Autoimmune Hemolytic Disease (Cold Type) (Warm Type)Phase 2NCT01345708
A Single-Arm Pilot Study With Low-Dose Rituximab Plus Standard Oral Prednisone In Idiopathic Autoimmune Hemolytic Anemia
A Phase 2 study testing prednisone, low dose rituximab in people with Autoimmune Hemolytic Disease (Cold Type) (Warm Type), run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico at 2 sites in Italy.
Trial summary
The aim of this prospective study was to evaluate the activity, safety and the duration of the response of low dose rituximab associated with standard oral prednisone as first line therapy in newly diagnosed warm autoimmune hemolytic anemia and cold hemagglutinin disease, and as second line therapy in warm autoimmune… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Hemolytic Disease (Cold Type) (Warm Type)
- What is being tested?
- prednisone, low dose rituximab
- Type of treatment
- Injection or infusion
- How many people
- 23 participants
- Where
- 2 sitesItaly
- Study dates
- Jan 2009to 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
- Newly diagnosed "warm" or "cold" AIHA, defined by symptomatic anemia and positive DAT, in the absence of underlying lymphoproliferative, infectious or neoplastic disease (according to the single Center diagnostic criteria).
- Idiopathic "warm" or "cold" AIHA relapsed after first line treatment with oral prednisone.
- Aged >18 years
- ECOG performance status grade 0, 1 or 2
- No psychiatric illness that precludes understanding concepts of the trial or signing informed consent
- Patients who have provided written informed consent prior to study participation, with the understanding that the consent may be withdrawn by the patient at any time without prejudice.
Who cannot join
- Cell or humoral immunologic deficit (congenital or acquired)
- Any other co-existing medical or psychological condition that would preclude participation in the study or compromise ability to give informed consent
- Active bacterial, viral, or fungal infection requiring systemic therapy HIV or HbsAg positive (with HBV-DNA+) or HCV-Ab positive (with HCV-RNA+) patients
- History of malignancies within 3 years prior to study entry
- Concomitant immunosuppressive or cytotoxic treatment
- Positive pregnancy test. Lactation.
- The presence of associated organ-specific autoimmune diseases do not constitute exclusion criteria. Previous splenectomy does not constitute exclusion criteria
Trial locations and contacts
2 sites in 1 country.
Italy2 sites
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122
Wilma Barcellini MD, UO Ematologia 2, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Milan, 20122
About this study
The aim of this prospective study was to evaluate the activity, safety and the duration of the response of low dose rituximab associated with standard oral prednisone as first line therapy in newly diagnosed warm autoimmune hemolytic anemia and cold hemagglutinin disease, and as second line therapy in warm autoimmune hemolytic anemia relapsed after standard oral prednisone. Further aim was to correlate the clinical response to biological parameters (cytokine and anti-erythrocyte antibody production in cultures).
- Being tested
- prednisone, low dose rituximab
- Keywords
- Low dose rituximab, WAIHA, CHD
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
prednisone, low dose rituximabDrug
Patients with "warm" AIHA will receive:
Rituximab 100 mg i.v.weekly (fixed dose irrespective of body surface area) for 4 consecutive weeks (days +7, +14, +21, +28), and prednisone 1 mg/kg/die p.o. days from day +1 to day +30, followed by tapering (10 mg/week until 0.5/mg/kg/die, then 5 mg/week until stop)
Patients with CHD will receive:
Rituximab 100 mg i.v.weekly (fixed dose irrespective of body surface area) for 4 consecutive weeks (days +7, +14, +21, +28)
What is being measured
The primary outcome is the main question the study is designed to answer.
overall response
Response will be evaluated at each clinic visit. Complete response (CR) will be defined as Hb increase >2 g/dL and Hb > 12 g/dL, normalization of all haemolytic markers and no transfusion requirement;Partial response (PR) will be defined as Hb increase >2 g/dL and Hb 10-12 g/dL, and 50% reduction of transfusion. In the remaining cases there is no response.
Measured: month +2
Secondary outcomes (1)
Sustained response (SR)
Sustained response (SR) was defined as Hb > 10 g/dL at month +6 and +12, in the absence of any treatment.
