Clinical trial NCT01066052: Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
Official title: Collaborative Study to Assess the Effects of Treatment With Recombinant Growth Hormone Saizen® in the Prevention of Short Stature in Young Girls Suffering From Turner Syndrome Before the Age of 4 Years. Original French Title: Etude Collaborative Pour apprécier Les Effets du Traitement Par l'Hormone de Croissance Recombinante SAIZEN® Dans le Retard de Croissance de la Fillette Atteinte de Syndrome de Turner Avant l'âge de 4 Ans
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Turner's Syndrome
Keywords: Growth hormone
Turner syndrome
r-hGH
early treatment
Lead sponsor: Merck KGaA, Darmstadt, Germany
Sponsor class: INDUSTRY
First posted: 2010-02-10
Enrollment: 115
Sex: FEMALE
Maximum age (years): 4
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years. After 4 years of treatment, height in these girls will be compared with an historical control group of untreated girls with Turner Syndrome, matched for age and height at baseline.


Eligibility criteria: Inclusion Criteria:

* Young girls with turner syndrome proved by karyotype
* Growth hormone secretion confirmed with ornithin stimulation test
* Normal glucidic metabolism confirmed by assessment of HbA1c
* None associated severe pathology which could have impact on growth (i.e. renal insufficiency, decompensated heart failure)
* No previous or associated treatment with anabolic or sexual steroids
* Known parental height

Exclusion Criteria:

* Severe associated pathology with impact on growth
* Concomitant treatment with impact on growth
* Previous or associated treatment with anabolic steroids
* Associated growth hormone deficiency

Primary outcomes:
measure: Height SDS at Year 4
timeFrame: Year 4
Secondary outcomes:
measure: Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels
timeFrame: Baseline up to Year 2
measure: Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)
timeFrame: Baseline, Year 1, Year 2
measure: Number of Participants With Anti r-hGH Antibodies
timeFrame: Baseline up to Year 2
measure: Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels
timeFrame: Baseline up to Year 2
measure: Number of Participants Who Reached Normal Height at Year 4
timeFrame: Year 4
Other outcomes:
Locations:
city: Paris
country: France
facility: Hôpital Robert Debré
Study officials:
affiliation: Merck KGaA, Darmstadt, Germany
name: Medical Responsible
role: STUDY_DIRECTOR
References:
citation: Linglart A, Cabrol S, Berlier P, Stuckens C, Wagner K, de Kerdanet M, Limoni C, Carel JC, Chaussain JL; French Collaborative Young Turner Study Group. Growth hormone treatment before the age of 4 years prevents short stature in young girls with Turner syndrome. Eur J Endocrinol. 2011 Jun;164(6):891-7. doi: 10.1530/EJE-10-1048. Epub 2011 Mar 11.