Combining tretinoin and arsenic trioxide with or without gemtuzumab ozogamicin for treating acute promyelocytic leukemia

Phase II Study of Treatment of Acute Promyelocytic Leukemia (APL) With ATRA, Arsenic Trioxide and Gemtuzumab Ozogamicin (GO)

Phase 2 Interventional M.D. Anderson Cancer Center · NCT01409161

This study is testing a new combination of treatments for people with acute promyelocytic leukemia to see if it helps them stay healthy longer and improves their chances of recovery without causing more side effects.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment151 (estimated)
Ages10 Years and up
SexAll
SponsorM.D. Anderson Cancer Center Academic / other
Drugs / interventionsgemtuzumab, chemotherapy
Locations5 sites (Houston, Texas and 4 other locations)
Trial IDNCT01409161 on ClinicalTrials.gov

What this trial studies

This phase II trial investigates the effectiveness of a combination treatment using tretinoin and arsenic trioxide, with the option of adding gemtuzumab ozogamicin, for patients with previously untreated acute promyelocytic leukemia (APL). The study aims to determine if this combination can lead to long-term event-free survival in low-risk patients and improve response rates in high-risk patients without increasing toxicity. Participants will receive these treatments over a specified schedule, followed by consolidation therapy for those who achieve a complete response. The trial includes regular follow-ups to monitor patient outcomes.

Who should consider this trial

Good fit: Ideal candidates for this study are patients diagnosed with acute promyelocytic leukemia who have not yet received treatment.

Not a fit: Patients with acute promyelocytic leukemia who have already undergone treatment or those with other types of leukemia may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment approach could significantly improve survival rates and outcomes for patients with acute promyelocytic leukemia.

How similar studies have performed: Other studies have shown promising results with similar treatment combinations in acute promyelocytic leukemia, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* A diagnosis of APL based on the presence of the PML-RAR-alpha fusion gene by cytogenetics, polymerase chain reaction (PCR), or POD test
* Ability to understand and the willingness to sign a written informed consent document indicating that they are aware of the investigational nature of the study
* Patients in whom therapy for APL was initiated on an emergent basis are eligible (patients may have already started treatment with ATRA, ATO, and/or one dose of idarubicin due to the urgency to start therapy early)
* Women of child-bearing potential must have a negative serum pregnancy test at screening; in addition to having a negative pregnancy test confirmed at screening, all female participants of childbearing potential must have a negative pregnancy test confirmed within 48 hours prior to dosing with the study drug
* 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

Exclusion Criteria:

* Fridericia corrected QT (QTcF) interval on the electrocardiogram (EKG) greater than 480 milliseconds
* Patients with creatinine \> 2.5 times upper limit of normal unless felt to be related the underlying leukemia by the treating physician or hemolysis or Gilbert's disease
* Patients with total bilirubin \>= 2.0 times upper limit of normal unless felt to be related the underlying leukemia by the treating physician or hemolysis or Gilbert's disease
* Patients with alanine aminotransferase (ALT)/aspartate aminotransferase (AST) \> 3 times upper limit of normal unless felt to be related the underlying leukemia by the treating physician or hemolysis or Gilbert's disease

Where this trial is running

Houston, Texas and 4 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Acute Promyelocytic Leukemia With PML-RARA
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.