Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
Growth Disorders
About this trial
This is an interventional treatment trial for Growth Disorders focused on measuring Insulin-like Growth Factor Deficiency, IGF-1, Short Stature
Eligibility Criteria
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
Sites / Locations
- Ipsen
Arms of the Study
Arm 1
Experimental
All rhIGF-1 Subjects
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.