Study of Velaglucerase Beta ERT in Children With Gaucher Disease
A Multi-center, Open-label Study to Evaluate the Safety and Efficacy of Velaglucerase Beta Enzyme Replacement Therapy in Children With Gaucher Disease
This is a Phase 3, multicenter, open-label, single-arm study to evaluate the safety, efficacy, PK, PD, and immunogenicity of velaglucerase beta in untreated children with GD1 or GD3 for 39 weeks.
연구 개요
연구 유형
등록 (추정된)
단계
- 3단계
연락처 및 위치
연구 연락처
- 이름: Charlene Song
- 전화번호: +86 18817946880
- 이메일: xiaoling.song@canbridgepharma.com
연구 연락처 백업
- 이름: Amelia Zhou
- 전화번호: +86 18016327369
- 이메일: feng.zhou@canrbdigepharma.com
연구 장소
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Shanghai Municipality
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Shanghai, Shanghai Municipality, 중국
- Room 202, 2nd Floor, 780 Cailun Road, Pilot Free Trade Zone
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연락하다:
- Amelia Zhou
- 전화번호: +86 18016327369
- 이메일: feng.zhou@canrbdigepharma.com
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
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Subject has a documented diagnosis of GD1 or GD3 according to the diagnostic criteria of the Expert Consensus on the Diagnosis and Treatment of Gaucher Disease in Chinese Children 2021;
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Written informed consent provided by the subject's parent/legally authorized representative;
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Subject is less than 12 years of age;
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Subject has GD-related splenomegaly, defined as at least 2 to 3 cm below the left costal margin by palpation, and one or more of the following:
- GD-related anemia, with a decrease of ≥1 g/dL in hemoglobin concentration below the lower limit of normal for age of the central laboratory; or
- GD-related hepatomegaly by palpation; or
- GD-related thrombocytopenia, with a platelet count < 100 × 10^9/L;
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Subject has not received ERT or SRT for at least 3 months prior to screening; Female subjects of childbearing age have a negative serum pregnancy test during screening.
Exclusion Criteria:
- Treatment with investigational drugs (including ambroxol) within 30 days or 5 half-lives, whichever is longer, prior to screening;
- Subjects have received erythropoietin, whole blood transfusion or transfusion of red blood cells, or long-term (continuous treatment for more than 3 months) systemic corticosteroids 3 months prior to screening; or received a platelet transfusion within 1 month prior to screening;
- Subject has non-Gaucher disease-related anemia (such as due to iron, folic acid, and/or vitamin B12 deficiency or infection/immune-mediated reasons);
- Subject has had a prior hepatectomy and/or splenectomy (including partial liver and/or splenectomy) or plans to have a hepatectomy and/or splenectomy (including partial liver and/or splenectomy) during the study;
- Subject has received organ transplantation, including hematopoietic stem cell transplantation;
- Subject has had a history of CTCAE Grade 3 or above infusion-related reaction or hypersensitivity reaction to imiglucerase or other ERTs (approved or experimental).
- Injection with a live vaccine within 30 days of the first study dose.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: velaglucerase beta 60 U/kg
This is a single-arm study. All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks. |
All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks with two weeks of follow-up through Week 39 (9 months). |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Primary Endpoints
기간: From Baseline to Week 39
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Incidence and number of TEAE, characterized by type, severity, seriousness (SAE), and relatedness to velaglucerase |
From Baseline to Week 39
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Mean percent change in MN spleen volume
기간: From Baseline to Week 39
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Normal spleen volume = 0.2% body weight. Multiples of normal (MN) spleen volume= actual MRI testing volume / normal spleen volume |
From Baseline to Week 39
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Mean change in hemoglobin concentration (g/dL)
기간: From baseline to Week 39
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From baseline to Week 39
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Mean percent change in MN liver volume
기간: From Baseline to Week 39
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Normal liver volume = 2.5% body weight. Multiples of normal (MN) liver volume= actual MRI testing volume / normal liver volume |
From Baseline to Week 39
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Mean percent change in platelet count ( 10^9/L)
기간: From Baseline to Week 39
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From Baseline to Week 39
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Mean change in height Z-score
기간: From Baseline to Week 39
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From Baseline to Week 39
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Mean change in weight Z-score
기간: From Baseline to Week 39
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From Baseline to Week 39
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공동 작업자 및 조사자
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
추가 관련 MeSH 약관
기타 연구 ID 번호
- CAN103-GD-301
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
only IPD used in the results publication
IPD 공유 기간
Beginning 3 months and ending 3 years after the publication of results
IPD 공유 액세스 기준
The criteria that must be met the types of analyses that qualify for data sharing.
A proposal that describes planned analyses must be submitted.
IPD 공유 지원 정보 유형
- 연구_프로토콜
- 수액
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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