Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency

Ipsen

Status and phase: Terminated; Phase 3

Conditions: Growth Disorders

Treatments: Drug: rh IGF-1 (mecasermin)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00330668

MS306

2019-000844-81 (EudraCT Number)

Details and patient eligibility

About

This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and adolescents treated for primary IGF-1 deficiency (IGFD). The secondary objective is to use the data collected to learn more about the relationship of IGF-1 exposure to the promotion of normal growth and pubertal development.

Full description

Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in GH action. In this protocol, subjects that have completed one year of mecasermin treatment on Tercica protocol MS301 (NCT00125164) will be allowed to enroll in this extension study. All subjects were planned to receive treatment.

This is a Phase IIIb open-label, multi-center, parallel dose, extension study conducted in approximately 40 centers across the United States.

Enrollment: 114 patients

Sex: All

Ages: 4 to 15 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Parents or legally authorized representatives must give signed informed consent before any trial related activities are conducted
• Where required, assent of the subject will be appropriately documented prior to any study related activities
• Completion of assessments at Visit 9 (Month 120 of Study MS301 [NCT00125164])

Exclusion criteria

• Incomplete participation in MS301 (NCT00125164)
• Known or suspected allergy to the trial product (mecasermin, recombinant human IGF-1 injection) or its formulation
• Development or presence of a chronic condition except as approved by the Medical Monitor
• Pregnancy
• Any social or medical condition that, in the opinion of the investigator, would be detrimental to either the subject or the study

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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114 participants in 1 patient group

All rhIGF-1 Subjects

Experimental group

Description:

All subjects entering MS306 began recombinant human insulin-like growth factor-1 (rhIGF-1) twice a day (BID) treatment. Each subject treated in MS301 had an MS306 starting dose that was based on their dose at the completion of MS301 (i.e. subcutaneous injections of rhIGF-1 at 40, 80, or 120 micrograms \[μg\]/ kilogram \[kg\] BID). MS301 untreated control subjects were randomised in MS306 in a 1:1 ratio to a dose of either 80 or 120 μg/kg rhIGF-1 BID. Following Protocol Amendment 1, all subjects received either 80 or 120 μg/kg rhIGF-1 BID until the implementation of Protocol Amendment 2. Following Protocol Amendment 2, all subjects were first switched to receive subcutaneous injections of 160 μg/kg rhIGF-1 once a day (QD), followed by individual dose-escalation first to 200 μg/kg rhIGF-1 QD and subsequently to a targeted maximum dose of 240 μg/kg rhIGF-1 QD. Subjects were treated QD until the early termination of the study.

Treatment:

Drug: rh IGF-1 (mecasermin)