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

NCT ID: NCT00330668

Last Updated: 2020-08-14

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 114 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-11-30
• Study Completion Date: 2010-03-31

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.

Conditions

Growth Disorders

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

rh IGF-1 (mecasermin)

Intervention Type: DRUG

Patients from untreated arm for prior study MS301 (NCT00125164) were randomized to a dose of either 80 or 120 mcg/kg twice daily. For patients receiving active treatment in previous study MS 301 (NCT00125164), they started on a dose of 80 or 120 mcg/kg twice daily based on the dose reached at end of the previous study. Following a protocol amendment in May 2009, all patients were switched to once daily doses of 160 µg/kg, escalated to a targeted maximum dose of 240 µg/kg.

Interventions

rh IGF-1 (mecasermin)

Patients from untreated arm for prior study MS301 (NCT00125164) were randomized to a dose of either 80 or 120 mcg/kg twice daily. For patients receiving active treatment in previous study MS 301 (NCT00125164), they started on a dose of 80 or 120 mcg/kg twice daily based on the dose reached at end of the previous study. Following a protocol amendment in May 2009, all patients were switched to once daily doses of 160 µg/kg, escalated to a targeted maximum dose of 240 µg/kg.

Intervention Type: DRUG

Other Intervention Names

Increlex

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

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 15 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ipsen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Sr Vice President, Clinical Development and Medical Affairs

Role: STUDY_DIRECTOR

Ipsen (formerly Tercica, Inc.)

Locations

Ipsen

Paris, , France

Countries

France

Other Identifiers

2019-000844-81

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MS306

Identifier Type: -

Identifier Source: org_study_id