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Safety, Tolerability and PK/PD Study in Young Adult Patients With Childhood Onset Growth Hormone Deficiency (GHD).

Phase 2
Completed
Conditions
Growth Hormone Deficiency
Interventions
Registration Number
NCT00778518
Lead Sponsor
Ambrx, Inc.
Brief Summary

Study to find the optimal dose of Growth Hormone Replacement in young adult patients suffering from childhood onset growth hormone deficiency (GHD).

Detailed Description

The primary objective of this study is to evaluate the safety, tolerability and pharmacodynamic response of three different ARX201 doses (PEG-ahGH) when administered repeatedly to young adult patients with childhood onset growth hormone deficiency (GHD).

Recruitment & Eligibility

Status
COMPLETED
Sex
Male
Target Recruitment
36
Inclusion Criteria
  • 18-30 years old
  • GHD of childhood onset
  • completed growth
  • IGF-1 <=2SDS
  • rhGH treatment naive
  • hGH levels below cut-off
Exclusion Criteria
  • History of malignancy or intracranial tumors
  • ECG abnormality
  • ICH
  • hepatic dysfunction
  • renal impairment
  • major medical conditions
  • inadequate T4
  • positive for HBV, HCV, or HIV
  • alcohol or drug abuse

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
3ARX201High Dose
1ARX201low dose
2ARX201Medium dose
Primary Outcome Measures
NameTimeMethod
Temporal profiling of circulating IGF-1 levels.3 period
Secondary Outcome Measures
NameTimeMethod
Body composition measurements at start of study and end of study6 months

Trial Locations

Locations (1)

Accelsiors CRO and Consultancy Services

🇭🇺

Budapest, Hungary

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