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

Phase 2
Terminated
Conditions
Thrombotic Thrombocytopenic Purpura
Interventions
Registration Number
NCT00953771
Lead Sponsor
Icahn School of Medicine at Mount Sinai
Brief Summary

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

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.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
9
Inclusion Criteria
  • TTP with platelets less than 100,000 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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Danazol, Plex, SteroidsDanazolEveryone will receive Danazol with plasma exchange and corticosteroids
Primary Outcome Measures
NameTimeMethod
Number of Plasma Exchangesup to 30 days

The total number of plasma exchanges performed after initiation of the first plasma exchange.

Secondary Outcome Measures
NameTimeMethod
Time to Remissionup to 30 days
Number of Participants With Relapsesup to 12 years

Number of participants with relapses

Time to Relapseup to 12 years
Number of Relapsesup to 12 years

Number of relapses

Number of Participants With Complete and Continuous Response RateAt 2 Years
Length of Stayup to 30 days

Trial Locations

Locations (1)

Mount Sinai St. Luke's-Roosevelt

🇺🇸

New York, New York, United States

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