Thrombotic Thrombocytopenic PurpuraPhase 2NCT00531089
Rituximab in Patients With Relapsed or Refractory TTP-HUS
A Phase 2 study testing Rituximab in people with Thrombotic Thrombocytopenic Purpura and Hemolytic Uremic Syndrome, run by Hamilton Health Sciences Corporation at 11 sites in Canada.
Trial summary
The general objective of this study is to assess the efficacy and safety of Rituximab in the management of patients with refractory or relapsed thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura, Hemolytic Uremic Syndrome
- What is being tested?
- Rituximab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 11 sitesCanada
- Study dates
- Dec 2007to Jan 2011
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- any patient 18 years or older diagnosed with relapsed or refractory TTP-HUS requiring therapy
Who cannot join
- alternate cause of hemolytic microangiopathy (evidence of DIC, malignant hypertension, vasculitis, anti-phospholipid antibody syndrome, post-partum acute renal failure)
- congenital or familial TTP
- TTP occuring post-stem cell, bone marrow, or solid organ transplant
- drug-induced TTP
- pregnancy or breast-feeding
- history of hepatitis B or C infection
Show all 14Show fewer
- prior rituximab treatment
- active or metastatic cancer
- other causes of thrombocytopenia such as ITP, myelodysplastic syndrome, confirmed or suspected drug-induced thrombocytopenia
- refusal to receive blood products
- hypersensitivity to blood products, plasma products, murine proteins, or any component of the Rituximab formulation
- geographic inaccessibility
- co-morbid illness limiting life expectancy to less than 2 months independent of TTP
- failure to provide written informed consent
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
Canada11 sites, 11 recruiting
- Not yet recruiting
Foothills Medical Centre, Calgary Health REgion Apheresis Service
Calgary, Alberta, T2N 2T9
John Klassen, MD, 403-944-4712, john.klassen@calgaryhealthregion.ca
John Klassen, MD
- Not yet recruiting
University of Alberta Hospital
Edmonton, Alberta
L Larratt, MD
- Not yet recruiting
Hopital Charles Lemoyne
Greenfield Park, Quebec
S Fox, MD, 450-466-5000
S Fox, MD
- Recruiting
Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5
Julie Carruthers, 905-525-9140 ext. 22942, carrutj@mcmaster.ca
Kathryn E Webert, MD
- Recruiting
London Health Sciences Centre, Westminister Campus
London, Ontario, N6A4G5
Clark F William, MD, 519-685-8500 ext. 57238, william.clark@lhsc.on.ca
William F Clark, MD
- Not yet recruiting
Hopital du Sacre-Coeur de Montreal
Montreal, Quebec, H4J1C5
J P Moquin, MD, 514-338-2222 ext. 3368, jp.moquin@videotron.ca
J P Moquin, MD
- Not yet recruiting
St. John Regional Hospital
Saint John, New Brunswick, E2K5S9
Sean Dolan, MD, 506-634-1201
Sean Dolan, MD
- Recruiting
St. Paul's Hospital Apheresis Unit
Saskatoon, Saskatchewan, S7M 0Z9
Ahmed Shoker, MD, 306-655-5934, ahmed.shoker@usask.ca
Ahmed Shoker, MD
- Recruiting
Princess Margaret Hospital, ABMT/Apheresis Unit
Toronto, Ontario, M5G2M9
David Barth, MD, 416-946-4688, david.barth@uhn.on.ca
David Barth, MD
- Recruiting
Vancouver General Hospital
Vancouver, British Columbia, V5Z1M9
Paul Yenson, Dr., 604-875-4863, pyenson@bccancer.bc.ca
Lisa Basque, 604-875-4111 ext. 69014, lbasque@bccancer.bc.ca
- Not yet recruiting
Winnipeg Regional Health Authority, Apheresis Department
Winnipeg, Manitoba, R3E 0T2
Cathy Moltzan, MD, 204-787-4269, cmoltzan@sbgh.mb.ca
Cathy Moltzan, MD
About this study
Official title: A Phase II Study Evaluating the Efficacy of Rituximab in the Management of Patients With Relapsed/Refractory Thrombotic Thrombocytopenic Purpura (TTP) - Hemolytic Uremic Syndrome (HUS)
The general objective of this study is to assess the efficacy and safety of Rituximab in the management of patients with refractory or relapsed thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS). There have been several case reports and case series describing the use of Rituximab in patients with TTP-HUS; however its use has not been studied in a large trial. It is hypothesized that Rituximab may ameliorate the severity of certain cases of TTP-HUS by decreasing the number of activity of B-cells which may result in decreased production of the ADAMTS13 protease inhibitor. Patients with TTP-HUS not responding to standard therapy or patients with relapsed disease may have particular benefit. Treatments that decrease the frequency of relapse or shorten the time to remission of TTP-HUS will be of benefit by decreasing the need for blood product support.
