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

Phase IIb Study to Evaluate the Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Profile of ARX201 Following Repeated Dosing to Young Adult Patients With Childhood Onset Growth Hormone Deficiency (GHD).

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00778518

Enrollment

Registered

2008-10-23

Start date

2008-07-31

Completion date

2009-10-31

Last updated

2009-10-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Growth Hormone Deficiency

Keywords

growth hormone, growth hormone deficiency, GHD, PEG, pegylated, IGF-1

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).

Interventions

DRUGARX201

Reconstituted lyophilized drug product at 2.5 mg/wk; 3 periods over six months.

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

SINGLE (Subject)

Eligibility

Sex/Gender

MALE

Age

18 Years to 30 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Temporal profiling of circulating IGF-1 levels.	3 period

Secondary

Measure	Time frame
Body composition measurements at start of study and end of study	6 months

Countries

Hungary

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026