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

Ambrx

Status and phase: Completed; Phase 2

Conditions: Growth Hormone Deficiency

Treatments: Drug: ARX201

Study type: Interventional

Funder types: Industry

Identifiers

NCT00778518

PRO-ARX201-701

EudraCT: 2007-001746-40

Details and patient eligibility

About

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

Full 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).

Enrollment: 36 estimated patients

Sex: Male

Ages: 18 to 30 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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36 participants in 3 patient groups

1

Active Comparator group

Description:

low dose

Treatment:

Drug: ARX201

Drug: ARX201

Drug: ARX201

2

Active Comparator group

Description:

Medium dose

Treatment:

Drug: ARX201

Drug: ARX201

Drug: ARX201

3

Active Comparator group

Description:

High Dose

Treatment:

Drug: ARX201

Drug: ARX201

Drug: ARX201