Health Condition 1: - Health Condition 2: D758- Other specified diseases of bloodand blood-forming organs
Conditions
Interventions
Intervention1: Drug: Crizanlizumab -Crizanlizumab (SEG101): SEG101 (crizanlizumab) drug administered at a dose of 5.0 mg/kg on Week 1 Day 1, Week 3 Day 1 and Day 1 of every 4-week cycle.
Control Inter
Sponsors
Novartis Healthcare Pvt Ltd
Eligibility
Inclusion criteria
Inclusion criteria: 1 Confirmed diagnosis of sickle cell disease (SCD) (e.g. any genotype including HbSS, HbSC, HbS�²0-thalassemia, HbS�²+-thalassemia, and others) by hemoglobin electrophoresis or high-performance liquid chromatography (HPLC) performed locally. 2 Experienced at least 1 VOC within the preceding 12 months, as determined by medical history. Prior VOC must have resolved at least 7 days prior to the first dose in the study and should include all the following: a.the occurrence of appropriate symptoms (see VOC definition in protocol Section 7.2.1.1) b.either a visit to a medical facility or healthcare professional, c.receipt of oral/parenteral opioid or other non-opioid parenteral analgesia. 3 If receiving HU/HC or erythropoietin stimulating agent, must have been receiving the drug for at least 6 months prior to Screening and plan to continue taking at the same dose and schedule during the trial. Dose alterations of HU/HC during Part A are not allowed, and if this occurs, the patient will enter directly to the Part B. 4 Received standard age-appropriate care for SCD, including penicillin prophylaxis, pneumococcal immunization, and parental education 5 Transcranial Doppler (TCD) considered low risk within the past 6 months (for 2 to 16 years).
Exclusion criteria
Exclusion criteria: 1 History of stem cell transplant. 2 Received any blood products within 30 days of Day 1 dosing. 3 Participating in a chronic transfusion program (preplanned series of transfusions for prophylactic purposes). 4 Patients with bleeding disorders 5 Planning on undergoing an exchange transfusion during the duration of the study. Patients requiring episodic transfusion in response to worsened anemia or VOC are permitted. 6 Contraindication or hypersensitivity to any drug from similar class as study drug or to any excipients of the study drug formulation. 7 Received a monoclonal antibody or immunoglobulin-based therapy within 6 months of Screening, or has documented immunogenicity to a prior monoclonal antibody. 8 Received active treatment on another investigational trial within 30 days (or 5 half lives of that agent, whichever is greater) prior to Screening or plans to participate in another investigational drug trial. 9 Pregnant females or females who have given birth within the past 90 days or who are breastfeeding. 10 Any documented history of a stroke or intracranial hemorrhage, or an uninvestigated neurologic finding within the past 12 months 11 Any conditional TCD within the past 12 months 12 Use of therapeutic anticoagulation (prophylactic doses permitted) or antiplatelet therapy (other than aspirin) within the 10 days prior to Week 1 Day 1 dosing 13 Hospitalized at Screening 14 Planning to undergo a major surgical procedure during the duration of the study 15 Planning to initiate or terminate HU/HC while on study, other than for safety reasons 16 Patient with active HIV infection (detectable viral load) 17 Patients with known active Hepatitis B infection. 18 Patients with known Hepatitis C history. 19 Significant active infection or immune deficiency (including chronic use of immunosuppressive drugs) in the opinion of the investigator. 20 Malignant disease. Exceptions to this exclusion include the following: malignancies that were treated curatively and have not recurred within 2 years prior to study treatment; any completely resected carcinoma in situ. 21 Has a serious mental or physical illness, which, in the opinion of the Investigator would compromise participation in the study. 22 Resting QTcF �450 msec at pretreatment (baseline) for patients under 12 years of age and �450 msec for males and �460 msec for female patients 12 years and older. 23 Cardiac or cardiac repolarization abnormality 24 Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome 25 Sexually active females who are unwilling to comply with reliable method of birth control until 15 weeks following last dose of study drug.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| ââ?¬Â¢AUC d15 for PK and PD parameters ââ?¬Â¢AUCtau for PK and PD parameters ââ?¬Â¢Cmax for PK parameters Timepoint: Week 1 and Week 15 | — |
Secondary
| Measure | Time frame |
|---|---|
| ââ?¬Â¢Number of Vaso Occusive Crisis (VOC) events leading to healthcare visit in clinic/ER/hospital ââ?¬Â¢Number of Vaso Occusive Crisis (VOC) events treated at home (based on documentation by health care provider following phone contact with the patient ââ?¬Â¢Number of each subcategory of VOC event (uncomplicated pain crisis, acute chest syndrome, hepatic sequestration, splenic sequestration, priapism ââ?¬Â¢Number of hospitalizations and ER visits (both overall and VOC-related) Timepoint: 6 months, 2 years | — |
Countries
Belgium, Brazil, Canada, Colombia, France, Germany, India, Italy, Lebanon, Netherlands, Oman, Spain, Switzerland, Turkey, United Kingdom, United States of America
Contacts
Public ContactMurugananthan K
Novartis Healthcare PVT LTD
Outcome results
None listed