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

Primary Purpose

Growth Hormone Deficiency

Status

Completed

Phase

Phase 2

Locations

Hungary

Study Type

Interventional

Intervention

ARX201

About this trial

This is an interventional treatment trial for Growth Hormone Deficiency focused on measuring growth hormone, growth hormone deficiency, GHD, PEG, pegylated, IGF-1

Eligibility Criteria

18 Years - 30 Years (Adult)MaleDoes not accept 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

Sites / Locations

Accelsiors CRO and Consultancy Services

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Active Comparator

Arm Label

Arm Description

low dose

Medium dose

High Dose

Outcomes

Primary Outcome Measures

Temporal profiling of circulating IGF-1 levels.

Secondary Outcome Measures

Body composition measurements at start of study and end of study

Full Information

NCT ID

NCT00778518

First Posted

October 22, 2008

Last Updated

October 9, 2009

Sponsor

Ambrx, Inc.

Collaborators

Merck Serono International SA

1. Study Identification

Unique Protocol Identification Number

NCT00778518

Brief Title

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

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 Type

Interventional

2. Study Status

Record Verification Date

October 2009

Overall Recruitment Status

Completed

Study Start Date

July 2008 (undefined)

Primary Completion Date

October 2009 (Actual)

Study Completion Date

October 2009 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Ambrx, Inc.

Collaborators

Merck Serono International SA

4. Oversight

Data Monitoring Committee

5. 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).

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Growth Hormone Deficiency

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

Participant

Allocation

Randomized

Enrollment

36 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Active Comparator

Arm Description

low dose

Arm Title

Arm Type

Active Comparator

Arm Description

Medium dose

Arm Title

Arm Type

Active Comparator

Arm Description

High Dose

Intervention Type

Drug

Intervention Name(s)

ARX201

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

ARX201

Intervention Description

Reconstituted lyophilized drug product at 5.0 mg/wk; 3 periods over six months.

Intervention Type

Drug

Intervention Name(s)

ARX201

Intervention Description

Reconstituted lyophilized drug product at 10.0 mg/wk; 3 periods over six months.

Primary Outcome Measure Information:

Title

Temporal profiling of circulating IGF-1 levels.

Time Frame

3 period

Secondary Outcome Measure Information:

Title

Body composition measurements at start of study and end of study

Time Frame

6 months

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

30 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Mihaly Juhasz, MD

Organizational Affiliation

Accelsiors CRO & Consultancy Services

Official's Role

Study Director

Facility Information:

Facility Name

Accelsiors CRO and Consultancy Services

City

Budapest

Country

Hungary

12. IPD Sharing Statement

Learn more about this trial

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

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