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Hairy Cell LeukemiaPhase 2NCT05388123

Low Dose Vemurafenib and Rituximab in Hairy Cell Leukemia

A Phase 2 study testing Low dose vemurafenib plus rituximab in people with Hairy Cell Leukemia, run by Scripps Health at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The current standard-of-care for Hairy Cell Leukemia involves chemotherapy, with agents such as cladribine or pentostatin. Chemotherapy is associated with infection, low blood counts and predisposition to future cancers. Read more about this study

Who is it for?
Ages 18 to 90 years
Conditions the study is for
Hairy Cell Leukemia
What is being tested?
Low dose vemurafenib plus rituximab
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
1 siteUnited States
Study dates
2 Mar 2022to 1 Apr 2026
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 90 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • ≥ 18 years of age
  • Histologically confirmed HCL that are BRAF V600E positive by IHC or NGS
  • Patient's must meet the standard treatment initiation criteria, as defined by ANC ≤1.0, Hgb ≤ 10.0 or PLT ≤100K
  • Patients can either have (1) not received any prior therapy for the disease or have had (2) failure to achieve any response to the initial purine analog-based therapy or (3) subsequent relapse after any prior therapy.
  • ECOG performance status of 0-2
  • Acceptable pre-study organ function during screening not exacerbated by Hairy Cell Leukemia. General thresholds should be a 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
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  • For women of childbearing potential, agreement to use acceptable methods of contraception
  • For men with female partners of childbearing potential, agreement to use barrier contraception
  • Negative serum pregnancy test within 7 days of commencement of treatment in premenopausal women.
  • 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.

Who cannot join

  • Pregnant or breast-feeding or intending to become pregnant during the study
  • Have had chemotherapy (including purine analogs), rituximab, and other investigational agents within six weeks prior to entering the study. The patients cannot have received BRAF inhibitor therapy within 6 months of 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
  • Active HIV, hepatitis B and hepatitis C or any clinically significant history of liver disease. Hepatitis B prior infection is not a contraindication though will require therapy.
  • Known hypersensitivity to any of the study drugs
  • Patients with HCL that are BRAF V600E mutation negative

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Scripps Cancer Center

    La Jolla, California, 92037

Other trials in United States

About this study

Official title: A Single Arm Phase II Pilot Study of Low Dose Vemurafenib Plus Rituximab in the Front-line and Relapsed/Refractory Treatment of Hairy Cell Leukemia

The current standard-of-care for Hairy Cell Leukemia involves chemotherapy, with agents such as cladribine or pentostatin. Chemotherapy is associated with infection, low blood counts and predisposition to future cancers. This study tests a new yet previously validated drug combination for the treatment of hairy cell leukemia. The treatment involves 8 weeks of treatment with an oral drug called vemurafenib and 8 doses of an intravenous medication called rituximab. The goal of this study is to see whether this treatment is better tolerated and more effective than the currently used treatment in this disease. In addition, this study uses a lower dose of vemurafenib than previous studies have used, with the goal of minimizing side effects from this medication.

Read the detailed description

This is a single-center, open label, single arm, investigator-initiated phase II trial of the oral BRAF inhibitor, vemurafenib, plus rituximab in patients with previously untreated or relapsed and refractory HCL. Eligible patients will receive vemurafenib at a dose of 240 mg orally twice daily (b.i.d.) continuously for 8 weeks. Rituximab 375 mg/m2 will be administered concomitantly with vemurafenib every 2 weeks from the first day of treatment. After completion of vemurafenib, the patient will receive rituximab 375 mg/m2 every 2 weeks for a total of 8 weeks. The entire duration of treatment will be 16 weeks. Six months after the initiation of the treatment, a bone marrow aspirate and biopsy will be performed for assessment of response and evaluation of minimal residual disease (MRD).

Being tested
Low dose vemurafenib plus rituximab
Medical subject headings
Leukemia, Hairy Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Low dose vemurafenib plus rituximabDrug

    Also called Vemurafenib, Rituximab

    Vemurafenib 240 mg twice daily for 8 weeks with concurrent rituximab 375 mg/m2 every 2 weeks followed by maintenance consolidative rituximab 4 times every 2 weeks post-vemurafenib

Study groups in NCT05388123
Study groupRoleWhat they receive
Low dose Vemurafenib and RituximabExperimentalEligible patients will receive vemurafenib at a dose of 240 mg orally twice daily (b.i.d.) continuously for 8 weeks. Rituximab 375 mg/m2 will be administered every 2 weeks for a total of 16 weeks. The entire duration of treatment will be 16 weeks.
Receives: Drug: Low dose vemurafenib plus rituximab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Complete Response

    Resolution of cytopenias and splenomegaly

    Measured: Up to 2 years from enrollment

Secondary outcomes (3)

  1. Time to hematologic response

    Days until resolution of cytopenias

    Measured: Up to 2 years from enrollment

  2. MRD Status

    At the time of bone marrow assessment by testing for BRAFV600E mutation status

    Measured: At 6 months, 1 year and 2 years from treatment

  3. Relapse-Free Survival

    Reapperance of Hairy-Cell related cytopenia

    Measured: From start of treatment until 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 May 2022
  2. Study start2 Mar 2022
  3. Primary completion1 Apr 2026
  4. Study completion1 Apr 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05388123
Sponsor's protocol ID
IRB-21-7787
Status last verified
Apr 2026
First submitted
16 May 2022
Last updated
24 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: Plan to publish aggregate data in manuscript

Publications

  • Tiacci E, De Carolis L, Simonetti E, Capponi M, Ambrosetti A, Lucia E, Antolino A, Pulsoni A, Ferrari S, Zinzani PL, Ascani S, Perriello VM, Rigacci L, Gaidano G, Della Seta R, Frattarelli N, Falcucci P, Foa R, Visani G, Zaja F, Falini B. Vemurafenib plus Rituximab in Refractory or Relapsed Hairy-Cell Leukemia. N Engl J Med. 2021 May 13;384(19):1810-1823. doi: 10.1056/NEJMoa2031298.

    PubMed 33979489

  • Grever M, Andritsos L, Banerji V, Barrientos JC, Bhat S, Blachly JS, Call T, Cross M, Dearden C, Demeter J, Dietrich S, Falini B, Forconi F, Gladstone DE, Gozzetti A, Iyengar S, Johnston JB, Juliusson G, Kraut E, Kreitman RJ, Lauria F, Lozanski G, Parikh SA, Park J, Polliack A, Ravandi F, Robak T, Rogers KA, Saven A, Seymour JF, Tadmor T, Tallman MS, Tam CS, Tiacci E, Troussard X, Zent C, Zenz T, Zinzani PL, Wormann B. Hairy cell leukemia and COVID-19 adaptation of treatment guidelines. Leukemia. 2021 Jul;35(7):1864-1872. doi: 10.1038/s41375-021-01257-7. Epub 2021 May 4.

    PubMed 33947938

  • Hermel DJ, Saven A. Low-Dose Vemurafenib Plus Rituximab in Front-Line and Relapsed or Refractory Hairy Cell Leukemia: The Scripps Regimen. Blood Adv. 2026 Jun 16:bloodadvances.2025019243. doi: 10.1182/bloodadvances.2025019243. Online ahead of print.

    PubMed 42308249

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Low dose vemurafenib plus rituximab in people with Hairy Cell Leukemia, run by Scripps Health at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Complete Response (time frame: Up to 2 years from enrollment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05388123, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2026.

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