A Study of Vismodegib in Patients With Relapsed/Refractory Acute Myelogenous Leukemia and Relapsed Refractory High-Risk Myleodysplastic Syndrome.
- Conditions
- Adult patients with acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).MedDRA version: 17.0Level: LLTClassification code 10024348Term: Leukemia myelogenousSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 17.0Level: PTClassification code 10028533Term: Myelodysplastic syndromeSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)Therapeutic area: Diseases [C] - Cancer [C04]
- Registration Number
- EUCTR2013-001570-14-DE
- Lead Sponsor
- F. Hoffmann-La Roche Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 60
- 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 NCI CTCAE Grade - Adequate hepatic and renal function
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 36
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 24
- 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 WBC counts - Current evidence of active 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 - 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: - To evaluate the efficacy of single-agent vismodegib in patients with acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) as measured by overall response rate (ORR).;Secondary Objective: - To evaluate the efficacy of single-agent vismodegib in patients with AML or MDS as measured by duration of overall response (DOR).<br>- To evaluate the efficacy of single-agent vismodegib in patients with AML or MDS as measured by overall survival (OS).;Primary end point(s): Overall response rate;Timepoint(s) of evaluation of this end point: Week 8
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Overall response rate<br>- Duration of Response<br>- Overall Survival ;Timepoint(s) of evaluation of this end point: - Anytime during the study treatment<br>- Time from the initial response to disease progression or death among patients who have experienced a response during study treatment<br>- Start of study until death