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.
연구 개요
상세 설명
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.
연구 유형
등록 (추정된)
단계
- 2 단계
연락처 및 위치
연구 연락처
- 이름: Grünenthal Clinical Trial Helpdesk
- 전화번호: +49 241 569 3223
- 이메일: clinical-trials@grunenthal.com
참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
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.
공부 계획
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디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: Tegacorat Low dose
Tegacorat Treatment Group 1 |
Oral solution |
|
실험적: Tegacorat Mid Dose
Tegacorat Treatment Group 2 |
Oral solution |
|
실험적: Tegacorat High dose
Tegacorat Treatment Group 3 |
Oral solution |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
기간: Baseline to Week 24
|
Change from baseline to Week 24 in TTSTAND velocity. |
Baseline to Week 24
|
|
Extension Phase: Incidence of Adverse Events
기간: First dose through Week 96
|
Safety and Tolerability. |
First dose through Week 96
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
기간: 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
기간: 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
기간: Baseline to Week 24
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 24
|
|
Core Phase: Incidence of Adverse Events
기간: First dose through Week 24
|
Safety and Tolerability. |
First dose through Week 24
|
|
Extension Phase: Time to Treatment Discontinuation Due to Adverse Events
기간: 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
기간: 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
기간: Baseline to Week 96
|
Change from baseline in Cushingoid features. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Glucose Metabolism
기간: 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
기간: 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
기간: 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
기간: 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
기간: Baseline to Week 96
|
Change from baseline in height Z-score. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Bone Age
기간: 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
기간: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in 10MWR Velocity
기간: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in TTCLIMB Velocity
기간: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in SV95C
기간: Baseline to Week 96
|
Change from baseline in SV95C measured using wearable sensor technology. |
Baseline to Week 96
|
공동 작업자 및 조사자
스폰서
연구 기록 날짜
연구 주요 날짜
연구 시작 (추정된)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- KF6019-02
- 2026-526326-40-00 (씨티스)
- U1111-1339-2419 (기타 식별자: Universal Trial Number)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
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 공유 지원 정보 유형
- 연구_프로토콜
- ICF
- CSR
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