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

Last updated: September 5, 2019
Sponsor: Ipsen
Overall Status: Completed

Phase

3

Condition

Severe Short Stature

Treatment

N/A

Clinical Study ID

NCT00125164
MS301
2019-001020-36
  • Ages 3-12
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Chronological age ≥ 3 and chronological or bone age less than or equal to 11 yearsinclusive in girls;

  • Chronological age ≥ 3 and chronological or bone age less than or equal to 12 yearsinclusive for boys

  • Prepubertal

  • Height SD score of < -2

  • IGF-1 SD score of < -2

Exclusion

Exclusion Criteria:

  • Prior treatment with rhGH, rhIGF-1, or other growth-influencing medications

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

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

Study Design

Total Participants: 137
Study Start date:
March 01, 2004
Estimated Completion Date:
July 31, 2008

Study 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.

Connect with a study center

  • Ipsen (formerly Tercica)

    Brisbane, California 94005
    United States

    Site Not Available

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