Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With IGF-1 Deficiency

Growth Disorders Clinical Trial

Official title:

Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With Primary IGF-1 Deficiency: A Multi-Center, Open Label, Concentration-Controlled Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00125190
• Other study ID #: MS308
• Secondary ID: 2019-001095-11
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: July 2005
• Est. completion date: January 2009

Study information

• Verified date: July 2020
• Source: Ipsen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study is intended to assess the effects of once daily dosing of recombinant human insulin-like growth factor (rhIGF-1) in increasing height velocity.

Description:

Growth failure associated with primary IGF-1 deficiency (IGFD). Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in growth hormone (GH) action. In this protocol, primary IGFD is defined as short stature (<-2 standard deviations [SDs] below the mean for age and gender), and abnormal serum IGF-1 (<-2 SDs below the mean for age and gender).

The trial is an open-label, concentration-controlled trial conducted at up to 20 centers throughout the United States.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 45
• Est. completion date: January 2009
• Est. primary completion date: January 2009
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

Inclusion Criteria:

• Chronological age = 3

• Chronological age or bone age = 12 for boys and = 11 for girls

• Prepubertal at Visit 1

• Height SD score of < -2

• IGF-1 SD score of < -2

Exclusion Criteria:

• Prior treatment with GH, IGF-1, or other growth-influencing medications

• Growth failure associated with other identifiable causes (e.g., syndromes, chromosomal abnormality)

• Chronic illness such as diabetes, cystic fibrosis, etc.

Related Conditions & MeSH terms

• Dwarfism
• Growth Disorders
• Insulin-Like Growth Factor-1 Deficiency

Intervention

Drug:
• rhIGF-1 (mecasermin) for a period of 86 weeks
Once a day rhIGF-1 injections

Locations

Country	Name	City	State
United States	Ipsen	Brisbane	California

Sponsors (1)

Lead Sponsor	Collaborator
Ipsen

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Height Velocity From Pretreatment (Week 0) to Week 34	Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 34 heights were imputed using the last height SD score carried forward.	Pretreatment to Week 34
Primary	Height Velocity From Week 34 to 86	Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 86 heights were imputed using the last height SD score carried forward.	Week 34 to 86
Secondary	Change in Height SD Score From Pretreatment to Week 34	Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. The SD score is calculated as the subject 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.	Pretreatment and Week 34
Secondary	Change in Height SD Score From Pretreatment to Week 86	Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. Subjects were repositioned between each measurement. The SD score is 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.	Pretreatment and Week 86
Secondary	Change in Bone Age From Pretreatment to Week 86 Minus Change in Chronological Age	Plain X-rays of the left hand and wrist were exposed for bone age appraisal. The films were sent to a central facility for standardized evaluation.	Pretreatment to Week 86
Secondary	Percent Change in Serum Concentration of IGFBP-1, IGFBP-2 and IGFBP-3 From Pretreatment to Week 86	Growth factor panels for measuring IGFBP-1, IGFBP-2 and IGFBP-3 were evaluated from screening and at each study visit up to Week 86. Inter-quartile range (Q1-Q3) is 10th to 90th percentile.	Pretreatment and Week 86
Secondary	Percent Change in Serum Concentration of ALS From Pretreatment to Week 86	Growth factor panels for measuring ALS were evaluated from screening and at each study visit up to Week 86. Inter-quartile range (Q1-Q3) is 10th to 90th percentile.	Pretreatment and Week 86