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

This study has been completed.
Sponsor:
Collaborator:
Merck Serono International SA
Information provided by:
Ambrx, Inc.
ClinicalTrials.gov Identifier:
NCT00778518
First received: October 22, 2008
Last updated: October 9, 2009
Last verified: October 2009
  Purpose

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


Condition Intervention Phase
Growth Hormone Deficiency
Drug: ARX201
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Pharmacokinetics/Dynamics Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
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).

Resource links provided by NLM:


Further study details as provided by Ambrx, Inc.:

Primary Outcome Measures:
  • Temporal profiling of circulating IGF-1 levels. [ Time Frame: 3 period ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Body composition measurements at start of study and end of study [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 36
Study Start Date: July 2008
Study Completion Date: October 2009
Primary Completion Date: October 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

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).

  Eligibility

Ages Eligible for Study:   18 Years to 30 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00778518

Locations
Hungary
Accelsiors CRO and Consultancy Services
Budapest, Hungary
Sponsors and Collaborators
Ambrx, Inc.
Merck Serono International SA
Investigators
Study Director: Mihaly Juhasz, MD Accelsiors CRO and Consultancy Services
  More Information

No publications provided

Responsible Party: Douglas W. Axelrod, MD, PhD, Sr. Vice President, Clinical Research and Development, Ambrx, Inc.
ClinicalTrials.gov Identifier: NCT00778518     History of Changes
Other Study ID Numbers: PRO-ARX201-701, EudraCT: 2007-001746-40
Study First Received: October 22, 2008
Last Updated: October 9, 2009
Health Authority: Belarus: Ministry of Health
Hungary: National Institute of Pharmacy
Romania: National Medicines Agency
Serbia and Montenegro: Agency for Drugs and Medicinal Devices
Ukraine: State Pharmacological Center - Ministry of Health

Keywords provided by Ambrx, Inc.:
growth hormone
growth hormone deficiency
GHD
PEG
pegylated
IGF-1

Additional relevant MeSH terms:
Dwarfism, Pituitary
Endocrine System Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 24, 2014