IV formulation with 30 min infusion, 5mg/kg dosage
Other Name: SEG101
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 signature.
- 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 these treatments.
- 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 permitted.
- 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 reaction.
- 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 immunosuppressive drugs)
- 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
Expanded Access program information for companies listed in the Expanded Access Company Directory is pulled daily from ClinicalTrials.gov, a resource provided by the National Institutes of Health in cooperation with the U.S. Food and Drug Administration. Click here for more information about ClinicalTrials.gov.