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MAXOMAT , biosynthetic growth hormone

Phase 3

Noonan Syndrome | Small molecule | Other |Sanofi|Last Updated: Oct 5, 2010

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
UNCONTROLLED
Total Trials1
Total Enrollment36
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00452725Effect of MAXOMAT ® on the Growth of Small Children to NOONAN's SyndromePHASE3 COMPLETED 36Oct 1, 1997Jul 1, 2010Oct 5, 20101 France
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Study Endpoints
Primary Endpoints
Evaluation of height : gain in height and rate of growth
at each visit (every 3 months) or every 6 months
Secondary Endpoints
Clinical and laboratory test safety
every 6 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Interventions
NameTypeDescription
MAXOMAT ®, biosynthetic growth hormoneDRUG2 posologies according to age (children and adolescents) treatment is planned for a 2 year duration
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Eligibility Criteria
Age Range3 Years — 15 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Children and adolescents with Noonan's syndrome with a height \< -2 SD and no progressive cardiopathy Exclusion Criteria: * Age \< 3 years * Height ≥ -2 SD The above information is not intended to contain all considerations relevant to a patient's potential participation in...

Countries:France
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