Clinical trial NCT00953771: Safety Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura (TTP)
Official title: Phase II Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Thrombotic Thrombocytopenic Purpura
Keywords: TTP
Thrombotic Thrombocytopenic Purpura
Lead sponsor: Icahn School of Medicine at Mount Sinai
Sponsor class: OTHER
Collaborators: name: Beth Israel Medical Center
First posted: 2009-08-06
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Plasma Exchanges
timeFrame: up to 30 days
Secondary outcomes:
measure: Time to Remission
timeFrame: up to 30 days
measure: Length of Stay
timeFrame: up to 30 days
measure: Number of Participants With Complete and Continuous Response Rate
timeFrame: At 2 Years
measure: Time to Relapse
timeFrame: up to 12 years
measure: Number of Participants With Relapses
timeFrame: up to 12 years
measure: Number of Relapses
timeFrame: up to 12 years
Other outcomes:
Locations:
city: New York
country: United States
facility: Mount Sinai St. Luke's-Roosevelt
state: New York
Study officials:
affiliation: Mount Sinai St. Luke's-Roosevelt
name: Ilan Shapira, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Torri V, Friedman M, Shapira I, Patel AA, Yoe J, Shah VP, Mirzoyev T, Machuca M, Varma M. Phase II study of danazol with plasma exchange and corticosteroids for the treatment of thrombotic thrombocytopenic purpura. Blood (2023) 142 (Supplement 1): 4007.