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

• Sponsor: Ambrx, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00778518
• Collaborator: Merck Serono International SA (Industry)
• Enrollment: 36
• Location: 1
• Arms: 3
• Duration (Months): 15
• Patients Per Site Per Month: 2.4

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Growth Hormone Deficiency	Drug: ARX201; Drug: ARX201; Drug: ARX201	Phase 2

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 Design

• Study Type: Interventional
• Anticipated Enrollment: 36 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Single (Participant)
• Primary Purpose: Treatment
• 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).
• Study Start Date: Jul 1, 2008
• Actual Primary Completion Date: Oct 1, 2009
• Actual Study Completion Date: Oct 1, 2009

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: 1 low dose	Drug: ARX201; Reconstituted lyophilized drug product at 2.5 mg/wk; 3 periods over six months.
Active Comparator: 2 Medium dose	Drug: ARX201; Reconstituted lyophilized drug product at 5.0 mg/wk; 3 periods over six months.
Active Comparator: 3 High Dose	Drug: ARX201; Reconstituted lyophilized drug product at 10.0 mg/wk; 3 periods over six months.

Outcome Measures

Primary Outcome Measures

1. Temporal profiling of circulating IGF-1 levels. [3 period]

Secondary Outcome Measures

1. Body composition measurements at start of study and end of study [6 months]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 30 Years
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Accelsiors CRO and Consultancy Services	Budapest		Hungary

Sponsors and Collaborators

• Ambrx, Inc.
• Merck Serono International SA

Investigators

• Study Director: Mihaly Juhasz, MD, Accelsiors CRO & Consultancy Services

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00778518
• Other Study ID Numbers: PRO-ARX201-701; EudraCT: 2007-001746-40
• First Posted: Oct 23, 2008
• Last Update Posted: Oct 12, 2009
• Last Verified: Oct 1, 2009

• Keywords provided by , ,: growth hormone; growth hormone deficiency; GHD; PEG; pegylated; IGF-1
• Additional relevant MeSH terms: Dwarfism, Pituitary; Endocrine System Diseases

Study Results

No Results Posted as of Oct 12, 2009