Clinical trial NCT02873338: Dociparstat Sodium (CX-01) Combined With Standard Induction Therapy for Newly Diagnosed Acute Myeloid Leukemia
Official title: A Randomized, Phase II Study of CX-01 Combined With Standard Induction Therapy for Newly Diagnosed Acute Myeloid Leukemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acute Myeloid Leukemia
Lead sponsor: Chimerix
Sponsor class: INDUSTRY
First posted: 2016-08-19
Enrollment: 75
Sex: ALL
Minimum age (years): 60
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This was an exploratory Phase 2, open label, randomized, multicenter, parallel group study to determine whether there was evidence that the addition of dociparstat (CX-01) at 2 different does levels to standard induction therapy (cytarabine+idarubicin, "7+3") and consolidation therapy had an additive therapeutic effect for subjects newly diagnosed with acute myeloid leukemia (AML) when compared with subjects receiving standard induction chemotherapy alone.

Detailed description: The primary efficacy endpoint was to assess whether dociparstat in conjunction with standard induction therapy for AML increased the complete remission rate based on the International Working Group AML response criteria.

A total of 75 subjects were to be randomized in a 1:1:1 ratio to 1 of the following treatment groups:

* Group 1: cytarabine + idarubicin
* Group 2: cytarabine + idarubicin + dociparstat 0.125 mg/kg/hr
* Group 3: cytarabine + idarubicin + dociparstat 0.25 mg/kg/hr

Subjects received up to 2 induction cycles and up to 2 consolidation cycles and participated in the study for up to 18 months. Clinical laboratory tests were conducted routinely, and bone marrow aspirates and biopsies were performed during the induction cycles. Safety was monitored through adverse events and clinical laboratory results.

Eligibility criteria: Inclusion Criteria:

Subjects had to meet all the following criteria to be eligible for enrollment in this study:

1. Had newly diagnosed, de novo or secondary, previously untreated acute myeloid leukemia (AML).
2. Had an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.

Exclusion Criteria:

Subjects who met any of the following criteria were not eligible for enrollment in this study:

1. Had acute promyelocytic leukemia
2. Had prior chemotherapy for AML.
3. Had prior intensive chemotherapy or stem cell transplantation for the treatment of myelodysplastic syndrome.
4. Had central nervous system (CNS) leukemia.

Primary outcomes:
measure: Number of Subjects Who Achieved Morphologic Complete Remission
timeFrame: During induction and re-induction phases of treatment (up to 60 days after the start of each treatment cycle)
Secondary outcomes:
measure: Duration of Event-free Survival
timeFrame: Randomization up to 30 months
measure: Time to Leukemia-free Survival
timeFrame: Randomization until disease relapse or patient death from any cause, whichever occurs first, assessed up to 30 months
measure: Number of Subjects Who Achieved Overall Survival
timeFrame: Randomization to end of study (18 months)
measure: Number of Subjects Who Achieved Composite Complete Remission
timeFrame: Up to 60 days after the start of each treatment cycle
measure: Duration of Morphologic Complete Remission
timeFrame: Randomization to end of study (18 months)
measure: Time to Recovery of Neutrophils
timeFrame: Randomization to ANC recovery, for up to 60 days after the start of each treatment cycle
measure: Time to Platelet Recovery
timeFrame: Randomization to platelet recovery, for up to 60 days after the start of each treatment cycle
measure: Number of Subjects Who Died by Day 30
timeFrame: 30 days (from first day of induction treatment to 30 days after)
measure: Number of Subjects Who Died by Day 60.
timeFrame: 60 days (from the first day of induction treatment to 60 days after)
measure: Number of Subjects Who Died by Day 90
timeFrame: 90 days (from the first day of induction treatment to 90 days after)
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: University of California, San Diego, Moores Cancer Center
state: California
city: Denver
country: United States
facility: Colorado Blood Cancer Institute
state: Colorado
city: Washington
country: United States
facility: George Washington University
state: District of Columbia
city: Indianapolis
country: United States
facility: Franciscan St. Francis Health
state: Indiana
city: Sioux City
country: United States
facility: June E. Nylen Cancer Center
state: Iowa
city: Louisville
country: United States
facility: Norton Cancer Institute
state: Kentucky
city: New Orleans
country: United States
facility: Tulane University/Tulane Cancer Center
state: Louisiana
city: Detroit
country: United States
facility: Karmanos Cancer Institute
state: Michigan
city: Minneapolis
country: United States
facility: Allina Health - Virginia Piper Cancer Institute
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine in St. Louis
state: Missouri
city: Albuquerque
country: United States
facility: New Mexico Cancer Care Alliance
state: New Mexico
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Lake Success
country: United States
facility: Northwell Health, Monter Cancer Center
state: New York
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University Knight Cancer Institute
state: Oregon
city: Providence
country: United States
facility: Rhode Island Hospital
state: Rhode Island
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Sioux Falls
country: United States
facility: Avera Cancer Institute
state: South Dakota
city: Nashville
country: United States
facility: Tennessee Oncology/Sarah Cannon Research Institute
state: Tennessee
city: Dallas
country: United States
facility: Baylor Research Institute/Baylor Sammons Cancer Center/Baylor University Medical Center
state: Texas
city: San Antonio
country: United States
facility: Methodist Healthcare System of San Antonio
state: Texas
city: Salt Lake City
country: United States
facility: Huntsman Cancer Institute
state: Utah
city: Salt Lake City
country: United States
facility: LDS Hospital
state: Utah
Study officials:
affiliation: Cantex Pharmaceuticals Inc.
name: Stephen Marcus, MD
role: STUDY_DIRECTOR
References: