Clinical trial NCT01306357: Evaluation of Overall Compliance and Duration of Zomacton® Treatment With the Zomajet® Needle-free Device
Official title: Evaluation De L'Observance Globale Et De La Duree De Traitement Par Zomacton® Avec Le Stylo Transjecteur Zomajet® Etude Pharmaco-Epidemiologique Observationnelle Longitudinale Prospective
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Turner's Syndrome
Human Growth Hormone Deficiency
Keywords: Turner's syndrome
somatropin
human growth hormone deficiency
Lead sponsor: Ferring Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2011-03-01
Enrollment: 87
Sex: ALL
Minimum age (years): 1
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients who meet the criteria of the Treatment Information Sheet (growth hormone deficiency or Turner's syndrome) for which treatment is initiated with Zomacton® 4 mg using the Zomajet® 2 Vision needle-free device or with Zomacton® 10 mg using the Zomajet® Vision X.

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.


Eligibility criteria: Inclusion Criteria:

* Growth hormone deficiency
* Turner's syndrome

Exclusion Criteria:

* Patients who do not meet the criteria in the treatment Information Sheet

Primary outcomes:
measure: Overall treatment compliance
timeFrame: up to three years
Secondary outcomes:
measure: 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)
timeFrame: Baseline (day 0)
measure: Description of the Dosages of Growth Hormone and way of use of needle-free device
timeFrame: up to 3 years
measure: Description of the evolution of the auxological and biochemical parameters (gain in height in SD, growth rate, IGF-1 if available)
timeFrame: Baseline (Day 0), up to three years
measure: Average Duration of Treatment
timeFrame: up to three years
Other outcomes:
Locations:
city: Angers
country: France
facility: Investigational site
city: Antibes Juan Les Pins
country: France
facility: Investigational site
city: Bordeaux
country: France
facility: Investigational site
city: Brive La Gaillarde
country: France
facility: Investigational site
city: Hyeres
country: France
facility: Investigational site
city: Le Mans
country: France
facility: Investigational site
city: Lille
country: France
facility: Investigational site
city: Lisieux
country: France
facility: Investigational site
city: Montivilliers
country: France
facility: Investigational site
city: Montpellier
country: France
facility: Investigational site
city: Nice
country: France
facility: Investigational site
city: Nieul Sur Mer
country: France
facility: Investigational site
city: Paris
country: France
facility: Investigational site
city: Puyricard
country: France
facility: Investigational site
city: Tarbes
country: France
facility: Investigational site
city: Toulon
country: France
facility: Investigational site
city: Toulouse
country: France
facility: Investigational site
Study officials:
affiliation: Ferring Pharmaceuticals
name: Clinical Development Support
role: STUDY_DIRECTOR
References: