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

Sponsor:	Ipsen
Information provided by (Responsible Party):	Ipsen
ClinicalTrials.gov Identifier:	NCT00572156

Purpose

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.

Condition	Intervention	Phase
Insulin-like Growth Factor-1 Deficiency	Drug: NutropinAq® (Somatropin [rDNA origin]) Drug: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Recombinant Human Growth Hormone (rhGH) and Recombinant Human Insulin-like Growth Factor-1 rhIGF-1) Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency: A Six-year, Randomized, Multi-center, Open-label, Parallel-group, Active Treatment Controlled, Dose Selection Trial

Resource links provided by NLM:

Genetics Home Reference related topics: metatropic dysplasia pseudoachondroplasia

MedlinePlus related topics: Diabetes Medicines Dwarfism

Drug Information available for: Somatropin Insulin-like growth factor I Mecasermin

U.S. FDA Resources

Further study details as provided by Ipsen:

Primary Outcome Measures:

Height velocity [ Time Frame: during the first year of treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Change in height SDS [ Time Frame: during the first year of treatment ] [ Designated as safety issue: No ]
Safety assessments including: physical exam, funduscopic exams, adverse events, safety and metabolic labs and anti-GH and anti-IGF-1 antibodies [ Time Frame: during treatment ] [ Designated as safety issue: Yes ]
Changes in serum concentrations of GH, IGF-1, IGFBP-1, IGFPB-3, ALS and GHBP [ Time Frame: during treatment ] [ Designated as safety issue: No ]
Change in bone age [ Time Frame: during treatment ] [ Designated as safety issue: No ]
Total change in height divided by total change in chronological age [ Time Frame: From year 2 until the end of the study ] [ Designated as safety issue: No ]
Total change in height SDS divided by total change in chronological age [ Time Frame: From year 2 until the end of the study ] [ Designated as safety issue: No ]
Total change in predicted adult height divided by the total change in chronological age [ Time Frame: From year 2 until the end of the study ] [ Designated as safety issue: No ]

Enrollment:	106
Study Start Date:	December 2007
Study Completion Date:	March 2012
Primary Completion Date:	April 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: 1. rhGH Alone	Drug: NutropinAq® (Somatropin [rDNA origin]) rhGH (Somatropin) 45µg/kg once daily injection Other Name: GH
Experimental: 2. Combination Dose	Drug: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin]) rhGH (Somatropin) 45µg/kg and rhIGF-1 (Mecasermin) 50µg/kg once daily injections Other Names: Increlex rhIGF-1 GH
Experimental: 3. Combination Dose	Drug: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin]) rhGH 45µg/kg and rhIGF-1 100µg/kg once daily injections Other Names: Increlex rhIGF-1 GH
Experimental: 4. Combination Dose	Drug: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin]) rhGH 45µg/kg and rhIGF-1 150µg/kg once daily injection Other Names: Increlex rhIGF-1 GH

Eligibility

Ages Eligible for Study:	5 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00572156

Locations

United States, California
Ipsen
Brisbane, California, United States, 94005

Sponsors and Collaborators

Ipsen

Investigators

Study Director:

Catherine Lesage, M.D.

Ipsen (formerly Tercica, Inc.)

More Information

Additional Information:

Company Trials Summary This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Ipsen
ClinicalTrials.gov Identifier:	NCT00572156 History of Changes
Other Study ID Numbers:	MS316
Study First Received:	December 10, 2007
Last Updated:	April 30, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by Ipsen:

IGF-1 Deficiency
growth
ISS

Constitutional growth delay
Primary IGFD
Primary IGF Deficiency

Additional relevant MeSH terms:

Dwarfism
Bone Diseases, Developmental
Bone Diseases

Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases

ClinicalTrials.gov processed this record on May 22, 2012