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

Mount Sinai Health System

Status and phase: Terminated; Phase 2

Conditions: Thrombotic Thrombocytopenic Purpura

Treatments: Drug: Danazol

Study type: Interventional

Funder types: Other

Identifiers

NCT00953771

055-08 (Other Identifier)

GCO 16-2608

Details and patient eligibility

About

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).

Full 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.

Enrollment: 9 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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9 participants in 2 patient groups

Danazol, Plex, Steroids

Experimental group

Description:

Everyone will receive Danazol with plasma exchange and corticosteroids

Treatment:

Drug: Danazol

Historic Control

No Intervention group