Prepubertal Children With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency

NCT ID: NCT00125164

Last Updated: 2019-09-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 137 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2004-03-31
• Study Completion Date: 2008-07-31

Brief Summary

This study is intended to determine whether twice daily weight based dosing with recombinant human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth of prepubertal children with short stature associated with low IGF-1 levels but who produce sufficient growth hormone (GH). Subjects will be randomized to either an observation arm or to active treatment.

Detailed Description

Prepubertal 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 GH action. In this protocol, primary IGFD is defined as short stature (height standard deviation score[SDS]<-2 below the mean for age and gender), low serum IGF-1 (IGF-1 SDS <-2 below the mean for age and gender), and levels of growth hormone (GH) that are normal (≥7ng/mL) after a GH stimulation test. Primary IGFD is believed to result from a lower than normal ability to produce IGF-1 when exposed to normal levels of GH, i.e., a type of GH insensitivity or GH resistance.

This trial is one year, randomized, open label, observation-controlled, parallel-dose comparison efficacy and safety study conducted in approximately 40 centers across the United States.

Conditions

Growth Disorders; Insulin-Like Growth Factor-1 Deficiency

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Untreated

Observational Group

Group Type: NO_INTERVENTION

No interventions assigned to this group

40 μg/kg BID (twice daily dosing)

Injection of rhIGF-1 40 μg/kg BID. Per protocol amendment these subjects were reassigned to receive 120 μg/kg BID. Due to the dose change, the efficacy results for these subjects were analysed in a separate subanalysis. For all outcome measures, mean and standard deviations were not calculated for this arm.

Group Type: EXPERIMENTAL

rhIGF-1 (mecasermin, Tercica, Inc.)

Intervention Type: DRUG

Twice Daily Injection

80 μg/kg BID (twice daily dosing)

Injection of rhIGF-1 80 μg/kg BID

Group Type: EXPERIMENTAL

rhIGF-1 (mecasermin, Tercica, Inc.)

Intervention Type: DRUG

Twice Daily Injection

120 μg/kg BID (twice daily dosing)

Injection of rhIGF-1 120 μg/kg BID

Group Type: EXPERIMENTAL

rhIGF-1 (mecasermin, Tercica, Inc.)

Intervention Type: DRUG

Twice Daily Injection

Interventions

rhIGF-1 (mecasermin, Tercica, Inc.)

Twice Daily Injection

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Chronological age ≥ 3 and chronological or bone age less than or equal to 11 years inclusive in girls;
• Chronological age ≥ 3 and chronological or bone age less than or equal to 12 years inclusive for boys
• Prepubertal
• Height SD score of < -2
• IGF-1 SD score of < -2

Exclusion Criteria

• Prior treatment with rhGH, rhIGF-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.

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ipsen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ipsen Medical Director

Role: STUDY_DIRECTOR

Ipsen

Locations

Ipsen (formerly Tercica)

Brisbane, California, United States

Countries

United States

Other Identifiers

2019-001020-36

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MS301

Identifier Type: -

Identifier Source: org_study_id