ThrombocytopeniaPhase 2NCT00049725
Daclizumab to Treat Chronic Immune Thrombocytopenia
A Phase 2 study testing Daclizumab in people with Thrombocytopenia, run by National Institutes of Health Clinical Center (CC) at 1 site in United States.
Trial summary
This study will evaluate the effectiveness of the drug daclizumab for treating patients with chronic immune thrombocytopenia (ITP), a disease in which the immune system destroys platelets (blood cells involved in the clotting process). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Thrombocytopenia
- What is being tested?
- Daclizumab
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2002to Dec 2004
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female greater than or equal to 18 years old
- Immune thrombocytopenia, and all of the following:
- at least three months since initial diagnosis
- lack of sustained response to initial treatment with prednisone, characterized by failure to maintain a platelet count of at least 30,000/ul for at least six weeks using prednisone at a dose of at least 10 mg per day
- baseline platelet count (as determined by an average of platelet count values over two months immediately prior to study entry) of less than 30,000/ul
- Note: In patients receiving IVIG or anti-D, platelet values immediately prior to infusion of the drug (i.e., at platelet nadir) will be considered in the determination of the baseline platelet value.
- Splenectomy or prior use of second-line immunomodulatory treatments (such as, but not limited to, CSA, danazol, azathioprine or cyclophosphamide) will not be considered a requirement for inclusion.
Who cannot join
- ECOG performance status greater than 1
- Concurrent symptomatic autoimmune hemolysis (Evans syndrome) characterized by hemoglobin less than 10 gm/dl or requirement for more than two units red cells within three months of enrollment, due to hemolysis
- Concurrent autoimmune disorders requiring treatment for involvement of organ systems other than cytopenias
- Initiation of any new immunomodulator agent, or increase in dose or frequency of any existing immunomodulator agent (such as, but not limited to, CSA, danazol, azathioprine or cyclophosphamide; IVIG and anti-D excepted) within two months of study entry
- Autologous transplantation for immune thrombocytopenia within one year of study entry
- Concurrent bleeding diathesis
Show all 12Show fewer
- Echinacea use within three months of study entry
- Pregnancy or lactation
- Chronic or current clinically significant infection, including HIV positivity and acute or persistent hepatitis B and C virus infection (characterized by elevated transaminases and positive hepatitis B surface antigen [HBsAg], or anti-hepatitis C virus [anti-HCV] antibody)
- History of active M. Tuberculosis infection
- Diagnosis of malignancy (with the exception of non-melanoma skin cancer and other malignancies which by virtue of surgical resection and no recurrence for at least five years prior to enrollment are considered to be cured)
- Inadequate mental capacity to give informed consent
Trial locations and contacts
1 site in 1 country.
United States1 site
Warren G. Magnuson Clinical Center (CC)
Bethesda, Maryland, 20892
About this study
Official title: A Study of Daclizumab in Chronic Immune Thrombocytopenia (ITP)
This study will evaluate the effectiveness of the drug daclizumab for treating patients with chronic immune thrombocytopenia (ITP), a disease in which the immune system destroys platelets (blood cells involved in the clotting process). Patients with ITP have abnormal bruising and bleeding; severe disease can be life-threatening. For many patients, standard drug treatments are not effective, and many of the drugs used may have significant side effects with long-term use. Daclizumab is a genetically engineered antibody that suppresses the immune system and has been used primarily to prevent rejection in patients who have had organ transplants. Daclizumab has fewer side effects than other immune suppressant drugs.
Patients with ITP 18 years of age or older who have platelet counts less than 30,000/microliter and have not responded to prednisone treatment may be eligible for this study. Candidates will be screened with a medical history, physical examination, and blood tests.
Read more
Participants will have a 15-minute infusion of daclizumab every 2 weeks for five doses. They will be seen by a physician at least once every 2 weeks while receiving the drug and then at weeks 12, 20, and 32 of the study. Blood will be drawn at the 4- and 8-week visits during treatment for diagnostic tests, and at each follow-up visit after treatment to assess the response to therapy.
Patients who respond well to treatment will have their pre-study immunosuppressive medicines tapered gradually one at a time starting with the 1-month follow-up visit. If their platelet count falls to pre-treatment levels at any time during the tapering, the dose reduction will stop and pre-study medications will be re-started, if necessary.
