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

NCT00572156 | Terminated Phase 2 Results DMC

• 2 other identifiers: MS3162019-000843-29
• Study Type: interventional
• Target: 106
• Locations: 1 country
• Sites: 1

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

Keywords

IGF-1 Deficiency; growth; ISS; Constitutional growth delay; Primary IGFD; Primary IGF Deficiency

Outcome Measures

Primary Outcomes (1)

• Height Velocity

  First year of treatment

Secondary Outcomes (12)

• Height Velocity

  Second, third and fourth year

• Cumulative Change in Height Standard Deviation Score (SDS)

  First, second, third and fourth year

• Predicted Adult Height (PAH)

  At baseline (Day 1), year 1,2,3 and 4

• Total Change From Baseline (Day 1) in BMI SDS

  At year 1,2,3,4 and end of study (visit 23) versus baseline (day 1)

• Skeletal Maturation

  At baseline(day 1), year 1,2,3 and 4

Study Arms (4)

1. rhGH Alone

ACTIVE COMPARATOR

Drug: NutropinAq® (Somatropin [rDNA origin])

2. Combination Dose

EXPERIMENTAL

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

3. Combination Dose

EXPERIMENTAL

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

4. Combination Dose

EXPERIMENTAL

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

Interventions

NutropinAq® (Somatropin [rDNA origin]) DRUG

rhGH (Somatropin) 45µg/kg once daily injection

Also known as: GH

1. rhGH Alone

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

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

Also known as: Increlex, rhIGF-1, GH

4. Combination Dose

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ipsen: lead

Study Sites (1)

Ipsen

Brisbane, California, 94005, United States

Location

MeSH Terms

Conditions

Insulin-Like Growth Factor I Deficiency; Puberty, Delayed

Interventions

Human Growth Hormone; mecasermin; Injections

Condition Hierarchy (Ancestors)

Gonadal Disorders; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins; Drug Administration Routes; Drug Therapy; Therapeutics

Limitations and Caveats

The study was prematurely terminated once the last subject had completed three years of treatment (compared to the six years planned in protocol Amendment) for strategic reasons.

Results Point of Contact

• Title: Medical Director, Endocrinology
• Organization: Ipsen

clinical.trials@ipsen.com

clinical.trials@ipsen.com

Study Officials

• Ipsen Medical Director

  Ipsen (formerly Tercica, Inc.)

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 10, 2007
• First Posted: December 12, 2007
• Study Start: December 1, 2007
• Primary Completion: April 1, 2010
• Study Completion: March 1, 2012
• Last Updated: March 30, 2023
• Results First Posted: December 15, 2015

Record last verified: 2023-03

Locations

US (1)