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ACP-001

Phase 2

Adult Growth Hormone Deficiency | Small molecule | Endocrine |Ascendis Pharma A/S|Last Updated: Mar 9, 2017

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 Enrollment37
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01247675A Safety, Pharmacokinetic and Pharmacodynamic Study of ACP-001 (TransCon hGH) in Adults With Growth Hormone DeficiencyPHASE2 COMPLETED 37Nov 1, 2010May 1, 2011Mar 9, 20174 Denmark, Germany +2
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Study Endpoints
Primary Endpoints
Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)
Start of study treatment through Week 4

Assessment of local tolerability was performed by examining injection sites by the investigator during study visits, and on the basis of records in the Patient Diary. Assessments included erythema, swelling, or pain.

Incidence of Treatment Emergent Anti-hGH Binding Antibody Formation
Start of study treatment through Day 42

Number of subjects with treatment emergent anti-hGH binding antibodies

Secondary Endpoints
Cmax of hGH
Days 22 to 29
Emax of IGF-I
Days 22 to 29
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ACP-001, 0.02 mg hGH/kg/wkEXPERIMENTALOnce weekly subcutaneous injection of ACP-001 equivalent to 0.02 mg hGH/kg/week for 4 weeks
ACP-001, 0.04 mg hGH/kg/wkEXPERIMENTALOnce weekly subcutaneous injection of ACP-001 equivalent to 0.04 mg hGH/kg/week for 4 weeks
ACP-001, 0.08 mg hGH/kg/wkEXPERIMENTALOnce weekly subcutaneous injection of ACP-001 equivalent to 0.08 mg hGH/kg/week for 4 weeks
Omnitrope, 0.04 mg hGH/kg/wkACTIVE_COMPARATOROnce daily subcutaneous injection of human Growth Hormone (Omnitrope) equivalent to 0.04 mg hGH/kg/week for 4 weeks
Interventions
NameTypeDescription
ACP-001 (TransCon hGH)DRUGs.c., weekly injection
OmnitropeDRUGs.c., daily injection
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Eligibility Criteria
Age Range20 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Male or female between 20 to 70 years * Body Mass Index (BMI, kg/m2) of 19.0 to 36.0 kg/m2, both inclusive * Adult Growth Hormone Deficient (AHGD) patients with documented growth hormone deficiency as defined in the Consensus guidelines for the diagnosis and treatment of adult...

Countries:DenmarkGermanyItalySweden
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