Clinical trial NCT02311894: A Post-Marketing Study of the Immunogenicity of Somatropin (Ribosomal Deoxyribo Nucleic Acid [rDNA] Origin) Injection (Nutropin AQ®) in Children With Growth Hormone Deficiency
Official title: A Phase IV, Multicenter, Open-Label Study of the Immunogenicity of Nutropin AQ® V1.1 [Somatropin (rDNA Origin) Injection] Administered Daily to Naïve Growth Hormone-Deficient Children (iSTUDY)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Growth Hormone Deficiency
Lead sponsor: Genentech, Inc.
Sponsor class: INDUSTRY
First posted: 2014-12-09
Enrollment: 82
Sex: ALL
Minimum age (years): 3
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Percentage of Participants Who Develop Anti-GH Antibodies After Treatment With Nutropin AQ v1.1
timeFrame: Baseline up to 1 year
Secondary outcomes:
measure: Percentage of Participants Who Exhibit Functional Growth Attenuation
timeFrame: Baseline up to 1 year
measure: Percentage of Participants With Neutralizing Antibodies
timeFrame: Baseline up to 1 year
measure: Annualized Growth Velocity at Months 6 and 12 (Change From Baseline)
timeFrame: Months 6, 12
measure: Height Standard Deviation Score (SDS) at Months 6 and 12 (Change From Baseline)
timeFrame: Months 6, 12
measure: Percentage of Participants With Adverse Events
timeFrame: Baseline up to 1 year
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital Research Institute
state: Arkansas
city: Orange
country: United States
facility: Children'S Hospital of Orange County
state: California
city: Sacramento
country: United States
facility: Center of Excellence in Diabetes & Endocrinology
state: California
city: San Diego
country: United States
facility: San Diego Medical Group; Pediatric Endocrinology
state: California
city: Centennial
country: United States
facility: Rocky Mountain Pediatric Endocrinology, PC
state: Colorado
city: Greenwood Village
country: United States
facility: Pediatric Endocrine Associates
state: Colorado
city: Jacksonville
country: United States
facility: Nemours Children's Clinic - of the Nemours Foundation
state: Florida
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Nemours Childrens Clinic
state: Florida
city: Tallahassee
country: United States
facility: The Pediatric Endocrine Office of Larry C. Deeb
state: Florida
city: Tampa
country: United States
facility: Pediatric Endrocine Assoc
state: Florida
city: Tampa
country: United States
facility: USF Diabetes Center
state: Florida
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
city: Louisville
country: United States
facility: University of Louisville
state: Kentucky
city: Baltimore
country: United States
facility: Barry J Reiner, MD, LLC
state: Maryland
city: Boston
country: United States
facility: Boston Childrens Hospital
state: Massachusetts
city: Springfield
country: United States
facility: Baystate Endocrinology and Diabetes; Baystate Children's Specialty Center, Pediatric Endocrinology
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota Childrens' Hospital
state: Minnesota
city: Saint Paul
country: United States
facility: Children's Healthcare d.b.a Children's Hospitals and Clinics of Minnesota
state: Minnesota
city: Kansas City
country: United States
facility: Children's Mercy Hospitals & Clinics; Pulmonology
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center PARTNER
state: New Jersey
city: New York
country: United States
facility: New York Presbyterian Hospital
state: New York
city: Chapel Hill
country: United States
facility: UNC General Pediatrics Clinic
state: North Carolina
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Hershey
country: United States
facility: Milton S Hershey Ped Sub Spclt
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University Hospital
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina; MUSC Pediatric Endocrinology
state: South Carolina
city: Dallas
country: United States
facility: Endocrine Associates of Dallas
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Hospital
state: Texas
city: Tacoma
country: United States
facility: MultiCare Health System Institute for Research and Innovation
state: Washington
city: Tacoma
country: United States
facility: MultiCare Institute for Research and Innovation
state: Washington
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: