Dociparstat Sodium (CX-01) Combined With Standard Induction Therapy for Newly Diagnosed Acute Myeloid Leukemia

NCT02873338 | Completed Phase 2 Results DMC

• 1 other identifier: CNTX-CX-01-2015-AML-1
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 23

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.

Conditions

Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Number of Subjects Who Achieved Morphologic Complete Remission

  Morphologic complete remission (CR) was evaluated by International Working Group (IWG) criteria and defined as absolute neutrophil count (ANC) \>1000/microliter; platelet count \>100,000, \<5% blasts in bone marrow aspirate, no blasts with Auer rods, and no evidence of extramedullary disease.

  During induction and re-induction phases of treatment (up to 60 days after the start of each treatment cycle)

Secondary Outcomes (10)

• Duration of Event-free Survival

  Randomization up to 30 months

• Time to Leukemia-free Survival

  Randomization until disease relapse or patient death from any cause, whichever occurs first, assessed up to 30 months

• Number of Subjects Who Achieved Overall Survival

  Randomization to end of study (18 months)

• Number of Subjects Who Achieved Composite Complete Remission

  Up to 60 days after the start of each treatment cycle

• Duration of Morphologic Complete Remission

  Randomization to end of study (18 months)

Study Arms (3)

Control (idarubicin+cytarabine)

ACTIVE COMPARATOR

Induction: * Idarubicin 12 mg/m2/day by slow (10 to 30 minutes) intravenous (IV) injection/infusion daily (Days 1, 2, and 3) * Cytarabine 100 mg/m2/day continuous 24-hour IV infusion (Days 1 to 7) Re-induction: * Idarubicin 12 mg/m2/day slow (10 to 30 minutes) IV injection/infusion daily (Days 1 and 2) * Cytarabine 100 mg/m2/day continuous 24-hour IV infusion (Days 1 to 5) Consolidation: • Cytarabine 1.0 g/m2 over 3 hours, every 12 hours (Days 1, 3, and 5)

Drug: Idarubicin; Drug: Cytarabine

Dociparstat 0.125 mg/kg

EXPERIMENTAL

Induction: * Dociparstat 4 mg/kg initial bolus 30 minutes post-idarubicin dose (Day 1), followed by dociparstat 0.125 mg/kg/hr continuous 24-hour intravenous (IV) infusion (Days 1 to 7) * Idarubicin 12 mg/m2/day slow IV injection/infusion daily (Days 1, 2, and 3) * Cytarabine 100 mg/m2/day continuous 24-hour IV infusion (Days 1 to 7) Re-induction: * Dociparstat 4 mg/kg initial bolus 30 minutes post-idarubicin dose (Day 1), followed by dociparstat 0.125 mg/kg/hr continuous 24-hour IV infusion (Days 1 to 5) * Idarubicin 12 mg/m2/day slow IV injection/infusion daily (Days 1 and 2) * Cytarabine 100 mg/m2/day continuous 24-hour IV infusion (Days 1 to 5) Consolidation: * Dociparstat 4 mg/kg initial bolus 30 minutes post-3-hour cytarabine infusion (Day 1), followed by dociparstat 0.125 mg/kg/hr continuous 24-hour IV infusion on (Days 1 to 5; total 120 hours) * Cytarabine 1.0 g/m2 over 3 hours, every 12 hours (Days 1, 3, and 5)

Drug: Dociparstat sodium; Drug: Idarubicin; Drug: Cytarabine

Dociparstat 0.25 mg/kg

EXPERIMENTAL

Induction: * Dociparstat 4 mg/kg initial bolus 30 minutes post-idarubicin dose (Day 1), followed by dociparstat 0.25 mg/kg/hr continuous 24-hour intravenous (IV) infusion (Days 1 to 7) * Idarubicin 12 mg/m2/day slow IV injection/infusion daily (Days 1, 2, and 3) * Cytarabine 100 mg/m2/day continuous 24-hour IV infusion (Days 1 to 7) Re-induction: * Dociparstat 4 mg/kg initial bolus 20 minutes post-idarubicin dose (Day 1), followed by dociparstat 0.25 mg/kg/hr continuous 24-hour IV infusion (Days 1 to 5) * Idarubicin 12 mg/m2/day slow IV injection/infusion daily (Days 1 and 2) * Cytarabine 100 mg/m2/day continuous 24-hour IV infusion (Days 1 to 5) Consolidation: * Dociparstat 4 mg/kg initial bolus 30 minutes post-3-hour cytarabine infusion (Day 1), followed by dociparstat 0.25 mg/kg/hr continuous 24-hour IV infusion (Days 1 to 5; total 120 hours) * Cytarabine 1.0 g/m2 over 3 hours, every 12 hours (Days 1, 3, and 5)

Drug: Dociparstat sodium; Drug: Idarubicin; Drug: Cytarabine

Interventions

Dociparstat sodium DRUG

Subjects received 4 mg/kg dociparstat intravenous (IV) bolus followed by doses of 0.125 or 0.25 mg/kg/hr dociparstat given on Days 1 through 7 with standard induction therapy, on Days 1 through 5 or 7 with standard re-induction therapy, and on Days 1, 3, and 5 with standard consolidation therapy.

