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

Sponsor

Icahn School of Medicine at Mount Sinai (Other)

Overall Status

Terminated

CT.gov ID

NCT00953771

Collaborator

Beth Israel Medical Center (Other)

Enrollment

Location

Arm

84.4

Duration (Months)

0.1

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Thrombotic Thrombocytopenic Purpura	Drug: Danazol	Phase 2

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.

Study Design

Study Type:

Interventional

Actual Enrollment :

8 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Oct 1, 2008

Actual Primary Completion Date :

Oct 14, 2015

Actual Study Completion Date :

Oct 14, 2015

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Danazol, Plex, Steroids Everyone will receive Danazol with plasma exchange and corticosteroids	Drug: Danazol Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment. Other Names: Plasma Exchange Corticosteroids

Arm

Intervention/Treatment

Experimental: Danazol, Plex, Steroids

Everyone will receive Danazol with plasma exchange and corticosteroids

Drug: Danazol

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

Other Names:

Plasma Exchange

Corticosteroids

Outcome Measures

Primary Outcome Measures

To assess the total number of plasma exchanges performed within 30 days of initiation of the first plasma exchange. [At 30 days]

Secondary Outcome Measures

Time to Remission [At 30 days]

Other Outcome Measures

Length of Stay [At 30 days]
Complete and Continuous Response Rate [At 2 Years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.