Read the detailed description
Immune thrombocytopenia (ITP) is an acquired blood disease in which the individual's immune system destroys platelets, the blood cells responsible for clotting. A number of standard treatments exist to decrease the destruction of platelets, including drugs such as the steroid hormone prednisone, or removal of the spleen. Over a third of adult patients will not maintain adequate platelet counts with these treatments. Alternative treatments may be indicated due to bleeding symptoms or baseline platelet counts less than 20,000/ul, a level at which spontaneous serious bleeding can occur. Therapy for chronic ITP is generally effective in less than 30-50% of patients, however, and most of these agents have significant toxicities with long-term use, are expensive, or their administration interferes with daily activities.
Daclizumab is a humanized anti-interleukin-2 receptor monoclonal antibody that works by targeting and impairing activated T lymphocytes, a subset of white blood cells that has been thought to be involved in the development and maintenance of ITP. Daclizumab is a well-tolerated and time-limited therapy, and is easily administered on an outpatient basis. The purpose of this study is to test the efficacy of daclizumab as either a sole agent in the treatment of chronic, symptomatic ITP, or as a treatment that might allow a decrease or discontinuation of medications such as prednisone.
- Conditions
- Thrombocytopenia
- Being tested
- Daclizumab
- Keywords
- Autoimmune Disease, Monoclonal Antibody Therapy, Anti-IL2 Receptor, Platelets, Monoclonal Antibody, Immune Thrombocytopenia, ITP
- Medical subject headings
- Thrombocytopenia, Autoimmune Diseases, Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DaclizumabDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2002 (estimated)
- Study startNov 2002
- Study completionDec 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for ThrombocytopeniaStudies 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 2
- Primary purpose
- Treatment
- Enrolment
- 24 participants
Sponsor and investigators
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00049725
- Sponsor's protocol ID
- 030046
- Other IDs
- 03-CC-0046
- Status last verified
- Dec 2004
- First submitted
- 12 Nov 2002
- Last updated
- 4 Mar 2008
- Results posted
- No
Publications
Berchtold P, Wenger M. Autoantibodies against platelet glycoproteins in autoimmune thrombocytopenic purpura: their clinical significance and response to treatment. Blood. 1993 Mar 1;81(5):1246-50.
PubMed 8443385He R, Reid DM, Jones CE, Shulman NR. Spectrum of Ig classes, specificities, and titers of serum antiglycoproteins in chronic idiopathic thrombocytopenic purpura. Blood. 1994 Feb 15;83(4):1024-32.
PubMed 8111044Kiefel V, Freitag E, Kroll H, Santoso S, Mueller-Eckhardt C. Platelet autoantibodies (IgG, IgM, IgA) against glycoproteins IIb/IIIa and Ib/IX in patients with thrombocytopenia. Ann Hematol. 1996 Apr;72(4):280-5. doi: 10.1007/s002770050173.
PubMed 8624385
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Daclizumab in Chronic Immune Thrombocytopenia (ITP)
- ClinicalTrials.gov ID
- NCT00049725
- Acronym / sponsor ID
- 030046
- Conditions
- Thrombocytopenia
- Keywords
- Autoimmune Disease, Monoclonal Antibody Therapy, Anti-IL2 Receptor, Platelets, Monoclonal Antibody, Immune Thrombocytopenia, ITP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants
- Lead sponsor
- National Institutes of Health Clinical Center (CC)US National Institutes of Health
- Study start
- Nov 2002
- Study completion
- Dec 2004
- First posted
- 13 Nov 2002 (estimated)
- Results
- Not posted in the source record
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 evaluate the effectiveness of the drug daclizumab for treating patients with chronic immune thrombocytopenia (ITP), a disease in which the immune system destroys platelets (blood cells involved in the clotting process). Patients with ITP have abnormal bruising and bleeding; severe disease can be life-threatening. For many patients, standard drug treatments are not effective, and many of the drugs used may have significant side effects with long-term use. Daclizumab is a genetically engineered antibody that suppresses the immune system and has been used primarily to prevent rejection in patients who have had organ transplants. Daclizumab has fewer side effects than other immune suppressant drugs.
Patients with ITP 18 years of age or older who have platelet counts less than 30,000/microliter and have not responded to prednisone treatment may be eligible for this study. Candidates will be screened with a medical history, physical examination, and blood tests.
