Targeted drug shows promise against rare leukemia
NCT ID NCT01711632
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a drug called vemurafenib (Zelboraf) in 36 people with hairy cell leukemia that had returned or not responded to standard therapy. The goal was to see how well the drug could clear leukemia from the blood and what side effects it caused. Vemurafenib works by blocking a specific protein (BRAF) that helps the cancer grow.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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36 people
The number who actually took part.
- Started
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Oct 2012
- Finished
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Aug 2024
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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)
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Dana Farber Cancer Institute
Boston, Massachusetts, 02115, United States
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Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
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Northwestern University
Evanston, Illinois, 60208, United States
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Ohio State University
Columbus, Ohio, 43210, United States
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Scripps Clinic
La Jolla, California, 92037, United States
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Other studies related to the condition(s) this trial covers.
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