Growth Hormone DeficiencyPhase 4NCT02311894
A Post-Marketing Study of the Immunogenicity of Somatropin (Ribosomal Deoxyribo Nucleic Acid [rDNA] Origin) Injection (Nutropin AQ®) in Children With Growth Hormone Deficiency
A Phase 4 study testing Somatropin in people with Growth Hormone Deficiency, run by Genentech, Inc. at 33 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 14 years
- Conditions the study is for
- Growth Hormone Deficiency
- What is being tested?
- Somatropin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 82 participants
- Where
- 33 sitesUnited States
- Study dates
- 31 Mar 2015to 8 Nov 2017
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 15Show fewer
- 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
Trial locations and contacts
33 sites in 1 country.
33 sites in 1 country
United States33 sites
University of Michigan
Ann Arbor, Michigan, 48109-0934
Emory Children's Center
Atlanta, Georgia, 20010
Barry J Reiner, MD, LLC
Baltimore, Maryland, 21229
Boston Childrens Hospital
Boston, Massachusetts, 02115
Rocky Mountain Pediatric Endocrinology, PC
Centennial, Colorado, 80112
UNC General Pediatrics Clinic
Chapel Hill, North Carolina, 27514
Medical University of South Carolina; MUSC Pediatric Endocrinology
Charleston, South Carolina, 29425
Endocrine Associates of Dallas
Dallas, Texas, 75231
Cook Children's Hospital
Fort Worth, Texas, 76104
Pediatric Endocrine Associates
Greenwood Village, Colorado, 80111
Hackensack University Medical Center PARTNER
Hackensack, New Jersey, 07601
Milton S Hershey Ped Sub Spclt
Hershey, Pennsylvania, 17033
Nemours Children's Clinic - of the Nemours Foundation
Jacksonville, Florida, 32207
Children's Mercy Hospitals & Clinics; Pulmonology
Kansas City, Missouri, 64108
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, 72202
University of Louisville
Louisville, Kentucky, 40202
Miami Children's Hospital
Miami, Florida, 33155-3009
University of Minnesota Childrens' Hospital
Minneapolis, Minnesota, 55455
New York Presbyterian Hospital
New York, New York, 10021
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Children'S Hospital of Orange County
Orange, California, 92868-3874
Nemours Childrens Clinic
Orlando, Florida, 32801
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107
Oregon Health and Science University
Portland, Oregon, 97239
Center of Excellence in Diabetes & Endocrinology
Sacramento, California, 95821
Children's Healthcare d.b.a Children's Hospitals and Clinics of Minnesota
Saint Paul, Minnesota, 55102
San Diego Medical Group; Pediatric Endocrinology
San Diego, California, 92123
Baystate Endocrinology and Diabetes; Baystate Children's Specialty Center, Pediatric Endocrinology
Springfield, Massachusetts, 01199
MultiCare Health System Institute for Research and Innovation
Tacoma, Washington, 98405
MultiCare Institute for Research and Innovation
Tacoma, Washington, 98405
The Pediatric Endocrine Office of Larry C. Deeb
Tallahassee, Florida, 32308
Pediatric Endrocine Assoc
Tampa, Florida, 33607
USF Diabetes Center
Tampa, Florida, 33612
About this study
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)
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.
- Conditions
- Growth Hormone Deficiency
- Being tested
- Somatropin
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SomatropinDrug
Also called Nutropin AQ v1.1
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.
| Study group | Role | What they receive |
|---|---|---|
| Somatropin | Experimental | 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. Receives: Drug: Somatropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Develop Anti-GH Antibodies After Treatment With Nutropin AQ v1.1
Participants who were tested positive to anti-GH antibody after initiation of study treatment.
Measured: Baseline up to 1 year
Secondary outcomes (5)
Percentage of Participants Who Exhibit Functional Growth Attenuation
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.
Measured: Baseline up to 1 year
Percentage of Participants With Neutralizing Antibodies
Among participants who developed positive anti-GH antibody post-baseline, participants who were tested positive to neutralizing anti-GH antibody during study participation.
Measured: Baseline up to 1 year
Annualized Growth Velocity at Months 6 and 12 (Change From Baseline)
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).
Measured: Months 6, 12
Height Standard Deviation Score (SDS) at Months 6 and 12 (Change From Baseline)
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.
Measured: Months 6, 12
Percentage of Participants With Adverse Events
Among participants who received at least one dose of study drug, those who reported at least one adverse event during study participation.
Measured: Baseline up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2014 (estimated)
- Study start31 Mar 2015
- Primary completion8 Nov 2017
- Study completion8 Nov 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- More trials run by Genentech, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 82 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genentech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02311894
- Sponsor's protocol ID
- ML29543
- Status last verified
- Dec 2018
- First submitted
- 4 Dec 2014
- Last updated
- 8 Jan 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02311894
- Acronym / sponsor ID
- ML29543
- Conditions
- Growth Hormone Deficiency
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 82 participants (actual)
- Lead sponsor
- Genentech, Inc.Industry (pharma, biotech, devices)
- Study start
- 31 Mar 2015 (actual)
- Primary completion
- 8 Nov 2017 (actual)
- Study completion
- 8 Nov 2017 (actual)
- First posted
- 9 Dec 2014 (estimated)
- Results
- Posted 15 Nov 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Somatropin | Experimental | Drug: SomatropinChildren 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. |
Who may qualify
- Ages
- 3 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SomatropinAlso: Nutropin AQ v1.1Somatropin 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. | Drug | Somatropin |
Primary outcome
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.
5 secondary outcomes
Percentage of Participants Who Exhibit Functional Growth Attenuation
Time frame: Baseline up to 1 year
Percentage of Participants With Neutralizing Antibodies
Time frame: Baseline up to 1 year
Annualized Growth Velocity at Months 6 and 12 (Change From Baseline)
Time frame: Months 6, 12
Height Standard Deviation Score (SDS) at Months 6 and 12 (Change From Baseline)
Time frame: Months 6, 12
Percentage of Participants With Adverse Events
Time frame: Baseline up to 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Growth Hormone Deficiency
RecruitingPhase 3
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
Turner Syndrome, Short Stature Homeobox Gene Mutation
RecruitingPhase 3
Randomized Clinical Trial of Two Different Initial Growth Hormone Doses in Children
Growth Disorders, Growth Failure
RecruitingPhase 3
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Growth Hormone Deficiency (GHD)
RecruitingPhase 3
Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)
Adult Growth Hormone Deficiency, Mild Traumatic Brain Injury
Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
33 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Somatropin in people with Growth Hormone Deficiency, run by Genentech, Inc. at 33 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 33 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Nov 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants Who Develop Anti-GH Antibodies After Treatment With Nutropin AQ v1.1 (time frame: Baseline up to 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2019.