NCT00953771

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).

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2008

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2008

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

August 4, 2009

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 6, 2009

Completed
6.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 19, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 19, 2015

Completed
7.7 years until next milestone

Results Posted

Study results publicly available

August 4, 2023

Completed
Last Updated

July 23, 2024

Status Verified

July 1, 2024

Enrollment Period

7.1 years

First QC Date

August 4, 2009

Results QC Date

June 23, 2023

Last Update Submit

July 11, 2024

Conditions

Thrombotic Thrombocytopenic Purpura

Keywords

TTPThrombotic Thrombocytopenic Purpura

Outcome Measures

Primary Outcomes (1)

  • Number of Plasma Exchanges

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

    up to 30 days

Secondary Outcomes (6)

  • 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

    up to 12 years

  • +1 more secondary outcomes

Study Arms (2)

Danazol, Plex, Steroids

EXPERIMENTAL

Everyone will receive Danazol with plasma exchange and corticosteroids

Drug: Danazol

Historic Control

NO INTERVENTION

Interventions

DanazolDRUG

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

Also known as: Plasma Exchange, Corticosteroids
Danazol, Plex, Steroids

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mount Sinai St. Luke's-Roosevelt

New York, New York, 10019, United States

Location

Related 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.

    RESULT

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Interventions

DanazolPlasma ExchangeAdrenal Cortex Hormones

Condition Hierarchy (Ancestors)

Purpura, ThrombocytopenicPurpuraBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesThrombotic MicroangiopathiesThrombocytopeniaBlood Platelet DisordersCytopeniaThrombophiliaHemorrhagePathologic ProcessesPathological Conditions, Signs and SymptomsSkin ManifestationsSigns and Symptoms

Intervention Hierarchy (Ancestors)

PregnadienesPregnanesSteroidsFused-Ring CompoundsPolycyclic CompoundsBlood TransfusionBiological TherapyTherapeuticsPlasmapheresisBlood Component RemovalSorption DetoxificationExtracorporeal CirculationSurgical Procedures, OperativeHormonesHormones, Hormone Substitutes, and Hormone Antagonists

Results Point of Contact

Title
Dr. Ilan Shapira
Organization
Mount Sinai Beth Israel
Ilan.Shapira@mountsinai.org
212-844-8948

Study Officials

  • Ilan Shapira, M.D.

    Mount Sinai St. Luke's-Roosevelt

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Assistant Professor

Study Record Dates

First Submitted

August 4, 2009

First Posted

August 6, 2009

Study Start

October 1, 2008

Primary Completion

November 19, 2015

Study Completion

November 19, 2015

Last Updated

July 23, 2024

Results First Posted

August 4, 2023

Record last verified: 2024-07

Locations

US(1)