Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years

Turner's Syndrome Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01066052
• Other study ID #: GF 5834
• Status: Completed
• Phase: Phase 4
• Start date: February 29, 1992
• Est. completion date: August 31, 2010

Study information

• Verified date: June 2018
• Source: Merck KGaA, Darmstadt, Germany
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 115
• Est. completion date: August 31, 2010
• Est. primary completion date: March 31, 1999
• Accepts healthy volunteers: No
• Gender: Female
• Age group: N/A to 4 Years

Eligibility

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

Related Conditions & MeSH terms

• Dwarfism
• Gonadal Dysgenesis
• Primary Ovarian Insufficiency
• Syndrome
• Turner Syndrome
• Turner's Syndrome

Intervention

Drug:
• r-hGH
Subcutaneous administration.

Locations

Country	Name	City	State
France	Hôpital Robert Debré	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Merck KGaA, Darmstadt, Germany

Country where clinical trial is conducted

France, 

References & Publications (1)

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 syn — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Height SDS at Year 4	Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender.	Year 4
Secondary	Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels	HbA1c develops when hemoglobin, a protein within red blood cells that carries oxygen throughout the body, joins with glucose in the blood, becoming glycated. The higher the level of glucose in the blood, the higher the level of HbA1c is detectable on red blood cells. The normal range for HbA1c is 4 percent (%) to 5.9%. Number of participants, who had abnormal HbA1c levels any time during the assessment, were reported.	Baseline up to Year 2
Secondary	Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)	BA was determined using left wrist and hand X-ray. CA was determined using the date of birth. Difference of BA and CA (BA-CA) was reported.	Baseline, Year 1, Year 2
Secondary	Number of Participants With Anti r-hGH Antibodies		Baseline up to Year 2
Secondary	Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels	The normal range for IGF1 levels is 45 to 117 nanogram per milliliter (ng/mL) for girls aged less than (<) 3 years and 80 to 236 ng/mL for girls aged 3 to 6 years. Values outside the normal range were considered abnormal. Number of participants, who had abnormal IGF1 levels any time during the assessment, were reported.	Baseline up to Year 2
Secondary	Number of Participants Who Reached Normal Height at Year 4	Participants with normal height were those who attained a height which was within +/- 2 height SDS of reference population standard. Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender.	Year 4