Arsenic Trioxide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Phase 2

Withdrawn

• Conditions: Adult Acute Monoblastic Leukemia (M5a); Adult Acute Myeloblastic Leukemia With Maturation (M2); Adult Acute Megakaryoblastic Leukemia (M7); Adult Acute Minimally Differentiated Myeloid Leukemia (M0); Adult Acute Myelomonocytic Leukemia (M4); Adult Erythroleukemia (M6a); Adult Pure Erythroid Leukemia (M6b); Recurrent Adult Acute Myeloid Leukemia; Adult Acute Monocytic Leukemia (M5b); Adult Acute Myeloblastic Leukemia Without Maturation (M1)
• Interventions: Drug: arsenic trioxide; Other: laboratory biomarker analysis

• Registration Number: NCT01835288
• Lead Sponsor: Stanford University

Brief Summary

This phase II trial studies how well arsenic trioxide works in treating patients with relapsed or refractory acute myeloid leukemia. Drugs used in chemotherapy, such as arsenic trioxide, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.

Detailed Description

PRIMARY OBJECTIVES:

I. Determine the complete remission rate of relapsed and refractory acute myeloid leukemia (AML) patients with Mutated Nucleophosmin 1 (NPM1) gene.

SECONDARY OBJECTIVES:

I. Determine the duration of remission in these patients. II. Determine the in vivo biological effect of arsenic trioxide in AML with mutated NPM1.

OUTLINE:

Patients receive arsenic trioxide intravenously (IV) over 1-2 hours daily for up to 45 days. Patients achieving complete remission, receive arsenic trioxide IV over 1-2 hours daily 5 days a week for 4 weeks. Treatment repeats every 8 weeks for up to 4 courses in the absence of disease progression or unacceptable toxicity.

Study Timeline

• Study First Submitted: 2013-04-15
• Study First Submitted QC: 2013-04-15
• Study First Posted: 2013-04-18
• Study Start: 2013-05
• Primary Completion: 2015-05
• Status Verified: 2018-05
• Last Update Submitted: 2018-05-16
• Last Update Posted: 2018-05-21

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• AML, any French- American- British (FAB) subtype except M3, with confirmed mutation in the NPM1 gene
• Relapsed and/or refractory AML from any duration of complete remission (CR); any number of prior therapies allowed
• Eastern Cooperative Oncology Group (ECOG) performance status 0-2, life expectancy > 3 months
• Serum creatinine =< 2.0 mg/dL
• Bilirubin =< 2.0 mg/dL
• Alanine aminotransferase (ALT)/aspartate aminotransferase (AST) =< 3 x upper limit of normal (ULN)
• Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria

• Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies; pregnancy tests must be obtained in women; sexually active males or females may not participate unless they have agreed to use an effective contraceptive method
• Patients who are currently receiving another investigational drug
• Patients who are currently receiving other anti-cancer agents
• Uncontrolled systemic fungal, bacterial, viral, or other infection (defined as exhibiting ongoing signs/symptoms related to the infection and without improvement, despite appropriate antibiotics or other treatment)
• Known hypersensitivity to arsenic trioxide

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment (arsenic trioxide)	arsenic trioxide	Patients receive arsenic trioxide IV over 1-2 hours daily for up to 45 days. Patients achieving complete remission, receive arsenic trioxide IV over 1-2 hours daily 5 days a week for 4 weeks. Treatment repeats every 8 weeks for up to 4 courses in the absence of disease progression or unacceptable toxicity.
Treatment (arsenic trioxide)	laboratory biomarker analysis	Patients receive arsenic trioxide IV over 1-2 hours daily for up to 45 days. Patients achieving complete remission, receive arsenic trioxide IV over 1-2 hours daily 5 days a week for 4 weeks. Treatment repeats every 8 weeks for up to 4 courses in the absence of disease progression or unacceptable toxicity.

Primary Outcome Measures

Name	Time	Method
Rate of complete remission following arsenic trioxide induction	After 4 weeks of therapy

Secondary Outcome Measures

Name	Time	Method
Median duration of remission	Time from documented complete remission until time of disease relapse, assessed up to 2 years