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| [email protected] |
| Novartis Pharmaceuticals | |
| 1-888-669-6682 | |
| [email protected] |
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> 16 Years
HbSS
Phase 2
Interventional
All
Drug
Unknown
The goal of the study is to compare the efficacy and safety of crizanlizumab + standard of
care to standard of care alone on renal function in sickle cell disease patients ≥ 16 years
with chronic kidney disease due to sickle cell nephropathy.
– Confirmed diagnosis of SCD (HbSS and HbSβ0-thal SCD genotypes are eligible)
– Patients with eGFR ≥ 45 to ≤ 140 mL/min/1.73 m2 based on CKD EPI formula (patients ≥
18) or the Creatinine-based “Bedside Schwartz” equation (patients < 18)
- Patients with ACR of ≥ 100 to < 2000 mg/g (taken as an average of the three screening
ACR values to determine eligibility)
- Receiving at least 1 standard of care drug(s) for SCD-related CKD: If receiving HU/HC,
the patient must have been receiving HU/HC for at least 6 months and on a stable dose
for 3 months, and/or an ACE inhibitor and/or ARB for 3 months and on a stable dose for
those 3 months.
- Hb ≥ 4.0 g/dL, absolute neutrophil count (ANC) ≥ 1.0 x 10^9/L, and platelet count ≥ 75
x 10^9/L
- Adequate hepatic function as defined by:
- Alanine aminotransferase (ALT) < 3.0 x upper limit of normal (ULN)
- Direct (conjugated) bilirubin ≤ 3.0 x ULN
- Written informed consent (or assent/ parental consent for minor subjects) prior to any
screening procedures
– History of stem cell transplant
– Patients with evidence of AKI within 3 months of study entry (can decrease interval to
within 6 weeks of study entry only if renal function has returned to pre-AKI values
prior to study entry)
– Blood pressure > 140/90 mmHg despite treatment
– Patients undergoing renal replacement therapy (ie. hemodialysis, peritoneal dialysis,
hemofiltration and kidney transplantation)
– Received blood products within 30 days of Week 1 Day 1
– Participating in a chronic transfusion program
– History of kidney transplant
– Patients with hypoalbuminemia
– Body mass index of ≥ 35
– Currently receiving or received voxelotor within 6 months of screening
– Patient has received crizanlizumab and/or other selectin inhibitor or plans to receive
it during the duration of the study.
Other protocol-defined inclusion/exclusion criteria may apply