Turner SyndromePhase 4NCT00134745
Defining the Optimal Hormonal Replacement Therapy in Turner Syndrome
A Phase 4 study testing estradiol in people with Turner Syndrome, run by Line Cleemann at 1 site in Denmark.
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. Read more about this study
- Who is it for?
- Ages 10 to 25 years, women only
- Conditions the study is for
- Turner Syndrome
- What is being tested?
- estradiol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 43 participants
- Where
- 1 siteDenmark
- Study dates
- Jun 2005to Mar 2013
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 25 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Verified Turner syndrome
- Ages 10-25 years
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Denmark1 site
Pediatric Unit, Hillerod Hospital
Hillerød, Frederiksborg County, 4300
About this study
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
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.
Read the 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.
- Conditions
- Turner Syndrome
- Being tested
- estradiol
- Keywords
- Turner Syndrome, Hormonal replacement therapy, Bone mineralization, Uterine size, aortic root dilatation
- Medical subject headings
- Turner Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
estradiolDrug
tablets, 2 mg day 1 through day 22 of the menstrual cycle for 5 years
| Study group | Role | What they receive |
|---|---|---|
| 4 mg estradiol | Active comparator | Not described Receives: Drug: estradiol |
| 2 mg estradiol | Placebo comparator | Not described Receives: Drug: estradiol |
What is being measured
The primary outcome is the main question the study is designed to answer.
The size of the uterus evaluated by magnetic resonance (MR)-scan
Measured: 5 years
Bones evaluated by dual-energy X-ray absorptiometry (DEXA)-scan
Measured: 5 years
Secondary outcomes (5)
The diameter of the aortic root evaluated by MR-scan
Measured: 5 years
Development of biochemical markers
Measured: 5 years
Body composition evaluated by DEXA-scan
Measured: 5 years
Emotional wellbeing and self-esteem evaluated by questionnaires
Measured: 5 years
The size of the uterus evaluated by ultrasound
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Aug 2005 (estimated)
- Study startJun 2005
- Primary completionMar 2013
- Study completionMar 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Turner SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Line Cleemann
- Sponsor type
- University, hospital or other
- 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
- Responsible party
- Sponsor-investigator, Line Cleemann, Hillerod Hospital, Denmark
- Investigators
- Line Cleemann, Doctor, Principal investigator, Pediatric Unit, Hillerod Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00134745
- Sponsor's protocol ID
- 120895
- Status last verified
- Jun 2013
- First submitted
- 24 Aug 2005
- Last updated
- 14 Jun 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Cleemann L, Holm K, Fallentin E, Moller N, Kristensen B, Skouby SO, Leth-Esbensen P, Jeppesen EM, Jensen AK, Gravholt CH. Effect of Dosage of 17ss-Estradiol on Uterine Growth in Turner Syndrome-A Randomized Controlled Clinical Pilot Trial. J Clin Endocrinol Metab. 2020 Mar 1;105(3):dgz061. doi: 10.1210/clinem/dgz061.
PubMed 31613320Brun S, Cleemann L, Holm K, Salskov G, Erlandsen M, Berglund A, Andersen NH, Gravholt CH. Five-Year Randomized Study Demonstrates Blood Pressure Increases in Young Women With Turner Syndrome Regardless of Estradiol Dose. Hypertension. 2019 Jan;73(1):242-248. doi: 10.1161/HYPERTENSIONAHA.118.11742.
PubMed 30571546
Study at a glance
From the registry record, last updated by the sponsor on 14 Jun 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00134745
- Acronym / sponsor ID
- 120895
- Conditions
- Turner Syndrome
- Keywords
- Turner Syndrome, Hormonal replacement therapy, Bone mineralization, Uterine size, aortic root dilatation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- Line CleemannUniversity, hospital or other
- 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
- Study start
- Jun 2005
- Primary completion
- Mar 2013 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 25 Aug 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 4 mg estradiol | Active comparator | Drug: estradiol |
| 2 mg estradiol | Placebo comparator | Drug: estradiol |
Who may qualify
- Ages
- 10 to 25 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Verified Turner syndrome
- Ages 10-25 years
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| estradioltablets, 2 mg day 1 through day 22 of the menstrual cycle for 5 years | Drug | 2 mg estradiol, 4 mg estradiol |
Primary outcome
The size of the uterus evaluated by magnetic resonance (MR)-scan
Time frame: 5 years
Bones evaluated by dual-energy X-ray absorptiometry (DEXA)-scan
Time frame: 5 years
5 secondary outcomes
The diameter of the aortic root evaluated by MR-scan
Time frame: 5 years
Development of biochemical markers
Time frame: 5 years
Body composition evaluated by DEXA-scan
Time frame: 5 years
Emotional wellbeing and self-esteem evaluated by questionnaires
Time frame: 5 years
The size of the uterus evaluated by ultrasound
Time frame: 5 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jun 2013.
- Derived from this study: PubMed 31613320Cleemann L, Holm K, Fallentin E, Moller N, Kristensen B, Skouby SO, Leth-Esbensen P, Jeppesen EM, Jensen AK, Gravholt CH. Effect of Dosage of 17ss-Estradiol on Uterine Growth in Turner Syndrome-A Randomized Controlled Clinical Pilot Trial. J Clin Endocrinol Metab. 2020 Mar 1;105(3):dgz061. doi: 10.1210/clinem/dgz061.
- Derived from this study: PubMed 30571546Brun S, Cleemann L, Holm K, Salskov G, Erlandsen M, Berglund A, Andersen NH, Gravholt CH. Five-Year Randomized Study Demonstrates Blood Pressure Increases in Young Women With Turner Syndrome Regardless of Estradiol Dose. Hypertension. 2019 Jan;73(1):242-248. doi: 10.1161/HYPERTENSIONAHA.118.11742.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Line Cleemann, Doctor principal investigator
Pediatric Unit, Hillerod Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing estradiol in people with Turner Syndrome, run by Line Cleemann at 1 site in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Jun 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The size of the uterus evaluated by magnetic resonance (MR)-scan (time frame: 5 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00134745, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jun 2013.