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

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ClinicalTrials.gov Identifier: NCT03720626
Expanded Access Status : Available
First Posted : October 25, 2018
Last Update Posted : December 4, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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.

Condition or disease Intervention/treatment
Sickle Cell Disease Drug: crizanlizumab

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Managed Access Program (MAP) to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients With History of Vaso-occlusive Crisis



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

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03720626


Contacts
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Contact: Novartis Pharmaceuticals +41613241111 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals

Sponsors and Collaborators
Novartis Pharmaceuticals

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03720626     History of Changes
Other Study ID Numbers: CSEG101A2001M
First Posted: October 25, 2018    Key Record Dates
Last Update Posted: December 4, 2018
Last Verified: December 2018
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
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
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn