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

Ipsen

Status and phase: Completed; Phase 3; Phase 2

Conditions: Growth Disorders; Insulin-Like Growth Factor-1 Deficiency

Treatments: Drug: rhIGF-1 (mecasermin) for a period of 86 weeks

Study type: Interventional

Funder types: Industry

Identifiers

NCT00125190

MS308

2019-001095-11 (EudraCT Number)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 45 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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45 participants in 1 patient group

rhIGF-1 QD

Experimental group

Description:

Subjects received subcutaneous injection (SC) injections of rhIGF-1 once a day.

Treatment:

Drug: rhIGF-1 (mecasermin) for a period of 86 weeks