Cold Agglutinin DiseasePhase 2DECADENCT01303952
Therapy of Chronic Cold Agglutinin Disease With Eculizumab
A Phase 2 study testing Eculizumab in people with Cold Agglutinin Disease, run by University Hospital, Essen at 1 site in Germany.
Trial summary
Evaluation of the efficacy and safety of eculizumab in symptomatic or transfusion-dependent patients with untreated or refractory hemolytic cold agglutinin disease(CAD) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cold Agglutinin Disease
- What is being tested?
- Eculizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteGermany
- Study dates
- Jan 2011to Jun 2015
- 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
- Individuals at least 18 years of age
- Diagnosis of CAD defined by the combination of chronic hemolysis, cold agglutinin titer (IgM or IgA) >64 at 4°C, positive direct antiglobulin test (DAT) when performed with polyspecific antiserum, negative (or only weakly positive) anti-IgG, and strongly positive with anti-C3d
- LDH level > 2 x upper limit of normal (ULN)
- Clinical symptoms requiring treatment, such as anemia, anemia-related symptoms or hemolysis-related symptoms
- Sufficient supplementation of iron, folic acid and vitamin B12 prior to treatment phase
- Patient must be willing and able to give written informed consent;
- Patients must be vaccinated against N. meningitidis at least 14 days prior treatment
- Patient must avoid conception during the trial using a method that is most appropriate for their physical state and culture
Who cannot join
- Active aggressive lymphoma requiring therapy or an active non-lymphatic malignant disease other than basal cell carcinoma or CIS of the cervix
- Liver disease with elevated LDH
- Absolute neutrophil count < 500/µL
- Presence or suspicion of active bacterial or viral infection, in the opinion of the Investigator, at treatment start or severe recurrent bacterial infections
- Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period
- Pregnant, breast-feeding, or intending to conceive during the course of the study, including the Post-treatment Phase
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- History of bone marrow/stem cell transplantation
- Inability to cooperate or any condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study
- Hypersensitivity to eculizumab, to murine proteins or to one of the excipients
Trial locations and contacts
1 site in 1 country.
Germany1 site
Department of Hematology, University Hospital Essen
Essen, 45122
About this study
Official title: Therapy for Chronic Cold Agglutinin Disease: A Prospective, Non-Randomized Multi-Center Study Demonstrating the Efficacy of Terminal Complement Inhibition in Patients With Cold Agglutinin Disease Using Eculizumab
Evaluation of the efficacy and safety of eculizumab in symptomatic or transfusion-dependent patients with untreated or refractory hemolytic cold agglutinin disease(CAD)
- Conditions
- Cold Agglutinin Disease
- Being tested
- Eculizumab
- Keywords
- cold agglutinin disease, hemolysis, eculizumab, terminal complement inhibition
- Medical subject headings
- Anemia, Hemolytic, Autoimmune, Hemolysis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EculizumabDrug
Weekly treatment with eculizumab (600 mg) via IV infusion for 4 doses followed the next week by eculizumab (900 mg) IV infusion and maintenance treatment with Eculizumab (900 mg) via IV infusion every other week for 21 weeks
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | Experimental | Not described Receives: Drug: Eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
LDH
Primary endpoint for this study is: lactate dehydrogenase (LDH) in U/l change from baseline to 26 weeks
Measured: From baseline to 26 weeks
Secondary outcomes (12)
Transfusion avoidance
Measured: From baseline to 26 weeks
PRBC units transfused
Measured: From baseline to 26 weeks
Change in hemoglobin levels
Measured: From baseline to 26 weeks
Haptoglobin
Measured: From baseline to 26 weeks
Hemopexin
Measured: From baseline to 26 weeks
Free hemoglobin
Measured: From baseline to 26 weeks
Reticulocytes
Measured: From baseline to 26 weeks
SF-36v2 QLQ
Measured: From baseline to 26 weeks
FACIT-F SCALE version 4
Measured: From baseline to 26 weeks
Six-Minute Walk Test
Measured: From baseline to 26 weeks
Circulatory symptoms
Measured: From baseline to 26 weeks
Thrombosis record
Measured: From baseline to 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2011 (estimated)
- Study startJan 2011
- Primary completionSep 2014
- Study completionJun 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Cold Agglutinin DiseaseStudies 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
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Essen
- Sponsor type
- University, hospital or other
- Collaborators
- Alexion Pharmaceuticals, Inc.
