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Crizanlizuamb

Phase 2

Sickle Cell Disease (SCD) | Small molecule | Hematology |Novartis AG|Last Updated: Oct 9, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment58
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04053764Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell DiseasePHASE2 COMPLETED 58Dec 10, 2019Mar 20, 2023Oct 9, 202424 United States, Brazil +9
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Study Endpoints
Primary Endpoints
Percentage of Participants With ≥ 30% Decrease in Albuminuria (ACR) at 12 Months
Baseline to 12 months

The effect of SEG101 on clinical disease activity was measured by at least 30% decrease in Albumin to Creatinine Ratio (ACR) from baseline to month 12. A reduction from baseline indicates improvement in patients.

Secondary Endpoints
Change From Baseline in Albuminuria (ACR) at 3, 6, 9 and 12 Months
Baseline to 3, 6, 9, and 12 months
Percentage of Participants With ≥ 30% Decrease in Albuminuria (ACR) at 6 Months
Baseline to 6 months
Percentage of Participants With Protein/Creatinine Ratio (PCR) Improvement and Stable PCR at 12 Months
Baseline to 12 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
crizanlizumab + standard of careEXPERIMENTAL5 mg/kg by intravenous (i.v.) infusion at Week 1 Day 1, Week 3 Day 1 and Day 1 of every 4-week cycle until Week 51 in addition to their usual standard of care treatment.
standard of careACTIVE_COMPARATORPatients in the standard of care alone arm will continue to receive their usual standard of care treatment.
Interventions
NameTypeDescription
CrizanlizuambDRUGCrizanlizumab is a concentrate for solution for infusion, i.v. use. Supplied in single use 10 mL vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab
Standard of CareDRUGHU/HC (hydroxyurea/hydroxycarbamide) and/or ACE (angiotensin-converting enzyme) inhibitors and/or ARBs (angiotensin-receptor blocker)
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites24

Inclusion Criteria: * 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 m...

Countries:United StatesBrazilFranceGreeceIrelandLebanonNetherlandsPanamaSpainTurkey (Türkiye)United Kingdom
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