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AEZS-130

Phase 3

Diagnosis of Adult Growth Hormone Deficiency (AGDH) | Small molecule | Endocrine |COSCIENS Biopharma Inc.|Last Updated: Jul 23, 2019

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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment101
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00448747Investigation of a New, Oral Growth Hormone Secretagogue, AEZS-130 as a Growth Hormone Stimulation Test.PHASE3 COMPLETED 101Jun 1, 2007Jul 1, 2011Jul 23, 201913 United States
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Study Endpoints
Primary Endpoints
Receiver Operating Characteristic (ROC) Analysis on Peak GH (Growth Hormon) Concentrations
GH sampling: pre-dose and 30, 45, 60, 75, 90, 120, 150 min post-dose

The primary endpoint for each individual is the peak GH concentration following AEZS-130 (macimorelin) administration.

Secondary Endpoints
Peak Insulin-Like Growth Factor (IGF)-1 Concentration Following Treatment
15 min. before macimorelin administration and at 150 min after macimorelin administration
Classification and Regression Tree (CART) Analysis of Peak Growth Hormone (GH) Following Macimorelin Administration
GH sampling: pre-dose and 30, 45, 60, 75, 90, 120, 150 min post-dose
Number of Participants With Drug Related Adverse Events (AEs)
14 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeDIAGNOSTIC
Treatment Arms
ArmTypeDescription
AEZS-130 ( formerly ARD-07)EXPERIMENTALA single oral administration of AEZS-130 (0.5 mg/kg po) as Growth Hormone Stimulation Test
L-ARG+GHRHACTIVE_COMPARATORThis trial was set up as a multi-center, randomized, cross-over study investigating AEZS-130 as a Growth Hormone Stimulation Tests in terms of safety and efficacy compared to L-ARG+GHRH. When GHRH became unavailable on the US market, this comparator arm was no longer available, which was addressed by Amendment No. 3 (version 27-March-2010). Control subject enrolled under Amendment No. 3 were not randomized as there was no cross-over due to unavailability of L-ARG+GHRH. These control subjects received only AEZS-130
Interventions
NameTypeDescription
AEZS-130 (formerly ARD-07)DRUGA single oral administration of AEZS-130 as Growth Hormone Stimulation Test
L-ARG+GHRHDRUGA single administration of L-ARG+GHRH (iv bolus) followed by a 30min infusion of L-ARG as Growth Hormone Stimulation Test
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersYes
Study Sites13

Inclusion for Matched Control Subjects: * Undergone normal growth and development * Normal serum prolactin (PRL) concentrations * Females should have a history of regular, age-appropriate menses * Males should have normal serum testosterone concentrations * Matched GHD subject already enrolled in s...

Countries:United States
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