Growth Hormone Deficiency
Conditions
Keywords
metabolic outcomes, auxological outcomes, recombinant human growth hormone, long acting growth hormone
Brief summary
The METAB-BELGROW-LAGH study aims to prospectively evaluate the metabolic outcomes of Belgian children diagnosed with growth hormone deficiency (GHD) over a three-year period following the initiation of treatment with either daily recombinant human growth hormone (rhGH) or weekly long-acting growth hormone (LAGH). Patients will be stratified according to their IGF-1 levels. The primary objective is to assess the metabolic outcomes of children treated with daily rhGH over the first three years. The secondary objectives include comparing the metabolic outcomes between patients treated with daily rhGH and those treated with weekly LAGH, and characterizing metabolic profiles based on IGF-1 levels. To achieve these objectives, both standard-of-care (SOC) and additional data will be collected at scheduled follow-up visits (baseline, 6, 12, 24, and 36 months), including clinical, auxological, and biological parameters. Additional metabolic markers, inflammatory and endothelial biomarkers will be assessed. In a subset of patients, carotid intima-media thickness (cIMT) and body fat distribution (via DEXA-scan) will also be measured.
Interventions
OTHERmetabolic and auxological outcomes
Evaluation over a three-year period following the initiation of treatment with daily recombinant human growth hormone (rhGH)
Sponsors
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
NONE
Intervention model description
Patients will be classified into two groups (depending on their treatment as decided by their pediatric endocrinologist): * patients with GHD who receive daily rhGH= rhGH cohort * patients with GHD who receive weekly LAGH= LAGH cohort
Eligibility
Sex/Gender
ALL
Age
5 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosed with growth hormone deficiency (GHD) * Treated with growth hormone (rhGH or LAGH)
Exclusion criteria
* No
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Auxological evaluation_1 | 3 years | Primary outcome measures included change in stature (in cm and SDS) over the course of the study. |
| Auxological evaluation_2 | 3 years | Primary outcome measures included change in weight (in kgs and SDS) over the course of the study. |
| Auxological evaluation_3 | 3 years | Primary outcome measures included change in BMI (in kg/m2) over the course of the study. |
| Evaluation of the blood pressure | 3 years | Measure of the systolic and diastolic blood pressure (in mmHg) with a sphygmomanometer |
| Evaluation of waist circumference | 3 years | Measure of waist circumference (in cm) with a meter |
| Evaluation of clinical signs of puberty (Tanner stage) and androgenism | 3 years | Evaluation of clinical signs of puberty (Tanner stage) and androgenism. |
| Metabolic evaluation_1 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of fasting glycemia (mg/dl). |
| Metabolic evaluation_2 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of fasting insulin (pmol/L). |
| Metabolic evaluation_3 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of hemoglobin A1c in diabetic patients (%). |
| Metabolic evaluation_4 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of transaminases (UI/L). |
| Metabolic evaluation_5 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of lipid profile (mg/dL). |
| Metabolic evaluation_6 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of creatinine (mg/dL). |
| Metabolic evaluation_7 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of alkaline phosphatase (UI/L). |
| Metabolic evaluation_8 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of calcium (mmol/L). |
| Metabolic evaluation_9 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of phosphorus (mmol/L). |
| Metabolic evaluation_10 | 3 years | Evaluation of the change of the metabolic profile using both SOC and non-SOC Blood tests samples. Measurements of insulin-like growth factor 1 levels (ng/mL). |
| Bone markers_1 | 3 years | Measurements in blood samples of bone markers such as procollagen type 1 N-terminal propeptide (µg/L). |
| Bone markers_2 | 3 years | Measurements in blood samples of bone markers such as insulin-like growth factor (ng/mL). |
| Bone markers_3 | 3 years | Measurements in blood samples of bone markers such as insulin-like growth factor binding protein (µL/mL). |
| Endothelial stress and inflammatory markers_1 | 3 years | Measurements in blood samples of endothelial stress and inflammatory markers such as asymmetric dimethylarginine (µmol/L). |
| Endothelial stress and inflammatory markers_2 | 3 years | Measurements in blood samples of endothelial stress and inflammatory markers such as TNF-a (pg/mL). |
| Endothelial stress and inflammatory markers_3 | 3 years | Measurements in blood samples of endothelial stress and inflammatory markers such as VEGF (pg/mL). |
| Endothelial stress and inflammatory markers_4 | 3 years | Measurements in blood samples of endothelial stress and inflammatory markers such as IL-6 (pg/mL). |
| Radiological evaluation of cIMT | 3 years | Measurements of cIMT (mm) using Doppler ultrasound. |
| Radiological evaluation of body fat distribution | 3 years | Measurements of body fat distribution (g/cm2) using DEXA-Scan. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| comparison with patients under weekly LAGH | 3 years | Data obtained for patients with daily rhGH will be compared to data obtained for patients with weekly LAGH treatment. Same measurement description as for the primary outcomes. |
| Stratification of the metabolic profile of the patients according to IGF-1 levels | 3 years | — |
Contacts
CONTACTLaure Boutsen, MD
CONTACTMicheline Grillet, PhD
PRINCIPAL_INVESTIGATORPhilippe Lysy, MD, PhD
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Outcome results
None listed