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PEG-Asparaginase

Phase 1

Acute Lymphoblastic Leukemia | Small molecule | Oncology |Novartis AG|Last Updated: Jul 18, 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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment22
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01523977Everolimus With Multiagent Re-Induction Chemotherapy in Pediatric Patients With ALLPHASE1 COMPLETED 22Nov 1, 2011Nov 1, 2018Jul 18, 20248 United States
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Study Endpoints
Primary Endpoints
Safety and Feasibility
2 years

To determine the safety and feasibility of treatment with everolimus in combination with vincristine, prednisone, PEG-asparaginase and doxorubicin in patients with relapsed acute lymphoblastic leukemia (ALL)

Secondary Endpoints
Clinical Activity
2 years
Impact on biologic markers
2 years
Determinants of Response
2 years
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Interventions
NameTypeDescription
EverolimusDRUGOrally, daily days 1-32 per assigned dose level
PrednisoneDRUG40 mg/m2/day orally 3 x daily days 4-32
VincristineDRUG1.5 mg/m2 IV daily on days 4, 11, 18, and 25
PEG-AsparaginaseDRUG2,500 U/m2 IV 1 x daily on days 5 and 18
DoxorubicinDRUG30 mg/m2 IV on days 4 and 5
DexrazoxaneDRUG300 mg/m2 IV on days 4 and 5
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Eligibility Criteria
Age Range18 Months — 21 Years
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * ALL in first bone marrow relapse occuring \> 18 months from initial diagnosis * Normal organ function * Maximum prior cumulative doxorubicin dose of \</= 360 mg/m2 or equivalent Exclusion Criteria: * Prior therapy for ALL except for intrathecal (IT) chemotherapy * Pregnant o...

Countries:United States
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