A Post-Marketing Study of the Immunogenicity of Somatropin (Ribosomal Deoxyribo Nucleic Acid [rDNA] Origin) Injection (Nutropin AQ®) in Children With Growth Hormone Deficiency
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| ClinicalTrials.gov Identifier: NCT02311894 |
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Recruitment Status :
Completed
First Posted : December 9, 2014
Results First Posted : November 15, 2018
Last Update Posted : January 8, 2019
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Sponsor:
Genentech, Inc.
Information provided by (Responsible Party):
Genentech, Inc.
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Growth Hormone Deficiency | Drug: Somatropin | Phase 4 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 82 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| 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) |
| Actual Study Start Date : | March 31, 2015 |
| Actual Primary Completion Date : | November 8, 2017 |
| Actual Study Completion Date : | November 8, 2017 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Metatropic dysplasia
Isolated growth hormone deficiency
Pseudoachondroplasia
MedlinePlus related topics:
Hormones
| Arm | Intervention/treatment |
|---|---|
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Experimental: 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.
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Drug: Somatropin
Somatropin will be administered as SC injections at a dose of up to 0.043 mg/kg/day. The dose may be adjusted for a change in body weight of at least (plus [+]/minus [-]) 2 kilograms (kg) from baseline at the Month 6 study visit or for a change in insulin-growth factor-1 (IGF-1), as per investigator assessment.
Other Name: Nutropin AQ v1.1 |
Primary Outcome Measures :
- Percentage of Participants Who Develop Anti-GH Antibodies After Treatment With Nutropin AQ v1.1 [ Time Frame: Baseline up to 1 year ]Participants who were tested positive to anti-GH antibody after initiation of study treatment.
Secondary Outcome Measures :
- Percentage of Participants Who Exhibit Functional Growth Attenuation [ Time Frame: 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 [ Time Frame: 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.
- Annualized Growth Velocity at Months 6 and 12 (Change From Baseline) [ Time Frame: 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).
- Height Standard Deviation Score (SDS) at Months 6 and 12 (Change From Baseline) [ Time Frame: 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.
- Percentage of Participants With Adverse Events [ Time Frame: 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.
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| Ages Eligible for Study: | 3 Years to 14 Years (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02311894
Locations
Show 33 study locations
Sponsors and Collaborators
Genentech, Inc.
Investigators
| Study Director: | Clinical Trials | Hoffmann-La Roche |
Study Documents (Full-Text)
Documents provided by Genentech, Inc.:
Documents provided by Genentech, Inc.:
Study Protocol [PDF] April 22, 2015
Statistical Analysis Plan [PDF] March 28, 2018
| Responsible Party: | Genentech, Inc. |
| ClinicalTrials.gov Identifier: | NCT02311894 |
| Other Study ID Numbers: |
ML29543 |
| First Posted: | December 9, 2014 Key Record Dates |
| Results First Posted: | November 15, 2018 |
| Last Update Posted: | January 8, 2019 |
| Last Verified: | December 2018 |
Additional relevant MeSH terms:
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Dwarfism, Pituitary Endocrine System Diseases Dwarfism Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Bone Diseases, Endocrine |
Hypopituitarism Pituitary Diseases Hypothalamic Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases |