Clinical Trials in Hairy Cell Leukemia (HCL)
Clinical trials are carefully controlled research studies, and are necessary to develop and study ways to improve treatment and quality of life for patients with hairy cell leukemia. Clinical trials may be available for patients at any stage of HCL. Your eligibility for a clinical trial depends on a variety of factors such as whether you have HCL or HCL variant, your stage of disease, your age and gender and the type of treatment, if any, that you’ve already received.
We encourage you to discuss clinical trials with your doctor and healthcare team to consider the availability of a trial for your unique diagnosis, treatment history and needs.
As you review your options with your doctor, you will want to understand the purpose and length of a given clinical study; potential benefits and risks; costs of the study, including any charges you will be responsible for; your responsibilities and limitations during the trial, including travel requirements; and whether you will continue to see your own doctor during the trial.
We will continue to add information about new studies as they are available. You are welcome to contact us at info@hairycellleukemia.org for additional information.
Clinical Trials at HCL Centers of Excellence
Low Dose Vemurafenib and Rituximab in Hairy Cell Leukemia
Learn more about this study. >>
Brief 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. 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.
Eligibility (Partial list is below. For complete inclusion criteria, visit the clinical trial page)
≥ 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.
Contacts
Alan Saven, MD Phone Number: 858-554-8788 Email: saven.alan@scrippshealth.org
David J Hermel, MD Phone Number: 858-554-8788 Email: hermel.david@scrippshealth.org
Binimetinib for people with relapsed/refractory BRAF Wild Type HCL and Variant
Learn more about this study. >>
Study objective
To determine the overall response rate (ORR) to binimetinib, in patients with BRAF WT HCL and HCLv.
Eligibility
BRAF WT HCL or HCLv with at least 1 prior purine analog treatment.
Need for treatment as evidenced by any one of the following: ANC <1 x10^3/mcL, Hgb <10g/dL, Platelet count <100 x10^3/mcL, leukemia cell count >5 x10^3/mcL, symptomatic splenomegaly, enlarging HCL mass > 2cm in short axis.
Greater than or equal to 18 years of age.
No uncontrolled infection, cardiopulmonary dysfunction, or secondary malignancy requiring treatment.
No chemotherapy, immunotherapy, investigational agent or radiotherapy within 4 weeks prior to the start of study treatment.
Contact
Holly Eager, R.N. Phone: (240) 858-7229 Email: holly.eager@nih.gov
Phase I Study of Anti-CD22 Chimeric Receptor T-Cells in patients with relapsed/refractory Hairy Cell Leukemia and Variant
Learn more about this study. >>
Objectives
To assess the safety and feasibility of administering escalating doses of autologous anti-CD22-CAR (M971BBz) engineered T-cells in subjects with HCL/HCLv following a cyclophosphamide/fludarabine lymphodepletion regimen.
Explore whether the administration of anti-CD22-CAR engineered T-cells can mediate antitumor effects in HCL/HCLv.
Eligibility
HCL/HCLv, after prior treatment with, ineligible for, refusal of, or inability to obtain 1) Rituximab given concurrently with or sequentially after purine analog, 2) moxetumomab pasudotox-tdft, and 3) BRAF-inhibition.
Need for treatment, either 1) ANC <1/nL, 2) Hgb <10g/dL, 3) Plt <100/nL, 4) HCL count >5/nL, 5) HCLv count doubling time <3 months, 6) symptomatic splenomegaly, 7) enlarging HCL mass > 2cm in short axis, 8) increasing lytic or blastic bone lesions.
> 18 years of age.
CD22 expression must be detected on greater than 15% of the malignant cells by immunohistochemistry or greater than 80% by flow cytometry.
No uncontrolled infection, cardiopulmonary dysfunction, or secondary malignancy requiring treatment.
No chemotherapy, immunotherapy, or radiation therapy less than or equal to 3 weeks prior to apheresis.
Contact
Theresa Yu, R.N. Phone: (301) 480-6195 Email: theresa.yu@nih.gov
Encorafenib + Binimetinib for people with BRAF V600E mutated relapsed/refractory HCL
Learn more about this study. >>
Background
Most people with HCL have a BRAF gene mutation. This can increase the growth of cancer cells. Vemurafenib has been tested to treat these people. However, researchers think a combination of drugs might work better.
Objective
To test if treatment with a combination of encorafenib and binimetinib in BRAF mutant HCL is more effective than treatment with vemurafenib.
Eligibility
People ages 18 and older with BRAF mutant HCL that did not respond to or came back after treatment.
