rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency

NCT ID: NCT00572156

Last Updated: 2023-03-30

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 106 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-12-31
• Study Completion Date: 2012-03-31

Brief Summary

IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in response to another hormone called growth hormone. Growth Hormone is produced by a small gland at the base of the brain (the pituitary). Together IGF-1 and GH are large contributors to growth during infancy, childhood, and adolescence.

Children with IGF Deficiency are short and have an imbalance in the levels of growth hormone and IGF-1 that their body produces. Their growth hormone levels are normal or even high, but IGF-1 levels do not increase normally in response to growth hormone. As a result, they have a type of growth hormone insensitivity and an inability to grow normally.

This study is a test to see whether daily dosing with a combination of rhIGF-1 and rhGH will help children with IGFD grow taller more quickly than children treated with rhGH alone. The study medications, rhIGF-1 and rhGH, are approved by the US Food and Drug Administration (FDA) for use in some growth disorders in children, but the combination of rhIGF-1 and rhGH in children with IGF-1 deficiency (IGFD) is investigational.

Conditions

Insulin-like Growth Factor-1 Deficiency

Study Design

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

Study Groups

1. rhGH Alone

Group Type: ACTIVE_COMPARATOR

NutropinAq® (Somatropin [rDNA origin])

Intervention Type: DRUG

rhGH (Somatropin) 45µg/kg once daily injection

2. Combination Dose

Group Type: EXPERIMENTAL

Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

Intervention Type: DRUG

rhGH (Somatropin) 45µg/kg and rhIGF-1 (Mecasermin) 50µg/kg once daily injections

3. Combination Dose

Group Type: EXPERIMENTAL

Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

Intervention Type: DRUG

rhGH 45µg/kg and rhIGF-1 100µg/kg once daily injections

4. Combination Dose

Group Type: EXPERIMENTAL

Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

Intervention Type: DRUG

rhGH 45µg/kg and rhIGF-1 150µg/kg once daily injection

Interventions

NutropinAq® (Somatropin [rDNA origin])

rhGH (Somatropin) 45µg/kg once daily injection

Intervention Type: DRUG

Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

rhGH 45µg/kg and rhIGF-1 150µg/kg once daily injection

Intervention Type: DRUG

Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

rhGH (Somatropin) 45µg/kg and rhIGF-1 (Mecasermin) 50µg/kg once daily injections

Intervention Type: DRUG

Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

rhGH 45µg/kg and rhIGF-1 100µg/kg once daily injections

Intervention Type: DRUG

Other Intervention Names

GH; Increlex; rhIGF-1; GH; Increlex; rhIGF-1; GH; Increlex; rhIGF-1; GH

Eligibility Criteria

Inclusion Criteria

• Parents or legally authorized representatives must give signed informed consent before any trial-related activities
• IGF-1 SDS of ≤ -1 for age and gender
• Short stature, as defined by a height SDS of ≤ -2 for age and gender
• Chronological age ≥ 5 years
• Bone age ≤ 11 years in boys and ≤ 9 years in girls
• GH sufficiency, defined as a maximal stimulated GH response of greater than or equal to 10 ng/mL at Visit 2 (note: upon approval of the Medical Monitor, the result of a prior GH stimulation test may satisfy this requirement).
• Prepubertal status
• Adequate nutrition as evidenced by a body mass index (BMI) greater than or equal to the 5th percentile for age and gender

Exclusion Criteria

• Severe Primary IGFD (defined as height and IGF-1 SDS ≤ 3, and stimulated GH response greater than or equal to 10 ng/mL)
• Prior or current use of medications with the potential to alter growth patterns including GH, IGF-1, IGFBP-3, gonadotrophin agonists (e.g., Lupron), aromatase inhibitors, androgens and estrogens
• Known or suspected allergy to rhGH, rhIGF-1 or a constituent of their formulations
• Current use of medications for attention deficit disorder
• A chronic health condition that requires anti-inflammatory steroids or daily medication unless approved by the Medical Monitor

• Minimum Eligible Age: 5 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 (formerly Tercica, Inc.)

Locations

Ipsen

Brisbane, California, United States

Countries

United States

Other Identifiers

2019-000843-29

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MS316

Identifier Type: -

Identifier Source: org_study_id