- Being tested
- Rituximab
- Keywords
- TTP, thrombotic thrombocytopenic purpura, HUS, hemolytic uremic syndrome, plasma exchange
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic, Hemolytic-Uremic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RituximabDrug
Also called Rituxan, rituximab
Rituximab will be administered on weeks 1, 2, 3, and 4 at a dose of 375 mg/m2 per infusion. Premedications (prednisone 50 mg, diphenhydramine 50 mg, acetaminophen) will be administered prior to study infusion. Patients will also be treated with plasma exchange as per institution/apheresis centre.
| Study group | Role | What they receive |
|---|---|---|
| Study group | Experimental | All patients in the study will be in the study group and will receive rituximab. There is no "control" arm. Receives: Drug: Rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of patients achieving all: (1) platelet count >150x109/L; (2) LDH < 1.5 x normal; (3) no requirement for plasma exchange therapy; (4) asymptomatic.
Measured: 8 weeks after initiation of therapy
Secondary outcomes (9)
proportion of patients with platelet count greater than 150 x 109/L
Measured: 8 weeks
proportion of patients with LDH < 1.5 X normal
Measured: 8 weeks
proportion of patients with no requirement for plasma exchange therapy
Measured: 8 weeks
proportion of patients who are asymptomatic (no new neurological symptoms ans stabilization of previous neurological symptoms
Measured: 8 weeks
clinical response (CR, PR, non-response)
Measured: 52 weeks
frequency of relapse
Measured: 52 weeks
mortality
Measured: 52 weeks
changes from baseline in platelet counts, LDH, ADAMTS13 protease level, ADAMTS13 inhibitor level
Measured: 8, 12, 24, 52 weeks
toxicity and clinical safety as assessed by monitoring of adverse events, laboratory parameters, vital signs during infusion, and immediate tolerability
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2007 (estimated)
- Study startDec 2007
- Primary completionJan 2011 (estimated)
- Study completionJan 2011 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombotic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hamilton Health Sciences Corporation
- Sponsor type
- University, hospital or other
- Collaborators
- Canadian Apheresis Group, Hoffmann-La Roche, McMaster University
- Investigators
- Kathryn E Webert, E, Principal investigator, Hamilton Health Sciences CorporationRonan Foley, MD, Principal investigator, Hamilton Health Sciences CorporationGail Rock, MD, Study director, Canadian Apheresis GroupWilliam Clark, MD, Study director, University of Western Ontario/London Health SciencesDavid Barth, MD, Study director, University of Toronto
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00531089
- Sponsor's protocol ID
- CAG-1
- Status last verified
- Sep 2007
- First submitted
- 17 Sep 2007
- Last updated
- 19 May 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study Evaluating the Efficacy of Rituximab in the Management of Patients With Relapsed/Refractory Thrombotic Thrombocytopenic Purpura (TTP) - Hemolytic Uremic Syndrome (HUS)
- ClinicalTrials.gov ID
- NCT00531089
- Acronym / sponsor ID
- CAG-1
- Keywords
- TTP, thrombotic thrombocytopenic purpura, HUS, hemolytic uremic syndrome, plasma exchange
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Hamilton Health Sciences CorporationUniversity, hospital or other
- Collaborators
- Canadian Apheresis Group, Hoffmann-La Roche, McMaster University
- Study start
- Dec 2007
- Primary completion
- Jan 2011 (estimated)
- Study completion
- Jan 2011 (estimated)
- First posted
