Treatment of Autoimmune Thrombocytopenia (AITP)

NCT00001630 | Completed Phase 1

• 2 other identifiers: 97015497-H-0154
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 1

Brief Summary

Platelets are particles found along with red and white blood cells in the blood that play a role in the process of blood clotting. Disorders affecting the platelets can lower the amount of platelets in the blood and put patients at risk of bleeding. The condition of low platelets is referred to as thrombocytopenia. Thrombocytopenia can be associated with a variety of diseases including cancer, leukemia, tuberculosis, or as a result of an autoimmune reaction. Autoimmune reactions are disorders in which the normal immune system begins attacking itself. Autoimmune thrombocytopenia (AITP) is a disorder of low blood platelet counts in which platelets are destroyed by antibodies produced by the immune system. Unfortunately, many patients with AITP do not respond to standard treatments for thrombocytopenia. Cyclophosphamide is a drug that works to suppress the activity of the immune system. Researchers believe that combining this drug with transplanted rescued blood stem cells may provide effective treatment for AITP. The purpose of this study is to explore the affordability and safety of this therapy for the treatment of AITP. The effectiveness of the therapy will be measured by the number of patients whose platelet levels rise greater than 100,000/m3. If this treatment approach appears affordable, this study will form the basis for a larger study to compare alternate treatment approaches.

Conditions

Autoimmune Disease; Autoimmune Hemolytic Anemia; Thrombocytopenia

Keywords

Immunosuppression; Thrombocytopenia; Autoimmune Disease; Evan's Syndrome; Autoimmune Hemolytic Anemia; Episodic Bleeding

Interventions

Isolex 300i DEVICE

Eligibility Criteria

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

You may qualify if:

• Male or female, ages 18-65 years old.
• Refractory severe chronic autoimmune thrombocytopenia, with or without autoimmune hemolytic anemia (Evan's syndrome), with all the following:
• Platelet count frequently below 20,000/mm(3) despite active
• treatment for a period of greater than 6 months.
• Normal or increased megakaryocytes on bone marrow
• aspirate/bx.
• No plausible alternative etiology such as drug-mediated
• thrombocytopenia, marrow failure syndrome or thrombocytopenia
• related to viral or bacterial infection.
• Failure of treatment with:
• i. conventional-dose steroids (e.g., prednisone or dosage of 40
• mg/day or equivalent, followed by dosage taper) for at least 3
• months.
• ii. intravenous immunoglobulin.
• iii. splenectomy.

You may not qualify if:

• ECOG performance status greater than 1.
• Cardiopulmonary disease including:
• History of coronary artery disease, angina pectoris or congestive heart failure.
• LV ejection fraction less than 40 percent by 2D echocardiogram.
• Renal disease, serum creatinine greater than 2.5 mg/dL or creatinine clearance less than 30 mL/min.
• Significant hepatic dysfunction, bilirubin greater than 2 mg/dL or transaminases greater than 2 times UNL.
• Uncorrected coagulopathy.
• Bone marrow aplasia (cellularity less than 10 percent), single or multilineage hematopoietic failure, myelodysplastic syndrome, or extensive marrow fibrosis.
• History or active diagnosis of malignancy (except treated non-melanoma skin cancer or cevical carcinoma in situ).
• HIV positive.
• Pregnancy or lactation, unwillingness to practice adequate birth control in the peritransplant period.
• Psychiatric illness or mental incapacity to understand and give informed consent.
• Other medical illness or condition which, in the opinion of the Investigators, may contraindicate participation in this study due to patients' risk or compromise of study integrity.

Sponsors & Collaborators

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

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Semple JW, Freedman J. Abnormal cellular immune mechanisms associated with autoimmune thrombocytopenia. Transfus Med Rev. 1995 Oct;9(4):327-38. doi: 10.1016/s0887-7963(05)80080-x. No abstract available.

  PMID: 8541715 BACKGROUND

• Karpatkin S. Autoimmune (idiopathic) thrombocytopenic purpura. Lancet. 1997 May 24;349(9064):1531-6. doi: 10.1016/S0140-6736(96)12118-8. No abstract available.

  PMID: 9167472 BACKGROUND

• George JN, el-Harake MA, Raskob GE. Chronic idiopathic thrombocytopenic purpura. N Engl J Med. 1994 Nov 3;331(18):1207-11. doi: 10.1056/NEJM199411033311807. No abstract available.

  PMID: 7935660 BACKGROUND

MeSH Terms

Conditions

Autoimmune Diseases; Anemia, Hemolytic, Autoimmune; Thrombocytopenia; Evans Syndrome

Condition Hierarchy (Ancestors)

Immune System Diseases; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Cytopenia

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: November 3, 1999
• First Posted: November 4, 1999
• Study Start: July 21, 1997
• Study Completion: June 11, 2009
• Last Updated: July 2, 2017

Record last verified: 2011-03-21

Locations

US (1)