Overview

This trial is active, not recruiting.

Condition hairy cell leukemia
Treatment vemurafenib
Phase phase 2
Target BRAF
Sponsor Memorial Sloan Kettering Cancer Center
Collaborator National Cancer Institute (NCI)
Start date October 2012
End date October 2017
Trial size 36 participants
Trial identifier NCT01711632, 12-200

Summary

The purpose of this study is to find out what effects, good and/or bad, treatment with vemurafenib (also known as Zelboraf™) has on the patient and on leukemia. Specifically, the researchers want to know how well vemurafenib eliminates leukemia from the blood.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Eligible patients will receive vemurafenib at a dose of 960mg orally twice daily (b.i.d.) continuously in cycles of 4 weeks (28 days).
vemurafenib Zelboraf™
Patients will receive vemurafenib at a dose of 960mg orally b.i.d. continuously in cycles of 4 weeks (28 days) as outpatient. A bone marrow aspirate and/or biopsy will be performed after the first cycle for research purposes only. After the completion of the third cycle, a repeat bone marrow aspirate and/or biopsy will be performed for assessment of response and evaluation of MRD. Following the third cycle assessments, patients who achieve complete response (CR) with detectable MRD or partial response (PR) may continue with vemurafenib for up to 3 additional cycles at the treating physician's discretion (Cycles 4-6). Patients who achieve CR without MRD will be observed as part of post-treatment followup, and may be re-treated with vemurafenib after relapse (as per below). Patients who achieve no response (NR) after the initial 3 cycles of vemurafenib will be removed from the study. They will be followed every 3 months as part of posttreatment followup for a total of 12 months.

Primary Outcomes

Measure
efficacy of vemurafenib
time frame: 3 months

Secondary Outcomes

Measure
Toxicity (safety and tolerability)
time frame: 2 years
To assess the pharmacodynamics
time frame: 2 years
evaluate biomarkers
time frame: 2 years

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - ≥ 18 years of age - Histologically confirmed classical HCL with one of the following: - Intolerance to purine analogs or considered to be poor candidates for purine analog-based therapy - Failure to achieve any response (CR or PR) to the initial purine analog-based therapy - Relapse ≤ 2 years of purine analog-based therapy - ≥ 2 relapses Histologic confirmation of diagnosis will be performed at MSKCC or a participating site. - Patients who meet the standard treatment initiation criteria, as defined by ANC ≤1.0, Hgb ≤ 10.0 or PLT ≤100K - ECOG performance status of 0-2 - Acceptable pre-study organ function during screening as defined as: Total bilirubin ≤ 1.5 times the upper limit of normal (ULN), aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5x ULN, and serum creatinine ≤ 1.5x ULN - Electrocardiogram (ECG) without evidence of clinically significant ventricular arrhythmias or ischemia as determined by the investigator and a rate-corrected QT interval (QTc, Bazett's formula) of < 480 msec. - For women of childbearing potential, agreement to the use of two acceptable methods of contraception, including one barrier method, during the study and for 6 months after discontinuation of vemurafenib - For men with female partners of childbearing potential, agreement to use a latex condom and to advise their female partner to use an additional method of contraception during the study and for 6 months after discontinuation of vemurafenib - Negative serum pregnancy test within 7 days of commencement of treatment in premenopausal women. - Agreement not to donate blood or blood products during the study and for at least 6 months after discontinuation of vemurafenib; for male partners, agreement not to donate sperm during the study and for at least 6 months after discontinuation of vemurafenib - Ability to understand and willingness to sign a written informed consent document. - Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures. Exclusion Criteria: - Pregnant or breast-feeding - Have had chemotherapy (including purine analogs, rituximab, and other investigational agents) within six weeks prior to entering the study - Major surgery within 4 weeks prior to entering the study - Invasive malignancy within the past 2 years prior to first study drug administration, except for adequately treated (with curative intent) basal or squamous cell carcinoma, melanoma, in situ carcinoma of the cervix, in situ ductal adenocarcinoma of the breast, in situ prostate cancer, or limited stage bladder cancer or other cancers from which the patient has been disease-free for at least 2 years - Refractory nausea or vomiting, malabsorption, external biliary shunt, or history of any type of gastrointestinal surgery that would preclude adequate absorption of study drug - Prior treatment with MEK or BRAF inhibitors - Active HIV, hepatitis B and hepatitis C - Patients with HCL variant (as defined by absence of expression of CD25 or absence of BRAF V600E mutation)

Additional Information

Official title A Phase II Study of the BRAF Inhibitor, Vemurafenib, in Patients With Relapsed or Refractory Hairy Cell Leukemia
Principal investigator Jae H. Park, MD
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Memorial Sloan Kettering Cancer Center.
Location data was received from the National Cancer Institute and was last updated in August 2016.