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Targeted drug shows promise against rare leukemia

NCT ID NCT01711632

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

36 people

The number who actually took part.

Started

Oct 2012

Finished

Aug 2024

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

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

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02115, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • Northwestern University

    Evanston, Illinois, 60208, United States

  • Ohio State University

    Columbus, Ohio, 43210, United States

  • Scripps Clinic

    La Jolla, California, 92037, United States

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Other studies related to the condition(s) this trial covers.