Thrombotic Thrombocytopenic PurpuraPhase 2TTPNCT00953771
Safety Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura (TTP)
A Phase 2 study testing Danazol in people with Thrombotic Thrombocytopenic Purpura, run by Icahn School of Medicine at Mount Sinai at 1 site in United States.
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura
- What is being tested?
- Danazol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2008to 19 Nov 2015
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 locations and contacts
1 site in 1 country.
United States1 site
Mount Sinai St. Luke's-Roosevelt
New York, New York, 10019
About this study
Official title: 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).
Read the 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.
- Conditions
- Thrombotic Thrombocytopenic Purpura
- Being tested
- Danazol
- Keywords
- TTP, Thrombotic Thrombocytopenic Purpura
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DanazolDrug
Also called Plasma Exchange, Corticosteroids
Danazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment.
| Study group | Role | What they receive |
|---|---|---|
| Danazol, Plex, Steroids | Experimental | Everyone will receive Danazol with plasma exchange and corticosteroids Receives: Drug: Danazol |
| Historic Control | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Plasma Exchanges
The total number of plasma exchanges performed after initiation of the first plasma exchange.
Measured: up to 30 days
Secondary outcomes (6)
Time to Remission
Measured: up to 30 days
Length of Stay
Measured: up to 30 days
Number of Participants With Complete and Continuous Response Rate
Measured: At 2 Years
Time to Relapse
Measured: up to 12 years
Number of Participants With Relapses
Number of participants with relapses
Measured: up to 12 years
Number of Relapses
Number of relapses
Measured: up to 12 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2009 (estimated)
- Study startOct 2008
- Primary completion19 Nov 2015
- Study completion19 Nov 2015
Why it stopped: low enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Thrombotic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- More trials run by Icahn School of Medicine at Mount SinaiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Sponsor type
- University, hospital or other
- Collaborators
- Beth Israel Medical Center
- Responsible party
- Principal investigator, Ilan Shapira, Icahn School of Medicine at Mount Sinai
- Investigators
- Ilan Shapira, M.D., Principal investigator, Mount Sinai St. Luke's-Roosevelt
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00953771
- Sponsor's protocol ID
- GCO 16-2608
- Other IDs
- 055-08
- Acronym
- TTP
- Status last verified
- Jul 2024
- First submitted
- 4 Aug 2009
- Last updated
- 23 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
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 at a glance
From the registry record, last updated by the sponsor on 23 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT00953771
- Acronym / sponsor ID
- TTP / GCO 16-2608
- Conditions
- Thrombotic Thrombocytopenic Purpura
- Keywords
- TTP, Thrombotic Thrombocytopenic Purpura
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: low enrollment
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Icahn School of Medicine at Mount SinaiUniversity, hospital or other
- Collaborators
- Beth Israel Medical Center
- Study start
- Oct 2008
- Primary completion
- 19 Nov 2015 (actual)
- Study completion
- 19 Nov 2015 (actual)
- First posted
- 6 Aug 2009 (estimated)
- Results
- Posted 4 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Danazol, Plex, Steroids | Experimental | Drug: DanazolEveryone will receive Danazol with plasma exchange and corticosteroids |
| Historic Control | No intervention | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DanazolAlso: Plasma Exchange, CorticosteroidsDanazol 600 mg PO will be initiated with plasma exchange and corticosteroids at the time of enrollment. | Drug | Danazol, Plex, Steroids |
Primary outcome
Number of Plasma Exchanges
Time frame: up to 30 days
The total number of plasma exchanges performed after initiation of the first plasma exchange.
6 secondary outcomes
Time to Remission
Time frame: up to 30 days
Length of Stay
Time frame: up to 30 days
Number of Participants With Complete and Continuous Response Rate
Time frame: At 2 Years
Time to Relapse
Time frame: up to 12 years
Number of Participants With Relapses
Time frame: up to 12 years
Number of Relapses
Time frame: up to 12 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publicationTorri 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ilan Shapira, M.D. principal investigator
Mount Sinai St. Luke's-Roosevelt
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Danazol in people with Thrombotic Thrombocytopenic Purpura, run by Icahn School of Medicine at Mount Sinai at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Aug 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Plasma Exchanges (time frame: up to 30 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00953771, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jul 2024.