Vodobatinib for Chronic Myeloid Leukemia

Recruiting at 67 trial locations

Overseen By Jayasree sreenivasan

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Sun Pharma Advanced Research Company Limited

Must be taking: TKIs

Disqualifiers: T315I, HIV, Hepatitis B, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Ongoing

122 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new medication, Vodobatinib, to determine its safety and effectiveness for people with chronic myeloid leukemia (CML), a type of blood cancer. The focus is on patients whose CML hasn't improved with at least three other treatments, including ponatinib. The trial seeks to understand how the body processes the medication and its impact on leukemia. Those who have tried multiple treatments without success and can manage regular doctor visits might be suitable for this trial. As a Phase 1 trial, this research explores how Vodobatinib works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I have to stop taking my current medications for the Vodobatinib trial?

The trial protocol does not specify if you must stop taking your current medications, but it mentions a washout period (time without taking certain medications) of at least 30 days or 5 half-lives for any other investigational agent. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that Vodobatinib (K0706) is likely to be safe for humans?

Research shows that Vodobatinib is under study as a treatment for chronic myeloid leukemia (CML) to assess its safety and effectiveness. In earlier studies, some patients experienced serious side effects. Out of seventy-eight patients, seven died, but researchers attributed only one death to the treatment. This indicates that while serious risks exist, most deaths were not directly linked to Vodobatinib.

Vodobatinib is undergoing early trials for individuals whose CML hasn't improved with other treatments. These trials primarily focus on safety, so unknown risks may still exist. However, testing in people suggests some initial confidence in its safety.

Joining a clinical trial is a personal decision. Understanding the possible risks and benefits is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Vodobatinib is unique because it targets chronic myeloid leukemia (CML) in a new way. Most current treatments for CML, like imatinib or dasatinib, are tyrosine kinase inhibitors that aim to block the BCR-ABL protein. Vodobatinib, however, is designed to be more selective, which could mean fewer side effects and better effectiveness against resistant forms of CML. Researchers are excited about vodobatinib because it has the potential to provide an option for patients who don’t respond well to existing therapies.

What evidence suggests that Vodobatinib might be an effective treatment for chronic myeloid leukemia?

Research has shown that Vodobatinib may help treat chronic myeloid leukemia (CML), particularly for patients unresponsive to other treatments. Studies found that 68% of patients with the chronic phase of CML experienced a significant reduction in cancer cells with Vodobatinib. Among patients with the accelerated phase of CML, 86% saw a major improvement in their blood counts. For those in the blast phase of CML, 50% had a major improvement in blood counts. Overall, survival rates were positive, with 100% of patients who couldn't tolerate previous treatments surviving. These findings suggest that Vodobatinib could be an effective option for those with treatment-resistant CML. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for adults over 18 with chronic myelogenous leukemia (CML) who haven't responded to at least three prior treatments, including ponatinib. They should be in a condition where they can perform daily activities with ease or have some limitation (ECOG status of 0-2). People ineligible include those unable to undergo blood draws, recent major surgery patients, candidates for curative therapy like stem cell transplant, other cancer diagnoses within 3 years, drug abuse history, T315I mutation presence in CML (Part C), and positive tests for pregnancy or certain infections.

Inclusion Criteria

I can take care of myself and perform daily activities.

Willing and able to comply with the scheduled visits

I have CML and my condition didn't improve after 3 different treatments, including ponatinib.

Exclusion Criteria

Positive exclusion tests: urine pregnancy tests (if applicable), HIV, hepatitis B surface antigen, or hepatitis C virus

You have a history of serious drug abuse according to the investigator's assessment.

I am eligible for a treatment that could potentially cure my condition, including a stem cell transplant.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Vodobatinib (K0706) to assess safety, tolerability, pharmacokinetics, and anti-leukemic activity

Ongoing

Follow-up

Participants are monitored for safety and effectiveness after treatment

60 months

What Are the Treatments Tested in This Trial?

Interventions

• Vodobatinib (K0706)

Trial Overview The study is testing Vodobatinib capsules' safety and effectiveness against resistant/intolerant chronic myelogenous leukemia. It's a Phase 1/2 trial which means it's early in the clinical research process focusing on how safe the treatment is (Phase 1) and starting to look at how well it works (Phase 2).

How Is the Trial Designed?

