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

NCT04938466 | Completed FDA Drug

• 1 other identifier: PEDS-2020-28945
• Study Type: observational
• Target: 19
• Locations: 1 country
• Sites: 1

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.

Conditions

Growth Hormone Deficiency; Growth Hormone Treatment

Outcome Measures

Primary Outcomes (7)

• Change in Insulin Resistance

  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

  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

  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

  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

  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

  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

  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 Outcomes (5)

• Change in PedsQL Questionnaire

  Every six months for approximately 2 years

• Change in QOLISSY Questionnaire

  Every six months for approximately 2 years

• Change in DID-EQ Questionnaire

  Every six months for approximately 2 years

• Change in DID-PQ Questionnaire

  Every six months for approximately 2 years

• Change in Adherence Survey

  Every six months for approximately 2 years

Study Arms (2)

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)

Consistent daily growth hormone (DGH)

Participants in this group will continue with daily growth hormone treatment.

Interventions

Long-Acting Growth Hormone (LAGH) DRUG

Long-acting growth hormone (LAGH) treatment

Transition to long-acting growth hormone (LAGH)

Eligibility Criteria

• Age: 2 Years - 13 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

Children receiving daily growth hormone treatment for growth hormone deficiency (GHD).

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Minnesota: lead

Study Sites (1)

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Blood serum samples for Growth Hormone Antibodies will be stored in the freezer storage of the M Health Fairview University of Minnesota Medical Center.

MeSH Terms

Conditions

Dwarfism, Pituitary

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Study Officials

• Bradley S Miller, MD, PhD

  University of Minnesota Department of Pediatric Endocrinology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 16, 2021
• First Posted: June 24, 2021
• Study Start: August 5, 2021
• Primary Completion: October 30, 2024
• Study Completion: October 30, 2024
• Last Updated: November 19, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)