Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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28 February 2019 |
Main ID: |
EUCTR2017-003538-10-ES |
Date of registration:
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07/11/2017 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Open-Label with BIVV009 in patients with Cold Agglutinin Disease
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Scientific title:
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A PIVOTAL, OPEN-LABEL, MULTICENTER STUDY TO ASSESS THE
EFFICACY AND SAFETY OF BIVV009 IN PATIENTS WITH PRIMARY COLD AGGLUTININ DISEASE WHO HAVE A RECENT HISTORY OF BLOOD TRANSFUSION
- Cardinal Study |
Date of first enrolment:
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19/01/2018 |
Target sample size:
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20 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-003538-10 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
Number of treatment arms in the trial: 1
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV): no
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Countries of recruitment
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Australia
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Austria
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Belgium
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Canada
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Denmark
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France
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Germany
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Hungary
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Israel
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Italy
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Japan
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Netherlands
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Norway
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Poland
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Spain
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Switzerland
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United Kingdom
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United States
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Contacts
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Name:
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Clinical Trial Dept
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Address:
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225 Second Avenue
02451
Waltham, MA
United States |
Telephone:
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003491 1459110 |
Email:
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regulatory.spain@pharm-olam.com |
Affiliation:
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Bioverativ USA Inc. |
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Name:
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Clinical Trial Dept
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Address:
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225 Second Avenue
02451
Waltham, MA
United States |
Telephone:
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003491 1459110 |
Email:
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regulatory.spain@pharm-olam.com |
Affiliation:
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Bioverativ USA Inc. |
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Key inclusion & exclusion criteria
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Inclusion criteria: 1.Adult males and females = 18 years of age at Screening 2.Body weight of = 39 kg at screening 3.Confirmed diagnosis of primary CAgD based on the following criteria: a.Chronic hemolysis, b.Polyspecific direct antiglobulin test (DAT) positive, c.Monospecific DAT strongly positive for C3d, d.Cold agglutinin titer = 64 at 4 degrees Celsius, e.IgG DAT = 1+, and f.No overt malignant disease 4.History of at least one documented blood transfusion within 6 months of enrollment 5.Hemoglobin level = 10.0 g/dL 6.Bilirubin level above the normal reference range 7.Ferritin level within the normal reference ranges unless outside normal range and deemed not clinically significant by the Investigator (or designee) 8.Presence of one or more of the following CAgD-related signs or symptoms within 3 months of Screening: a.Symptomatic anemia defined as: i.Fatigue, ii.Weakness, iii.Shortness of breath, iv.Palpitations, fast heart beat v.Light headedness and/or vi.Chest pain b.Acrocyanosis c.Raynaud’s syndrome d.Hemoglobinuria e.Disabling circulatory symptoms, and/or f.Major adverse vascular event (including thrombosis) 9.Bone marrow biopsy within 6 months of Screening with no overt evidence of lymphoproliferative disease or other hematological malignancy. An additional bone marrow biopsy will be required if the prior bone marrow is deemed unsuitable for analysis by the Sponsor. 10.Vaccinations against encapsulated bacterial pathogens (Neisseria meningitis, Meningitis B, Haemophilus influenzae, and Streptococcus pneumoniae) within 5 years of enrollment or as specified in Appendix B. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 15 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria: 1.Cold agglutinin syndrome secondary to infection, rheumatologic disease, or active hematologic malignancy 2.Clinically relevant infection of any kind within the month preceding enrollment (eg, active hepatitis C, pneumonia) 3.Clinical diagnosis of systemic lupus erythematosus (SLE); or other autoimmune disorders with anti-nuclear antibodies at Screening 4.Positive hepatitis panel (including hepatitis B surface antigen and/or hepatitis C virus antibody) prior to or at Screening 5.Positive human immunodeficiency virus (HIV) antibody at Screening 6.Treatment with rituximab monotherapy within 3 months or rituximab combination therapies (eg, with bendamustine, fludarabine, ibrutinib, or cytotoxic drugs) within 6 months prior to enrollment 7.Concurrent treatment with corticosteroids other than a stable daily dose equivalent to = 10 mg/day prednisone for previous 3 months 8.Erythropoietin deficiency. Concurrent treatment with erythropoietin is permitted if the patient has been on a stable dose for the previous 3 months. 