Defining the Optimal Hormonal Replacement Therapy in Turner Syndrome

Sponsor

Line Cleemann (Other)

Overall Status

Completed

CT.gov ID

NCT00134745

Collaborator

Novo Nordisk A/S (Industry), The County of Frederiksborg (Other), The foundation of Kaptajnløjtnant Harald Jensen and Wife (Other), The foundation of Mrs. Olga Bryde (Other), The foundation of Mr. Ivan Nielsen (Other)

Enrollment

Location

Arms

Duration (Months)

0.5

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Turner Syndrome	Drug: estradiol	Phase 4

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.

Study Design

Study Type:

Interventional

Actual Enrollment :

43 participants

Allocation:

Randomized

Intervention Model:

Single Group Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

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

Study Start Date :

Jun 1, 2005

Actual Primary Completion Date :

Mar 1, 2013

Actual Study Completion Date :

Mar 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: 4 mg estradiol	Drug: estradiol tablets, 2 mg day 1 through day 22 of the menstrual cycle for 5 years
Placebo Comparator: 2 mg estradiol	Drug: estradiol tablets, 2 mg day 1 through day 22 of the menstrual cycle for 5 years

Arm

Intervention/Treatment

Active Comparator: 4 mg estradiol

Drug: estradiol

tablets, 2 mg day 1 through day 22 of the menstrual cycle for 5 years

Placebo Comparator: 2 mg estradiol

Drug: estradiol

tablets, 2 mg day 1 through day 22 of the menstrual cycle for 5 years

Outcome Measures

Primary Outcome Measures

The size of the uterus evaluated by magnetic resonance (MR)-scan [5 years]
Bones evaluated by dual-energy X-ray absorptiometry (DEXA)-scan [5 years]

Secondary Outcome Measures

The diameter of the aortic root evaluated by MR-scan [5 years]
Development of biochemical markers [5 years]
Body composition evaluated by DEXA-scan [5 years]
Emotional wellbeing and self-esteem evaluated by questionnaires [5 years]
The size of the uterus evaluated by ultrasound [5 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

10 Years to 25 Years

Sexes Eligible for Study:

Female

Accepts Healthy Volunteers:

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