Baseline to Week 24
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 24
|
|
Core Phase: Incidence of Adverse Events
Zeitfenster: First dose through Week 24
|
Safety and Tolerability. |
First dose through Week 24
|
|
Extension Phase: Time to Treatment Discontinuation Due to Adverse Events
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: Baseline to Week 96
|
Change from baseline in Cushingoid features. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Glucose Metabolism
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: Baseline to Week 96
|
Change from baseline in height Z-score. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Bone Age
Zeitfenster: 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
Zeitfenster: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in 10MWR Velocity
Zeitfenster: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in TTCLIMB Velocity
Zeitfenster: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in SV95C
Zeitfenster: Baseline to Week 96
|
Change from baseline in SV95C measured using wearable sensor technology. |
Baseline to Week 96
|
Mitarbeiter und Ermittler
Sponsor
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Geschätzt)
Primärer Abschluss (Geschätzt)
Studienabschluss (Geschätzt)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
Zusätzliche relevante MeSH-Bedingungen
- Erkrankungen des Bewegungsapparates
- Erkrankungen des Nervensystems
- Muskelerkrankungen
- Neuromuskuläre Erkrankungen
- Genetische Krankheiten, angeboren
- Genetische Krankheiten, X-gebunden
- Muskelerkrankungen, atrophisch
- Angeborene, erbliche und neonatale Krankheiten und Anomalien
- Muskeldystrophien
- Muskeldystrophie, Duchenne
Andere Studien-ID-Nummern
- KF6019-02
- 2026-526326-40-00 (Ctis)
- U1111-1339-2419 (Andere Kennung: Universal Trial Number)
Plan für individuelle Teilnehmerdaten (IPD)
Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?
Beschreibung des IPD-Plans
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
Art der unterstützenden IPD-Freigabeinformationen
- STUDIENPROTOKOLL
- ICF
- CSR
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Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
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