Midostaurin in Treating Older Patients With Mutated Acute Myeloid Leukemia Post-Transplant

NCT02723435 | Withdrawn Phase 2 DMC FDA Drug

• 4 other identifiers: IRB-31582NCI-2016-00424HEMAML0022-EXTNCT02723435
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This phase 2 trial studies the side effects and how well midostaurin works in treating older patients with acute myeloid leukemia with change in genetic material post-hematopoietic cell transplantation. Midostaruin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving midostaruin post-transplant may improve patient outcomes.

Conditions

Acute Myeloid Leukemia With Gene Mutations; Adult Acute Myeloid Leukemia in Remission

Outcome Measures

Primary Outcomes (4)

• Event-free survival

  Up to 1 year

• Incidence of adverse events using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0

  Up to 30 days

• Overall survival

  Calculated and reported with Kaplan Meier curves. The statistical analyses will focus on estimation rather than hypothesis testing. Two-sided 95% confidence intervals will be presented, using the Clopper-Pearson method for proportions and using Greenwood's formula for time to event outcomes.

  Up to 1 year

• Relapse free survival

  Calculated and reported with Kaplan Meier curves. The statistical analyses will focus on estimation rather than hypothesis testing. Two-sided 95% confidence intervals will be presented, using the Clopper-Pearson method for proportions and using Greenwood's formula for time to event outcomes.

  Up to 1 year

Secondary Outcomes (1)

• Relapse rate after allogeneic transplant

  Up to 1 year

Study Arms (1)

Midostaurin

EXPERIMENTAL

Beginning 30 days post-HCT, participants receive oral midostaurin twice-a-day in 28-day treatment cycles, continuing up to 12 cycles in the absence of disease progression or unacceptable toxicity.

Drug: Midostaurin

Interventions

Midostaurin DRUG

Given PO

Also known as: Rydapt, CGP 41251, N-benzoyl-staurosporine, PKC-412

Midostaurin

Eligibility Criteria

• Age: 60 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Elderly patients with FLT3-mutated acute myeloid leukemia (AML)
• Prior enrollment in Stanford study IRB-25737
• In continued complete remission
• ≥ 30 days but ≤ 90 days post allogeneic hematopoietic cell transplant (HCT); treatment on this study protocol must begin before day 90 post-HCT
• Absolute neutrophil count (ANC) ≥ 1000 cells/uL
• Hemoglobin ≥ 8.0 g/dL and not requiring regular transfusions
• Platelets ≥ 50,000 cells/uL and not requiring regular transfusions
• Aspartate aminotransferase (AST) ≤ 2.5 times upper limit of normal (ULN)
• Alanine aminotransferase (ALT) ≤ 2.5 X ULN
• Serum bilirubin ≤ 2.5 times ULN
• Ability to give written informed consent, including via legally authorized representative
• Corrected QT (QTc) ≤ 450 msec
• Ejection fraction (EF) ≥ 45% by 2-dimensional transthoracic echocardiography (TTE) or multiple-gated acquisition (MUGA)
• Sexually active males, including vasectomized males, must agree via informed consent to use a condom during vaginal, anal, or oral intercourse, while taking midostaurin and for 5 months after stopping midostaurin
• Females must have or be:

You may not qualify if:

• Uncontrolled acute graft-vs-host disease (GVHD) grade 3 to 4
• Uncontrolled active infection
• Evidence of active AML (eg, circulating peripheral blasts on complete blood count)
• Known confirmed diagnosis of human immunodeficiency virus (HIV) infection
• Known confirmed diagnosis of active viral hepatitis
• QTc \> 450 msec
• Congenital long QT syndrome
• History of presence of sustained ventricular tachycardia, history of ventricular fibrillation or torsades de pointes
• Bradycardia defined as heart rate (HR) \< 50 beats per minute (bpm)
• Bifascicular block (right bundle branch block plus left anterior hemiblock)
• Congestive heart failure (CHF) New York Heart Association (NYHA) class 3 or 4
• Cardiac ejection fraction (EF) \< 45% within 28 days prior to starting cycle 1
• Other known malignancy (except carcinoma in situ)
• Other concurrent severe and/or uncontrolled medical condition which could compromise participation in the study, eg:
• Uncontrolled diabetes

Sponsors & Collaborators

• Stanford University: lead

MeSH Terms

Interventions

midostaurin

Study Officials

• David Iberri, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Assistant Professor

Study Record Dates

• First Submitted: March 25, 2016
• First Posted: March 30, 2016
• Primary Completion: April 1, 2018
• Study Completion: April 1, 2018
• Last Updated: May 4, 2018

Record last verified: 2018-05

Data Sharing

• IPD Sharing: Will not share