Validation study for genetic markers for growth hormone treatment in growth hormone deficient and turner syndrome patients

• Register: EUCTR
• Last refreshed on: 3 December 2012
• Main ID: EUCTR2011-000460-10-CZ
• Date of registration: 22/06/2011
• Prospective Registration: Yes
• Primary sponsor: Merck Serono S.A. Geneva
• Public title: Validation study for genetic markers for growth hormone treatment in growth hormone deficient and turner syndrome patients
• Scientific title: First year growth response associated genetic markers validation Phase IV open-label study in Growth Hormone Deficient and Turner Syndrome pre-pubertal children : the PREDICT Pharmacogenetics Validation Study - PREDICT PGx Validation Study
• Date of first enrolment: 22/08/2011
• Target sample size: 395
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-000460-10
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 0

Countries of recruitment

Argentina, Canada, Czech Republic, France, Italy, Spain, United Kingdom

Contacts

• Name: Communication Center Merck KGaA
• Affiliation: Merck KGaA

• Name: Communication Center Merck KGaA
• Affiliation: Merck KGaA

Key inclusion & exclusion criteria

Inclusion criteria:
Pre-established diagnosis of IGHD or TS based on classical criteria
with at least 1 year of r-hGH therapy and with Tanner stage 1 at
treatment start
Retrospective availability of a complete set of clinical, auxological
and biological parameters necessary for building the predictive
model
Are the trial subjects under 18? yes
Number of subjects for this age range: 350
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 45
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Acquired GHD
Any drug or disease that could affect growth


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Idiopathic Growth Hormone Deficiency and Turner Syndrome
MedDRA version: 13.1 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders
MedDRA version: 13.1 Level: PT Classification code 10045181 Term: Turner's syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Trade Name: Saizen 8mg click.easy powder for solution for injection
Product Name: NA
Product Code: NA
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: somatropin
Current Sponsor code: NA
Other descriptive name: RECOMBINANT HUMAN GROWTH HORMONE
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 5.83-

Primary Outcome(s)

Secondary Objective: To evaluate the contribution of validated genetic markers related
to the amplitude of first year growth in IGHD children in
response to r-hGH therapy using a model derived from the
growth hormone deficiency (GHD) Kabi-Pharmacia
International Growth Study (KIGS) predictive model of the first
year growth response to r-hGH.
To evaluate the contribution of validated genetic markers related
to the amplitude of first year growth in TS girls in response to rhGH
therapy in TS girls using a model derived from the TS
KIGS predictive model of the first year growth response to r-hGH.

Primary end point(s): The list of genetic markers associated to the first
year growth response to r-hGH measured by the 3 following growth
parameters:
Change from baseline to year 1 in Height (cm)
Change from baseline to year 1 in Height standard deviation score
(SDS)
Height Velocity (HV) (cm/year) SDS at 1 year

Timepoint(s) of evaluation of this end point: at time of final report

Main Objective: To confirm that at least one of the genetic markers associated to the
amplitude of first year growth response to recombinant human
growth hormone (r-hGH) treatment identified in PREDICT Long-
Term Follow-Up (LTFU) Study (28614) is replicated in an
independent population of this study of prepubertal children with
either idiopathic growth hormone deficiency (IGHD) or Turner
Syndrome (TS).

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: at time of final report

Secondary end point(s): The first year growth predictive value of the models including the
validated genetic markers on HV (cm/year) at 1 year in IGHD
children and in TS girls treated with r-hGH combined to wellestablished
clinical, auxological and biological markers derived
from the KIGS GHD predictive model of the first year growth
response to r-hGH therapy

Secondary ID(s)

2011-000460-10-GB

EMR200104_010

Source(s) of Monetary Support

Merck Serono SA