A Pilot Study of Dociparstat Sodium (ODSH) in Acute Myeloid Leukemia

NCT02056782 | Completed Phase 1 Results DMC

• 1 other identifier: PGX-ODSH-2013-AML-1
• Study Type: interventional
• Target: 12
• Locations: 0 countries
• Sites: N/A

Brief Summary

This was an open-label pilot study that evaluated the safety and preliminary evidence of a therapeutic effect of dociparstat in conjunction with standard induction and consolidation therapy for acute myeloid leukemia (AML).

Conditions

Acute Myeloid Leukemia

Keywords

Acute Myeloid Leukemia; AML; ODSH; ODSH-AML; CX-01; dociparstat; dociparstat sodium; 2-O, 3-O desulfated heparin

Outcome Measures

Primary Outcomes (1)

• Time (Days) to Transfusion-independent Platelet Recovery (Platelet Counts Values ≥ 20,000/μL and ≥ 50,000/μL Without a Platelet Transfusion)

  A primary endpoint of this study was evidence of an effect of dociparstat on transfusion independent platelet recovery time. The time (days) to transfusion-independent platelet recovery will be defined as the number of days from the first day of chemotherapy until the first of 5 consecutive days with platelet counts values ≥ 20,000/μL and ≥ 50,000/μL without a platelet transfusion.

  Day 1 to Day 35 (35 days)

Secondary Outcomes (1)

• Number of Subjects Who Achieved a Morphologic Complete Remission

  Day 1 to Day 35 (35 days)

Study Arms (1)

Dociparstat

EXPERIMENTAL

The following induction regimen was administered: * Cytarabine (100 mg/m2/day) via continuous intravenous (IV) infusion 24 hours daily for 7 days. * Idarubicin (12 mg/m2/day) IV on Days 1, 2, and 3. * Dociparstat (4 mg/kg) given over 5 minutes IV, immediately after the idarubicin dose on Day 1, followed by a continuous IV infusion (0.25 mg/kg/hr for 24 hours daily) for a total of 7 days.

Drug: Dociparstat sodium

Interventions

Dociparstat sodium DRUG

The following induction regimen was administered: * Cytarabine (100 mg/m2/day) via continuous intravenous (IV) infusion 24 hours daily for 7 days. * Idarubicin (12 mg/m2/day) IV on Days 1, 2, and 3. * Dociparstat (4 mg/kg) given over 5 minutes IV, immediately after the idarubicin dose on Day 1, followed by a continuous IV infusion (0.25 mg/kg/hr for 24 hours daily) for a total of 7 days.

Also known as: dociparstat, ODSH, CX-01, 2-O, 3-O desulfated heparin

Dociparstat

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• All patients had to meet the following criteria to be eligible for this study:
• Had newly diagnosed, previously untreated acute myeloid leukemia (AML). Acute promyelocytic leukemia and acute megakaryoblastic leukemia subtypes were excluded
• Had no prior chemotherapy for AML; however, prior hydroxyurea to control white blood cell count was allowed
• Was aged 18 years or older.
• Had an Eastern Cooperative Oncology Group (ECOG) Performance status of 0-2.
• Had a cardiac ejection fraction ≥ 50% (echocardiography or Multi-Gated Acquisition Scan \[MUGA\]).
• Had adequate hepatic and renal function (aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\], bilirubin and creatinine \< 2.5 x upper normal limit).
• Was able to provide informed consent and signed an approved consent form that conformed to federal and institutional guidelines.

You may not qualify if:

• Patients who met any of the following criteria were not eligible to be enrolled in this study:
• Had acute promyelocytic leukemia.
• Had acute megakaryoblastic leukemia.
• Had central nervous system (CNS) leukemia
• Had the presence of uncontrolled bleeding.
• Had the presence of significant active infection that was uncontrolled, as judged by the Investigator.
• Had a history of severe congestive heart failure or other cardiac disease that contraindicated the use of anthracyclines, including idarubicin.
• Had pre-existing liver disease.
• Had renal insufficiency, which, in the opinion of the Investigator, might have adversely affected the schedule and dose of therapy with cytarabine, as well as the management of tumor lysis syndrome. Patients with creatinine levels ≥2 mg/dL were not eligible.
• Had use of recreational drugs or history of drug addiction, within the prior 6 months.
• Had known history of positive hepatitis B surface antigens or hepatitis C virus (HCV) antibodies.
• Had known history of positive test for human immunodeficiency virus (HIV) antibodies
• Had psychiatric or neurologic conditions that could have compromised patient safety or compliance, or interfered with the ability to give proper informed consent.
• Had history of other active malignant disease within 5 years, other than cured basal cell carcinoma of the skin, cured in situ carcinoma of the cervix, or localized prostate cancer that had received definitive therapy. Such prostate cancer patients who were receiving hormonal therapy were eligible.
• Had the presence of disseminated intravascular coagulation, as confirmed by laboratory studies demonstrating evidence of both increased thrombin generation (decreased fibrinogen, prolonged prothrombin time \[PT\] and partial thromboplastin time \[aPTT\]), as well as increased fibrinolysis (elevated D-dimer level).

Sponsors & Collaborators

• Jazz Pharmaceuticals: lead
• Translational Drug Development: collaborator

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MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

N-desulfated,2-O,3-O-desulfated heparin

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: CBertrand
• Organization: Cantex

cbertrand@cantex.com

954-315-3660

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 22, 2014
• First Posted: February 6, 2014
• Study Start: December 1, 2013
• Primary Completion: February 1, 2015
• Study Completion: June 1, 2015
• Last Updated: February 21, 2023
• Results First Posted: February 21, 2023

Record last verified: 2022-05