Initial Treatment of Patients With Immune Thrombocytopenic Purpura (ITP^2)
This study has been terminated.
(The study was closed due to accrual futility there were only a total of 8 subjects enrolled.)
Sponsor:
New England Research Institutes
Collaborator:
Information provided by (Responsible Party):
New England Research Institutes
ClinicalTrials.gov Identifier:
NCT00991939
First received: October 7, 2009
Last updated: March 11, 2013
Last verified: March 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Immune Thrombocytopenic Purpura |
Drug: Dexamethasone USP Micronized Drug: Prednisone |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | Initial Treatment of Patients With Immune Thrombocytopenic Purpura: The ITP^2 Study |
Resource links provided by NLM:
Genetics Home Reference related topics:
thrombotic thrombocytopenic purpura
MedlinePlus related topics:
Steroids
Drug Information available for:
Dexamethasone
Prednisone
Dexamethasone acetate
Dexamethasone sodium phosphate
U.S. FDA Resources
Further study details as provided by New England Research Institutes:
Primary Outcome Measures:
- 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. [ Time Frame: From 60 days through 365 days after study entry. ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- 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 [ Time Frame: From 60 days through 365 days after study entry ] [ Designated as safety issue: No ]
- 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 [ Time Frame: 365 days after study entry ] [ Designated as safety issue: No ]
- 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 [ Time Frame: From 180 days through 365 days after study entry ] [ Designated as safety issue: No ]
- 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 [ Time Frame: From 180 days through 365 days after study entry ] [ Designated as safety issue: No ]
- The percentage of patients receiving acute therapeutic intervention during the first 60 days after study entry [ Time Frame: Through 60 days after study entry ] [ Designated as safety issue: No ]
- The percentage of patients receiving acute therapeutic intervention beyond the first 60 days after study entry [ Time Frame: From 60 days through 365 days after study entry ] [ Designated as safety issue: No ]
- The percentage of platelet counts ≥ 50,000/μl after day 60 (If a subject receives an acute therapeutic intervention, the next protocol-specified platelet count will be excluded from this analysis, as it may be influenced by the intervention.) [ Time Frame: From 60 days through 365 days after study entry ] [ Designated as safety issue: No ]
- The percentage of platelet counts ≥ 150,000/μl after day 60 (If a subject receives an acute therapeutic intervention, the next protocol-specified platelet count will be excluded from this analysis, as it may be influenced by the intervention.) [ Time Frame: From 60 days through 365 days after study entry ] [ Designated as safety issue: No ]
- The percentage of patients undergoing splenectomy [ Time Frame: Through 365 days after study entry ] [ Designated as safety issue: No ]
- Change in the quality of life from randomization to weeks 4, 8 and end of study, determined using the SF-36 health survey [ Time Frame: Weeks 4, 8, and 52 after study entry ] [ Designated as safety issue: No ]
- The incidence and severity of bleeding as defined by a customized bleeding score [ Time Frame: Through 365 days after study entry ] [ Designated as safety issue: No ]
- The percentage of patients not completing study therapy [ Time Frame: 49 days after study entry ] [ Designated as safety issue: No ]
- The percentage of patients with severe adverse events attributable to steroid therapy [ Time Frame: Through 1 year after study entry ] [ Designated as safety issue: Yes ]
| Enrollment: | 8 |
| Study Start Date: | January 2010 |
| Estimated Study Completion Date: | February 2014 |
| Estimated Primary Completion Date: | February 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: High dose pulse dexamethasone |
Drug: Dexamethasone USP Micronized
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.
|
| Active Comparator: Standard prednisone therapy |
Drug: Prednisone
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.
|
Detailed Description:
ITP is a common disorder associated with significant morbidity. For more than 40 years it has been recognized that this disorder was responsive to corticosteroid therapy. As corticosteroids are easily obtainable and inexpensive, they have become the standard first-line therapy for adult patients with newly-diagnosed ITP. Generally, patients are treated with prednisone at a dose of approximately 1 mg/kg, or 60 mg/day, and once a response is obtained the daily dosage is gradually tapered. While approximately 70% of patients treated in this manner respond initially, most will relapse as the corticosteroid dose is lowered; ultimately only 15-20% of patients achieve a complete or partial remission of their ITP at an "acceptable" dose of prednisone. Recently, several studies have suggested that the use of high dose corticosteroids, specifically pulse dexamethasone, may be a more efficacious initial therapy for ITP, capable of causing a higher initial response rate and a significantly longer duration of remission despite a shorter course of initial therapy.
