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

Primary Purpose

Thrombotic Thrombocytopenic Purpura

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Danazol
Sponsored by
Icahn School of Medicine at Mount Sinai
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Thrombotic Thrombocytopenic Purpura focused on measuring TTP, Thrombotic Thrombocytopenic Purpura

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

  • Mount Sinai St. Luke's-Roosevelt

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Danazol, Plex, Steroids

Historic Control

Arm Description

Everyone will receive Danazol with plasma exchange and corticosteroids

Outcomes

Primary Outcome Measures

Number of Plasma Exchanges
The total number of plasma exchanges performed after initiation of the first plasma exchange.

Secondary Outcome Measures

Time to Remission
Length of Stay
Number of Participants With Complete and Continuous Response Rate
Time to Relapse
Number of Relapses

Full Information

First Posted
August 4, 2009
Last Updated
July 18, 2023
Sponsor
Icahn School of Medicine at Mount Sinai
Collaborators
Beth Israel Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT00953771
Brief Title
Safety Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura (TTP)
Acronym
TTP
Official Title
Phase II Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Terminated
Why Stopped
low enrollment
Study Start Date
October 2008 (undefined)
Primary Completion Date
November 19, 2015 (Actual)
Study Completion Date
November 19, 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Icahn School of Medicine at Mount Sinai
Collaborators
Beth Israel Medical Center

4. Oversight

Data Monitoring Committee
No

5. 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).
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thrombotic Thrombocytopenic Purpura
Keywords
TTP, Thrombotic Thrombocytopenic Purpura

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
29 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Danazol, Plex, Steroids
Arm Type
Experimental
Arm Description
Everyone will receive Danazol with plasma exchange and corticosteroids
Arm Title
Historic Control
Arm Type
No Intervention
Intervention Type
Drug
Intervention Name(s)
Danazol
Other Intervention Name(s)
Plasma Exchange, Corticosteroids
Intervention Description
Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
Primary Outcome Measure Information:
Title
Number of Plasma Exchanges
Description
The total number of plasma exchanges performed after initiation of the first plasma exchange.
Time Frame
up to 60 days
Secondary Outcome Measure Information:
Title
Time to Remission
Time Frame
up to 30 days
Title
Length of Stay
Time Frame
up to 30 days
Title
Number of Participants With Complete and Continuous Response Rate
Time Frame
At 2 Years
Title
Time to Relapse
Time Frame
up to 12 years
Title
Number of Relapses
Time Frame
up to 12 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ilan Shapira, M.D.
Organizational Affiliation
Mount Sinai St. Luke's-Roosevelt
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mount Sinai St. Luke's-Roosevelt
City
New York
State/Province
New York
ZIP/Postal Code
10019
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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