- ICH GCP
- 미국 임상 시험 레지스트리
- 임상시험 NCT01911039
Phase 1 Infused Donor T Regulatory Cells in Steroid Dependent/Refractory Chronic GVHD
A Phase 1 Safety and Tolerability Study of Infused Donor T Regulatory Cells in Steroid Dependent/Refractory Chronic Graft Versus Host Disease
연구 개요
상태
개입 / 치료
상세 설명
PRIMARY OBJECTIVES:
Determine the safety and tolerability of donor T regulatory (Treg) cell infusions in subjects with steroid dependent/refractory chronic graft versus host disease.
SECONDARY OBJECTIVES:
- Determine the quantitative blood Treg cell changes following the cell infusions
- Determine clinical efficacy of donor Treg cells as failure-free survival (FFS) defined by the absence of a new immunosuppressive therapy added, non-relapse mortality, and recurrent malignancy at Day 180 after the first Treg infusion
In addition to FFS, the study will measure the change in:
- cGVHD symptom burden measured by the Lee cGVHD Symptom Scale by increase in >7 points
- NIH organ-specific cGVHD scale
- The reduction in daily corticosteroid requirement of prednisone to <=0.25 mg/kg-day at Day 180 after the first Treg infusion
연구 유형
등록 (실제)
단계
- 1단계
연락처 및 위치
연구 장소
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California
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Palo Alto, California, 미국, 94305
- Stanford University, School of Medicine
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
Steroid dependent/refractory cGVHD defined as:
- Steroid dependent disease: Persistent cGVHD manifestations requiring a glucocorticoid dose >= prednisone 0.25 mg/kg/day (0.5 mg/kg orally [po] every other day) for at least 12 weeks
- Steroid refractory disease: Progressive cGVHD manifestations despite treatment with a glucocorticoid dose >= prednisone 0.5 mg/kg/day (1 mg/kg po every other day) for at least 4 weeks
- Participants must be receiving systemic glucocorticoid therapy for cGVHD; all immunosuppressive therapy may include but not be limited to tacrolimus, sirolimus, CellCept, cyclosporine, and systemic corticosteroid must be at stable doses for 28 days prior to the first cell infusion
Chronic GVHD manifestations that can be followed on physical or laboratory exam; these include but are not necessarily limited to:
- Skin changes
- Oral mucosa changes
- Bronchiolitis obliterans
- Ocular changes
- Karnofsky performance status >= 60
- Serum creatinine =< 2 mg/dL
- Absolute neutrophil count (ANC) > 1 x 10^9/L
- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =< 20 x upper limit of normal (ULN) or
- Total bilirubin =< 10 x ULN
- Allogeneic hematopoietic cell transplant recipient
- Transfusion independent
- Oxygen saturation during exertion is maintained at >= 88% on room air
- Does not have clinically significant, symptomatic uncontrolled heart disease (e.g., unstable angina, congestive heart failure, or uncontrolled hypertension)
- DONOR: Age >= 18 to =< 75 years old
- DONOR: Karnofsky performance status of >= 70% defined by institutional standards
- DONOR: Must be the same sibling donor from whom the recipient's blood and marrow graft was collected for the original allogeneic transplant that is human leukocyte antigen (HLA) 7/8 or 8/8 matched at the HLA-A, B,C, DRB1
- DONOR: Serologies for human immunodeficiency virus (HIV) antigen (Ag), HIV 1 and HIV 2 antibody (Ab), human T-lymphotropic virus type I (HTLV 1) and HTLV 2 Ab, hepatitis B surface antigen (sAg) or polymerase chain reaction positive (PCR+), or hepatitis C Ab or PCR+, Syphilis (Treponema) screen and HIV 1 and hepatitis C by nucleic acid testing (NAT) have been collected prior to apheresis
- DONOR: Female donors of child-bearing potential must have a negative serum or urine beta-human chorionic gonadotropin (HCG) test within three weeks of apheresis
- DONOR: Capable of undergoing leukapheresis, have adequate venous access, and be willing to undergo insertion of a central catheter should leukapheresis via peripheral vein be inadequate
- DONOR: Donor selection will be in compliance with 21 Code of Federal Regulations (CFR) 1271
Exclusion Criteria:
- Original transplant utilized an unrelated donor graft
- Uncontrolled infections that are not responsive to antimicrobial therapy
- Progressive malignant disease, including post-transplant lymphoproliferative disease unresponsive to therapy
- Second malignancy except for skin cancer within the last 5 years
- Received any investigational agent =< 28 days before Treg infusions
- Received filgrastim (GCSF) treatment within one month of enrollment
- Received a donor lymphocyte infusion (DLI) or hematopoietic cell transplantation (HCT) within 3 months of enrollment
- DONOR: Evidence of active infection or viral hepatitis
- DONOR: HIV positive
- DONOR: Pregnant donor
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: Regulatory T Cells
Cohort 1 at 1x105 Treg cells/kg, Cohort 2 at 5x105 Treg cells/kg and Cohort 3 at 1.5x106 Treg cells/kg with an extension phase at the MTD (or maximum administered dose if the MTD is not reached).
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다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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The frequency of adverse events related to the donor Treg infusions (e.g., grade III-IV aGVHD by the modified Keystone criteria and grade 3 or higher infusional toxicities graded according to the CTCAE v. 4)
기간: Up to day 180
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For infusion-related toxicities, recipients will be monitored for 1 hour after the Treg infusion.
Additional toxicities which may occur during the first 28 days after the Treg infusions will count towards the assessment of safety and tolerability (DLT assessment) (e.g., development of aGVHD).
Acute GVHD will be assessed using the modified Keystone criteria on Days 14, 28, 42, 56, 84 and 180 after the Treg infusion (or if the subject is exhibiting signs of aGVHD in-between study visits).
Dose limiting toxicities are defined in Section 8.
Only toxicities which occur during the first 28 days after the cell infusion will count towards the assessment of DLTs.
A dose of Treg will be considered safe if DLTs occur in only 1/6 or 0/3 members of the cohort during the dose-escalation phase.
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Up to day 180
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Change in absolute blood Treg levels
기간: Baseline to day 42
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The change in Treg cell counts from baseline to post infusion will be depicted in boxplots of both relative proportion and absolute numbers.
Mean log (fold change) and confidence intervals will be calculated.
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Baseline to day 42
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Improvement in Failure Free Survival (FFS) over cGVHD
기간: At day 180
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FFS is defined as the absence of a third line therapy (treatment failure).
Estimated by the Kaplan-Meier product-limit method, with standard confidence limits.
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At day 180
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Successful achievement of cGVHD partial response or Complete response by the NIH consensus criteria
기간: Up to day 180
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The results will be summarized in tabular form, with confidence intervals for the trinomial proportions. |
Up to day 180
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The ability to reduce steroid requirements to <0.25 mg/kg-day
기간: At day 180
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At day 180
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Change in >7 points on the Lee cGVHD Symptom scale relates to improvement in quality of life
기간: Baseline to day 180
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A one-sample t-test will be used on the change in scale from baseline to months 1, 3, and 6.
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Baseline to day 180
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공동 작업자 및 조사자
스폰서
수사관
- 수석 연구원: Laura Johnston, Stanford University
연구 기록 날짜
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