Acute Promyelocytic Leukemia (APL) Treated With ATRA, Arsenic Trioxide and Gemtuzumab Ozogamicin

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01409161
Recruitment Status : Recruiting
First Posted : August 4, 2011
Last Update Posted : August 6, 2018
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:

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The goal of this clinical research study is to learn if the combination of all-trans retinoic acid (ATRA), arsenic trioxide (ATO), and gemtuzumab ozogamicin (GO) can help to control acute promyelocytic leukemia (APL). The safety of this drug combination will also be studied.

This is an investigational study. ATRA and ATO are FDA approved and commercially available. GO is not FDA approved or commercially available. At this time, it is being used for research purposes only.

Up to 150 patients will take part in the study. All will be enrolled at MD Anderson.

Condition or disease Intervention/treatment Phase
Leukemia Drug: ATRA Drug: ATO Drug: GO (Gemtuzumab ozogamicin) Drug: Methylprednisolone Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 150 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Study of Treatment of Acute Promyelocytic Leukemia (APL) With ATRA, Arsenic Trioxide and Gemtuzumab Ozogamicin (GO)
Actual Study Start Date : October 2011
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : October 2019

Arm Intervention/treatment
Experimental: ATRA + ATO
Induction: All-trans retinoic acid (ATRA) 45 mg/m2 daily orally and Arsenic trioxide (ATO) 0.15 mg/kg by vein daily beginning on day 1 with Gemtuzumab ozogamicin (GO) 9 mg/m2 by vein. Methylprednisolone 50 mg by vein daily for 5 days followed by rapid taper starting on day 6 (Induction).
Drug: ATRA
45 mg/m2 daily by mouth beginning Day 1 (in 2 divided doses approximately 12 hours apart) for Course 1 (Induction) and during Weeks 1-2 and 5-6 of Courses 2-5 (Consolidation).
Other Names:
  • Tretinoin (oral)
  • All-trans retinoic acid
  • Vesanoid

Drug: ATO
0.15 mg/kg by vein over 1 hour (+/- 10 minutes) daily beginning on day 1, Course 1 (Induction) then over 1-2 hours for 5 days total during Weeks 1-4 of Courses 2-5 (Consolidation). Each course is about 8 weeks.
Other Names:
  • Arsenic trioxide
  • Trissenox

Drug: GO (Gemtuzumab ozogamicin)
9 mg/m2 by vein may be given one time during weeks 1-4 (Induction).
Other Names:
  • Gemtuzumab ozogamicin
  • Mylotarg

Drug: Methylprednisolone
50 mg by vein daily for 5 days followed by rapid taper starting on day 6 (Induction).
Other Names:
  • Depo-Medrol
  • Medrol
  • Solu-Medrol

Primary Outcome Measures :
  1. Event Free Survival (EFS) [ Time Frame: Day 21-28 ]
    EFS defined as the time from start of treatment to first documentation of disease relapse or death.

Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. A diagnosis of APL based on the presence of the PML-RAR-alpha fusion gene by cytogenetics, PCR, or POD test.
  2. 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.
  3. 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).
  4. Patients age 10 years and older are eligible.
  5. 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.
  6. 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:

  1. QTcF interval on the EKG greater than 480 milliseconds.
  2. Patients with creatinine > 2.5 and total bilirubin >/= 2.0 and ALT/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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01409161

Contact: Kiran Naqvi, MD 713-745-5073

United States, Texas
University of Texas MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Principal Investigator: Kiran Naqvi, MD M.D. Anderson Cancer Center

Additional Information:
Responsible Party: M.D. Anderson Cancer Center Identifier: NCT01409161     History of Changes
Other Study ID Numbers: 2010-0981
NCI-2011-02767 ( Registry Identifier: NCI CTRP )
First Posted: August 4, 2011    Key Record Dates
Last Update Posted: August 6, 2018
Last Verified: August 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by M.D. Anderson Cancer Center:
Acute Promyelocytic Leukemia
All-trans retinoic acid
Arsenic trioxide
Gemtuzumab ozogamicin

Additional relevant MeSH terms:
Leukemia, Promyelocytic, Acute
Neoplasms by Histologic Type
Leukemia, Myeloid, Acute
Leukemia, Myeloid
Prednisolone acetate
Methylprednisolone acetate
Methylprednisolone Hemisuccinate
Prednisolone hemisuccinate
Prednisolone phosphate
Arsenic trioxide
Anti-Inflammatory Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Neuroprotective Agents
Protective Agents
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Keratolytic Agents