- 18 Sep 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The general objective of this study is to assess the efficacy and safety of Rituximab in the management of patients with refractory or relapsed thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS). There have been several case reports and case series describing the use of Rituximab in patients with TTP-HUS; however its use has not been studied in a large trial. It is hypothesized that Rituximab may ameliorate the severity of certain cases of TTP-HUS by decreasing the number of activity of B-cells which may result in decreased production of the ADAMTS13 protease inhibitor. Patients with TTP-HUS not responding to standard therapy or patients with relapsed disease may have particular benefit. Treatments that decrease the frequency of relapse or shorten the time to remission of TTP-HUS will be of benefit by decreasing the need for blood product support.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study group | Experimental | Drug: RituximabAll patients in the study will be in the study group and will receive rituximab. There is no "control" arm. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- any patient 18 years or older diagnosed with relapsed or refractory TTP-HUS requiring therapy
Key exclusion criteria (14 listed)
- alternate cause of hemolytic microangiopathy (evidence of DIC, malignant hypertension, vasculitis, anti-phospholipid antibody syndrome, post-partum acute renal failure)
- congenital or familial TTP
- TTP occuring post-stem cell, bone marrow, or solid organ transplant
- drug-induced TTP
- pregnancy or breast-feeding
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 |
|---|---|---|
| RituximabAlso: Rituxan, rituximabRituximab will be administered on weeks 1, 2, 3, and 4 at a dose of 375 mg/m2 per infusion. Premedications (prednisone 50 mg, diphenhydramine 50 mg, acetaminophen) will be administered prior to study infusion. Patients will also be treated with plasma exchange as per institution/apheresis centre. | Drug | Study group |
Primary outcome
The proportion of patients achieving all: (1) platelet count >150x109/L; (2) LDH < 1.5 x normal; (3) no requirement for plasma exchange therapy; (4) asymptomatic.
Time frame: 8 weeks after initiation of therapy
9 secondary outcomes
proportion of patients with platelet count greater than 150 x 109/L
Time frame: 8 weeks
proportion of patients with LDH < 1.5 X normal
Time frame: 8 weeks
proportion of patients with no requirement for plasma exchange therapy
Time frame: 8 weeks
proportion of patients who are asymptomatic (no new neurological symptoms ans stabilization of previous neurological symptoms
Time frame: 8 weeks
clinical response (CR, PR, non-response)
Time frame: 52 weeks
frequency of relapse
Time frame: 52 weeks
mortality
Time frame: 52 weeks
changes from baseline in platelet counts, LDH, ADAMTS13 protease level, ADAMTS13 inhibitor level
Time frame: 8, 12, 24, 52 weeks
toxicity and clinical safety as assessed by monitoring of adverse events, laboratory parameters, vital signs during infusion, and immediate tolerability
Time frame: 8 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2010.
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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.
Kathryn E Webert, E principal investigator
Hamilton Health Sciences Corporation
Ronan Foley, MD principal investigator
Hamilton Health Sciences Corporation
Gail Rock, MD study director
Canadian Apheresis Group
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rituximab in people with Thrombotic Thrombocytopenic Purpura and Hemolytic Uremic Syndrome, run by Hamilton Health Sciences Corporation at 11 sites in Canada.
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 1 inclusion and 14 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: status not confirmed, as of the sponsor's last update on 19 May 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Canada. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Kathryn E Webert, MD (905-521-2100 ext. 76733, webertk@mcmaster.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of patients achieving all: (1) platelet count >150x109/L; (2) LDH < 1.5 x normal; (3) no requirement for plasma exchange therapy; (4) asymptomatic. (time frame: 8 weeks after initiation of therapy). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 May 2010.