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

NCT00125164 | Completed Phase 3 Results DMC

• 2 other identifiers: MS3012019-001020-36
• Study Type: interventional
• Target: 137
• Locations: 1 country
• Sites: 1

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.

Conditions

Growth Disorders; Insulin-Like Growth Factor-1 Deficiency

Keywords

Primary IGF-1 Deficiency

Outcome Measures

Primary Outcomes (1)

• Height Velocity During the First Year - Intent to Treat (ITT)Population

  Height to be 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. Reposition subject between each measurement.

  Measured at baseline and at one year

Secondary Outcomes (8)

• Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population

  Measured at baseline and at one year

• Changes in Bone Age From Baseline to One Year

  Measured at baseline and at one year

• Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year

  Measured at baseline and at one year

• Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year

  Measured at baseline and at one year

• Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-2 (IGFBP-2) at One Year

  Measured at baseline and at one year

Study Arms (4)

Untreated

NO INTERVENTION

Observational Group

40 μg/kg BID (twice daily dosing)

EXPERIMENTAL

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.

Drug: rhIGF-1 (mecasermin, Tercica, Inc.)

80 μg/kg BID (twice daily dosing)

EXPERIMENTAL

Injection of rhIGF-1 80 μg/kg BID

Drug: rhIGF-1 (mecasermin, Tercica, Inc.)

120 μg/kg BID (twice daily dosing)

EXPERIMENTAL

Injection of rhIGF-1 120 μg/kg BID

Drug: rhIGF-1 (mecasermin, Tercica, Inc.)

Interventions

rhIGF-1 (mecasermin, Tercica, Inc.) DRUG

Twice Daily Injection

120 μg/kg BID (twice daily dosing); 40 μg/kg BID (twice daily dosing); 80 μg/kg BID (twice daily dosing)

Eligibility Criteria

• Age: 3 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Ipsen: lead

Study Sites (1)

Ipsen (formerly Tercica)

Brisbane, California, 94005, United States

Location

MeSH Terms

Conditions

Growth Disorders

Interventions

mecasermin

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Medical Director, Pediatric Endocrinology
• Organization: Ipsen

clinical.trials@ipsen.com

clinical.trials@ipsen.com

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 27, 2005
• First Posted: July 29, 2005
• Study Start: March 1, 2004
• Primary Completion: July 1, 2008
• Study Completion: July 1, 2008
• Last Updated: September 18, 2019
• Results First Posted: April 30, 2010

Record last verified: 2019-09

Locations

US (1)