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

Growth Hormone Deficiency Clinical Trial

Official title:

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).

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00778518
• Other study ID #: PRO-ARX201-701
• Secondary ID: EudraCT: 2007-00
• Status: Completed
• Phase: Phase 2
• First received: October 22, 2008
• Last updated: October 9, 2009
• Start date: July 2008
• Est. completion date: October 2009

Study information

• Verified date: October 2009
• Source: Ambrx, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Belarus: Ministry of HealthHungary: National Institute of PharmacyRomania: National Medicines AgencySerbia and Montenegro: Agency for Drugs and Medicinal DevicesUkraine: State Pharmacological Center - Ministry of Health
• Study type: Interventional

Clinical Trial Summary

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

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 information / eligibility

• Status: Completed
• Enrollment: 36
• Est. completion date: October 2009
• Est. primary completion date: October 2009
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 30 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Pharmacokinetics/Dynamics Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Subject), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency

Intervention

Drug:
• ARX201
Reconstituted lyophilized drug product at 2.5 mg/wk; 3 periods over six months.
• ARX201
Reconstituted lyophilized drug product at 5.0 mg/wk; 3 periods over six months.
• ARX201
Reconstituted lyophilized drug product at 10.0 mg/wk; 3 periods over six months.

Locations

Country	Name	City	State
Hungary	Accelsiors CRO and Consultancy Services	Budapest

Sponsors (2)

Lead Sponsor	Collaborator
Ambrx, Inc.	Merck Serono International SA

Country where clinical trial is conducted

Hungary, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Temporal profiling of circulating IGF-1 levels.		3 period	Yes
Secondary	Body composition measurements at start of study and end of study		6 months	No