What side effects are associated with this type of intervention?

The most common treatments for Acute Promyelocytic Leukemia (APL) include Tretinoin, Arsenic Trioxide, and Gemtuzumab Ozogamicin. Tretinoin can cause headaches, fever, dry skin, and increased white blood cell counts. Arsenic Trioxide is associated with fatigue, nausea, vomiting, diarrhea, and QT prolongation affecting heart rhythm. Gemtuzumab Ozogamicin may lead to infusion-related reactions, liver toxicity, and myelosuppression. Combining these treatments can potentially amplify these side effects.

What prior FDA approvals exist?

Tretinoin (all-trans retinoic acid, ATRA) and Arsenic Trioxide are both FDA-approved for the treatment of Acute Promyelocytic Leukemia (APL). Tretinoin works by promoting the differentiation of promyelocytes, while Arsenic Trioxide induces apoptosis and partial differentiation of APL cells. Gemtuzumab Ozogamicin, an immunotoxin targeting CD33, has also been studied in combination with these agents to enhance treatment efficacy by specifically targeting and killing cancer cells. These treatments aim to stop the growth of cancer cells by various mechanisms, including killing the cells, stopping their division, or preventing their spread.

What other types of research exist?

Promising novel alternatives for treating Acute Promyelocytic Leukemia (APL) in 2024 include FLT3 inhibitors (e.g., midostaurin), BCL-2 inhibitors (e.g., venetoclax), and newer antibody-drug conjugates. These therapies target specific pathways or proteins involved in APL, potentially offering improved efficacy and safety profiles.

← Back to Search

Arsenic-containing Agent

Tretinoin + Arsenic Trioxide (+/- Gemtuzumab) for Leukemia

Phase 2

Recruiting

Led By Farhad Ravandi-Kashani

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

All sexually active subjects (males and females of child-bearing potential) agree to use 2 effective methods of contraception for the duration of the study

A diagnosis of APL based on the presence of the PML-RAR-alpha fusion gene by cytogenetics, polymerase chain reaction (PCR), or POD test

Timeline

Screening 3 weeks

Treatment Varies

Follow Up the time from the start of treatment to first documentation of disease relapse or death, assessed up to 2 years

Awards & highlights

No Placebo-Only Group

Summary

This study is evaluating whether a combination of drugs may help treat acute promyelocytic leukemia.

Who is the study for?

This trial is for patients with a new diagnosis of acute promyelocytic leukemia (APL) confirmed by specific genetic tests. Participants must understand the study and agree to sign consent forms. Those who have started emergency APL treatment can join, and women of childbearing age need negative pregnancy tests before and during the study. Contraception use is required for all sexually active participants.

What is being tested?

The trial is testing how well tretinoin and arsenic trioxide work together, with or without an additional drug called gemtuzumab ozogamicin, in treating newly diagnosed APL patients. The goal is to see if adding gemtuzumab ozogamicin can kill more cancer cells without harming normal cells.

What are the potential side effects?

Possible side effects include reactions related to chemotherapy such as fatigue, nausea, hair loss, increased risk of infection; heart rhythm problems; liver function changes; or kidney issues. Gemtuzumab may cause targeted cell death but also might affect non-cancerous cells.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I agree to use two effective birth control methods during the study.

Select...

My APL is confirmed by a specific gene test.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ the time from the start of treatment to first documentation of disease relapse or death, assessed up to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~the time from the start of treatment to first documentation of disease relapse or death, assessed up to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and the time from the start of treatment to first documentation of disease relapse or death, assessed up to 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Event free survival

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (tretinoin, arsenic trioxide, gemtuzumab ozogamicin)Experimental Treatment4 Interventions

INDUCTION: Patients receive tretinoin PO BID, arsenic trioxide IV over 1-2 hours daily, and gemtuzumab ozogamicin IV over 2 hours once at weeks 1-4. CONSOLIDATION: Patients achieving CR receive arsenic trioxide IV 5 days per week during weeks 1-4, 9-12, 17-20, and 25-28 and tretinoin PO BID for 2 weeks on and 2 weeks off. Treatment repeats every 8 weeks for up to 4 courses in the absence of disease progression or unacceptable toxicity.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Tretinoin

2013

Completed Phase 4

~2040

Gemtuzumab Ozogamicin

2006

Completed Phase 4

~460

Arsenic Trioxide

2011

Completed Phase 3

~240

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Acute Promyelocytic Leukemia (APL) include Tretinoin and Arsenic Trioxide, which are chemotherapy agents that induce differentiation and apoptosis of leukemic cells, thereby stopping their growth and spread. Gemtuzumab Ozogamicin is an immunotoxin that targets the CD33 antigen on leukemic cells, delivering a cytotoxic agent to kill these cells specifically. These treatments are vital for APL patients as they effectively target and eliminate the cancerous cells, leading to remission and improving survival rates.

Promyelocytic leukemia retinoid signaling targets regulate apoptosis,tissue factor and thrombomodulin expression.