Tretinoin and Arsenic Trioxide With or Without Gemtuzumab Ozogamicin in Treating Patients With Previously Untreated Acute Promyelocytic Leukemia
May 18, 2026 updated by: M.D. Anderson Cancer Center
Phase II Study of Treatment of Acute Promyelocytic Leukemia (APL) With ATRA, Arsenic Trioxide and Gemtuzumab Ozogamicin (GO)
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.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Detailed Description
PRIMARY OBJECTIVES:
I. Assess whether a combination of all-trans retinoic acid (ATRA [tretinoin]), and arsenic trioxide (ATO) can produce long-term event-free survival in patients with low-risk untreated acute promyelocytic leukemia (APL).
II. Assess whether administration of gemtuzumab ozogamicin (GO) at the diagnosis in patients with high-risk APL (white blood cell [WBC] > 10,000) and if the WBC rises to > 10,000 after start of treatment (in patients with low-risk disease) will improve complete response (CR) rate without increasing toxicity in high-risk untreated APL.
OUTLINE:
INDUCTION: Patients receive tretinoin orally (PO) twice daily (BID), arsenic trioxide intravenously (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.
After completion of study treatment, patients are followed up every 6-12 months.
Study Type
Interventional
Enrollment (Estimated)
151
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Farhad Ravandi-Kashani
- Phone Number: 713-745-0394
- Email: fravandi@mdanderson.org
Study Locations
-
-
Texas
-
Houston, Texas, United States, 77030
- Recruiting
- M D Anderson Cancer Center
-
Contact:
- Farhad Ravandi-Kashani
- Phone Number: 713-745-0394
-
Principal Investigator:
- Farhad Ravandi-Kashani
-
Houston, Texas, United States, 77094
- Recruiting
- MD Anderson Regional Care Center-Katy
-
Contact:
- Farhad Ravandi-Kashani
- Phone Number: 713-745-0394
-
Principal Investigator:
- Farhad Ravandi-Kashani
-
Nassau Bay, Texas, United States, 77058
- Recruiting
- MD Anderson Regional Care Center-Bay Area
-
Contact:
- Farhad Ravandi-Kashani
- Phone Number: 713-745-0394
-
Principal Investigator:
- Farhad Ravandi-Kashani
-
Sugar Land, Texas, United States, 77478
- Recruiting
- MD Anderson Regional Care Center-Sugar Land
-
Contact:
- Farhad Ravandi-Kashani
- Phone Number: 713-745-0394
-
Principal Investigator:
- Farhad Ravandi-Kashani
-
The Woodlands, Texas, United States, 77384
- Recruiting
- MD Anderson Regional Care Center-The Woodlands
-
Contact:
- Farhad Ravandi-Kashani
- Phone Number: 713-745-0394
-
Principal Investigator:
- Farhad Ravandi-Kashani
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
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. |
Correlative studies
Given IV
Other Names:
Given IV
Other Names:
Given PO
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
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
|
Monitored using a Bayesian time-to-event model.
|
The time from the start of treatment to first documentation of disease relapse or death, assessed up to 2 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Farhad Ravandi-Kashani, M.D. Anderson Cancer Center
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
October 5, 2011
Primary Completion (Estimated)
December 18, 2027
Study Completion (Estimated)
December 18, 2027
Study Registration Dates
First Submitted
August 2, 2011
First Submitted That Met QC Criteria
August 2, 2011
First Posted (Estimated)
August 4, 2011
Study Record Updates
Last Update Posted (Actual)
May 20, 2026
Last Update Submitted That Met QC Criteria
May 18, 2026
Last Verified
May 1, 2026
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Neoplasms
- Neoplasms by Histologic Type
- Hematologic Diseases
- Leukemia, Myeloid
- Leukemia
- Leukemia, Myeloid, Acute
- Hemic and Lymphatic Diseases
- Leukemia, Promyelocytic, Acute
- Amino Acids, Peptides, and Proteins
- Proteins
- Organic Chemicals
- Retinoids
- Carotenoids
- Polyenes
- Alkenes
- Hydrocarbons, Acyclic
- Hydrocarbons
- Cyclohexenes
- Cyclohexanes
- Cycloparaffins
- Hydrocarbons, Alicyclic
- Hydrocarbons, Cyclic
- Terpenes
- Pigments, Biological
- Biological Factors
- Carbohydrates
- Glycosides
- Antibodies, Monoclonal, Humanized
- Antibodies, Monoclonal
- Antibodies
- Immunoglobulins
- Immunoproteins
- Blood Proteins
- Serum Globulins
- Globulins
- Inorganic Chemicals
- Diterpenes
- Aminoglycosides
- Oxides
- Oxygen Compounds
- Calicheamicins
- Vitamin A
- Arsenicals
- Arsenic Trioxide
- Gemtuzumab
- Tretinoin
- arsenous acid
Other Study ID Numbers
- 2010-0981 (Other Identifier: M D Anderson Cancer Center)
- NCI-2011-02767 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
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