Defining the Optimal Hormonal Replacement Therapy in Turner Syndrome

This study has been completed.
Sponsor:
Collaborators:
Novo Nordisk A/S
The County of Frederiksborg
The foundation of Kaptajnløjtnant Harald Jensen and Wife
The foundation of Mrs. Olga Bryde
The foundation of Mr. Ivan Nielsen
Information provided by (Responsible Party):
Line Cleemann, Hillerod Hospital, Denmark
ClinicalTrials.gov Identifier:
NCT00134745
First received: August 24, 2005
Last updated: June 13, 2013
Last verified: June 2013
  Purpose

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 Intervention Phase
Turner Syndrome
Drug: estradiol
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Pharmacodynamics Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Hillerod Hospital, Denmark:

Primary Outcome Measures:
  • The size of the uterus evaluated by magnetic resonance (MR)-scan [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
  • Bones evaluated by dual-energy X-ray absorptiometry (DEXA)-scan [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • The diameter of the aortic root evaluated by MR-scan [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
  • Development of biochemical markers [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
  • Body composition evaluated by DEXA-scan [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
  • Emotional wellbeing and self-esteem evaluated by questionnaires [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
  • The size of the uterus evaluated by ultrasound [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]

Enrollment: 43
Study Start Date: June 2005
Study Completion Date: March 2013
Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   10 Years to 25 Years
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00134745

Locations
Denmark
Pediatric Unit, Hillerod Hospital
Hillerod, Frederiksborg County, Denmark, 4300
Sponsors and Collaborators
Line Cleemann
Novo Nordisk A/S
The County of Frederiksborg
The foundation of Kaptajnløjtnant Harald Jensen and Wife
The foundation of Mrs. Olga Bryde
The foundation of Mr. Ivan Nielsen
Investigators
Principal Investigator: Line Cleemann, Doctor Pediatric Unit, Hillerod Hospital
  More Information

No publications provided

Responsible Party: Line Cleemann, Doctor, PhD, Hillerod Hospital, Denmark
ClinicalTrials.gov Identifier: NCT00134745     History of Changes
Other Study ID Numbers: 120895
Study First Received: August 24, 2005
Last Updated: June 13, 2013
Health Authority: Denmark: The Danish National Committee on Biomedical Research Ethics

Keywords provided by Hillerod Hospital, Denmark:
Turner Syndrome
Hormonal replacement therapy
Bone mineralization
Uterine size
aortic root dilatation

Additional relevant MeSH terms:
Gonadal Dysgenesis
Primary Ovarian Insufficiency
Turner Syndrome
Syndrome
Adnexal Diseases
Cardiovascular Abnormalities
Cardiovascular Diseases
Chromosome Disorders
Congenital Abnormalities
Disease
Disorders of Sex Development
Endocrine System Diseases
Genetic Diseases, Inborn
Genital Diseases, Female
Gonadal Disorders
Heart Defects, Congenital
Heart Diseases
Ovarian Diseases
Pathologic Processes
Sex Chromosome Disorders
Sex Chromosome Disorders of Sex Development
Urogenital Abnormalities
Estradiol
Estradiol 17 beta-cypionate
Estradiol 3-benzoate
Estradiol valerate
Polyestradiol phosphate
Contraceptive Agents
Contraceptive Agents, Female
Estrogens

ClinicalTrials.gov processed this record on October 21, 2014