RecruitingPhase 1 / Phase 2

Testing the Combination of Anti-cancer Drugs, Tovorafenib Plus Rituximab, in Patients With Hairy Cell Leukemia

A Phase 1 Study of Combination Tovorafenib (DAY101) and Rituximab Treatment in Relapsed or Refractory Classical Hairy Cell Leukemia and Phase 2 Randomized Study Comparing Tovorafenib (DAY101) and Rituximab With Cladribine and Rituximab for Front-Line Treatment of Classical Hairy Cell Leukemia

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: Blood draws, CT scans, Biopsies

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Patients also undergo blood sample collection, computed tomography (CT) as clinically indicated, and bone marrow biopsy and aspiration throughout the study."
  • "Patients also undergo blood sample collection, CT as clinically indicated, and bone marrow biopsy and aspiration throughout the study."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the safety, side effects, and effects of tovorafenib plus rituximab in adults with classical hairy cell leukemia, a blood cancer. It includes people whose cancer came back or did not respond to treatment. For people who have not been treated, it compares this drug pair with the standard treatment, cladribine plus rituximab.

This phase I/II trial tests the safety, side effects, and effectiveness of tovorafenib in combination with rituximab in patients with classical hairy cell leukemia (cHCL) that has come back after a period of improvement (recurrent) or that has not responded to previous treatment (refractory) and compares the effect of tovorafenib and rituximab to current standard treatment of cladribine and rituximab in cHCL patients that have not yet received treatment. Tovorafenib blocks certain proteins made by the mutated BRAF gene, which may help keep cancer cells from growing. It is a type of kinase inhibitor. Rituximab is a monoclonal antibody. It binds to a protein called CD20, which is found on B cells (a type of white blood cell) and some types of cancer cells. This may help the immune system kill cancer cells. Cladribine damages the cell's deoxyribonucleic acid and may kill cancer cells. It is a type of antimetabolite. Giving tovorafenib in combination with rituximab may be safe and tolerable and more effective than cladribine with rituximab in treating patients with untreated, recurrent or refractory cHCL.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be age 18 or older and have confirmed classical hairy cell leukemia with a gene change called BRAF V600E.
  • For phase 1, have had fludarabine, pentostatin, or cladribine unless these drugs were unsafe for you. For the phase 2 first-treatment group, have had no treatment for this cancer.
  • Need cancer treatment because of low blood counts, repeated infections, growing or painful disease, enlarged organs, or symptoms such as weight loss, tiredness, fever, or night sweats.
  • Be well enough for daily activities, unless limits are caused by the leukemia. Meet the study's liver, kidney, and heart test requirements.
  • If you have HIV or hepatitis B, the virus must be controlled as required. Hepatitis C must be cured, or undetectable for more than 6 months while on treatment.
  • Any other cancer must not interfere with checking this treatment's safety or effects.
  • If you or your partner could become pregnant, use two required birth control methods before and during treatment and for 12 months afterward. Women must use non-hormonal methods; men must not father a child or donate sperm during the study.
  • Be able to understand and sign a consent form, or have a legally authorized person give consent for you.

