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

NCT ID: NCT01880437

Last Updated: 2015-12-15

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

38 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-09-30

Study Completion Date

2014-11-30

Brief Summary

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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.

Detailed Description

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Conditions

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Myelodysplastic Syndromes, Myelogenous Leukemia, Acute

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Vismodegib

Group Type EXPERIMENTAL

cytarabine

Intervention Type DRUG

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

vismodegib

Intervention Type DRUG

Cohorts 1 and 2: 150 mg orally daily

Interventions

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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

Intervention Type DRUG

vismodegib

Cohorts 1 and 2: 150 mg orally daily

Intervention Type DRUG

Other Intervention Names

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RO5450815

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Stanford, California, United States

Site Status

Ann Arbor, Michigan, United States

Site Status

Kansas City, Missouri, United States

Site Status

New York, New York, United States

Site Status

Houston, Texas, United States

Site Status

Edmonton, Alberta, Canada

Site Status

Toronto, Ontario, Canada

Site Status

Montreal, Quebec, Canada

Site Status

Montreal, Quebec, Canada

Site Status

Braunschweig, , Germany

Site Status

Essen, , Germany

Site Status

Hamburg, , Germany

Site Status

Heidelberg, , Germany

Site Status

Münster, , Germany

Site Status

Countries

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United States Canada Germany

Other Identifiers

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2013-001570-14

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GO28852

Identifier Type: -

Identifier Source: org_study_id