NCT04938466

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 19 (actual)
• Sponsor: University of Minnesota · Academic / Other
• Sex: All
• Age: 2 Years – 13 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Growth Hormone Deficiency
• Growth Hormone Treatment

Interventions

Type	Name	Description
DRUG	Long-Acting Growth Hormone (LAGH)	Long-acting growth hormone (LAGH) treatment

Timeline

• Start date: 2021-08-05
• Primary completion: 2024-10-30
• Completion: 2024-10-30
• First posted: 2021-06-24
• Last updated: 2024-11-19

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study