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

Ambrx, Inc.36 enrolled

Overview

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

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Growth Hormone Deficiency

Interventions

ARX201DRUG

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

ARX201DRUG

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

ARX201DRUG

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

Eligibility

Sex: MALEMin age: 18 YearsMax age: 30 Years

Medical Language ↔ Plain English

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

Locations (1)

Accelsiors CRO and Consultancy Services

Budapest, Hungary

Outcomes

Primary Outcomes

Temporal profiling of circulating IGF-1 levels.

Time frame: 3 period

Secondary Outcomes

Body composition measurements at start of study and end of study

Time frame: 6 months

Data from ClinicalTrials.gov

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