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

Merck KGaA (EMD Serono)

Status and phase: Completed; Phase 4

Conditions: Turner's Syndrome

Treatments: Drug: r-hGH

Study type: Interventional

Funder types: Industry

Identifiers

NCT01066052

GF 5834

Details and patient eligibility

About

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.

Enrollment: 115 patients

Sex: Female

Ages: Under 4 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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115 participants in 2 patient groups

r-hGH

Experimental group

Description:

Participants (girls) will receive r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received will depend on participants' baseline height standard deviation score (SDS) relative to the general population standard: participants with a height SDS of -2 standard deviation (SD) or lower will receive 0.05 milligrams per kilogram (mg/kg) per day r-hGH and those with a height SDS between -1 and -2 SD will receive 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants will receive a fixed dose of 0.05 mg/kg per day for a further 2 years.

Treatment:

Drug: r-hGH

Historical Control

No Intervention group

Description:

This arm will include matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated.