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

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

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).

Study Overview

• Status: Terminated
• Conditions: Thrombotic Thrombocytopenic Purpura
• Intervention / Treatment: Drug: Danazol

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 Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10019
      • Mount Sinai St. Luke's-Roosevelt

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / 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
No Intervention: Historic Control

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Plasma Exchanges	The total number of plasma exchanges performed after initiation of the first plasma exchange.	up to 30 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Remission		up to 30 days
Length of Stay		up to 30 days
Number of Participants With Complete and Continuous Response Rate		At 2 Years
Time to Relapse		up to 12 years
Number of Participants With Relapses	Number of participants with relapses	up to 12 years
Number of Relapses	Number of relapses	up to 12 years

Other Outcome Measures

Outcome Measure	Time Frame
Length of Stay	At 30 days
Complete and Continuous Response Rate	At 2 Years

Collaborators and Investigators

• Sponsor: Icahn School of Medicine at Mount Sinai
• Collaborators: Beth Israel Medical Center
• Investigators: Principal Investigator: Ilan Shapira, M.D., Mount Sinai St. Luke's-Roosevelt

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: October 1, 2008
• Primary Completion (Actual): November 19, 2015
• Study Completion (Actual): November 19, 2015

Study Registration Dates

• First Submitted: August 4, 2009
• First Submitted That Met QC Criteria: August 5, 2009
• First Posted (Estimated): August 6, 2009

Study Record Updates

• Last Update Posted (Actual): July 23, 2024
• Last Update Submitted That Met QC Criteria: July 11, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: TTP; Thrombotic Thrombocytopenic Purpura
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Hematologic Diseases; Hemorrhage; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombophilia; Thrombotic Microangiopathies; Cytopenia; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombotic Thrombocytopenic; Physiological Effects of Drugs; Hormones, Hormone Substitutes, and Hormone Antagonists; Hormone Antagonists; Estrogen Antagonists; Danazol
• Other Study ID Numbers: GCO 16-2608; 055-08 (Other Identifier: Beth Israel Medical Center)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No