Novartis Pharmaceuticals

MAP to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients

Official Title: 
Managed Access Program (MAP) to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients With History of Vaso-occlusive Crisis
Brief Summary: 
The purpose of this Cohort Treatment Plan is to allow access to crizanlizumab (SEG101) for eligible patients diagnosed with sickle cell disease (SCD) to prevent or reduce the frequency of vaso-occlusive crises (VOC). The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
Available
EA Type
Treatment IND/Protocol
Sickle Cell Disease

Drug: crizanlizumab
IV formulation with 30 min infusion, 5mg/kg dosage
Other Name: SEG101

Eligibility Criteria: 

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

Eligibility Gender: 
All
Eligibility Age: 
Minimum: 18 Years
Contacts: 

Novartis Pharmaceuticals
+41613241111
novartis.email@novartis.com

Novartis Pharmaceuticals

Novartis Pharmaceuticals
EA Number: 
Keywords: 
Sickle cell disease, sickle cell anemia, vaso-occlusive crisis, P-selectin, SEG101, crizanlizumab, monoclonal antibody, Anemia, Sickle Cell, HbS Disease, Hemoglobin SC Disease, Sickle Cell Disorders, Sickling Disorder Due to Hemoglobin S, Adult, Hb SS, Hb SC, Hb Sβ+-thalassemia, Hb Sβ0-thalassemia, Expanded Access Use, Pain Crisis, Compassionate Use
MeSH Terms: 
Anemia, Sickle Cell
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