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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 oppdatert 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.

Studieoversikt

Status

Har ikke rekruttert ennå

Forhold

Intervensjon / Behandling

Detaljert 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.

Studietype

Intervensjonell

Registrering (Antatt)

36

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

4 år til 6 år (Barn)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Mann

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Tegacorat Low dose

Tegacorat Treatment Group 1
Legemiddel: Tegacorat

Oral solution
Eksperimentell: Tegacorat Mid Dose

Tegacorat Treatment Group 2
Legemiddel: Tegacorat

Oral solution
Eksperimentell: Tegacorat High dose

Tegacorat Treatment Group 3
Legemiddel: Tegacorat

Oral solution

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
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
Tiltaksbeskrivelse
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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Grünenthal GmbH

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Antatt)

9. november 2026

Primær fullføring (Antatt)

4. januar 2030

Studiet fullført (Antatt)

4. januar 2030

Datoer for studieregistrering

Først innsendt

27. oktober 2026

Først innsendt som oppfylte QC-kriteriene

27. oktober 2026

Først lagt ut (Faktiske)

3. november 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

3. november 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

27. oktober 2026

Sist bekreftet

1. oktober 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

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

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (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 Støtteinformasjonstype

  • STUDY_PROTOCOL
  • ICF
  • CSR

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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