Initial Treatment of Patients With Immune Thrombocytopenic Purpura

NCT00991939 | Terminated Phase 3 Results DMC

• 20 other identifiers: 675U01HL072268HL072268HL072033HL072291HL072196HL072289HL072248HL072191HL072299HL072305HL072274HL072028HL072359HL072072HL072355HL072283HL072346HL072331HL072290
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 18

Brief Summary

This study will compare treatment with 3 courses of high-dose dexamethasone versus treatment with prednisone, for patients recently diagnosed with immune thrombocytopenic purpura (ITP). The primary hypothesis is that patients treated with high-dose dexamethasone will obtain a more durable remission than patients treated with prednisone.

Conditions

Immune Thrombocytopenic Purpura

Keywords

Immune thrombocytopenic purpura; ITP; Dexamethasone; Prednisone

Outcome Measures

Primary Outcomes (1)

• The Percentage of Patients in Each Treatment Arm Who Remain Free of All ITP Therapy With a Platelet Count ≥ 50,000/μl From 60 Days Through 365 Days After Study Entry.

  From 60 days through 365 days after study entry.

Secondary Outcomes (13)

• The Percentage of Patients Who Remain Free of All ITP Therapy With a Platelet Count ≥ 150,000/μl From 60 Days Through 365 Days After Study Entry

  From 60 days through 365 days after study entry

• The Percentage of Patients With Platelets ≥ 50,000/μl at 365 Days Who Are Off All Treatment, Have Received ≤ 2 Acute Therapeutic Interventions for Thrombocytopenia, and Whose Last Acute Therapeutic Intervention Occurred at Least 90 Days Before Day 365

  365 days after study entry

• The Percentage of Patients Who Remain Free of All ITP Therapy With a Platelet Count of ≥ 150,000 From 180 Through 365 Days After Study Entry

  From 180 days through 365 days after study entry

• The Percentage of Patients Who Remain Free of All ITP Therapy With a Platelet Count of ≥ 50,000 From 180 Through 365 Days After Study Entry

  From 180 days through 365 days after study entry

• The Percentage of Patients Receiving Acute Therapeutic Intervention During the First 60 Days After Study Entry

  Through 60 days after study entry

Study Arms (2)

High dose pulse dexamethasone

EXPERIMENTAL

Drug: Dexamethasone USP Micronized

Standard prednisone therapy

ACTIVE COMPARATOR

Drug: Prednisone

Interventions

Dexamethasone USP Micronized DRUG

The dose for dexamethasone is 30 mg/day for patients \< 60 kg and 40 mg/day for patients \> 60 kg. The patient will be dosed on days 1-4, 15-18 and 29-32. On the remaining days during the treatment phase of the study, the patient will receive placebo capsules.

High dose pulse dexamethasone

Prednisone DRUG

Prednisone will be administered to study patients at a dose of 60 mg/day for patients less than 60 kg and 80 mg/day for patients \> 60 kg for 21 days. The following schedule for tapering of prednisone will be used: after three weeks of treatment at either 60 mg/day (for patients \< 60 kg) or 80 mg/day (for patients ≥ 60 kg), the dose will be reduced to 40 mg/day for 1 week, then 20 mg/day for 1 week, then 10 mg/day for 1 week, then 5 mg/day for 1 week and then stopped. Placebo capsules will be added as necessary during the treatment phase of the study, to maintain blinding.

Standard prednisone therapy

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Must meet criteria for a diagnosis of ITP as specified by ASH guidelines
• Must be within 30 days after diagnosis of ITP at the time of randomization (diagnosis of ITP starts with first platelet count ≤ 100,000/μl)
• Platelet count ≤ 30,000/μl at the time ITP is diagnosed, and/or at some time between the diagnosis of ITP and study entry
• Platelet count ≤ 150,000/μl at the time of randomization
• Age ≥ 15 years
• If bone marrow examination is available, it must be compatible with ITP
• Subjects, or their legal guardians, must have the ability to provide informed consent

You may not qualify if:

• Rituximab therapy or splenectomy for ITP or for any other cause within the previous 8 weeks.
• Known HIV infection
• Known HCV infection
• Known systemic lupus erythematosus
• Pregnancy or breastfeeding
• Insulin-requiring diabetes mellitus
• Previous exposure to prednisone for ITP at a dose ≥ 1.5 mg/kg prednisone/day for ≥ 1 week prior to study entry
• Ongoing use of treatments that are known to inhibit platelet function, e.g. aspirin
• Anything that in the opinion of the investigator is likely to interfere with participation in the study
• Persons previously randomized in the ITP\^2 study
• Persons currently enrolled in other interventional clinical trials
• Exposure to thrombopoietic agent prior to study entry
• Previous exposure to dexamethasone for the treatment of ITP at a dose of 30 mg/day or greater for subjects \< 60 kg or 40 mg/day or greater for subjects \>= 60 kg for at least four days

Sponsors & Collaborators

• Carelon Research: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (18)

Tulane University

New Orleans, Louisiana, 70112, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Brigham & Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

Weill Medical College, Cornell University

New York, New York, 10021, United States

Location

University of North Carolina Hospitals

Chapel Hill, North Carolina, 27514, United States

Location

Duke University

Durham, North Carolina, 27710, United States

Location

Case Western Reserve University

Cleveland, Ohio, 44106, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

The University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburgh Presbyterian and Shadyside Hospital

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Washington Medical Center

Seattle, Washington, 98195, United States

Location

Gundersen Clinic

La Crosse, Wisconsin, 54601, United States

Location

University of Wisconsin

Madison, Wisconsin, 53792, United States

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Prednisone

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Results Point of Contact

• Title: Susan F. Assmann, PhD
• Organization: New England Research Institutes, Inc.

sassmann@neriscience.com

617-923-7747

Study Officials

• Susan F Assmann, PhD

  Carelon Research

  PRINCIPAL INVESTIGATOR

• James Bussel, MD

  Weill Medical College, Cornell University

  PRINCIPAL INVESTIGATOR

• Alvin Schmaier, MD

  Case Western Reserve University

  PRINCIPAL INVESTIGATOR

• Jodi Segal, MD

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

• Terry Gernsheimer, MD

  University of Washington

  PRINCIPAL INVESTIGATOR

• Eliot Williams, MD

  University of Wisconsin, Madison

  PRINCIPAL INVESTIGATOR

• Ellis Neufeld, MD

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

• Judith Lin, MD

  Brigham and Women's Hospital

  PRINCIPAL INVESTIGATOR

• Thomas Ortel, MD

  Duke University

  PRINCIPAL INVESTIGATOR

• David Kuter, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

• Cindy Leissinger, MD

  Tulane University

  PRINCIPAL INVESTIGATOR

• Ann Zimrin, MD

  University of Maryland

  PRINCIPAL INVESTIGATOR

• Nigel Key, MD

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

• James George, MD

  The University of Oklahoma

  PRINCIPAL INVESTIGATOR

• Michele Lambert, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

• Joseph Kiss, MD

  University of Pittsburgh

  PRINCIPAL INVESTIGATOR

• Bruce Sachais, MD, PhD

  University of Pennsylvania

  PRINCIPAL INVESTIGATOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 7, 2009
• First Posted: October 8, 2009
• Study Start: January 1, 2010
• Primary Completion: March 1, 2013
• Study Completion: March 1, 2013
• Last Updated: February 14, 2014
• Results First Posted: February 14, 2014

Record last verified: 2014-01

Locations

US (18)