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: Drug: ARX201

• 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).

Study Timeline

• Study Start: 2008-07
• Study First Submitted: 2008-10-22
• Study First Submitted QC: 2008-10-22
• Study First Posted: 2008-10-23
• Status Verified: 2009-10
• Primary Completion: 2009-10
• Study Completion: 2009-10
• Last Update Submitted: 2009-10-09
• Last Update Posted: 2009-10-12

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

Group	Intervention	Description
3	ARX201	High Dose
1	ARX201	low dose
2	ARX201	Medium dose

Primary Outcome Measures

Name	Time	Method
Temporal profiling of circulating IGF-1 levels.	3 period

Secondary Outcome Measures

Name	Time	Method
Body composition measurements at start of study and end of study	6 months

Trial Locations

Locations (1)

Accelsiors CRO and Consultancy Services; 🇭🇺 Budapest, Hungary