Recent Updates
Recently added Catalysts

perifosine + temsirolimus

Phase 1

Pediatric Solid Tumors | Small molecule | Oncology |COSCIENS Biopharma Inc.|Last Updated: Mar 10, 2017

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment23
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01049841Perifosine With Temsirolimus for Recurrent Pediatric Solid TumorsPHASE1 COMPLETED 23Jan 1, 2010Mar 1, 2017Mar 10, 20171 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Maximum Tolerated Dose (MTD) of perifosine + temsirolimus combination in children with cancer.
2 years
Secondary Endpoints
To determine whether pharmacokinetic serum levels of both perifosine and temsirolimus correlate with toxicity.
2 years
To record preliminary data on the efficacy of the perifosine + temsirolimus combination.
2 years
If previously resected tissue is available, determine whether molecular features predict response including, Elevated PI3K/AKT/mTOR signaling, Elevated RAS/MAPK signaling, Cell cycle markers
2 years
Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
perifosine + temsirolimusEXPERIMENTALThis is a single arm, phase I study. Eligible patients will receive a loading dose of oral perifosine on the first day, followed by a maintenance dose starting on the second day until progression. Each patient is assigned to a group according to their body surface area (BSA). Temsirolimus will be combined with perifosine at four dose levels to determine the MTD for the combination therapy. Temsirolimus dosing will start on the same day as the perifosine load.
Interventions
NameTypeDescription
perifosine + temsirolimusDRUGThe patient will take oral tablets of perifosine at a dose and frequency to be determined their height, weight and time when they enter the study as those entering early will be treated with lower dose levels (dosing frequency will vary from once a week to daily). The patient will receive an intravenous injection of temsirolimus once a week at a dose to be determined by their height, weight and time when they enter the study as those entering early will be treated with lower doses. In addition they will be asked to keep a medicine diary.
Unlock Study Design Details
Eligibility Criteria
Age RangeN/A — 21 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Any solid tumor that has failed standard therapy * Patient must have evidence of tumor by CT, MRI, MIBG scan, serum markers, or tissue sampling. * Age ≤ 21 years (age ≤ 35 years for biopsy proven medulloblastoma or neuroblastoma) * Karnofsky/Lansky performance status ≥ 50% (Ka...

Countries:United States
Unlock Eligibility Criteria