Evaluation of Overall Compliance and Duration of Zomacton® Treatment With the Zomajet® Needle-free Device

Completed

• Conditions: Turner's Syndrome; Human Growth Hormone Deficiency
• Interventions: Drug: Somatropin

• Registration Number: NCT01306357
• Lead Sponsor: Ferring Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2007-09
• Study First Submitted: 2011-02-28
• Study First Submitted QC: 2011-02-28
• Study First Posted: 2011-03-01
• Primary Completion: 2011-10
• Study Completion: 2011-11
• Status Verified: 2012-02
• Last Update Submitted: 2023-11-15
• Last Update Posted: 2023-11-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 87

Inclusion Criteria

• Growth hormone deficiency
• Turner's syndrome

Exclusion Criteria

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Zomacton® with Zomajet® needle-free device	Somatropin	Zomacton® 4 mg delivered by percutaneous transjection (needle-free) using the Zomajet® 2 Vision device or Zomacton® 10 mg delivered by percutaneous transjection (needle-free) using the Zomajet® Vision X needle-free device.

Primary Outcome Measures

Name	Time	Method
Overall treatment compliance	up to three years

Secondary Outcome Measures

Name	Time	Method
Average Duration of Treatment	up to three years
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

Trial Locations

Locations (1)

Investigational site; 🇫🇷 Toulouse, France