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

Thrombotic Thrombocytopenic Purpura Clinical Trial

— TTP  

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00953771
• Other study ID #: GCO 16-2608
• Secondary ID: 055-08
• Status: Terminated
• Phase: Phase 2
• Start date: October 2008
• Est. completion date: November 19, 2015

Study information

• Verified date: January 2024
• Source: Icahn School of Medicine at Mount Sinai
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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 information / eligibility

• Status: Terminated
• Enrollment: 9
• Est. completion date: November 19, 2015
• Est. primary completion date: November 19, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombotic Thrombocytopenic
• Thrombotic Thrombocytopenic Purpura

Intervention

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

Locations

Country	Name	City	State
United States	Mount Sinai St. Luke's-Roosevelt	New York	New York

Sponsors (2)

Lead Sponsor	Collaborator
Icahn School of Medicine at Mount Sinai	Beth Israel Medical Center

Country where clinical trial is conducted

United States, 

References & Publications (1)

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.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Plasma Exchanges	The total number of plasma exchanges performed after initiation of the first plasma exchange.	up to 30 days
Secondary	Time to Remission		up to 30 days
Secondary	Length of Stay		up to 30 days
Secondary	Number of Participants With Complete and Continuous Response Rate		At 2 Years
Secondary	Time to Relapse		up to 12 years
Secondary	Number of Participants With Relapses	Number of participants with relapses	up to 12 years
Secondary	Number of Relapses	Number of relapses	up to 12 years