Phase 2
N=17
Combined Tretinoin and Arsenic Trioxide for Patients With Newly Diagnosed Acute Promyelocytic Leukemia Followed by Risk-Adapted Postremission Therapy
Acute Promyelocytic Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT01404949 ↗Enrolled (actual)
17
Serious AEs
41.2%
Results posted
Nov 2021
Primary outcome: Primary: To Determine the Rate of Molecular Remission
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Tretinoin and Arsenic Trioxide (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Memorial Sloan Kettering Cancer Center
- Primary completion
- Feb 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY To Determine the Rate of Molecular Remission |
— | — |
| SECONDARY Participants Who Experienced a Complete Remission |
16 | — |
| SECONDARY To Determine the Proportion of Patients in Molecular Remission |
— | — |
| SECONDARY To Determine the Disease-free and Event-free Survival of Patients |
— | — |
| SECONDARY To Determine the Toxicity of This Treatment Program |
— | — |
| SECONDARY To Characterize the Differentiation of APL Cells During Treatment |
— | — |
| SECONDARY Explore the in Vivo Induction of Telomerase-dependent Cell Death |
— | — |
Summary
The purpose of this study is to find what effects, good and/or bad, treatment with two drugs has on leukemia. The first medicine is tretinoin (also called all-trans retinoic acid, ATRA, or Vesanoid). It is an approved medicine that causes the leukemia cells in APL to mature. It is related to vitamin A. The second is arsenic trioxide (Trisenox). It is an approved medicine for APL that comes back after earlier treatment.
APL is most often treated with tretinoin and standard chemotherapy drugs. These chemotherapy drugs can cause infection and bleeding. They can also damage the heart and normal bone marrow cells. This can lead to a second leukemia years later.
In this study, the investigators are using tretinoin and arsenic trioxide together. Both drugs work to treat APL. They have been used together in only a limited number of people. The investigators want to use these drugs together to reduce the amount of standard chemotherapy and decrease side effects. The patient will receive standard chemotherapy with a drug called idarubicin only if they have a higher chance of the leukemia coming back or a higher risk of side effects.
Eligibility Criteria
Inclusion Criteria
- Previously untreated patients with a morphologic diagnosis of APL, confirmed by demonstration of t(15;17) using conventional cytogenetics OR florescence in situ hybridization (FISH), OR a positive RT-PCR assay for PML-RAR at the subject's local institution.
- Age ≥18 years. Karnofsky performance status of ≥ 60%.
- Adequate renal function as demonstrated by a serum creatinine ≤ 2.0 mg/dl or a creatinine clearance of > 60 ml/min.
- Adequate hepatic function as demonstrated by a bilirubin < 2.0 mg/dl (unless attributable to Gilbert's disease) and an alkaline phosphatase, AST, and ALT ≤ 2.5 times the upper limit of normal.
- Normal cardiac function as demonstrated by a left ventricular ejection fraction ≥ 50% on echocardiogram or MUGA scan.
- QTc ≤ 500 msec on baseline ECG.
- Negative serum pregnancy test in women of childbearing potential.
- Ability to swallow oral medication.
- Men and women of child-bearing potential must be willing to practice an effective method of birth control during treatment and at least 4 months after treatment is finished.
- Patients with central nervous system involvement by APL are eligible and may receive concomitant treatment with radiation therapy and/or intrathecal chemotherapy in accordance with standard medical practice.
Exclusion Criteria
- Previous treatment for APL, except tretinoin, which may be given for up to 7 days prior to study entry.
- Active serious infections not controlled by antibiotics.
- Pregnant women or women who are breast-feeding.
- Concurrent active malignancy requiring immediate therapy.
- Clinically significant cardiac disease (NY Heart Association Class III or IV), including chronic arrhythmias, or pulmonary disease.
- Other serious or life-threatening conditions deemed unacceptable by the principal investigator.
Data sourced from ClinicalTrials.gov (NCT01404949). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.