Who can't join

  • Have the variant form of hairy cell leukemia, lack the BRAF V600E gene change, or have platelets below 50,000. Platelets help blood clot.
  • Have cancer involving the brain or spinal cord, unless treated brain cancer spots show no growth on follow-up scans.
  • Take warfarin, direct oral blood thinners, other experimental drugs, or certain medicines that interact with the study drugs.
  • Still have side effects worse than mild from earlier cancer treatment, except hair loss.
  • Be pregnant, breastfeeding, or unwilling to follow the birth control rules.
  • Have an allergy to a study drug or a similar drug, including a past severe allergic reaction during a rituximab infusion.
  • Be unable to swallow pills, have digestive tract problems, or have an uncontrolled illness or another condition that makes the study too risky.
  • Have received a live or weakened-live vaccine within 28 days before being assigned to a treatment group.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: * Patients must have histologically or cytologically confirmed diagnosis of classical hairy cell leukemia (HCL), including demonstration of BRAF V600E mutation by immunohistochemistry, molecular diagnostic testing, or polymerase chain reaction (PCR) * PHASE 1 ONLY: Prior therapy with at least one purine nucleoside analog-containing regimen (fludarabine, pentostatin, or cladribine) unless contraindicated. Prior vemurafenib alone is allowed in the relapsed/refractory cohort * PHASE 2 ONLY: No prior HCL-directed treatment for front-line cohort. The design of this cohort is such that the patients will need to be treatment naïve * Age ≥ 18 years. Because no dosing or adverse event (AE) data are currently available on the use of tovorafenib (DAY101) or cladribine in combination with rituximab in patients \< 18 years of age, children are excluded from this study * Patients must meet indications for treatment of cHCL: * Absolute neutrophil count \< 1,000/mcL * Platelets \< 100,000/mcL * Hemoglobin \< 10 g/dL * Recurrent infections * Symptomatic and/or progressive extramedullary disease including lymph nodes and bone lesions * Progressive or symptomatic splenomegaly or hepatomegaly * Disease-related constitutional symptoms consisting of unexplained weight loss exceeding 10% body weight during the preceding 6 months, Cancer Therapy Evaluation Program (CTEP) active version of the Common Terminology Criteria for Adverse Events (CTCAE) grade 2 or 3 fatigue, and/or fever \> 100.5 F or night sweats for \> 2 weeks without evidence of active infection * Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 (Karnofsky ≥ 60%); ECOG performance status \> 2 (Karnofsky \< 60%) will be allowed if considered due to HCL * Total bilirubin ≤ 1.5 x upper limit of normal (ULN) * Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) ≤ 3 x institutional ULN (unless related to Gilbert's disease or HCL; patients with documented Gilbert's disease may be enrolled with sponsor approval provided total bilirubin is ≤ 2.0 x ULN) * Creatinine clearance (ClCr) ≥ 30 mL/min * Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial * For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated * Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load for \> 6 months * Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial * 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 * The effects of tovorafenib (DAY101), cladribine, and rituximab on the developing human fetus are unknown. For this reason and because BRAF kinase inhibitor agents as well as other therapeutic agents used in this trial are known to be teratogenic, women of child-bearing potential (WOCBP) and men must agree to use two forms of adequate contraception (including a highly effective birth control method in addition to a barrier method) during treatment prior to study entry and for the duration of study treatment participation and 12 months after the last dose of the study medication * WOCBP should use effective non-hormonal contraception during treatment and for 12 months after the last dose of the study medication. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. For male patients with a female partner of childbearing potential, a condom should be used for contraception in addition to one of the highly effective contraception methods prior to the study, for the duration of study treatment, and 12 months after the last dose of the study medication. Male patients must not father a child or donate sperm while participating in this study * Ability to understand and the willingness to sign a written informed consent document. Legally authorized representatives (LARs) may sign and give informed consent on behalf of study participants Exclusion Criteria: * Central nervous system (CNS) involvement with HCL is very rare, and therefore the biology of the disease in patients with CNS involvement may not be representative of the disease under study as a whole. Patients with treated brain metastases are eligible if follow-up brain imaging after CNS-directed therapy shows no evidence of progression * Patients with HCL who are BRAF V600E mutation negative and those with the variant HCL * Patients with platelets \< 50,000/mCL * Patients on warfarin and direct oral anticoagulants (due to risk of bleeding) * Patients who have not recovered from AEs as a result of prior anti-cancer therapy (i.e., have residual toxicities \> grade 1), with the exception of alopecia * Patients who are receiving any other investigational agents * Patients who are receiving strong CYP2C8 inhibitors, inducers, and breast cancer resistance protein (BCRP) substrates with narrow therapeutic index * Patients who are pregnant, breastfeeding, and/or unwilling to use adequate contraception during the study period and for 12 months after completion of the study * Patients with a history of allergic reactions attributed to compounds of similar chemical or biologic composition to tovorafenib (DAY101) or other agents used in the study, including those with a previous history of severe infusion-related reaction (anaphylaxis) with rituximab administration * Patients with known hypersensitivity to any of the study drugs * Patients with an inability to swallow oral medications or with gastrointestinal impairment * Live or live-attenuated vaccines within 28 days of randomization * Patients with uncontrolled intercurrent illness or any other significant condition(s) that would make participation in this protocol unreasonably hazardous * Pregnant women are excluded from this study because tovorafenib (DAY101) is a BRAF kinase inhibitor agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for AEs in nursing infants secondary to treatment of the mother with tovorafenib (DAY101), breastfeeding should be discontinued if the mother is treated with tovorafenib (DAY101). These potential risks may also apply to other agents used in this study

Study sites (4)

MD

OH

PA

VA