An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Vaso-occlusive Crises.

Study point of contact

Novartis Pharmaceuticals
Novartis Pharmaceuticals
+41613241111
[email protected]

Age

> 16 Years

Phase

Phase 4

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

Sickle cell disease (SCD) is a genetic blood disorder. Crizanlizumab has been approved in
India and other countries to reduce the frequency of vaso-occlusive crises (VOCs) in patients
with SCD aged 16 years and older.

The purpose of this local Phase IV study is to evaluate the safety of crizanlizumab
specifically in Indian patients with SCD aged 16 years or older with a history of VOC leading
to healthcare visit.

participation requirements

– Signed informed consent

– Male or female participant aged 16 years and older

– Confirmed diagnosis of SCD by hemoglobin electrophoresis or high performance liquid
chromatography (HPLC). All SCD genotypes are eligible.

– History of VOC leading to healthcare visit prior to screening visit

– Participants must meet the following central laboratory values at the screening visit:

Absolute Neutrophil Count ≥1.0 x 109/L Platelet count ≥75 x 109/L Hemoglobin: for adults
(Hb) ≥4.0 g/dL and for adolescents (Hb) ≥5.5 g/dL Glomerular filtration rate ≥ 45
mL/min/1.73 m2 using CKD-EPI formula Direct (conjugated) bilirubin < 2.0 x ULN Alanine Aminotransferase (ALT) < 3.0 x ULN - ECOG performance status ≤2 for adults and Karnofsky Performance Scale ≥ 50% for adolescents.

participation restrictions

– Contraindication or hypersensitivity to any drug or metabolites from similar class as
study drug. History of severe hypersensitivity reaction to other monoclonal antibodies
which in the opinion of the investigator may pose an increased risk of serious
infusion reaction.

– Participant has received crizanlizumab and/or other P-selectin inhibitor prior to the
study or plans to receive it during the duration of the study.

– Concurrent severe and/or uncontrolled medical conditions which, in the opinion of the
Investigator, could cause unacceptable safety risks or compromise participation in the
study.

– Any condition which, in the opinion of the investigator, is likely to interfere with
the successful collection of the measurements required for the study.

– Participant has documented immunogenicity to a prior biological drug.

– Participants who are on active treatment with Voxelotor, other investigational drug or
other monoclonal antibody, or intend to initiate the same during the course of the
trial.

– Pregnant females or females who have given birth within the past 90 days prior
screening or who are breastfeeding.

– Women of childbearing potential unless using highly effective methods of contraception
during dosing and for 15 weeks after stopping treatment

– Significant bleeding disorder

– Active HIV infection

– Active Hepatitis B infection

– Positive test for Hepatitis C RNA

– Malignant disease

– Active infection or immune deficiency