18 to 70 Years
Written patient informed consent must be obtained prior to start of treatment.
– Male or female, 18 to 70 years of age (inclusive) on the day of informed consent
– Confirmed diagnosis of sickle cell disease by hemoglobin electrophoresis or high
performance liquid chromatography (HPLC) [performed locally]. All sickle cell disease
genotypes are eligible (HbSS, HbSβ0, HbSC, HbSβ+, and others).
– History of reoccurring VOC as assessed by the Treating Physician.
– Patients receiving HU/HC, L-glutamine (Endari), erythropoietin stimulating agents or
other therapies as prevention therapy and continue to experience VOC while on any of
– Patients can continue taking the preventive therapy.
– Patient is not a candidate to be treated with alternative treatment options or has
discontinued alternative treatments due to unacceptable benefit risk as documented by
the Treating Physician.
– Patient must meet the following laboratory values prior to treatment:
– Absolute Neutrophil Count ≥1.0 x 109/L
– Platelets ≥75 x 109/L
– Hemoglobin (Hgb) ≥4.0 g/dL
– Estimated glomerular filtration rate ≥ 45 mL/min
– Direct (conjugated) bilirubin ≤2.0 x ULN
– Alanine transaminase (ALT) ≤ 3.0 x ULN
– ECOG performance status ≤ 2
Patients eligible for this Treatment Plan must not meet any of the following criteria:
– Is on a chronic transfusion program as defined by participating in a scheduled
(pre-planned) series of transfusions (simple or exchange). Episodic transfusions are
– Contraindication or hypersensitivity to any drug or metabolites from similar class as
crizanlizumab drug or to any excipients of the drug formulation.
– History of severe hypersensitivity reaction to other monoclonal antibodies, which in
the opinion of the Treating Physician may pose an increased risk of serious infusion
– Use of therapeutic anticoagulation (prophylactic doses permitted) or antiplatelet
therapy (other than aspirin or NSAIDs) within the 10 days prior to starting treatment
– Patient has no acute pathologic processes.
– Received a monoclonal antibody or immunomodulatory agent within 1 year of starting
treatment, or has documented immunogenicity to a prior biologic.
– Pregnant or nursing women
– Patients with bleeding disorders
– Known history of testing positive for Human Immunodeficiency Virus (HIV) infection
– Patients with active Hepatitis B infections (HBsAg positive)
o Note: Patients with antecedent but no active Hepatitis B (i.e. anti-HBc positive,
HBsAg and HBV-DNA negative) are eligible
– Significant active infection or immune deficiency (including chronic use of
– Has a serious mental or physical illness, which, in the opinion of the Treating
Physician would compromise compliance to treatment.
– QTcF ≥470 msec prior to treatment
Other protocol-defined inclusion/exclusion criteria may apply