A Post-Marketing Study of the Immunogenicity of Somatropin (Ribosomal Deoxyribo Nucleic Acid [rDNA] Origin) Injection (Nutropin AQ®) in Children With Growth Hormone Deficiency
- Registration Number
- NCT02311894
- Lead Sponsor
- Genentech, Inc.
- Brief Summary
This is a Phase IV, multicenter, open-label, single-arm study of somatropin (rDNA origin) (Nutropin AQ v1.1) in pre-pubertal children with growth hormone deficiency (GHD) naïve to prior recombinant human growth hormone (rhGH) treatment. The study is designed to characterize the immunogenicity profile of somatropin (rDNA origin) injection when administered daily subcutaneously for 12 months. The clinical impact of immunogenicity will also be assessed.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 82
- Bone age less than equal to (</=) 9 years (females) or </= 11 years (males) as determined by X-ray of the left hand and wrist using Greulich and Pyle method and obtained within the 12 months prior to enrollment
- Prepubertal (Tanner I) males and females by physical examination
- Diagnosis of GHD (stimulated GH less than [<] 10 nanograms per milliliter [ng/mL]) by two standard pharmacologic tests obtained up to 12 months prior to informed consent/assent
- Normal thyroid function test within the 12 months prior to informed consent/assent
- Normal complete blood counts within 12 months prior to informed consent/assent
- Documentation of prior height and weight measurements, with height standard deviation score (SDS) </= 5th percentile for idiopathic isolated GHD participants
- Any previous rhGH treatment
- Short stature etiologies other than GHD
- Acute critical illness or uncontrolled chronic illness, which in the opinion of the investigator and medical monitor, would interfere with participation in this study, interpretation of the data, or pose a risk to participant safety
- Chronic illnesses such as inflammatory bowel disease, celiac disease, heart disease, and diabetes
- Bone diseases such as achondroplasia or hypochondroplasia, intracranial tumor, irradiation, and traumatic brain injury
- Participants receiving oral or inhaled chronic corticosteroid therapy (greater than [>] 3 months) for other medical conditions other than central adrenal insufficiency
- Participants who require higher (2 times or greater than maintenance) doses of corticosteroids for more than 5 days in the 6 months prior to enrollment in the study
- Participants with active malignancy or any other condition that the investigator believes would pose a significant hazard to the participant if rhGH were initiated
- Females with Turner syndrome regardless of their GH status
- Prader-Willi syndrome regardless of GH status
- Born small for gestational age regardless of GH status
- Presence of scoliosis requiring monitoring
- Previous participation in another clinical trial or investigation of GH, treatment for growth failure, or treatment with a biologic agent
- Participants with closed epiphyses
- Participants with a known hypersensitivity to somatropin, excipients, or diluent
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Somatropin Somatropin Children will receive daily SC injections of somatropin at a dose of up to 0.043 milligrams per kilogram per day (mg/kg/day) for 1 year.
- Primary Outcome Measures
Name Time Method Percentage of Participants Who Develop Anti-GH Antibodies After Treatment With Nutropin AQ v1.1 Baseline up to 1 year Participants who were tested positive to anti-GH antibody after initiation of study treatment.
- Secondary Outcome Measures
Name Time Method Annualized Growth Velocity at Months 6 and 12 (Change From Baseline) Months 6, 12 Annualized growth velocity is defined as (height - baseline height) / (date of height assessment - date of baseline)\*365.25. Results are presented according to anti-GH antibody status (positive included all participants that were anti-GH antibody positive at least once post-baseline visit and anti-GH antibody negative population included all participants that were anti-GH antibody negative at all post-baseline visits).
Percentage of Participants With Adverse Events Baseline up to 1 year Among participants who received at least one dose of study drug, those who reported at least one adverse event during study participation.
Percentage of Participants Who Exhibit Functional Growth Attenuation Baseline up to 1 year Growth attenuation is defined as initial growth response greater than pretreatment velocity followed by reduction in growth response to below the pretreatment velocity in the subsequent 6- to 12-month treatment period or reaching ≤ 2 cm per year.
Percentage of Participants With Neutralizing Antibodies Baseline up to 1 year Among participants who developed positive anti-GH antibody post-baseline, participants who were tested positive to neutralizing anti-GH antibody during study participation.
Height Standard Deviation Score (SDS) at Months 6 and 12 (Change From Baseline) Months 6, 12 Height Standard Deviation Score (SDS) allows for the comparison of a participants height to that of others in the same age group. Therefore, the average height for that age group will have the SDS of 0. In this study, the starting Height SDS score was ≤ -1.5 (≤ 5th percentile). Results are presented according to anti-GH antibody status (positive included all participants that were anti-GH antibody positive at least once post-baseline visit and anti-GH antibody negative population included all participants that were anti-GH antibody negative at all post-baseline visits.
Trial Locations
- Locations (33)
Barry J Reiner, MD, LLC
🇺🇸Baltimore, Maryland, United States
Endocrine Associates of Dallas
🇺🇸Dallas, Texas, United States
Miami Children's Hospital
🇺🇸Miami, Florida, United States
Boston Childrens Hospital
🇺🇸Boston, Massachusetts, United States
Rocky Mountain Pediatric Endocrinology, PC
🇺🇸Centennial, Colorado, United States
Pediatric Endocrine Associates
🇺🇸Greenwood Village, Colorado, United States
New York Presbyterian Hospital
🇺🇸New York, New York, United States
Children'S Hospital of Orange County
🇺🇸Orange, California, United States
Arkansas Children's Hospital Research Institute
🇺🇸Little Rock, Arkansas, United States
Center of Excellence in Diabetes & Endocrinology
🇺🇸Sacramento, California, United States
San Diego Medical Group; Pediatric Endocrinology
🇺🇸San Diego, California, United States
Nemours Children's Clinic - of the Nemours Foundation
🇺🇸Jacksonville, Florida, United States
Nemours Childrens Clinic
🇺🇸Orlando, Florida, United States
USF Diabetes Center
🇺🇸Tampa, Florida, United States
Pediatric Endrocine Assoc
🇺🇸Tampa, Florida, United States
Emory Children's Center
🇺🇸Atlanta, Georgia, United States
Baystate Endocrinology and Diabetes; Baystate Children's Specialty Center, Pediatric Endocrinology
🇺🇸Springfield, Massachusetts, United States
University of Minnesota Childrens' Hospital
🇺🇸Minneapolis, Minnesota, United States
Children's Mercy Hospitals & Clinics; Pulmonology
🇺🇸Kansas City, Missouri, United States
Hackensack University Medical Center PARTNER
🇺🇸Hackensack, New Jersey, United States
UNC General Pediatrics Clinic
🇺🇸Chapel Hill, North Carolina, United States
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Medical University of South Carolina; MUSC Pediatric Endocrinology
🇺🇸Charleston, South Carolina, United States
Cook Children's Hospital
🇺🇸Fort Worth, Texas, United States
MultiCare Health System Institute for Research and Innovation
🇺🇸Tacoma, Washington, United States
MultiCare Institute for Research and Innovation
🇺🇸Tacoma, Washington, United States
Thomas Jefferson University Hospital
🇺🇸Philadelphia, Pennsylvania, United States
The Pediatric Endocrine Office of Larry C. Deeb
🇺🇸Tallahassee, Florida, United States
Milton S Hershey Ped Sub Spclt
🇺🇸Hershey, Pennsylvania, United States
Children's Healthcare d.b.a Children's Hospitals and Clinics of Minnesota
🇺🇸Saint Paul, Minnesota, United States
University of Louisville
🇺🇸Louisville, Kentucky, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States