- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04938466
Assessment of Adherence, Quality of Life, Clinical Response and Safety of Daily and Long-Acting Growth Hormone Therapy (LAuGH TRACK)
July 26, 2023 updated by: University of Minnesota
The purpose of the study is to compare quality of life, adherence, insulin resistance, body composition and efficacy of long-acting growth hormone (LAGH) to daily growth hormone (DGH) in children with growth hormone deficiency (GHD).
These objectives will be evaluated every 6 months for subjects prior to switch from DGH to LAGH, and 6 months after.
Study Overview
Status
Recruiting
Intervention / Treatment
Detailed Description
Daily subcutaneous injections of recombinant human growth hormone (GH) is the standard of care therapy for children with GH deficiency (GHD).
A potential impediment to long-term efficacy of daily GH (DGH) is a lack of adherence and persistence.
Multiple Long-Acting Growth Hormone (LAGH) molecules are being developed with the expectation that reduced frequency of injections will improve the patient and caregiver experience leading to improved adherence and, ultimately, improve efficacy.
However, the connection between reduced injection frequency, improved adherence and improved efficacy have not yet been demonstrated.
GH has metabolic effects that impact insulin resistance, body composition and lipid levels.
There is concern that persistently elevated levels of GH provided by LAGH may have a negative effect on metabolism.
There is also concern that the larger size of the LAGH molecules in development may limit their access to the target tissues leading to an imbalance of the linear growth and metabolic effects.
The purpose of the study is to compare quality of life, adherence, insulin resistance, body composition and efficacy of long-acting growth hormone (LAGH) to daily growth hormone (DGH) in children with growth hormone deficiency (GHD).
These objectives will be evaluated every 6 months for subjects prior to switch from DGH to LAGH, and 6 months after.
Study Type
Observational
Enrollment (Estimated)
80
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Bradley S Miller, MD, PhD
- Phone Number: (612)-624-5409
- Email: mille685@umn.edu
Study Locations
-
-
Minnesota
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Minneapolis, Minnesota, United States, 55455
- Recruiting
- University of Minnesota
-
Contact:
- Bradley S Miller, MD, PhD
- Phone Number: 612-624-5409
- Email: mille685@umn.edu
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
2 years to 13 years (Child)
Accepts Healthy Volunteers
No
Sampling Method
Probability Sample
Study Population
Children receiving daily growth hormone treatment for growth hormone deficiency (GHD).
Description
Inclusion Criteria:
- Girls must be between the ages of 2 and 11 years, and boys must be between the ages of 2 and 13 years
- Have established diagnosis of pediatric growth hormone deficiency (GHD). For the purposes of the study, GHD is defined as peak growth hormone response to clonidine/arginine stimulation testing of <10 ng/mL
- Either treatment-naive or currently treated with a daily growth hormone as approved by health insurance
Exclusion Criteria:
- Any medical condition which, in the opinion of the Investigator, can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment
- Current treatment with long-acting growth hormone
- Currently pregnant or breastfeeding
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Transition to long-acting growth hormone (LAGH)
Participants in this arm will transition between daily growth hormone treatment and long-acting growth hormone treatment.
|
Long-acting growth hormone (LAGH) treatment
|
Consistent daily growth hormone (DGH)
Participants in this group will continue with daily growth hormone treatment.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Insulin Resistance
Time Frame: Every six months for approximately 2 years
|
Fasting glucose and insulin will be measured using standard laboratory tests in order to calculate homeostatic model assessment of insulin resistance (HOMA-IR).
The equation is as follows, fasting plasma glucose (mmol/l) multiplied by fasting serum insulin (mU/l) divided by 22.5.
HOMA-IR is a unitless measure with no defined range.
Higher scores indicate lower insulin sensitivity/higher insulin resistance.
Insulin resistance will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Change in Bone Mineral Density - DXA
Time Frame: Every six months for approximately 2 years
|
Bone mineral density will be measured using dual energy X-ray absorptiometry (DXA) reported in units of grams per centimeter^2.
Bone density will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Change in Bone Mineral Density - pQCT
Time Frame: Every six months for approximately 2 years
|
Bone mineral density will be measured using peripheral quantitative computed tomography (pQCT) and reported in units of grams per centimeter^3.
Bone density will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Change in Percent Fat
Time Frame: Every six months for approximately 2 years
|
Percent fat will be measured using dual energy X-ray absorptiometry (DXA), peripheral quantitative computed tomography (pQCT), and BodPod and reported in units of percent kilograms.
Percent fat will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Change in Fat Free Mass
Time Frame: Every six months for approximately 2 years
|
Fat free mass will be measured using dual energy X-ray absorptiometry (DXA), peripheral quantitative computed tomography (pQCT), and BodPod and reported in units of kilograms.
Fat free mass will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Change in Muscle Content - DXA
Time Frame: Every six months for approximately 2 years
|
Muscle content will be measured using dual energy X-ray absorptiometry (DXA) and BodPod and reported in units of percent kilograms.
Muscle content will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Change in Muscle Content - pQCT
Time Frame: Every six months for approximately 2 years
|
Muscle content will be measured using peripheral quantitative computed tomography (pQCT) and reported in units of grams per centimeter^3.
Muscle content will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in PedsQL Questionnaire
Time Frame: Every six months for approximately 2 years
|
The PedsQL Questionnaire contains 23 items rated on a 3- or 5-point Likert scale.
Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0.
Total score is calculated as the unweighted average of item scores.
Higher scores indicate better health-related quality of life.
PedsQL will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Change in QOLISSY Questionnaire
Time Frame: Every six months for approximately 2 years
|
The QOLISSY Questionnaire contains 66 items rated on a 5-point Likert scale.
Total score is calculated as the mean of item scores.
Total scores range from 66 to 330 with higher scores indicating better quality of life.
QOLISSY will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Change in DID-EQ Questionnaire
Time Frame: Every six months for approximately 2 years
|
The Diabetes Injection Device - Experience Questionnaire (DID-EQ) contains 10 items on a 4-point Likert scale.
Total score is calculated as a weighted average of item scores.
Total scores range from 10 to 40 with higher score indicating more positive perceptions of injection device characteristics.
DID-EQ will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Change in DID-PQ Questionnaire
Time Frame: Every six months for approximately 2 years
|
The Diabetes Injection Device - Preference Questionnaire (DID-EQ) contains 10 items on a 5-point Likert scale (-2 to 2), with a total range of -20 to 20.
Scores on each end of the scale equal a more positive perception of the device.
A higher score indicates a stronger preference for LAGH device and a lower (negative) score indicates a preference for daily growth hormone device.
Results will be reported descriptively and categorically as the frequency and percentage of patients reporting each response to each item.
DID-EQ will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to
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Every six months for approximately 2 years
|
Change in Adherence Survey
Time Frame: Every six months for approximately 2 years
|
An adherence survey will be administered and medication possession ratio (MPR) data will be obtained.
Likert scale items will be measured with a range of 26 to 121, with a higher score indicating a greater burden to growth hormone treatment.
Non-Likert scale items (2 items with percent report and 2 items with Yes/No responses) will not be added to a total score but will be reported descriptively.
Adherence will be assessed at baseline, 6 months prior to beginning LAGH treatment, and 6 months following the start of LAGH treatment, or every 6 months for two years for those who do not switch to LAGH.
Change in values will be reported, for participants on Daily Growth Hormone who never switch to LAGH, from baseline to values obtained at 6 months, 12 months, 18 months, and 24 months.
For participants who switch to LAGH, baseline values pre-LAGH treatment will be compared to those obtained 6 months following the start of LAGH treatment.
|
Every six months for approximately 2 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Bradley S Miller, MD, PhD, University of Minnesota Department of Pediatric Endocrinology
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
August 5, 2021
Primary Completion (Estimated)
October 1, 2024
Study Completion (Estimated)
October 1, 2024
Study Registration Dates
First Submitted
June 16, 2021
First Submitted That Met QC Criteria
June 16, 2021
First Posted (Actual)
June 24, 2021
Study Record Updates
Last Update Posted (Actual)
July 27, 2023
Last Update Submitted That Met QC Criteria
July 26, 2023
Last Verified
July 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- PEDS-2020-28945
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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