Participants will have a 15-minute infusion of daclizumab every 2 weeks for five doses. They will be seen by a physician at least once every 2 weeks while receiving the drug and then at weeks 12, 20, and 32 of the study. Blood will be drawn at the 4- and 8-week visits during treatment for diagnostic tests, and at each follow-up visit after treatment to assess the response to therapy.
Patients who respond well to treatment will have their pre-study immunosuppressive medicines tapered gradually one at a time starting with the 1-month follow-up visit. If their platelet count falls to pre-treatment levels at any time during the tapering, the dose reduction will stop and pre-study medications will be re-started, if necessary.
Detailed description
Immune thrombocytopenia (ITP) is an acquired blood disease in which the individual's immune system destroys platelets, the blood cells responsible for clotting. A number of standard treatments exist to decrease the destruction of platelets, including drugs such as the steroid hormone prednisone, or removal of the spleen. Over a third of adult patients will not maintain adequate platelet counts with these treatments. Alternative treatments may be indicated due to bleeding symptoms or baseline platelet counts less than 20,000/ul, a level at which spontaneous serious bleeding can occur. Therapy for chronic ITP is generally effective in less than 30-50% of patients, however, and most of these agents have significant toxicities with long-term use, are expensive, or their administration interferes with daily activities.
Daclizumab is a humanized anti-interleukin-2 receptor monoclonal antibody that works by targeting and impairing activated T lymphocytes, a subset of white blood cells that has been thought to be involved in the development and maintenance of ITP. Daclizumab is a well-tolerated and time-limited therapy, and is easily administered on an outpatient basis. The purpose of this study is to test the efficacy of daclizumab as either a sole agent in the treatment of chronic, symptomatic ITP, or as a treatment that might allow a decrease or discontinuation of medications such as prednisone.
Study design
- Primary purpose
- Treatment
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Male or female greater than or equal to 18 years old
- at least three months since initial diagnosis
- lack of sustained response to initial treatment with prednisone, characterized by failure to maintain a platelet count of at least 30,000/ul for at least six weeks using prednisone at a dose of at least 10 mg per day
- baseline platelet count (as determined by an average of platelet count values over two months immediately prior to study entry) of less than 30,000/ul
- Note: In patients receiving IVIG or anti-D, platelet values immediately prior to infusion of the drug (i.e., at platelet nadir) will be considered in the determination of the baseline platelet value.
Key exclusion criteria (12 listed)
- ECOG performance status greater than 1
- Concurrent symptomatic autoimmune hemolysis (Evans syndrome) characterized by hemoglobin less than 10 gm/dl or requirement for more than two units red cells within three months of enrollment, due to hemolysis
- Concurrent autoimmune disorders requiring treatment for involvement of organ systems other than cytopenias
- Initiation of any new immunomodulator agent, or increase in dose or frequency of any existing immunomodulator agent (such as, but not limited to, CSA, danazol, azathioprine or cyclophosphamide; IVIG and anti-D excepted) within two months of study entry
- Autologous transplantation for immune thrombocytopenia within one year of study entry
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 |
|---|---|---|
| Daclizumab | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2008.
- Background: PubMed 8443385Berchtold P, Wenger M. Autoantibodies against platelet glycoproteins in autoimmune thrombocytopenic purpura: their clinical significance and response to treatment. Blood. 1993 Mar 1;81(5):1246-50.
- Background: PubMed 8111044He R, Reid DM, Jones CE, Shulman NR. Spectrum of Ig classes, specificities, and titers of serum antiglycoproteins in chronic idiopathic thrombocytopenic purpura. Blood. 1994 Feb 15;83(4):1024-32.
- Background: PubMed 8624385Kiefel V, Freitag E, Kroll H, Santoso S, Mueller-Eckhardt C. Platelet autoantibodies (IgG, IgM, IgA) against glycoproteins IIb/IIIa and Ib/IX in patients with thrombocytopenia. Ann Hematol. 1996 Apr;72(4):280-5. doi: 10.1007/s002770050173.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Daclizumab in people with Thrombocytopenia, run by National Institutes of Health Clinical Center (CC) at 1 site 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 12 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: finished, as of the sponsor's last update on 4 Mar 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00049725, which also lists 3 linked publications.
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 4 Mar 2008.