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

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

Sponsors

Lead sponsor: Icahn School of Medicine at Mount Sinai

Collaborator: Beth Israel Medical Center

Source 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.

Overall Status Terminated
Start Date October 2008
Completion Date October 14, 2015
Primary Completion Date October 14, 2015
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
To assess the total number of plasma exchanges performed within 30 days of initiation of the first plasma exchange. At 30 days
Secondary Outcome
Measure Time Frame
Time to Remission At 30 days
Enrollment 8
Condition
Intervention

Intervention type: Drug

Intervention name: Danazol

Description: Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.

Arm group label: Danazol, Plex, Steroids

Eligibility

Criteria:

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.

Gender: All

Minimum age: 18 Years

Maximum age: N/A

Healthy volunteers: No

Overall Official
Last Name Role Affiliation
Ilan Shapira, M.D. Principal Investigator Mount Sinai St. Luke's-Roosevelt
Location
facility Mount Sinai St. Luke's-Roosevelt
Location Countries

United States

Verification Date

December 2018

Responsible Party

Responsible party type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Arm group label: Danazol, Plex, Steroids

Arm group type: Experimental

Description: Everyone will receive Danazol with plasma exchange and corticosteroids

Acronym TTP
Study Design Info

Allocation: N/A

Intervention model: Single Group Assignment

Primary purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov