Human Growth Hormone Deficiency, Turner's Syndrome
Conditions
Keywords
Turner's syndrome, somatropin, human growth hormone deficiency
Brief summary
The purpose of this strictly observational, prospective, longitudinal study is to evaluate with sufficient precision the rate of overall treatment compliance from one year to 3 years of follow-up of the patients. Somatotropin is indicated in the long-term treatment of children with growth retardation related to a deficiency in secretion of growth hormone and in the long-term treatment of growth retardation related to Turner's syndrome confirmed by chromosomal analysis. These are the two indications of Zomacton® 4 mg and 10 mg injection solution. The use of the Zomajet® needle-free device (Zomajet® 2 Vision, reserved for the administration of Zomacton® 4 mg or of the Zomajet® Vision X needle-free device, reserved for the administration of Zomacton® 10 mg), allows the product to be administered by percutaneous transjection (needle-free) and can be used by the child directly or by the family after an initial training. In April 2004, the CEPP (Commission for the Evaluation of Products and Services) requested a follow-up of the cohort of patients using the Zomajet® 2 Vision system measuring the compliance and duration of use of the device. The number of patients initiated on Zomacton treatment using the Zomajet® needle-free device is estimated to 30. Over a period of inclusion of 3 years, we therefore estimate that 90 patients will be treated. In the cohort studied the patients will be followed-up for 1 year at least and for 3 years at the maximum. The rate of treatment compliance will be evaluated according to the ratio of the actual duration of administration over the total duration recommended by the physician during the observation period.
Interventions
DRUGSomatropin
4 mg or 10 mg delivered by needle-free device
Sponsors
Ferring Pharmaceuticals
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
1 Years to 16 Years
Healthy volunteers
No
Inclusion criteria
* Growth hormone deficiency * Turner's syndrome
Exclusion criteria
* Patients who do not meet the criteria in the treatment Information Sheet
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall treatment compliance | up to three years |
Secondary
| Measure | Time frame |
|---|---|
| Description of the auxological and biochemical characteristics of the population at inclusion (notably the exploration of GH deficiency, the height, the difference in height from the average in SD, the rate of growth prior to treatment) | Baseline (day 0) |
| Description of the Dosages of Growth Hormone and way of use of needle-free device | up to 3 years |
| Description of the evolution of the auxological and biochemical parameters (gain in height in SD, growth rate, IGF-1 if available) | Baseline (Day 0), up to three years |
| Average Duration of Treatment | up to three years |
Countries
France
Outcome results
None listed