Contact
Dr. Robert J. Kreitman Email: kreitmar@mail.nih.gov Phone: 301-648-7375
Monica E. Epstein Email: monica.epstein@nih.gov Phone: (301) 435-2375
Investigation of the B- and T-cell repertoire and immune response in patients with acute and resolved COVID-19 infection
Learn more about this study. >>
This is a NIH-approved COVID-19 related study that examines the characteristics of immune cells (T- and B-cells) in patients who are or were infected with COVID-19. In order to better understand the research results, the study will also look at individuals who are not known to have been infected with COVID-19.
The goals of this study are to:
1) determine a pattern of immune response which may help predict whether a patient might be able recover at home or would need closer monitoring, such as in the hospital;
2) determine if immune response to COVID19 is primarily B- or T-cell driven, and
3) provide investigators conducting future vaccine trials with information that indicates a favorable immune response, to more quickly assess whether the vaccine tested is sufficient.
Contact
Dr. Robert J. Kreitman. kreitmar@mail.nih.gov. 301-648-7375
Cladribine and rituximab for patients with once-relapsed HCL
Learn more about this study. >>
Purpose
To achieve complete remission without minimal residual disease, to prevent relapse.
Eligibility
HCL after one course of either pentostatin or cladribine. Need for retreatment due to abnormal blood counts, growing lymph nodes, painful spleen or infections.
Contact
Julie Feurtado julie.feurtado@nih.gov 301-480-6186
Dr. Robert J. Kreitman kreitmar@mail.nih.gov 301-648-7375
Randomized Phase II Trial of Rituximab With Either Pentostatin or Bendamustine for Multiply Relapsed or Refractory HCL
Learn more about this study. >>
Purpose
To achieve complete remission without minimal residual disease, to prevent relapse.
Eligibility
HCLv with or without prior treatment, or HCL with at least 2 prior treatments; Treatment required due to abnormal blood counts, growing lymph nodes, painful spleen or infections.
Contact
Theresa Yu theresa.yu@nih.gov 301-480-6195
Laura Wisch Laura.Wisch@nih.gov 301-480-6194
Dr. Robert J. Kreitman kreitmar@mail.nih.gov 301-648-7375
Clinical Trials Not Currently Recruiting Patients
Moxetumomab Pasudotox and rituximab for relapsed Hairy Cell Leukemia
Learn more about this study. >>
Purpose:
To test the safety of Moxetumomab pasudotox (Lumoxiti) taken with Rituximab for people with HCL or HCL variant, both agents already approved for HCL or commonly used for HCL.
This study is no longer recruiting patients.
A Phase II study of the BRAF inhibitor, vemurafenib, plus obinutuzumab in patients with previously untreated HCL
Learn more about this study. >>
Purpose:
Although HCL patients experience excellent responses to initial purine analog based chemotherapy (e.g., cladribine or pentostatin), relapses are not uncommon and the disease remains incurable. There is a need for novel therapies for HCL patients. To this end, we are conducting a phase II clinical trial combining the BRAF inhibitor, vemurafeinb, and anti-CD20 antibody, obinutuzumab, in patients with previously untreated HCL to achieve a deeper molecular response with more favorable side effect profile and durable remissions.
Eligibility:
Adult patients, ≥18 years old, with histologically confirmed classical HCL and who have not received any prior therapy for the disease. Patients must meet the treatment initiation criteria as defined by ANC ≤1.0, OR Hgb ≤10.0 OR PLT ≤100K. Patients must have at least one of these indications.
This study is active, but not currently recruiting patients.
Multicenter phase 2 study of the bruton’s tyrosine kinase inhibitor pci-32765 (ibrutinib) for treatment of relapsed HCL
Learn more about this study. >>
Purpose:
Determine the response rate of HCL after 32 weeks of single-agent ibrutinib treatment.
Eligibility:
Adults with classical hairy cell leukemia or the hairy cell leukemia variant who require treatment. Classical hairy cell leukemia patients must have received prior purine nucleoside analogue treatment (cladribine or pentostatin) or be unable to receive it. Anyone with the hairy cell leukemia variant is eligible whether or not they have had prior treatment.
Participating Sites:
The Ohio State University, National Institutes of Health Clinical Center, Karmanos Cancer Institue, Mayo clinic, and M D Anderson Cancer Center
This study is no longer recruiting patients.
ClinicalTrials.gov
You may want to explore www.ClinicalTrials.gov, a registry of federally and privately supported clinical trials in the United States and around the world. This website is a service of the U.S. National Institutes of Health (NIH). Once on the www.ClinicalTrials.gov website, you can enter “hairy cell leukemia” in the “Condition or Disease” box and click “Search” for a list of relevant trials. You can also specify a “Country” to narrow your search.