Measured: month +6, month +12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2011 (estimated)
- Study startJan 2009
- Primary completionJan 2010
- Study completionJan 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Autoimmune Hemolytic Disease (Cold Type) (Warm Type)Studies 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
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
- Sponsor type
- University, hospital or other
- Collaborators
- University Hospital, Udine, Italy
- Investigators
- PierMannuccio Mannucci, MD, Study director, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F. Sforza 28 20122, Milano.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01345708
- Sponsor's protocol ID
- Eudra CT 2008-006713-25
- Status last verified
- Apr 2011
- First submitted
- 28 Apr 2011
- Last updated
- 2 May 2011
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Provan D, Butler T, Evangelista ML, Amadori S, Newland AC, Stasi R. Activity and safety profile of low-dose rituximab for the treatment of autoimmune cytopenias in adults. Haematologica. 2007 Dec;92(12):1695-8. doi: 10.3324/haematol.11709.
PubMed 18055995Zaja F, Vianelli N, Volpetti S, Battista ML, Defina M, Palmieri S, Bocchia M, Medeot M, De Luca S, Ferrara F, Isola M, Baccarani M, Fanin R. Low-dose rituximab in adult patients with primary immune thrombocytopenia. Eur J Haematol. 2010 Oct;85(4):329-34. doi: 10.1111/j.1600-0609.2010.01486.x. Epub 2010 Jul 28.
PubMed 20546023Gomez-Almaguer D, Solano-Genesta M, Tarin-Arzaga L, Herrera-Garza JL, Cantu-Rodriguez OG, Gutierrez-Aguirre CH, Jaime-Perez JC. Low-dose rituximab and alemtuzumab combination therapy for patients with steroid-refractory autoimmune cytopenias. Blood. 2010 Dec 2;116(23):4783-5. doi: 10.1182/blood-2010-06-291831. Epub 2010 Sep 14.
PubMed 20841509Barcellini W, Zaja F, Zaninoni A, Imperiali FG, Battista ML, Di Bona E, Fattizzo B, Consonni D, Cortelezzi A, Fanin R, Zanella A. Low-dose rituximab in adult patients with idiopathic autoimmune hemolytic anemia: clinical efficacy and biologic studies. Blood. 2012 Apr 19;119(16):3691-7. doi: 10.1182/blood-2011-06-363556. Epub 2012 Jan 20.
PubMed 22267606
Study at a glance
From the registry record, last updated by the sponsor on 2 May 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm Pilot Study With Low-Dose Rituximab Plus Standard Oral Prednisone In Idiopathic Autoimmune Hemolytic Anemia
- ClinicalTrials.gov ID
- NCT01345708
- Acronym / sponsor ID
- Eudra CT 2008-006713-25
- Keywords
- Low dose rituximab, WAIHA, CHD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore PoliclinicoUniversity, hospital or other
- Collaborators
- University Hospital, Udine, Italy
- Study start
- Jan 2009
- Primary completion
- Jan 2010 (actual)
- Study completion
- Jan 2011 (actual)
- First posted
- 2 May 2011 (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 aim of this prospective study was to evaluate the activity, safety and the duration of the response of low dose rituximab associated with standard oral prednisone as first line therapy in newly diagnosed warm autoimmune hemolytic anemia and cold hemagglutinin disease, and as second line therapy in warm autoimmune hemolytic anemia relapsed after standard oral prednisone. Further aim was to correlate the clinical response to biological parameters (cytokine and anti-erythrocyte antibody production in cultures).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Newly diagnosed "warm" or "cold" AIHA, defined by symptomatic anemia and positive DAT, in the absence of underlying lymphoproliferative, infectious or neoplastic disease (according to the single Center diagnostic criteria).
- Idiopathic "warm" or "cold" AIHA relapsed after first line treatment with oral prednisone.