- Responsible party
- Principal investigator, Alexander Roeth, MD, University Hospital, Essen
- Investigators
- Alexander Roeth, MD, Principal investigator, Department of Hematology, University Hospital Essen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01303952
- Sponsor's protocol ID
- CAD01
- Other IDs
- 2009-016966-97, 2009
- Acronym
- DECADE
- Status last verified
- Jul 2017
- First submitted
- 24 Feb 2011
- Last updated
- 19 Jul 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Roth A, Huttmann A, Rother RP, Duhrsen U, Philipp T. Long-term efficacy of the complement inhibitor eculizumab in cold agglutinin disease. Blood. 2009 Apr 16;113(16):3885-6. doi: 10.1182/blood-2009-01-196329. No abstract available.
PubMed 19372265Roth A, Duhrsen U. Cold agglutinin disease. Eur J Haematol. 2010 Jan 1;84(1):91. doi: 10.1111/j.1600-0609.2009.01320.x. Epub 2009 Jul 14. No abstract available.
PubMed 19614954Roth A, Bommer M, Huttmann A, Herich-Terhurne D, Kuklik N, Rekowski J, Lenz V, Schrezenmeier H, Duhrsen U. Eculizumab in cold agglutinin disease (DECADE): an open-label, prospective, bicentric, nonrandomized phase 2 trial. Blood Adv. 2018 Oct 9;2(19):2543-2549. doi: 10.1182/bloodadvances.2018024190.
PubMed 30291112
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Therapy for Chronic Cold Agglutinin Disease: A Prospective, Non-Randomized Multi-Center Study Demonstrating the Efficacy of Terminal Complement Inhibition in Patients With Cold Agglutinin Disease Using Eculizumab
- ClinicalTrials.gov ID
- NCT01303952
- Acronym / sponsor ID
- DECADE / CAD01
- Conditions
- Cold Agglutinin Disease
- Keywords
- cold agglutinin disease, hemolysis, eculizumab, terminal complement inhibition
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- University Hospital, EssenUniversity, hospital or other
- Collaborators
- Alexion Pharmaceuticals, Inc.
- Study start
- Jan 2011 (actual)
- Primary completion
- Sep 2014 (actual)
- Study completion
- Jun 2015 (actual)
- First posted
- 25 Feb 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.
