Open-Label with BIVV009 in patients with Cold Agglutinin Disease

Phase 1

• Conditions: 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; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2017-003538-10-ES
• Lead Sponsor: Bioverativ USA Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-11-07
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 20

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified