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CSJ137

Phase 1

Anemia, Iron-Deficiency | Monoclonal antibody | Hematology |Novartis AG|Last Updated: Oct 12, 2021

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
RandomizedPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment40
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02570854A Two-part Single- and Repeat-dose Study of CSJ137 in Anemic Chronic Hemodialysis PatientsPHASE1 COMPLETED 40Sep 22, 2015May 13, 2020Oct 12, 202113 United States, Czechia +2
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Study Endpoints
Primary Endpoints
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
baseline through 115 days after CSJ137 is administered

safety and tolerability following administration of CSJ137

Minimum active dose of CSJ137 determined by assessment of levels of hemoglobin in blood, without evidence of liver dysfunction or other safety concerns.
Hemoglobin response at 28 days post-dose

to determine the minimum dose of CSJ137 that is active for treatment

Secondary Endpoints
Peak concentration (Cmax) of CSJ137 in serum
before CSJ137 is administered, then 0.5 hours and 6 hours after CSJ137 is administered on Day 1. Also 1, 2, 3, 5, 12, 19, 28, and 84 days after CSJ137 is administered
Area under the serum concentration versus time curve (AUC)
before CSJ137 is administered, then 0.5 hours and 6 hours after CSJ137 is administered on Day 1. Also 1, 2, 3, 5, 12, 19, 28, and 84 days after CSJ137 is administered
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CSJ137EXPERIMENTALIn Part 1 up to 48 subjects will receive a single dose of CSJ137. In Part 2, up to 40 patients will be randomized to one of 2 arms with equal allocation: one CSJ137 dose arm from Part 1 and a placebo arm. Each patient in Part 2 will receive up to 2 doses (repeat dose).
PlaceboPLACEBO_COMPARATORIn Part 2, up to 40 patients will be randomized to one of 2 arms with equal allocation: one CSJ137 dose arm from Part 1 and a placebo arm. Each patient in Part 2 will receive up to 2 doses (repeat dose).
Interventions
NameTypeDescription
CSJ137BIOLOGICALStarting dose is 0.010 mg/kg and dose escalation will proceed with semi-log increase steps to a maximum dose level 10 mg/kg. Subjects receive the treatment via up to 30 minutes intravenous infusion.
PlaceboDRUGSubjects will be dosed with a matching placebo (vehicle control) via up to 30 minutes intravenous infusion.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: 1. Hemodialysis-dependent for at least 2 months prior to screening. 2. Receiving hemodialysis at least 2 times per week 3. Receiving erythropoietin (EPO) therapy. 4. Hemoglobin (Hgb) ≥ 8.5 and \< 11.5 g/dL at screening. 5. Ferritin \>500 ng/mL and ≤ 2000 ng/mL at screening. 6. T...

Countries:United StatesCzechiaIsraelUnited Kingdom
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