Clinical trial NCT04938466: Assessment of Adherence, Quality of Life, Clinical Response and Safety of Daily and Long-Acting Growth Hormone Therapy
Official title: Assessment of Adherence, Quality of Life, Clinical Response and Safety of Daily and Long-Acting Growth Hormone Therapy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Growth Hormone Deficiency
Growth Hormone Treatment
Lead sponsor: University of Minnesota
Sponsor class: OTHER
First posted: 2021-06-24
Enrollment: 19
Sex: ALL
Minimum age (years): 2
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Children receiving daily growth hormone treatment for growth hormone deficiency (GHD).

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in Insulin Resistance
timeFrame: Every six months for approximately 2 years
measure: Change in Bone Mineral Density - DXA
timeFrame: Every six months for approximately 2 years
measure: Change in Bone Mineral Density - pQCT
timeFrame: Every six months for approximately 2 years
measure: Change in Percent Fat
timeFrame: Every six months for approximately 2 years
measure: Change in Fat Free Mass
timeFrame: Every six months for approximately 2 years
measure: Change in Muscle Content - DXA
timeFrame: Every six months for approximately 2 years
measure: Change in Muscle Content - pQCT
timeFrame: Every six months for approximately 2 years
Secondary outcomes:
measure: Change in PedsQL Questionnaire
timeFrame: Every six months for approximately 2 years
measure: Change in QOLISSY Questionnaire
timeFrame: Every six months for approximately 2 years
measure: Change in DID-EQ Questionnaire
timeFrame: Every six months for approximately 2 years
measure: Change in DID-PQ Questionnaire
timeFrame: Every six months for approximately 2 years
measure: Change in Adherence Survey
timeFrame: Every six months for approximately 2 years
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
Study officials:
affiliation: University of Minnesota Department of Pediatric Endocrinology
name: Bradley S Miller, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: