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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

27 oktober 2026 uppdaterad av: Grünenthal GmbH

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.

Studieöversikt

Status

Har inte rekryterat ännu

Betingelser

Intervention / Behandling

Detaljerad beskrivning

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.

Studietyp

Interventionell

Inskrivning (Beräknad)

36

Fas

  • Fas 2

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

4 år till 6 år (Barn)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Manlig

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: Randomiserad
  • Interventionsmodell: Parallellt uppdrag
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: Tegacorat Low dose

Tegacorat Treatment Group 1
Läkemedel: Tegacorat

Oral solution
Experimentell: Tegacorat Mid Dose

Tegacorat Treatment Group 2
Läkemedel: Tegacorat

Oral solution
Experimentell: Tegacorat High dose

Tegacorat Treatment Group 3
Läkemedel: Tegacorat

Oral solution

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
Tidsram: Baseline to Week 24

Change from baseline to Week 24 in TTSTAND velocity.
Baseline to Week 24
Extension Phase: Incidence of Adverse Events
Tidsram: First dose through Week 96

Safety and Tolerability.
First dose through Week 96

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
Tidsram: 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
Tidsram: 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
Tidsram: Baseline to Week 24

Change From Baseline in Functional Test Performance.
Baseline to Week 24
Core Phase: Incidence of Adverse Events
Tidsram: First dose through Week 24

Safety and Tolerability.
First dose through Week 24
Extension Phase: Time to Treatment Discontinuation Due to Adverse Events
Tidsram: 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
Tidsram: 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
Tidsram: Baseline to Week 96

Change from baseline in Cushingoid features.
Baseline to Week 96
Extension Phase: Change From Baseline in Glucose Metabolism
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: Baseline to Week 96

Change from baseline in height Z-score.
Baseline to Week 96
Extension Phase: Change From Baseline in Bone Age
Tidsram: 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
Tidsram: Baseline to Week 96

Change From Baseline in Functional Test Performance.
Baseline to Week 96
Extension Phase: Change From Baseline in 10MWR Velocity
Tidsram: Baseline to Week 96

Change From Baseline in Functional Test Performance.
Baseline to Week 96
Extension Phase: Change From Baseline in TTCLIMB Velocity
Tidsram: Baseline to Week 96

Change From Baseline in Functional Test Performance.
Baseline to Week 96
Extension Phase: Change From Baseline in SV95C
Tidsram: Baseline to Week 96

Change from baseline in SV95C measured using wearable sensor technology.
Baseline to Week 96

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Grünenthal GmbH

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Beräknad)

9 november 2026

Primärt slutförande (Beräknad)

4 januari 2030

Avslutad studie (Beräknad)

4 januari 2030

Studieregistreringsdatum

Först inskickad

27 oktober 2026

Först inskickad som uppfyllde QC-kriterierna

27 oktober 2026

Första postat (Faktisk)

3 november 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

3 november 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

27 oktober 2026

Senast verifierad

1 oktober 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • KF6019-02
  • 2026-526326-40-00 (Ctis)
  • U1111-1339-2419 (Annan identifierare: Universal Trial Number)

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

JA

IPD-planbeskrivning

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-delning som stöder informationstyp

  • STUDY_PROTOCOL
  • ICF
  • CSR

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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