Assessment of Adherence, Quality of Life, Clinical Response and Safety of Daily and Long-Acting Growth Hormone Therapy (LAuGH TRACK)

November 18, 2024 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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
19

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

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Bradley S Miller, MD, PhD
  • Phone Number: (612)-624-5409
  • Email: mille685@umn.edu

Study Locations

  • United States
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Transition to long-acting growth hormone (LAGH)
Participants in this arm will transition between daily growth hormone treatment and long-acting growth hormone treatment.
Drug: Long-Acting Growth Hormone (LAGH)
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University of Minnesota

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 5, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 30, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 30, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 16, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 16, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 24, 2021

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 19, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 18, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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