Daclizumab to Treat Chronic Immune Thrombocytopenia

NCT00049725 | Completed Phase 2

• 2 other identifiers: 03004603-CC-0046
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief 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). 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.

Conditions

Thrombocytopenia

Keywords

Autoimmune Disease; Monoclonal Antibody Therapy; Anti-IL2 Receptor; Platelets; Monoclonal Antibody; Immune Thrombocytopenia; ITP

Interventions

Daclizumab DRUG

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• National Institutes of Health Clinical Center (CC): lead

Study Sites (1)

Warren G. Magnuson Clinical Center (CC)

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• 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.

  PMID: 8443385 BACKGROUND

• He 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.

  PMID: 8111044 BACKGROUND

• Kiefel 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.

  PMID: 8624385 BACKGROUND

MeSH Terms

Conditions

Thrombocytopenia; Autoimmune Diseases; Purpura, Thrombocytopenic, Idiopathic

Interventions

Daclizumab

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Design

• Study Type: interventional
• Phase: phase 2
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: November 12, 2002
• First Posted: November 13, 2002
• Study Start: November 1, 2002
• Study Completion: December 1, 2004
• Last Updated: March 4, 2008

Record last verified: 2004-12

Locations

US (1)