Immune Thrombocytopenic PurpuraPhase 3ITP^2NCT00991939
Initial Treatment of Patients With Immune Thrombocytopenic Purpura
A Phase 3 study testing Dexamethasone USP Micronized and Prednisone in people with Immune Thrombocytopenic Purpura, run by Carelon Research at 18 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Immune Thrombocytopenic Purpura
- What is being tested?
- Dexamethasone USP Micronized and Prednisone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 18 sitesUnited States
- Study dates
- Jan 2010to Mar 2013
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 13Show fewer
- 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
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
United States18 sites
University of Maryland
Baltimore, Maryland, 21201
Johns Hopkins Hospital
Baltimore, Maryland, 21287
Massachusetts General Hospital
Boston, Massachusetts, 02114
Brigham & Women's Hospital
Boston, Massachusetts, 02115
Children's Hospital Boston
Boston, Massachusetts, 02115
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27514
Case Western Reserve University
Cleveland, Ohio, 44106
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Duke University
Durham, North Carolina, 27710
Gundersen Clinic
La Crosse, Wisconsin, 54601
University of Wisconsin
Madison, Wisconsin, 53792
Tulane University
New Orleans, Louisiana, 70112
Weill Medical College, Cornell University
New York, New York, 10021
The University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of Pittsburgh Presbyterian and Shadyside Hospital
Pittsburgh, Pennsylvania, 15213
University of Washington Medical Center
Seattle, Washington, 98195
About this study
Official title: Initial Treatment of Patients With Immune Thrombocytopenic Purpura: The ITP^2 Study
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.
Read the 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.
- Conditions
- Immune Thrombocytopenic Purpura
- Being tested
- Dexamethasone USP Micronized, Prednisone
- Keywords
- Immune thrombocytopenic purpura, ITP, Dexamethasone, Prednisone
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dexamethasone USP MicronizedDrug
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.
PrednisoneDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| High dose pulse dexamethasone | Experimental | Not described Receives: Drug: Dexamethasone USP Micronized |
| Standard prednisone therapy | Active comparator | Not described Receives: Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: Through 60 days after study entry
The Percentage of Patients Receiving Acute Therapeutic Intervention Beyond the First 60 Days After Study Entry
Measured: From 60 days through 365 days after study entry
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.)
Measured: From 60 days through 365 days after study entry
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.)
Measured: From 60 days through 365 days after study entry
The Percentage of Patients Undergoing Splenectomy
Measured: Through 365 days after study entry
Change in the Quality of Life From Randomization to Weeks 4, 8 and End of Study, Determined Using the SF-36 Health Survey
Measured: Weeks 4, 8, and 52 after study entry
The Incidence and Severity of Bleeding as Defined by a Customized Bleeding Score
Measured: Through 365 days after study entry
The Percentage of Patients Not Completing Study Therapy
Measured: 49 days after study entry
The Percentage of Patients With Severe Adverse Events Attributable to Steroid Therapy
Measured: Through 1 year after study entry
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2009 (estimated)
- Study startJan 2010
- Primary completionMar 2013
- Study completionMar 2013
Why it stopped: The study was closed due to accrual futility there were only a total of 8 subjects enrolled.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Immune Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Carelon Research
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Susan F Assmann, PhD, Principal investigator, Carelon ResearchJames Bussel, MD, Principal investigator, Weill Medical College, Cornell UniversityAlvin Schmaier, MD, Principal investigator, Case Western Reserve UniversityJodi Segal, MD, Principal investigator, Johns Hopkins UniversityTerry Gernsheimer, MD, Principal investigator, University of WashingtonEliot Williams, MD, Principal investigator, University of Wisconsin, MadisonEllis Neufeld, MD, Principal investigator, Boston Children's HospitalJudith Lin, MD, Principal investigator, Brigham and Women's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00991939
- Sponsor's protocol ID
- 675
- Other IDs
- U01HL072268, HL072268, HL072033, HL072291, HL072196, HL072289, HL072248, HL072191
- Acronym
- ITP^2
- Status last verified
- Jan 2014
- First submitted
- 7 Oct 2009
- Last updated
- 14 Feb 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Initial Treatment of Patients With Immune Thrombocytopenic Purpura: The ITP^2 Study
- ClinicalTrials.gov ID
- NCT00991939
- Acronym / sponsor ID
- ITP^2 / 675
- Conditions
- Immune Thrombocytopenic Purpura
- Keywords
- Immune thrombocytopenic purpura, ITP, Dexamethasone, Prednisone
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was closed due to accrual futility there were only a total of 8 subjects enrolled.
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Carelon ResearchUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Jan 2010
- Primary completion
- Mar 2013 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 8 Oct 2009 (estimated)
- Results
- Posted 14 Feb 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| High dose pulse dexamethasone | Experimental | Drug: Dexamethasone USP Micronized |
| Standard prednisone therapy | Active comparator | Drug: Prednisone |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dexamethasone USP MicronizedThe 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. | Drug | High dose pulse dexamethasone |
| PrednisonePrednisone 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. | Drug | Standard prednisone therapy |
Primary outcome
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.
13 secondary outcomes
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
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
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
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
The Percentage of Patients Receiving Acute Therapeutic Intervention During the First 60 Days After Study Entry
Time frame: Through 60 days after study entry
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
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
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
The Percentage of Patients Undergoing Splenectomy
Time frame: Through 365 days after study entry
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
The Incidence and Severity of Bleeding as Defined by a Customized Bleeding Score
Time frame: Through 365 days after study entry
The Percentage of Patients Not Completing Study Therapy
Time frame: 49 days after study entry
The Percentage of Patients With Severe Adverse Events Attributable to Steroid Therapy
Time frame: Through 1 year after study entry
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Feb 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Susan F Assmann, PhD principal investigator
Carelon Research
James Bussel, MD principal investigator
Weill Medical College, Cornell University
Alvin Schmaier, MD principal investigator
Case Western Reserve University
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dexamethasone USP Micronized and Prednisone in people with Immune Thrombocytopenic Purpura, run by Carelon Research at 18 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Feb 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Feb 2014.