Group I: Vodobatinib (K0706) capsules Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Sun Pharma Advanced Research Company Limited

Lead Sponsor

Trials

33

Recruited

4,600+

Published Research Related to This Trial

Imatinib Mesylate has significantly improved outcomes for chronic myeloid leukemia (CML) patients in the chronic phase, leading to high rates of complete hematologic and cytogenetic responses.

Long-term studies indicate that while Imatinib is effective, there is a need for early molecular markers to identify which patients will benefit most from this treatment, especially with the availability of generic options and newer therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Imatinib mesylate in chronic myeloid leukemia: frontline treatment and long-term outcomes. Stagno, F., Stella, S., Spitaleri, A., et al.[2016]

In a study of 163 newly diagnosed patients with chronic-phase chronic myeloid leukemia (CML), dasatinib and nilotinib showed significantly higher rates of complete cytogenetic response (CCyR) and major molecular response (MMR) compared to imatinib after 3 and 12 months of treatment.

The safety profiles of dasatinib, nilotinib, and imatinib were similar, indicating that while dasatinib and nilotinib are more effective, they do not present additional safety concerns compared to imatinib.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical Efficacy of Dasatinib, Nilotinib and Imatinib in Newly Diagnosed Patients with Chronic-Phase Chronic Myeloid Leukemia: A Three-year Retrospective Analysis]. Wang, YY., Zhao, HG., Cui, ZG., et al.[2022]

In a study of 26 chronic myelogenous leukemia (CML) patients treated with nilotinib for nearly 5 years, 61.5% achieved a complete hematologic response, demonstrating its efficacy for patients resistant or intolerant to imatinib.

Nilotinib was generally safe, with most adverse events being mild (grade 1 or 2), though some patients experienced significant hematologic side effects, particularly those in advanced stages of the disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Long-term outcomes of nilotinib treatment for chronic myelogenous leukemia patients with imatinib resistance or intolerance]. Wei, Y., Zhang, X., Chen, W., et al.[2022]

In a study of 35 chronic myelogenous leukemia (CML) patients who were resistant or intolerant to imatinib, nilotinib demonstrated a high overall survival rate of 93.5% at 18 months, indicating its efficacy as a treatment option.

Nilotinib was well-tolerated, with most adverse events being mild (grade 1-2), although some patients experienced more serious hematologic issues, particularly in advanced CML, highlighting the importance of monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Nilotinib treatment for imatinib resistant or intolerant chronic myelogenous leukemia.]. Wang, AH., Zhou, L., You, JH., et al.[2018]

Citations

1. thelancet.com thelancet.com/journals/lanhae/article/PIIS2352-3026(24)00354-5/abstract

Vodobatinib for patients with Philadelphia chromosome- ... For patients with accelerated-phase chronic myeloid leukaemia, six (86%) of seven patients had a major haematological response (median duration ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2352302624003545

Vodobatinib for patients with Philadelphia chromosome ... Major haematological response was reached by two (50%) of four patients with blast-phase chronic myeloid leukaemia and the median duration of response was 6·2 ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11534886/

Outcomes of chronic myeloid leukemia patients after ... With a median follow-up of 11.2 months, overall survival of the global cohort was 73%; 100% for intolerant patients, 71% for resistant patients ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT02629692

Study Details | NCT02629692 | Safety and Anti-leukemic ... Phase 1/2 study to determine safety, tolerability, pharmacokinetics, and anti-leukemic activity of Vodobatinib (K0706) in treatment-refractory/intolerant CML.

5. oncpracticemanagement.com oncpracticemanagement.com/issues/2021/february-2021-vol-11-no-2/vodobatinib-effective-in-patients-with-cml-regardless-of-previous-ponatinib-therapy

Vodobatinib Effective in Patients with CML Regardless of ... Across all doses, MCyR to vodobatinib was observed in 21 of the 31 (68%) evaluable patients with chronic-phase CML. The efficacy according to previous ponatinib ...

6. cmladvocates.net cmladvocates.net/clinical_trials/clr_15_03-safety-and-anti-leukemic-activity-of-vodobatinib-k0706-for-refractory-intolerant-cml-asia-europe-usa/

CLR_15_03 = Safety and anti-leukemic activity of ... This is an open label, Phase 2 study to determine the efficacy and safety of the novel tyrosine kinase inhibitor vodobatinib (K0706).

7. mayo.edu mayo.edu/research/clinical-trials/cls-20522394

Safety and Anti-leukemic Activity of Vodobatinib (K0706) ... The purpose of this study is determine the safety, tolerability, pharmacokinetics and activity of K0706.