- ICH GCP
- 미국 임상 시험 레지스트리
- 임상시험 NCT02127892
SCID Bu/Flu/ATG Study With T Cell Depletion
Phase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling Donor
연구 개요
상태
정황
상세 설명
The study is being conducted to assess the following:
- overall survival
- event-free survival (events are defined as: death,non-engraftment/2nd transplant, immune reconstitution failure)
- acute toxicity of the conditioning regimen
- engraftment frequency immune reconstitution frequency and tempo acute and chronic graft-versus-host disease (GVHD), frequency and severity.
The outcome from this protocol will be compared to the retrospective cohort consisting of all patients who have undergone haplo-identical HSCT for SCID at CHLA from 1984-2006 based on the assessment of the above-listed endpoints.
The CliniMACS device will be used for CD34+ selection in place of the Isolex 300i. The CliniMACS CD34 Reagent System is an investigational medical device that has not yet been approved by the FDA. This device is used in vitro to select and enrich specific cell populations. When using the CliniMACS CD34 Reagent, the system selects CD34+ cells from heterogenous hematological cell populations for transplantation in cases where this is clinically indicated.
연구 유형
등록 (실제)
단계
- 2 단계
- 1단계
연락처 및 위치
연구 장소
-
-
California
-
Los Angeles, California, 미국, 90027
- Children's Hospital Los Angeles
-
-
참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- All patients with SCID who lack a histocompatible sibling or HLA-matched related donor will be considered as candidates for this study protocol.
Eligible patients must have adequate physical function to tolerate the chemotherapy conditioning regimen and the HSCT, as measure by:
- Renal: creatinine clearance or GFR ≥50 ml/min/1.73m2, and not requiring dialysis
- Pulmonary: Because patients with SCID frequently present with infectious pneumonia causing ventilatory failure, patients will be considered for enrollment in the study even if respiratory failure requiring mechanical ventilatory support is present. In patients recently diagnosed with pneumonia, efforts to stabilize the respiratory status will be made prior to enrollment in the study.
- Infectious disease status. The presence of infection per se will not be a reason for exclusion from the study. Patients with SCID are frequently infected with both routine pathogens as well as opportunistic infections. Antibiotic, antifungal and antiviral prophylaxis and therapy will be instituted as clinically indicated. Despite the use of antimicrobial therapy, the ability to control infections will not be achieved unless HSCT is performed. Therefore, subjects may be enrolled in the study, even though infection is present, because control of infection may depend on engraftment of a donor immune system.
- Patients will be 0-21 years of age.
Exclusion Criteria:
- Patient with histocompatible sibling or other related donor
- End-organ failure that precludes the ability to tolerate the transplant procedure, including conditioning.
- Renal failure requiring dialysis
- Congenital heart disease resulting in congestive heart failure
- Severe CNS disease, e.g., coma or intractable seizures
- Ventilatory failure due to non-infectious etiology
- Major congenital anomalies that adversely affect survival, eg CNS malformations
- Metabolic diseases that would affect transplant survival, eg urea cycle disorders
- HIV infection
Since the only chance of survival for patients with SCID is successful transplantation, all patients with SCID will be considered to be potential subjects for the study, regardless of end-organ dysfunction.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위화되지 않음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
---|---|
다른: unrelated BM with T cell depletion
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
|
Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
다른 이름들:
|
다른: unrelated cord blood
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g.
not mismatched for both HLA A alleles).
|
Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
다른 이름들:
|
다른: haplo BM with T cell depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
|
haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
다른 이름들:
|
다른: unrelated PBSC with T cell depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
|
peripheral blood stem cell will be processed for CD34+ cell isolation.
다른 이름들:
|
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
---|---|---|
Number of Participants With Engraftment
기간: 100 day
|
Engraftment is defined as recovery of blood counts (neutrophil and platelet engraftment) with cells of donor origin, documented by either bone marrow or peripheral blood chimerism assays after hematopoietic stem cell transplant.
|
100 day
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
---|---|---|
Number of Participants With Donor-derived CD3+ T Lymphocytes >/= 100/mm3
기간: 1 year
|
Absolute number of donor-derived CD3+ T lymphocytes >/= 100/mm3 in participating subjects.
|
1 year
|
기타 결과 측정
결과 측정 |
측정값 설명 |
기간 |
---|---|---|
Number of Participants With Veno-occlusive Disease (VOD) - Moderate and Severe
기간: 100 days
|
Evaluation of veno-occlusive disease determined by the presence of the following features; fluid retention, weight gain, leaky capillary syndrome, painful liver enlargement, refractoriness to platelet tranfusion and hyperbilirubinemia
|
100 days
|
Number of Participants With Graft Versus Host Disease (GVHD) - Grade III or IV
기간: 1 year
|
GVHD disease surveillance done by clinical evaluation, to include history, physical examination, specifically for rash, jaundice, liver dysfunction, nausea and vomiting, diarrhea and failure to thrive.
|
1 year
|
Overall Survival
기간: 1 year
|
Overalls survival of patient at 1 year post transplant
|
1 year
|
공동 작업자 및 조사자
수사관
- 수석 연구원: Neena Kapoor, M.D., Children's Hospital Los Angeles, University of Southern California
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (실제)
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 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- CCI-06-00243
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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. .