9.Concurrent usage of iron supplementation unless the patient has been on a stable dose for at least 4 weeks. 10.Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the patient or compromise the quality of the data derived from his/her participation in this study (as determined by the Investigator [or designee]) at Screening 11.Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 half lives, whichever is greater, prior to treatment start 12.Females who are pregnant, lactating, or, if having reproductive potential, are considered potentially unreliable with respect to contraceptive practice
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
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Health Condition(s) or Problem(s) studied
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Therapeutic area: Diseases [C] - Immune System Diseases [C20]
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Primary Cold Agglutinin Disease
MedDRA version: 20.0
Level: PT
Classification code 10073785
Term: Autoimmune haemolytic anaemia
System Organ Class: 10005329 - Blood and lymphatic system disorders
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Intervention(s)
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Product Name: BIVV009 Product Code: BIVV009 Pharmaceutical Form: Solution for infusion INN or Proposed INN: BIVV009 Current Sponsor code: BIVV009 Other descriptive name: COMPLEMENT C1 ESTERASE INHIBITOR Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 18-
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Primary Outcome(s)
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Secondary Objective: Part A: Efficacy: •To assess the effect of BIVV009 on clinical events and laboratory parameters related to hemolysis and anemia in patients with primary CAgD •To assess the effect of BIVV009 on quality of life (QOL) in patients with primary CAgD Safety: •To evaluate the overall safety and tolerability of BIVV009 in patients with primary CAgD Exploratory: •To assess the effect of BIVV009 on specific complications of CAgD •To evaluate the effect of BIVV009 on certain disease-related biomarkers in patients with primary CAgD •To evaluate the pharmacokinetics of BIVV009
Part B: •The secondary objective of Part B is to investigate the durability of response during long-term treatment with BIVV009 in patients with primary CAgD.
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Timepoint(s) of evaluation of this end point: Week 26 (EOT)
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Primary end point(s): The primary efficacy endpoint is the responder rate. A patient will be considered a responder if he or she did not receive a blood transfusion from Week 5 through Week 26 (EOT) and did not receive treatment for CAgD beyond what is permitted per protocol. Additionally, the patient’s Hgb level must meet either of the following criteria: • Hgb level is = 12 g/dL at the treatment assessment endpoint (defined as mean value from Weeks 23, 25, and 26), or • Hgb increased = 2 g/dL from baseline (defined as the last Hgb value before administration of the first dose of study drug) at treatment assessment endpoint
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Main Objective: Part A: The primary objective of Part A is to determine whether BIVV009 administration results in a = 2 g/dL increase in hemoglobin (Hgb) levels or increases Hgb to = 12 g/dL and obviates the need for blood transfusion during treatment in patients with primary CAgD who have a recent history of transfusion
Part B: The primary objective of Part B is to evaluate the long-term safety and tolerability of BIVV009 in patients with primary CAgD.
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Secondary Outcome(s)
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Secondary end point(s): •Mean change from baseline in bilirubin (excluding patients with Gilbert’s Syndrome) at the treatment assessment endpoint (mean of values at Week 23, 25, and 26) •Mean change from baseline in QOL, as assessed by the change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale scores at the treatment assessment endpoint •Mean change from baseline in lactate dehydrogenase (LDH) at the treatment assessment endpoint •Number of transfusions and number of units after the first 5 weeks of study drug administration •Mean change from baseline in Hgb at the treatment assessment endpoint
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Timepoint(s) of evaluation of this end point: Week 26 (EOT)
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Secondary ID(s)
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BIVV009-03
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128,190
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2017-003538-10-AT
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Source(s) of Monetary Support
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Bioverativ USA Inc.
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Ethics review
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Status: Approved
Approval date:
Contact:
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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