Evaluation of the efficacy and safety of eculizumab in symptomatic or transfusion-dependent patients with untreated or refractory hemolytic cold agglutinin disease(CAD)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eculizumab | Experimental | Drug: Eculizumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Individuals at least 18 years of age
- Diagnosis of CAD defined by the combination of chronic hemolysis, cold agglutinin titer (IgM or IgA) >64 at 4°C, positive direct antiglobulin test (DAT) when performed with polyspecific antiserum, negative (or only weakly positive) anti-IgG, and strongly positive with anti-C3d
- LDH level > 2 x upper limit of normal (ULN)
- Clinical symptoms requiring treatment, such as anemia, anemia-related symptoms or hemolysis-related symptoms
- Sufficient supplementation of iron, folic acid and vitamin B12 prior to treatment phase
Key exclusion criteria (9 listed)
- Active aggressive lymphoma requiring therapy or an active non-lymphatic malignant disease other than basal cell carcinoma or CIS of the cervix
- Liver disease with elevated LDH
- Absolute neutrophil count < 500/µL
- Presence or suspicion of active bacterial or viral infection, in the opinion of the Investigator, at treatment start or severe recurrent bacterial infections
- Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period
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 |
|---|---|---|
| EculizumabWeekly treatment with eculizumab (600 mg) via IV infusion for 4 doses followed the next week by eculizumab (900 mg) IV infusion and maintenance treatment with Eculizumab (900 mg) via IV infusion every other week for 21 weeks | Drug | Eculizumab |
Primary outcome
LDH
Time frame: From baseline to 26 weeks
Primary endpoint for this study is: lactate dehydrogenase (LDH) in U/l change from baseline to 26 weeks
12 secondary outcomes
Transfusion avoidance
Time frame: From baseline to 26 weeks
PRBC units transfused
Time frame: From baseline to 26 weeks
Change in hemoglobin levels
Time frame: From baseline to 26 weeks
Haptoglobin
Time frame: From baseline to 26 weeks
Hemopexin
Time frame: From baseline to 26 weeks
Free hemoglobin
Time frame: From baseline to 26 weeks
Reticulocytes
Time frame: From baseline to 26 weeks
SF-36v2 QLQ
Time frame: From baseline to 26 weeks
FACIT-F SCALE version 4
Time frame: From baseline to 26 weeks
Six-Minute Walk Test
Time frame: From baseline to 26 weeks
Circulatory symptoms
Time frame: From baseline to 26 weeks
Thrombosis record
Time frame: From baseline to 26 weeks
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 19 Jul 2017.
- Background: PubMed 19372265Roth A, Huttmann A, Rother RP, Duhrsen U, Philipp T. Long-term efficacy of the complement inhibitor eculizumab in cold agglutinin disease. Blood. 2009 Apr 16;113(16):3885-6. doi: 10.1182/blood-2009-01-196329. No abstract available.
- Background: PubMed 19614954Roth A, Duhrsen U. Cold agglutinin disease. Eur J Haematol. 2010 Jan 1;84(1):91. doi: 10.1111/j.1600-0609.2009.01320.x. Epub 2009 Jul 14. No abstract available.
- Derived from this study: PubMed 30291112Roth A, Bommer M, Huttmann A, Herich-Terhurne D, Kuklik N, Rekowski J, Lenz V, Schrezenmeier H, Duhrsen U. Eculizumab in cold agglutinin disease (DECADE): an open-label, prospective, bicentric, nonrandomized phase 2 trial. Blood Adv. 2018 Oct 9;2(19):2543-2549. doi: 10.1182/bloodadvances.2018024190.
Related trials for Cold Agglutinin Disease
RecruitingPhase 1
OM336 in Autoimmune Cytopenias
AIHA - Warm Autoimmune Hemolytic Anemia, AIHA - Cold Autoimmune Hemolytic Anemia
Active, not recruitingObservational study
Cold Agglutinin Disease Real World Evidence Registry
Cold Agglutinin Disease (CAD), Cold Agglutinin Syndrome (CAS)
RecruitingPhase 1
Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Male and Female Subjects and Safety, Tolerability, Pharmacokinetics, and Pilot Efficacy Biomarkers in Subjects With Cold Agglutinin Disease
First in Man Study to Evaluate Initial Safety
Active, not recruitingPhase 2
Zanubrutinib in Primary Cold Agglutinin Disease
Lymphoma
Not yet recruitingPhase 1
Inaticabtagene Autoleucel Injection in the Treatment of Autoimmune Hemolytic Anemia After Three or More Lines of Therapy
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.
Alexander Roeth, MD principal investigator
Department of Hematology, University Hospital Essen
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 Eculizumab in people with Cold Agglutinin Disease, run by University Hospital, Essen at 1 site in Germany.
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 9 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 19 Jul 2017. 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 Germany. 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?
LDH (time frame: From baseline to 26 weeks). 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 NCT01303952, which also lists 3 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 19 Jul 2017.