Recruiting

Phase 2

Tretinoin, Arsenic Trioxide, Gemtuzumab OzogamicinTretinoin and Arsenic Trioxide With or Without Gemtuzumab Ozogamicin in Treating Patients With Previously Untreated Acute Promyelocytic Leukemia

Sponsor:

M.D. Anderson Cancer Center

Code:

NCT01409161

Conditions

Acute Promyelocytic Leukemia With PML-RARA

Eligibility Criteria

Sex: All

Age: 10+

Healthy Volunteers: Not accepted

Interventions

Arsenic Trioxide

Gemtuzumab Ozogamicin

Laboratory Biomarker Analysis

Tretinoin

Study Details

Brief summary:

This phase II trial studies how well tretinoin and arsenic trioxide with or without gemtuzumab ozogamicin works in treating patients with previously untreated acute promyelocytic leukemia. Drugs used in chemotherapy, such as tretinoin and arsenic trioxide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Immunotoxins, such as gemtuzumab ozogamicin, may find certain cancer cells and kill them without harming normal cells. Giving tretinoin and arsenic trioxide together with gemtuzumab ozogamicin may kill more cancer cells.

Conditions

Acute Promyelocytic Leukemia With PML-RARA

Study ID

NCT01409161

Start date

Oct 5, 2011

Status verified date

May, 2026

Completion date

Dec 18, 2027

Anticipated

Primary completion date

Dec 18, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 10+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

151 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (tretinoin, arsenic trioxide, gemtuzumab ozogamicin)

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.

Interventions

Arsenic Trioxide

Given IV

Gemtuzumab Ozogamicin

Given IV

Laboratory Biomarker Analysis

Correlative studies

Tretinoin

Given PO

Primary outcome measure

Event free survival [ Time Frame: The time from the start of treatment to first documentation of disease relapse or death, assessed up to 2 years ]

Central Contacts and Locations

Central contacts

Farhad Ravandi-Kashani

713-745-0394 fravandi@mdanderson.org

Locations

M D Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Farhad Ravandi-Kashani

713-745-0394

Principal Investigator:

Farhad Ravandi-Kashani

MD Anderson Regional Care Center-Katy

Recruiting

Houston, Texas, United States, 77094

Contacts

Farhad Ravandi-Kashani

713-745-0394

Principal Investigator:

Farhad Ravandi-Kashani

MD Anderson Regional Care Center-Bay Area

Recruiting

Nassau Bay, Texas, United States, 77058

Contacts

Farhad Ravandi-Kashani

713-745-0394

Principal Investigator:

Farhad Ravandi-Kashani

MD Anderson Regional Care Center-Sugar Land

Recruiting

Sugar Land, Texas, United States, 77478

Contacts

Farhad Ravandi-Kashani

713-745-0394

Principal Investigator:

Farhad Ravandi-Kashani

MD Anderson Regional Care Center-The Woodlands

Recruiting

The Woodlands, Texas, United States, 77384

Contacts

Farhad Ravandi-Kashani

713-745-0394

Principal Investigator:

Farhad Ravandi-Kashani

More Information

Sponsor

M.D. Anderson Cancer Center

Last update posted

May 20, 2026

Last verified

May, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by M.D. Anderson Cancer Center on 2026-05-20.