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Defining the Optimal Hormonal Replacement Therapy in Turner Syndrome

Phase 4
Completed
Conditions
Turner Syndrome
Interventions
Registration Number
NCT00134745
Lead Sponsor
Line Cleemann
Brief Summary

The purpose of this study is to examine whether a larger dosage of estrogen than the one used today will secure the development of a normal sized uterus and increase the strength of the bones in girls and young women with Turner syndrome.

The purpose is also to evaluate whether aortic dilatation is present in this group of patients, and if the estrogen dosage will influence the emotional well-being and self-esteem of the patients.

Detailed Description

Turner syndrome is a common chromosomal disorder with only one X-chromosome or partial deletions in one X-chromosome in all or some of the cell-lines in the body. Appr. 18 girls/year are being born with the syndrome in Denmark. The syndrome is mostly known for reduced final height and the lack of pubertal development with infertility, but it is also known for diseases influencing other parts of the body like the heart with the risk of development of aortic dilatation and subsequently dissection with the risk of rupture, profuse bleeding and sudden death. Reduced bone strength and increased risk of bone fracture are also features of the syndrome. Treatment with growth hormone and female sex-hormones are well-established treatments.

Girls and young women with Turner syndrome ages 10 to 21 years will participate. The girls ages 15 to 21 years will randomly and double blindly receive treatment with either 2 mg or 4 mg estrogen for 5 years. Yearly examinations with blood tests, physical examinations, questionnaires, ultrasound and MR-scan of the internal female genitalia, DEXA-scan of the bones and MR-scan of the heart will be performed.

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
43
Inclusion Criteria
  • Verified Turner syndrome
  • Ages 10-25 years
Exclusion Criteria
  • Contraindications to the MR-scan
  • Contraindications to the trial medication
  • Severe or chronic sickness with impact on the parameters of the study or incompatibility with the trial medication
  • Intake of medications with interactions with trial medication

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
4 mg estradiolestradiol-
2 mg estradiolestradiol-
Primary Outcome Measures
NameTimeMethod
The size of the uterus evaluated by magnetic resonance (MR)-scan5 years
Bones evaluated by dual-energy X-ray absorptiometry (DEXA)-scan5 years
Secondary Outcome Measures
NameTimeMethod
The diameter of the aortic root evaluated by MR-scan5 years
Development of biochemical markers5 years
Body composition evaluated by DEXA-scan5 years
Emotional wellbeing and self-esteem evaluated by questionnaires5 years
The size of the uterus evaluated by ultrasound5 years

Trial Locations

Locations (1)

Pediatric Unit, Hillerod Hospital

🇩🇰

Hillerod, Frederiksborg County, Denmark

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