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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 opdateret af: 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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

36

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

4 år til 6 år (Barn)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Han

Beskrivelse

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

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Tegacorat Low dose

Tegacorat Treatment Group 1
Medicin: Tegacorat

Oral solution
Eksperimentel: Tegacorat Mid Dose

Tegacorat Treatment Group 2
Medicin: Tegacorat

Oral solution
Eksperimentel: Tegacorat High dose

Tegacorat Treatment Group 3
Medicin: Tegacorat

Oral solution

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
Tidsramme: Baseline to Week 24

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

Safety and Tolerability.
First dose through Week 96

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
Tidsramme: 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
Tidsramme: 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
Tidsramme: Baseline to Week 24

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

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

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

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

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

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

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

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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Grünenthal GmbH

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

9. november 2026

Primær færdiggørelse (Anslået)

4. januar 2030

Studieafslutning (Anslået)

4. januar 2030

Datoer for studieregistrering

Først indsendt

27. oktober 2026

Først indsendt, der opfyldte QC-kriterier

27. oktober 2026

Først opslået (Faktiske)

3. november 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

3. november 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

27. oktober 2026

Sidst verificeret

1. oktober 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

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

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

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-deling Understøttende informationstype

  • STUDY_PROTOCOL
  • ICF
  • CSR

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

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