Managed Access Program (MAP) to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients With History of Vaso-occlusive Crisis

Study point of contact

Novartis Pharmaceuticals
Novartis Pharmaceuticals
+41613241111
[email protected]

Age

18 to 70 Years

Genotypes

HbSS, HbSC

Study type

Expanded Access

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

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.

participation requirements

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

participation restrictions

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