Phase 3 Clinical Trial Evaluating the Efficacy and Safety of Navepegritide and Lonapegsomatropin in Achondroplasia (cAtCH)
A Phase 3, Randomized, Double-blind, Active Controlled, Multi-center Trial Investigating the Efficacy and Safety of Dual Therapy With Once-weekly Navepegritide and Once-weekly Lonapegsomatropin Compared to Navepegritide Monotherapy and Lonapegsomatropin Monotherapy in Children and Adolescents With Achondroplasia
This pivotal trial is being conducted to evaluate the efficacy, safety and tolerability of dual therapy with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 17 years.
연구 개요
상태
정황
연구 유형
등록 (추정된)
단계
- 3단계
연락처 및 위치
연구 연락처
- 이름: Ascendis Registry Inquiries
- 전화번호: +45 61242484
- 이메일: asnd_registryinquiries@ascendispharma.com
참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- 1. Age 2 to <18 years at randomization
- 2. Clinical diagnosis of ACH with historical genetic confirmation
- 3. At least 1 height measurement available from medical records
Exclusion Criteria:
- 1. Treatment with growth hormone within 1 year prior to randomization
- 2. Prior treatment with certain medication(s) which potentially could have affected growth (prior treatment with vosoritide is not exclusive)
- 3. History of any surgery intended to affect stature, growth, or body proportionality except procedures for leg bowing
- 4. Unable to stand without assistance
- 5. Known or suspected medical conditions other than achondroplasia that could affect growth
- 6. Known history or presence of injury of the growth plates that affects growth potential of long bones
- 7. Known history or presence of malignant disease
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 네 배로
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: Once weekly navepegritide SC and lonapegsomatropin SC
|
Administered weekly as two separate SC injections. Treatment duration of up to 104 weeks |
|
실험적: Once weekly navepegritide SC and placebo for lonapegsomatropin SC
|
Administered as two separate SC injections. Treatment duration of up to 52 weeks of navepegritide and placebo for lonapegsomatropin followed by 52 weeks of navepegritide and lonapegsomatropin. |
|
실험적: Once weekly lonapegsomatropin SC and placebo for navepegritide SC
|
Administered as two separate SC injections. Treatment duration of up to 52 weeks of lonapegsomatropin and placebo for navepegritide followed by 52 weeks of navepegritide and lonapegsomatropin. |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Annualized Growth Velocity (AGV)
기간: 52 weeks
|
To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to navepegritide alone |
52 weeks
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Treatment-emergent Adverse Events (AE)
기간: Throughout trial duration 104 weeks
|
Number of events |
Throughout trial duration 104 weeks
|
|
Annualized Growth Velocity (AGV)
기간: 52 weeks
|
To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to lonapegsomatroptin alone |
52 weeks
|
|
Annualized Growth Velocity (AGV) change from baseline
기간: 52 weeks
|
To evaluate the change from baseline in AGV, cm per year |
52 weeks
|
|
ACH-specific height Z-score
기간: 52 weeks
|
To evaluate the change from baseline in ACH-specific height Z-score |
52 weeks
|
|
CDC-based height Z-score
기간: 52 weeks
|
To evaluate the change from baseline in CDC-based height Z-score |
52 weeks
|
|
Bone safety
기간: Throughout trial duration 104 weeks
|
Qualitative assessment with radiology |
Throughout trial duration 104 weeks
|
|
Arm span Z-score
기간: 52 weeks
|
Change from baseline in arm span Z-score. |
52 weeks
|
|
Upper to lower body segment ratio
기간: 52 weeks
|
Change from baseline in upper to lower body segment ratio. |
52 weeks
|
|
Arm span
기간: 52 weeks
|
Change from baseline in arm span, cm. |
52 weeks
|
|
Interpedicular distance (IDP)
기간: 52 weeks
|
Change from baseline in IDP (L1-L4 average), mm |
52 weeks
|
공동 작업자 및 조사자
수사관
- 연구 책임자: Medical Director, MD, Ascendis Pharma A/S
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
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