Safety Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura (TTP)

Study Overview
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Brief Summary
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The purpose of the study is to find out if administration of danazol with plasma exchange and corticosteroids will reduce the number of plasma exchanges required to control Thrombotic Thrombocytopenic Purpura (TTP).

Detailed Description
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Danazol is a synthetic steroid hormone structurally resembling a group of natural hormones (androgens)found in the body. Danazol has immune modifying activity and is effective in treatment of blood disorders with low platelet counts such as idiopathic thrombocytopenic purpura (however FDA has not yet approved danazol for this disorder). A study of danazol in conjunction with plasma exchange for thrombotic thrombocytopenic purpura showed that danazol decreased the number of plasma exchanges required by approximately 80% and reduced the time needed to control the disease. It's not clear how danazol works in TTP. It is not approved by the FDA for the treatment of TTP.

Official Title
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Phase II Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura

Conditions
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Thrombotic Thrombocytopenic Purpura

Intervention / Treatment
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* Drug: Danazol


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: TTP with platelets less than 10,000 and microangiopathic hemolytic anemia Age greater than 18 and less than 60 LDH > 2x upper limit of normal PT and PTT normal Patients must give signed informed consent Pre-menopausal woman must have negative pregnancy test. TTP not related to underlying cancer, treatment of cancer or transplantation. TTP not associated with drugs. Exclusion Criteria: LFTs AST/ALT > 2x upper limit of normal Hepatitis B and Hepatitis C infection. HIV with active opportunistic infections Acute or chronic Disseminated Intravascular Coagulation (DIC), defined as D-dimer 8ug/ml and fibrinogen<100 mg/dl TTP related to drugs, malignancy and transplantation. Pregnancy Concurrent other investigational drug use during this study. Porphyria.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Danazol, Plex, Steroids<br>Everyone will receive Danazol with plasma exchange and corticosteroids | Drug: Danazol<br>* Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.<br>* Other names: Corticosteroids;|
| No Intervention: Historic Control<br> | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Plasma Exchanges | The total number of plasma exchanges performed after initiation of the first plasma exchange. | up to 60 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to Remission |  | up to 30 days | 
| Length of Stay |  | up to 30 days | 
| Number of Participants With Complete and Continuous Response Rate |  | At 2 Years | 
| Time to Relapse |  | up to 12 years | 
| Number of Relapses |  | up to 12 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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TTP, Thrombotic Thrombocytopenic Purpura