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

University of Minnesota (UMN)

Status

Enrolling

Conditions

Growth Hormone Treatment

Growth Hormone Deficiency

Treatments

Drug: Long-Acting Growth Hormone (LAGH)

Study type

Observational

Funder types

Other

Identifiers

NCT04938466

PEDS-2020-28945

Details and patient eligibility

About

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.

Full 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.

Enrollment

80 estimated patients

Sex

All

Ages

2 to 13 years old

Volunteers

No Healthy Volunteers

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