Managed Access Program (MAP) to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients With History of Vaso-occlusive Crisis
MAP to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients
Sponsors
Source
Novartis
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.
Overall Status
Available
Start Date
N/A
Completion Date
N/A
Primary Completion Date
N/A
Study Type
Expanded Access
Condition
Intervention
Intervention Type
Drug
Intervention Name
Description
Enter formulation, drug 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
Gender
All
Minimum Age
18 Years
Maximum Age
N/A
Healthy Volunteers
No
Overall Contact
Verification Date
2018-10-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Keywords
Condition Browse
Firstreceived Results Date
N/A
Overall Contact Backup
Last Name
Novartis Pharmaceuticals
Firstreceived Results Disposition Date
N/A
Expanded Access Info
Expanded Access Type Treatment
Yes
Study First Submitted
October 24, 2018
Study First Submitted Qc
October 24, 2018
Study First Posted
October 25, 2018
Last Update Submitted
October 24, 2018
Last Update Submitted Qc
October 24, 2018
Last Update Posted
October 25, 2018
ClinicalTrials.gov processed this data on October 26, 2018
Conditions
Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov,
conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.