ICH GCP | Clinical Trials Registry

Novartis Pharmaceuticals 

Drug

crizanlizumab 

IV formulation with 30 min infusion, 5mg/kg dosage

SEG101

Inclusion criteria 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 Exclusion criteria 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

All

18 Years

N/A

No

Novartis Pharmaceuticals

+41613241111

Sponsor

Novartis Pharmaceuticals

Yes