- Aged >18 years
- ECOG performance status grade 0, 1 or 2
- No psychiatric illness that precludes understanding concepts of the trial or signing informed consent
Key exclusion criteria (7 listed)
- Cell or humoral immunologic deficit (congenital or acquired)
- Any other co-existing medical or psychological condition that would preclude participation in the study or compromise ability to give informed consent
- Active bacterial, viral, or fungal infection requiring systemic therapy HIV or HbsAg positive (with HBV-DNA+) or HCV-Ab positive (with HCV-RNA+) patients
- History of malignancies within 3 years prior to study entry
- Concomitant immunosuppressive or cytotoxic treatment
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 |
|---|---|---|
| prednisone, low dose rituximabPatients with "warm" AIHA will receive: Rituximab 100 mg i.v.weekly (fixed dose irrespective of body surface area) for 4 consecutive weeks (days +7, +14, +21, +28), and prednisone 1 mg/kg/die p.o. days from day +1 to day +30, followed by tapering (10 mg/week until 0.5/mg/kg/die, then 5 mg/week until stop) Patients with CHD will receive: Rituximab 100 mg i.v.weekly (fixed dose irrespective of body surface area) for 4 consecutive weeks (days +7, +14, +21, +28) | Drug | – |
Primary outcome
overall response
Time frame: month +2
Response will be evaluated at each clinic visit. Complete response (CR) will be defined as Hb increase >2 g/dL and Hb > 12 g/dL, normalization of all haemolytic markers and no transfusion requirement;Partial response (PR) will be defined as Hb increase >2 g/dL and Hb 10-12 g/dL, and 50% reduction of transfusion. In the remaining cases there is no response.
1 secondary outcome
Sustained response (SR)
Time frame: month +6, month +12
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 May 2011.
- Background: PubMed 18055995Provan D, Butler T, Evangelista ML, Amadori S, Newland AC, Stasi R. Activity and safety profile of low-dose rituximab for the treatment of autoimmune cytopenias in adults. Haematologica. 2007 Dec;92(12):1695-8. doi: 10.3324/haematol.11709.
- Background: PubMed 20546023Zaja F, Vianelli N, Volpetti S, Battista ML, Defina M, Palmieri S, Bocchia M, Medeot M, De Luca S, Ferrara F, Isola M, Baccarani M, Fanin R. Low-dose rituximab in adult patients with primary immune thrombocytopenia. Eur J Haematol. 2010 Oct;85(4):329-34. doi: 10.1111/j.1600-0609.2010.01486.x. Epub 2010 Jul 28.
- Background: PubMed 20841509Gomez-Almaguer D, Solano-Genesta M, Tarin-Arzaga L, Herrera-Garza JL, Cantu-Rodriguez OG, Gutierrez-Aguirre CH, Jaime-Perez JC. Low-dose rituximab and alemtuzumab combination therapy for patients with steroid-refractory autoimmune cytopenias. Blood. 2010 Dec 2;116(23):4783-5. doi: 10.1182/blood-2010-06-291831. Epub 2010 Sep 14.
- Derived from this study: PubMed 22267606Barcellini W, Zaja F, Zaninoni A, Imperiali FG, Battista ML, Di Bona E, Fattizzo B, Consonni D, Cortelezzi A, Fanin R, Zanella A. Low-dose rituximab in adult patients with idiopathic autoimmune hemolytic anemia: clinical efficacy and biologic studies. Blood. 2012 Apr 19;119(16):3691-7. doi: 10.1182/blood-2011-06-363556. Epub 2012 Jan 20.
Related trials for Autoimmune Hemolytic Disease (Cold Type) (Warm Type)
RecruitingObservational study
National Longitudinal Cohort of Hematological Diseases- Autoimmune Hemolytic Anemia
AIHA - Warm Autoimmune Hemolytic Anemia, AIHA - Cold Autoimmune Hemolytic Anemia
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.
PierMannuccio Mannucci, MD study director
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F. Sforza 28 20122, Milano.
2 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 prednisone, low dose rituximab in people with Autoimmune Hemolytic Disease (Cold Type) (Warm Type), run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico at 2 sites in Italy.
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 6 inclusion and 7 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: finished, as of the sponsor's last update on 2 May 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
overall response (time frame: month +2). 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 NCT01345708, which also lists 4 linked publications.
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 2 May 2011.