Also known as: 2-O, 3-O desulfated heparin, ODSH, PGX-100, CX-01, Dociparstat

Dociparstat 0.125 mg/kg; Dociparstat 0.25 mg/kg

Idarubicin DRUG

Subjects received 12 mg/m2/day idarubicin by slow (10 to 15 minutes) intravenous (IV) injection daily on Days 1, 2 and 3 of induction therapy, and on Days 1 and 2 of re-induction therapy.

Also known as: Idamycin

Control (idarubicin+cytarabine); Dociparstat 0.125 mg/kg; Dociparstat 0.25 mg/kg

Cytarabine DRUG

Subjects received 100 mg/m2/day cytarabine by continuous intravenous (IV) infusion on Days 1 through 7 of induction therapy and on Days 1 through 5 of re-induction therapy. During consolidation therapy, subjected received 1.0 g/m2 cytarabine IV infusion given over 3 hours every 12 hours on Days 1, 3, and 5.

Control (idarubicin+cytarabine); Dociparstat 0.125 mg/kg; Dociparstat 0.25 mg/kg

Eligibility Criteria

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

You may qualify if:

• Subjects had to meet all the following criteria to be eligible for enrollment in this study:
• Had newly diagnosed, de novo or secondary, previously untreated acute myeloid leukemia (AML).
• Had an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.

You may not qualify if:

• Subjects who met any of the following criteria were not eligible for enrollment in this study:
• Had acute promyelocytic leukemia
• Had prior chemotherapy for AML.
• Had prior intensive chemotherapy or stem cell transplantation for the treatment of myelodysplastic syndrome.
• Had central nervous system (CNS) leukemia.

Sponsors & Collaborators

• Jazz Pharmaceuticals: lead

Study Sites (23)

University of California, San Diego, Moores Cancer Center

La Jolla, California, 92093, United States

Location

Colorado Blood Cancer Institute

Denver, Colorado, 80218, United States

Location

George Washington University

Washington D.C., District of Columbia, 20037, United States

Location

Franciscan St. Francis Health

Indianapolis, Indiana, 46237, United States

Location

June E. Nylen Cancer Center

Sioux City, Iowa, 51101, United States

Location

Norton Cancer Institute

Louisville, Kentucky, 40207, United States

Location

Tulane University/Tulane Cancer Center

New Orleans, Louisiana, 70112, United States

Location

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Allina Health - Virginia Piper Cancer Institute

Minneapolis, Minnesota, 55407, United States

Location

Washington University School of Medicine in St. Louis

St Louis, Missouri, 63110, United States

Location

New Mexico Cancer Care Alliance

Albuquerque, New Mexico, 87131, United States

Location

Northwell Health, Monter Cancer Center

Lake Success, New York, 11042, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45267, United States

Location

Oregon Health & Science University Knight Cancer Institute

Portland, Oregon, 97239, United States

Location

Rhode Island Hospital

Providence, Rhode Island, 02903, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Avera Cancer Institute

Sioux Falls, South Dakota, 57105, United States

Location

Tennessee Oncology/Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

Baylor Research Institute/Baylor Sammons Cancer Center/Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

Methodist Healthcare System of San Antonio

San Antonio, Texas, 78229, United States

Location

Huntsman Cancer Institute

Salt Lake City, Utah, 84112, United States

Location

LDS Hospital

Salt Lake City, Utah, 84143, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

N-desulfated,2-O,3-O-desulfated heparin; Idarubicin; Cytarabine

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Daunorubicin; Anthracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds; Aminoglycosides; Glycosides; Carbohydrates; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides

Results Point of Contact

• Title: Chief Medical Officer
• Organization: Chimerix, Inc.

dmoore@chimerix.com

919-806-1074

Study Officials

• Stephen Marcus, MD

  Cantex Pharmaceuticals Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 12, 2016
• First Posted: August 19, 2016
• Study Start: August 1, 2016
• Primary Completion: June 1, 2019
• Study Completion: June 1, 2019
• Last Updated: September 7, 2023
• Results First Posted: October 29, 2021

Record last verified: 2023-08

Locations

US (23)