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A Study of Vismodegib in Patients With Relapsed/Refractory Acute Myelogenous Leukemia and Relapsed Refractory High-Risk Myelodysplastic Syndrome

2015년 11월 11일 업데이트: Hoffmann-La Roche

A PHASE IB/II STUDY TO EVALUATE THE SAFETY AND EFFICACY OF VISMODEGIB IN RELAPSED/REFRACTORY ACUTE MYELOGENOUS LEUKEMIA (AML) AND RELAPSED/REFRACTORY HIGH-RISK MYELODYSPLASTIC SYNDROME (MDS)

This study will assess the safety and efficacy of vismodegib in patients with relapsed/refractory acute myelogenous leukemia (AML) and relapsed/refractory high-risk myelodysplastic syndrome (MDS). Patients in Cohort 1 will receive single-agent vismodegib 150 mg orally daily. In Cohort 2, patients will receive vismodegib 150 mg orally daily in combination with cytarabine 20 mg subcutaneously for 10 days.

Anticipated time on study treatment is until disease progression, intolerable toxicity, or patient withdrawal of consent.

연구 개요

상태

종료됨

정황

개입 / 치료

연구 유형

중재적

등록 (실제)

38

단계

  • 2 단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 장소

  • 독일
      • Braunschweig, 독일, 38114
      • Essen, 독일, 45122
      • Hamburg, 독일, 20246
      • Heidelberg, 독일, 69120
      • Münster, 독일, 48149
  • 미국
    • California
      • Stanford, California, 미국, 94305
    • Michigan
      • Ann Arbor, Michigan, 미국, 48109
    • Missouri
      • Kansas City, Missouri, 미국, 64131
    • New York
      • New York, New York, 미국, 10065
    • Texas
      • Houston, Texas, 미국, 77030
  • 캐나다
    • Alberta
      • Edmonton, Alberta, 캐나다, T6L5X8
    • Ontario
      • Toronto, Ontario, 캐나다, M5G 2M9
    • Quebec
      • Montreal, Quebec, 캐나다, H1T 2M4
      • Montreal, Quebec, 캐나다, H3T 1E2

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

18년 이상 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

Inclusion Criteria:

  • Adult patients, >/= 18 years of age
  • Patients with documented relapsed or refractory AML, except acute promyelocytic leukemia (APL [M3 subtype]), or relapsed or refractory high-risk MDS (high-risk MDS defined as International Prognostic Scoring System (IPSS) Int-2 or high and >/= 10% blasts in bone marrow)
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
  • Negative serum pregnancy test for women of childbearing potential and use of two forms of contraception while enrolled in the study and for 7 months after the patient discontinues from study
  • Male patients with female partners of childbearing potential must agree to use a latex condom and to advise their female partner to use an additional method of contraception during the study and for 2 months after the last dose of vismodegib
  • All non-hematological adverse events of any prior chemotherapy, surgery, or radiotherapy must have resolved to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Grade </= 2 prior to starting therapy
  • Adequate hepatic and renal function

Exclusion Criteria:

  • Prior treatment with a Hh pathway inhibitor
  • Prior therapy for the treatment of malignancy within 14 days of Day 1, with the exception of:

Hydroxyurea in patients who need to continue this agent to maintain white blood cell (WBC) counts </= 50,000/mL. Hydroxyurea must be discontinued by Day 14 of the study

  • Current evidence of active central nervous system (CNS) leukemia
  • Any other active malignancy (except non-melanoma skin cancer or carcinoma in situ of the cervix)
  • Any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as:

Unstable angina, symptomatic or otherwise uncontrolled arrhythmia requiring medication (does not include stable, lone atrial fibrillation), or myocardial infarction </= 6 months before study treatment start Any active (acute or chronic) or uncontrolled infection/disorders that impair the ability to evaluate the patient or for the patient to complete the study

  • Pregnant or breast-feeding women
  • Patients who refuse to potentially receive blood products and/or have a severe hypersensitivity to blood products

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

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: 비스모데깁
의약품: cytarabine
Cohort 2: 20 mg sc daily for 10 days starting Day 1, with a possible further cycle of 20 mg sc daily for 5 days starting no earlier than Day 29
의약품: vismodegib
Cohorts 1 and 2: 150 mg orally daily
다른 이름들:
  • RO5450815

