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 TRANSFUSIO

Phase 3

Completed

• Conditions: Primary Cold Agglutinin Disease; 10018911; 10003816

• Registration Number: NL-OMON50716
• Lead Sponsor: Bioverativ USA, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 6

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.
11. Adequate IV access
12. If female, must be post-menopausal, surgically sterile, or be established
on (>= 3 months prior to Screening)
and agree to continue to use the same highly effective methods of birth control
throughout the study and for 6
weeks following administration of the last dose of study drug
13. Males must be surgically sterile for at least 90 days or when sexually
active with female partners of childbearing
potential will agree to use highly effective contraception from Day 0 until 6
weeks days following
administration of the last dose of study drug.
14. Able to comprehend and give informed consent
15. Able to comply with the requirements of the study and to complete
the full sequence of protocol-related procedures.

Exclusion Criteria

1.Cold agglutinin syndrome secondary to infection, rheumatologic
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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
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary efficacy endpoint is the responder rate as defined in Table 2.</p><br>