Isolated growth hormone deficiency in pubertal children Nutritional, Metabolic, Endocrine
Conditions
Interventions
Control arm: Standard care - Continuation of growth hormone medication (somatropin) as daily injections, until final height. Treatment will last from the point of randomisation, up until 6 weeks befor
Sponsors
University College London
Eligibility
Sex/Gender
All
Age
8 Years to 17 Years
Inclusion criteria
Inclusion criteria: 1. Initial diagnosis of I-GHD will have been made by either two GH stimulation tests (peak GH <6.7µg/L) or one abnormal stimulation test with low IGF-1 (below normal range for sex & age), irrespective of sex-hormone priming for GH stimulation tests 2. Children with reversed I-GHD (peak GH =6.7 µg/L and a serum IGF-1 within normal reference range for sex and age) and a normal brain MRI (incl. small anterior pituitary) 3. Children in established puberty – Tanner stages B2/3 in girls & 6-12ml testes* in boys (as measured by orchidometer**) 4. Children will have discontinued GH treatment for a minimum of 6 weeks prior to re-testing 5. Children will have remained off GH therapy from time of re-test until randomisation 6. Ability to tolerate the administration of GH therapy 7. Ability to comply with trial schedule and follow up 8. Written informed consent obtained from the patient’s parent/guardian and written assent obtained from patient (where age appropriate). Patients aged 16 years or older will provide their own written informed consent *In the event of discrepancy between the size of an individual’s testicles, the larger testicle should be used **In the event that the size of a patient’s testicle falls between the measuring beads of the orchidometer and it is not clear which bead the testicle is most similar to, the larger bead should be used
Exclusion criteria
Exclusion criteria: 1. Multiple pituitary hormone deficiency (hypopituitarism) with or without additional pituitary hormone supplementation 2. Known genetic cause of I-GHD 3. Organic GHD (mid-brain tumours, congenital mid-brain malformations, septo-optic dysplasia; radiotherapy to the total body or brain) 4. Ectopic posterior pituitary 5. Other indications for GH therapy 6. Receiving GH treatment during the (minimum 6 week) discontinuation period 7. Receiving prednisolone or dexamethasone for a period of 4 weeks or longer in the time period immediately prior to randomisation 8. Known history of persistent non-compliance with prescribed medication regimens 9. Pregnant or lactating 10. Any malignancy 11. Currently participating in another Clinical Trial of an Investigational Medicinal Product (CTIMP)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Height (cm) measured in Standard Deviation Score (FH SDS) at end of follow-up | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Bone-related: 1.1. Bone age delay measured using BoneXpert X-ray analysis at end of follow-up 1.2. Bone age acceleration measured using BoneXpert X-ray analysis between enrolment and end of follow-up 1.3. Bone health index measured using BoneXpert X-ray analysis at end of follow-up 2. Biochemistry: 2.1. Serum IGF-1 and lipid profiles measured using trial site's usual testing methods at end of follow-up 2.2. Peak stimulated GH measured using insulin tolerance test or argnine test at end of follow-up 3. Adverse events measured using GHD Reversal Trial CRFs over follow-up duration 4. Health Economics: 4.1. Cost per percentage achieving Target Height measured using healthcare contacts costs (captured via GHD Reversal Trial CRFs) over follow-up duration 4.2. Cost per Quality Adjusted Life Year (QALY) gained, measured using CHU-9D questionnaire over follow-up duration 5. Qualitative Research: Trial acceptability (parents, patients and staff); reasons to decline the trial; parent and patient experience of the trial and treatment pathways, measured via interviews with parents, patients and site staff during the pilot phase of the trial | — |
Countries
Austria, England, United Kingdom
Contacts
Public ContactAdam Khan
Outcome results
None listed