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Percentage of Participants With a Complete Response (CR) or CR With Incomplete Blood Count Recovery (CRi) or Morphologic Leukemia Free State (MLFS) or Partial Response (PR) at Week 8
기간: Week 8
CR was defined as achieved if the neutrophils count was greater than (>) 1000 cells per microliter (µL), platelets count >100000/µL, bone marrow blasts percentage (%) less than (<) 5, no Auer rods (clumps of azurophilic granular material that form elongated needles seen in the cytoplasm of leukemic blasts), no transfusion requirements and no signs of extra medullary disease (EMD). CRi was defined if either of the cell (neutrophil or platelet) lineage was not recovered (neutrophils >1000 cells/µL or Not applicable [NA] or platelets count >100000/µL or NA), bone marrow blasts <5% with no Auer rods and confirmed by flow cytometry with no signs of EMD. MLFS (neutrophil and platelet criteria were NA) was defined as bone marrow blasts <5% with no Auer rods and confirmed by flow cytometry with no signs of EMD. PR was defined as neutrophils count >1000 cells/µL, platelets count >100000/µL, and >50% decrease from baseline to a range of 5-25% of bone marrow blasts or blasts <5% with Auer rods.
Week 8

2차 결과 측정

결과 측정
측정값 설명
기간
Percentage of Participants With CR, CRi, MLFS or PR at Anytime During Study Treatment
기간: Up to 30 days of last dose of study drug (maximum treatment duration = 225 days)
CR was defined as achieved if the neutrophils count >1000 cells/µL, platelets count >100000/µL, bone marrow blasts <5%, no Auer rods (clumps of azurophilic granular material that form elongated needles seen in the cytoplasm of leukemic blasts), no transfusion requirements and no signs of EMD. CRi was defined if either of the cell (neutrophil or platelet) lineage was not recovered (neutrophils > 1000 cells/µL or NA or platelets count >100000/µL or NA, bone marrow blasts <5% with no Auer rods and confirmed by flow cytometry with no signs of EMD. MLFS (neutrophil and platelet criteria were NA) was defined as bone marrow blasts <5% with no Auer rods and confirmed by flow cytometry with no signs of EMD. PR was defined as neutrophils count >1000 cells/µL, platelets count >100000/µL, and >50% decrease from baseline to a range of 5-25% of bone marrow blasts or blasts <5% with Auer rods. The 95% confidence intervals (CI) were constructed using Blyth-Still-Cassella method.
Up to 30 days of last dose of study drug (maximum treatment duration = 225 days)
Duration of Overall Response (DOR)
기간: Up to 30 days of last dose of study drug (maximum treatment duration = 225 days)
DOR is defined as the time from the first occurrence of a documented overall response to the time of relapse, as determined by the investigator using International Working Group (IWG) criteria (Participants not falling under any of the response criteria [CR or CRi or MLFS or PR] described under outcome measure 1 were considered as non-responders) or death from any cause during the study (defined as death within 30 days after the last dose of study drug).
Up to 30 days of last dose of study drug (maximum treatment duration = 225 days)
Median Overall Survival (OS) Time
기간: Up to death or 30 days of last dose of study drug (maximum treatment duration = 225 days)
OS was defined as the time from start of study drug to death from any cause. OS was estimated using Kaplan-Meier analysis. Participants alive at the last date known to be alive were censored for the analysis.
Up to death or 30 days of last dose of study drug (maximum treatment duration = 225 days)
Percentage of Participants With an Event of Death During the Study
기간: Up to death or 30 days of last dose of study drug (maximum treatment duration = 225 days)
Up to death or 30 days of last dose of study drug (maximum treatment duration = 225 days)
Pharmacokinetics (PK): Steady-state Plasma Concentration of Vismodegib
기간: Predose on Days 8, 29 and 57
PK data was planned to be reported only if the results of Cohort 2 are available.
Predose on Days 8, 29 and 57
Area Under the Concentration-time Curve (AUC) of Cytarabine
기간: Predose, 0.25, 0.5, 1, 3, 6 hours post-dose on Days 1, 8 and 29
PK data was planned to be reported only if the results of Cohort 2 are available.
Predose, 0.25, 0.5, 1, 3, 6 hours post-dose on Days 1, 8 and 29

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Hoffmann-La Roche

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작

2013년 9월 1일

기본 완료 (실제)

2014년 11월 1일

연구 완료 (실제)

2014년 11월 1일

연구 등록 날짜

최초 제출

2013년 6월 11일

QC 기준을 충족하는 최초 제출

2013년 6월 14일

처음 게시됨 (추정)

2013년 6월 19일

연구 기록 업데이트

마지막 업데이트 게시됨 (추정)

2015년 12월 15일

QC 기준을 충족하는 마지막 업데이트 제출

2015년 11월 11일

마지막으로 확인됨

2015년 11월 1일

추가 정보

이 연구와 관련된 용어

추가 관련 MeSH 약관

기타 연구 ID 번호

  • GO28852
  • 2013-001570-14 (EudraCT 번호)

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. .

cytarabine에 대한 임상 시험

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약물 개입

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정황

희귀 질병

약물 개입

식이 보충제

스폰서 / 협력자

위치

구독하다