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 standard oral corticosteroids. This may reflect the ability of high dose corticosteroids to eradicate a sensitive pathogenic lymphoid clone that may be transiently susceptible to aggressive immunosuppressive therapy early in the course of disease.
Eligibility| Ages Eligible for Study: | 15 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00991939
Locations
| United States, Louisiana | |
| Tulane University | |
| New Orleans, Louisiana, United States, 70112 | |
| United States, Maryland | |
| Johns Hopkins Hospital | |
| Baltimore, Maryland, United States, 21287 | |
| University of Maryland | |
| Baltimore, Maryland, United States, 21201 | |
| United States, Massachusetts | |
| Children's Hospital Boston | |
| Boston, Massachusetts, United States, 02115 | |
| Brigham & Women's Hospital | |
| Boston, Massachusetts, United States, 02115 | |
| Massachusetts General Hospital | |
| Boston, Massachusetts, United States, 02114 | |
| United States, New York | |
| Weill Medical College, Cornell University | |
| New York, New York, United States, 10021 | |
| United States, North Carolina | |
| University of North Carolina Hospitals | |
| Chapel Hill, North Carolina, United States, 27514 | |
| Duke University | |
| Durham, North Carolina, United States, 27710 | |
| United States, Ohio | |
| Case Western Reserve University | |
| Cleveland, Ohio, United States, 44106 | |
| Cleveland Clinic Foundation | |
| Cleveland, Ohio, United States, 44195 | |
| United States, Oklahoma | |
| The University of Oklahoma Health Sciences Center | |
| Oklahoma City, Oklahoma, United States, 73104 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| University of Pennsylvania | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| University of Pittsburgh Presbyterian and Shadyside Hospital | |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| United States, Washington | |
| University of Washington Medical Center | |
| Seattle, Washington, United States, 98195 | |
| United States, Wisconsin | |
| Gundersen Clinic | |
| La Crosse, Wisconsin, United States, 54601 | |
| University of Wisconsin | |
| Madison, Wisconsin, United States, 53792 | |
Sponsors and Collaborators
New England Research Institutes
Investigators
| Principal Investigator: | Susan F Assmann, PhD | New England Research Institutes |
| 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 | Children's Hospital Boston |
| 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 |
More Information
No publications provided
| Responsible Party: | New England Research Institutes |
| ClinicalTrials.gov Identifier: | NCT00991939 History of Changes |
| Other Study ID Numbers: | 675, U01HL072268, HL072268, HL072033, HL072291, HL072196, HL072289, HL072248, HL072191, HL072299, HL072305, HL072274, HL072028, HL072359, HL072072, HL072355, HL072283, HL072346, HL072331, HL072290 |
| Study First Received: | October 7, 2009 |
| Last Updated: | March 11, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by New England Research Institutes:
|
Immune thrombocytopenic purpura ITP Dexamethasone Prednisone |
Additional relevant MeSH terms:
|
Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Blood Coagulation Disorders Hematologic Diseases Hemorrhage Pathologic Processes Skin Manifestations Signs and Symptoms Thrombotic Microangiopathies Thrombocytopenia Blood Platelet Disorders Immune System Diseases Hemorrhagic Disorders Autoimmune Diseases |
Dexamethasone acetate Dexamethasone Prednisone Dexamethasone 21-phosphate BB 1101 Anti-Inflammatory Agents Therapeutic Uses Pharmacologic Actions Antiemetics Autonomic Agents Peripheral Nervous System Agents Physiological Effects of Drugs Central Nervous System Agents Gastrointestinal Agents Glucocorticoids |
ClinicalTrials.gov processed this record on June 17, 2013