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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00330668
Recruitment Status : Terminated (Unacceptable frequency of hypoglycemia observed at and above 200 ug/kg/day)
First Posted : May 29, 2006
Results First Posted : June 27, 2011
Last Update Posted : August 14, 2020
Sponsor:
Information provided by (Responsible Party):
Ipsen

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.

Condition or disease Intervention/treatment Phase
Growth Disorders Drug: rh IGF-1 (mecasermin) Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 114 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : November 2005
Actual Primary Completion Date : February 2010
Actual Study Completion Date : March 2010


Arm Intervention/treatment
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.

Drug: 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.
Other Name: Increlex




Primary Outcome Measures :
  1. Height Velocity During BID Dosing Period [ Time Frame: At Years 1, 2 and 3 in BID dosing period. ]
    Height was measured standing, without shoes, as the average of 3 measurements by the same observer identical technique with a Harpenden or other wall-mounted stadiometer at baseline and each study visit up to 3 years. Height velocity (during any interval of time (annualised) is computed as (height on date 2 - height on date 1)/(age on date 2 - age on date 1) where height is expressed as centimetres so that height velocity is expressed as centimetres per year (cm/yr). Height velocity is presented for subjects completing each year of BID treatment (i.e. Year 1 [0-1 years], Year 2 [1-2 years], Year 3 [2-3 years]).


Secondary Outcome Measures :
  1. Mean Change From Baseline in Height Standard Deviation (SD) Score During BID Dosing Period [ Time Frame: At baseline and Years 1, 2 and 3 in BID dosing period. ]
    Height was measured standing, without shoes, as the average of 3 measurements by the same observer using identical technique with a Harpenden or other wall-mounted stadiometer at baseline and each study visit up to 3 years. Height SD score was determined using the National Center for Health Statistics 2000 data as provided by the Center for Disease Control. The SD score was calculated as the patient value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child. Mean change from baseline in height SD score is presented for all subjects completing each year of BID treatment (i.e. Year 1 [0-1 years], Year 2 [1-2 years], Year 3 [2-3 years]).

  2. Mean Change From Baseline in Body Mass Index (BMI) SD Score During BID Dosing Period [ Time Frame: At baseline and Years 1, 2 and 3 in BID dosing period. ]
    BMI SD score was calculated using the National Center for Health Statistics 2000 data as provided by the Center for Disease Control. The SD score was calculated as the patient value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child. Mean change from baseline in BMI SD score is presented for all subjects completing each year of BID treatment (i.e. Year 1 [0-1 years], Year 2 [1-2 years], Year 3 [2-3 years]).

  3. Mean Change From Baseline in Bone Age During BID Dosing Period [ Time Frame: At baseline and Years 1, 2 and 3 in BID dosing period. ]
    Radiographs of the left hand and wrist were taken on an approximately annual basis for determination of bone (skeletal) age. The films were sent to a central facility for standardised evaluation. Mean change from baseline in bone age is presented for all subjects completing each year of BID treatment (i.e. Year1 [0-1 years], Year 2 [1-2 years], Year 3 [2-3 years]).

  4. Mean Change From Baseline in Predicted Adult Height During BID Dosing Period [ Time Frame: At baseline and Years 1, 2 and 3 in BID dosing period. ]
    Predicted adult heights were estimated using the Roche-Wainer-Theissen method which takes into account changes in age, height and bone age. Mean change from baseline in predicted adult height is presented for all subjects completing each year of BID treatment (i.e. Year 1 [0-1 years], Year 2 [1-2 years], Year 3 [2-3 years]).



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Ages Eligible for Study:   4 Years to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00330668


Locations
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France
Ipsen
Paris, France
Sponsors and Collaborators
Ipsen
Investigators
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Study Director: Sr Vice President, Clinical Development and Medical Affairs Ipsen (formerly Tercica, Inc.)
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Responsible Party: Ipsen
ClinicalTrials.gov Identifier: NCT00330668    
Other Study ID Numbers: MS306
2019-000844-81 ( EudraCT Number )
First Posted: May 29, 2006    Key Record Dates
Results First Posted: June 27, 2011
Last Update Posted: August 14, 2020
Last Verified: August 2020
Keywords provided by Ipsen:
Insulin-like Growth Factor Deficiency
IGF-1
Short Stature
Additional relevant MeSH terms:
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Growth Disorders
Failure to Thrive
Pathologic Processes
Mecasermin
Growth Substances
Physiological Effects of Drugs