Defining the Optimal Hormonal Replacement Therapy in Turner Syndrome

Turner Syndrome Clinical Trial

Official title:

The Growth of Genitalia Interna and the Bone Mineralization Under Hormonal Replacement Therapy and the Presence of Aortic Root Dilatation in Girls With Turner Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00134745
• Other study ID #: 120895
• Status: Completed
• Phase: Phase 4
• First received: August 24, 2005
• Last updated: June 13, 2013
• Start date: June 2005
• Est. completion date: March 2013

Study information

• Verified date: June 2013
• Source: Hillerod Hospital, Denmark
• Contact: n/a
• Is FDA regulated: No
• Health authority: Denmark: The Danish National Committee on Biomedical Research Ethics
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 43
• Est. completion date: March 2013
• Est. primary completion date: March 2013
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 10 Years to 25 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Pharmacodynamics Study, Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Gonadal Dysgenesis
• Primary Ovarian Insufficiency
• Syndrome
• Turner Syndrome

Intervention

Drug:
• estradiol
tablets, 2 mg day 1 through day 22 of the menstrual cycle for 5 years

Locations

Country	Name	City	State
Denmark	Pediatric Unit, Hillerod Hospital	Hillerod	Frederiksborg County

Sponsors (6)

Lead Sponsor	Collaborator
Line Cleemann	Novo Nordisk A/S, The County of Frederiksborg, The foundation of Kaptajnløjtnant Harald Jensen and Wife, The foundation of Mr. Ivan Nielsen, The foundation of Mrs. Olga Bryde

Country where clinical trial is conducted

Denmark, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The size of the uterus evaluated by magnetic resonance (MR)-scan		5 years	Yes
Primary	Bones evaluated by dual-energy X-ray absorptiometry (DEXA)-scan		5 years	Yes
Secondary	The diameter of the aortic root evaluated by MR-scan		5 years	Yes
Secondary	Development of biochemical markers		5 years	Yes
Secondary	Body composition evaluated by DEXA-scan		5 years	Yes
Secondary	Emotional wellbeing and self-esteem evaluated by questionnaires		5 years	Yes
Secondary	The size of the uterus evaluated by ultrasound		5 years	Yes