- ICH GCP
- 미국 임상 시험 레지스트리
- 임상시험 NCT03019809
A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
A Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome.
연구 개요
상태
상세 설명
Severe graft dysfunction, such as the degree of donor chimerism predominantly in the myeloid compartment is one of major problem in patients with Wiskott-Aldrich syndrome (WAS), especially after hematopoietic stem cell transplantation (HSCT) from alternative donor. It often leads to the development of severe thrombocytopenia or even transplants rejection. In this study the hypothesis is that the use of plerixafor and G-CSF as additional agents in conditioning regimen would offers advantages due to lowing risk of mixed chimerism after HSCT. This effect is based on the fact that simultaneous use of plerixafor with G-CSF is efficient in inducing stem cell release and opening of bone marrow (BM) niches. Moreover, stem cell release probably leads to liberation of host stem cells from the anti-apoptotic effects of the BM stroma for the more powerful effect of chemotherapy.
In this study, the investigators use TCR alpha/beta grafts depletion of the grafts as basic technology for HSCT from haploidentical and unrelated donors approved in Institution.
Thus, the purpose of this study is to evaluate the safety and efficiency of myeloablative conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patients with Wiskott-Aldrich syndrome.
연구 유형
등록 (예상)
단계
- 2 단계
연락처 및 위치
연구 연락처
- 이름: Dmitry Balashov, MD, PhD
- 전화번호: 6534 +7(495)287-6570
- 이메일: bala8@yandex.ru
연구 연락처 백업
- 이름: Michael Maschan, Professor
- 전화번호: +7(926)651-2145
- 이메일: mmaschan@yandex.ru
연구 장소
-
-
-
Moscow, 러시아 연방, 117997
- 모병
- Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
-
연락하다:
- Dmitry Balashov, MD, PhD
- 전화번호: 6534 +7(495)287-6570
- 이메일: bala8@yandex.ru
-
연락하다:
- Michael Maschan, Professor
- 전화번호: +7(926)287-6570
- 이메일: mmaschan@yandex.ru
-
부수사관:
- Michael Maschan, Professor
-
부수사관:
- Alexandra Laberko, MD
-
부수사관:
- Svetlana Kozlovskaya, MD
-
부수사관:
- Elena Gutovskaya, MD
-
부수사관:
- Anna Shcherbina, Professor
-
-
참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Patients aged ≥ 1 months and < 19 years
- Patients diagnosed with Wiskott-Aldrich syndrome eligible for an allogeneic transplantation and lacking a related HLA-matched donor
- Lansky/Karnofsky score > 40, WHO > 4
- Signed written informed consent
Exclusion Criteria:
- Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
- Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction <40%)
- Serious concurrent uncontrolled medical disorder
- Lack of parents' informed consent.
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 |
---|---|
실험적: Plerixafor/G-CSF for HSCT conditioning
Myeloablative conditioning regimen with Plerixafor and G-CSF as addition agents before stem cell transplantation in WAS patients.
|
Mobilization of hematopoietic stem (HSC) into circulation
Directed inhibition of CXC chemokine receptor type 4 (CXCR4) for opening enough BM niches for adequate donor HSC engraftment.
|
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
---|---|---|
Event free survival (EFS)
기간: 24 months
|
The EFS probability compared with historical control.
We mean event as patient's death, second transplantation or persistence of severe thrombocytopenia
|
24 months
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
---|---|---|
Overall survival (OS)
기간: 24 months
|
The OS probability compared with historical control.
|
24 months
|
Percentage of patients with full/mixed donor chimerism
기간: 12 months
|
Evaluation of the percentage of patients with the full/mixed donor chimerism (whole blood and CD3+ lineage).
In addition, patients will be divided in accordance with % of donors cells: >95%; 50%-95%; 10%-49%; <10%.
All data will be compared with historical control
|
12 months
|
Transplant related mortality (TRM)
기간: 24 months
|
The TRM probability compared with historical control.
|
24 months
|
Severe thrombocytopenia (ST)
기간: 24 months
|
The ST probability after HSCT compared with historical control
|
24 months
|
Autoimmune complications (AC)
기간: 24 months
|
The AC probability after HSCT compared with historical control
|
24 months
|
Acute Graft Versus Host Diseases (aGVHD)
기간: 12 months
|
Cumulative Incidence and severity of aGVHD
|
12 months
|
Chronic Graft Versus Host Diseases (cGVHD)
기간: 24 months
|
Cumulative Incidence and severity of cGVHD
|
24 months
|
Plerixafor related complications (PRC)
기간: 2 week
|
PRC: severity, features, incidence
|
2 week
|
공동 작업자 및 조사자
수사관
- 연구 의자: Alexei Maschan, Professor, Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
- 수석 연구원: Dmitry Balashov, Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (예상)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (추정)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .
G-CSF for Conditioning before HSCT.에 대한 임상 시험
-
New Approaches to Neuroblastoma Therapy ConsortiumNational Cancer Institute (NCI)완전한
-
City of Hope Medical CenterNational Cancer Institute (NCI)모병급성 골수성 백혈병 | 호지킨 림프종 | 골수 섬유증 | 급성 림프구성 백혈병 | 만성림프구성백혈병 | 비호지킨 림프종 | 골수 형성 이상 증후군 | 조혈 및 림프계 신생물 | 골수 증식성 신생물 | 만성 골수성 백혈병, BCR-ABL1 양성미국
-
Medical College of WisconsinNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute (NHLBI); National... 그리고 다른 협력자들완전한
-
Ronald LevyNational Institutes of Health (NIH)완전한
-
Stanford University종료됨림프종, 비호지킨 | 미만성 거대 B세포 림프종(DLBCL) | 림프종, B세포 | 악성 림프종, 비호지킨미국
-
National Institute of Allergy and Infectious Diseases...빼는