Olutasidenib for Acute Myeloid Leukemia

MI
Overseen ByMassey IIT Research Operations
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: Virginia Commonwealth University
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called olutasidenib for individuals with acute myeloid leukemia (AML) that has a specific genetic mutation known as IDH1. The goal is to determine if olutasidenib is safe and can maintain cancer remission or improve survival after initial treatments. Participants must have confirmed IDH1 mutant AML and have completed their initial treatment rounds, achieving some level of recovery, such as a complete response. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.

Will I have to stop taking my current medications?

The trial requires that you stop taking any medications that might interfere with the study drug, including those that affect its effectiveness, metabolism, or safety. This includes drugs known to cause QT prolongation and other targeted therapies for AML. It's best to discuss your current medications with the study team to see if any need to be stopped.

Is there any evidence suggesting that olutasidenib is likely to be safe for humans?

Research has shown that olutasidenib is generally well-tolerated by people with acute myeloid leukemia (AML) who have a specific gene change called IDH1. In earlier studies, patients taking olutasidenib experienced long-lasting remissions, meaning their cancer stayed away for some time. Most patients found the treatment's side effects manageable.

The FDA has already approved olutasidenib for treating adults with relapsed or refractory AML with the IDH1 mutation. This approval indicates that the treatment has been reviewed and found safe for certain conditions.

Overall, evidence suggests that olutasidenib is generally safe for people with AML and the IDH1 mutation. However, individual experiences can differ, so discussing potential risks with a healthcare provider is important.12345

Why do researchers think this study treatment might be promising?

Olutasidenib is unique because it specifically targets mutations in the IDH1 enzyme, which is involved in many cases of acute myeloid leukemia (AML). Unlike traditional chemotherapy treatments that broadly attack rapidly dividing cells, olutasidenib offers a more targeted approach, potentially reducing side effects. Researchers are excited about its ability to precisely inhibit the mutated enzyme, which could lead to better outcomes for patients who have these specific genetic mutations. This precision in targeting makes olutasidenib a promising option for those with AML who may not respond well to conventional therapies.

What evidence suggests that olutasidenib might be an effective treatment for acute myeloid leukemia?

Research has shown that olutasidenib, which participants in this trial will receive, may effectively treat acute myeloid leukemia (AML) with an IDH1 mutation. In earlier studies, patients who received olutasidenib demonstrated better response rates, including complete recovery, particularly if they had fewer previous treatments. For example, one study found a 54% overall response rate in these patients. Additionally, olutasidenib has been associated with longer survival times compared to some other treatments, such as ivosidenib. Specifically, the average time patients remained in complete remission with olutasidenib was reported as 28.1 months, suggesting it could help keep the cancer away and possibly improve survival.678910

Who Is on the Research Team?

KM

Keri Maher, DO

Principal Investigator

Virginia Commonwealth University

Are You a Good Fit for This Trial?

This trial is for individuals with acute myeloid leukemia (AML) that has a specific mutation called IDH1. Participants should have completed the standard initial treatments known as induction and consolidation therapy.

Inclusion Criteria

AST/ALT ≤3 × ULN
I will use barrier methods during sex to prevent pregnancy.
Calculated creatinine clearance ≥30 mL/min
See 7 more

Exclusion Criteria

Participation in another clinical trial within 4 weeks
Medical, psychological, or social conditions impacting study participation
I am scheduled for a stem cell transplant within the next 2 months.
See 13 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive olutasidenib 150 mg by mouth twice daily for up to 2 years

Up to 2 years
Regular monitoring visits for toxicities, adverse events, QOL, and disease status

Follow-up

Participants are monitored for survival endpoints after treatment

Up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

  • Olutasidenib
Trial Overview The study is testing olutasidenib, an investigational drug, to see if it's safe and can help maintain remission or improve survival in AML patients with the IDH1 mutation after they've finished their main treatments.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Olutasidenib Investigational Agent AdministrationExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Virginia Commonwealth University

Lead Sponsor

Trials
732
Recruited
22,900,000+

Rigel Pharmaceuticals

Industry Sponsor

Trials
37
Recruited
4,000+

Citations

A phase 2 study of olutasidenib in relapsed/refractory acute ...Conclusions: Higher response rates (including CR and CRh) and greater survival were observed in patients receiving OLU following 1-2 versus ≥3 ...
2.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40511494/
Effectiveness of olutasidenib versus ivosidenib in patients ...Following weighting, treatment with OLU versus IVO was associated with significantly higher rates of complete response (RD: 0.25; 95%CI: 0.01, ...
AML-629: A Phase 2 Study of Olutasidenib in Relapsed ...Patients treated after 1–2 prior regimens had higher ORR (54% vs 39%) and CR/CRh rates (41% vs 24%), a greater proportion achieving CR (38% vs 22%), and longer ...
Functional cure with single agent olutasidenib in relapsed ...In the pivotal Phase II trial of olutasidenib, the median duration of complete response (CR) was 28.1 months. Here we report the first patient ...
A phase 2 study of olutasidenib in relapsed/refractory acute ...Higher response rates (including CR and CRh) and greater survival were observed in patients receiving OLU following 1-2 versus ≥3 prior treatment regimens.
FDA Approval Summary: Olutasidenib for Adult Patients with ...FDA Approval Summary: Olutasidenib for Adult Patients with Relapsed or Refractory Acute Myeloid Leukemia with an Isocitrate Dehydrogenase-1 Mutation. Ashley C ...
Safety and efficacy of olutasidenib treatment in elderly ...Olutasidenib was generally well tolerated in elderly patients with R/R mIDH1AML and induced durable remissions.
Acute Myeloid Leukemia AML-619: Olutasidenib as ...Evaluate the efficacy and tolerability of olutasidenib as maintenance therapy in patients with mIDH1 AML who achieved complete remission (CR) to prior therapy.
Olutasidenib in combination with azacitidine induces durable ...Olutasidenib plus azacitidine induced high response rates and durable remissions with a tolerable side effect profile in patients with R/R AML with diverse ...
Five-Year Follow-Up Data Confirm Long-Term Benefits of ...Justin M. Watts, MD, discusses 5-year efficacy and safety data for olutasidenib in IDH1-mutant, relapsed/refractory AML.
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