Clinical trial NCT00330668: Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
Official title: Recombinant Human Insulin-Like Growth Factor-1 (IGF-1) Treatment of Children With Growth Failure Associated With Primary IGF-1 Deficiency: An Open-Label, Multi-Center, Extension Study
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Growth Disorders
Keywords: Insulin-like Growth Factor Deficiency
IGF-1
Short Stature
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2006-05-29
Enrollment: 114
Sex: ALL
Minimum age (years): 4
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

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

Primary outcomes:
measure: Height Velocity During BID Dosing Period
timeFrame: At Years 1, 2 and 3 in BID dosing period.
Secondary outcomes:
measure: Mean Change From Baseline in Height Standard Deviation (SD) Score During BID Dosing Period
timeFrame: At baseline and Years 1, 2 and 3 in BID dosing period.
measure: Mean Change From Baseline in Body Mass Index (BMI) SD Score During BID Dosing Period
timeFrame: At baseline and Years 1, 2 and 3 in BID dosing period.
measure: Mean Change From Baseline in Bone Age During BID Dosing Period
timeFrame: At baseline and Years 1, 2 and 3 in BID dosing period.
measure: Mean Change From Baseline in Predicted Adult Height During BID Dosing Period
timeFrame: At baseline and Years 1, 2 and 3 in BID dosing period.
Other outcomes:
Locations:
city: Paris
country: France
facility: Ipsen
Study officials:
affiliation: Ipsen (formerly Tercica, Inc.)
name: Sr Vice President, Clinical Development and Medical Affairs
role: STUDY_DIRECTOR
References: