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Gonadal DysgenesisPhase 2NCT00001221

Effect of Biosynthetic Growth Hormone and/or Ethinyl Estradiol on Adult Height in Patients With Turner Syndrome

A Phase 2 study testing Growth Hormone (Humatrope) in people with Gonadal Dysgenesis and Turner's Syndrome, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Turners Syndrome is a genetic condition in females that is a result of abnormal chromosomes. Girls with Turner syndrome are very short as children and as adults. Although their growth hormone secretion is almost always normal, giving injections of growth hormone to Turner syndrome girls may increase their rate of… Read more about this study

Who is it for?
Any age, women only
Conditions the study is for
Gonadal Dysgenesis, Turner's Syndrome
What is being tested?
Growth Hormone (Humatrope)
How many people
160 participants
Where
1 siteUnited States
Study dates
Sep 1987to Sep 2003
Phase
Phase 2
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 Any age
  • Women only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • All subjects will be outpatients.
  • Patients must be females with Turner's syndrome diagnosed by leukocyte karyotype
  • Chronologic age greater than or equal to 5 years.
  • Prepubertal Tanner I status. If breast development is Tanner II or III, demonstration of castrate status through measurement of serum FSH (greater than 12 MIU/ML).
  • All patients must be below the 10th percentile for chronologic age.
  • Patients must have at least 6 months accurate, growth measurements available for calculation of pre-study growth velocity. Pretreatment measurements must be obtained during a time when the patient is not receiving a potential growth-promoting agent.
  • Patients judged to be thyroxine deficient must have replacement which has resulted in normal thyroid function tests over the three-month period prior to enrollment (T4, T3, Thyroid Stimulating Hormone).
  • Patients and/or parents or legal guardians of patients must sign an informed consent statement. Assent should be obtained from all patients competent to understand the protocol. Local IRB requirements apply.

Who cannot join

  • Patients who have received any form of human growth hormone within the past 3 months, or who have received a cumulative course of therapy totaling greater than one year.
  • Patients who have received treatment with estrogen or androgen in the past three months or who have received a cumulative course of therapy totaling greater than one year.
  • Patients who have any Y component in their chromosome analysis.
  • Patients with a chronologic age greater than 12 years.
  • Patients with a bone age greater than 12 years.
  • Patients who have clinically significant cardiac, pulmonary, gastrointestinal, hepatic or renal disease or who have had any malignancy.
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  • Patients who have significant hematuria or proteinuria in pretherapy evaluation.
  • Patients who have diabetes mellitus.
  • Patients who have any active chronic infection (tuberculosis).
  • Patients who are taking amphetamines or any other drugs known to interfere with growth hormone secretion or actions.
  • Patients who are poor medical, psychological, or psychiatric risks for whom, in the opinion of the principal investigator, the investigational drug would be unwise.
  • Patients whose parents are substance abusers, nor those who come from homes in which appropriate emotional development may be limited.
  • Patients who cannot be seen on the schedule required by the protocol.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • National Institute of Child Health and Human Development (NICHD)

    Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: A Double-Blind, Randomized, Placebo-Controlled Trial of the Effect of Biosynthetic Growth Hormone and/or Ethinyl Estradiol on Adult Height in Patients With Turner Syndrome

Turners Syndrome is a genetic condition in females that is a result of abnormal chromosomes. Girls with Turner syndrome are very short as children and as adults. Although their growth hormone secretion is almost always normal, giving injections of growth hormone to Turner syndrome girls may increase their rate of growth. In addition, most girls with Turner syndrome do not have normal ovaries.

In normal girls the ovaries begin producing small amounts of the female sex hormone, estrogen at about 11 - 12 years of age. As girls grow older the level of estrogen increases. Estrogen is responsible for the changes in girls known as feminization. During feminization the hips grow wider, the breasts develop, there is an increase in the rate of growth, and eventually girls experience their first menstrual period.

Read more

This study was designed to evaluate the effect of low dose estrogen, growth hormone, and the combination of low dose estrogen and growth hormone on adult height in girls with Turner syndrome. Patients will be entered into the study from ages 5 to 12 and will be randomly placed into one of four groups.

  1. Group one will receive low dose estrogen
  2. Group two will receive growth hormone
  3. Group three will receive both low dose estrogen and growth hormone
  4. Group four will receive a placebo "sugar pill"

Once started, the treatment will continue until the patients approach their adult height, and growth slows to less than 1/2 inch over the preceding year. This usually occurs by the age of 15 or 16.

Patients will be seen at the outpatient clinic every 6 months during the study and will receive a routine check-up with blood and urine tests, and hand/wrist X-rays to determine bone age. On patient's yearly visits they will have the density of bone measured in their spine and forearm.

Read the detailed description

Adult women with Turner's syndrome are quite short. Several treatments have been used to increase growth rate in these patients. They include oxandrolone (1), growth hormone (2, 3), and low dose estrogen (4). However, the ability of these hormone treatments to increase adult height has never been evaluated in controlled clinical trials.

We propose to evaluate the effect on adult height of low dose estrogen, growth hormone, and the combination of low dose estrogen and growth hormone. Patients will be entered into the study from ages 5 to 12 and will be assigned randomly to one of the four groups (the 3 above treatment groups or placebo). The randomized assignment will be double-blind throughout the study. Treatment will be maintained to adult height (the first height measurement at which the preceding annual growth rate was < 1.5 cm). Beginning at age 12, the estrogen treatment in all 4 groups will be changed to a standardized regimen of increasing estrogen dose, so that all children in the study will undergo secondary sexual changes at an appropriate age.

In addition, the study will assess the effect of growth hormone and estrogen treatment on bone density and, in a parallel, separate, study, the effect of treatment on cognition and learning ability.

Being tested
Growth Hormone (Humatrope)
Keywords
Growth, Gonadal Dysgenesis, Estrogen, Somatotropin, Turner's Syndrome, Growth Hormone
Medical subject headings
Gonadal Dysgenesis, Turner Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Growth Hormone (Humatrope)Drug

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Nov 1999 (estimated)
  2. Study startSep 1987
  3. Study completionSep 2003

Resources

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 2
Primary purpose
Treatment
Enrolment
160 participants
IDs, dates and publications
ClinicalTrials.gov ID
NCT00001221
Sponsor's protocol ID
870152
Other IDs
87-CH-0152
Status last verified
Sep 2003
First submitted
3 Nov 1999
Last updated
4 Mar 2008
Results posted
No

Publications

  • Rosenfeld RG, Hintz RL, Johanson AJ, Brasel JA, Burstein S, Chernausek SD, Clabots T, Frane J, Gotlin RW, Kuntze J, et al. Methionyl human growth hormone and oxandrolone in Turner syndrome: preliminary results of a prospective randomized trial. J Pediatr. 1986 Dec;109(6):936-43. doi: 10.1016/s0022-3476(86)80272-4.

    PubMed 3537249

  • Raiti S, Moore WV, Van Vliet G, Kaplan SL. Growth-stimulating effects of human growth hormone therapy in patients with Turner syndrome. J Pediatr. 1986 Dec;109(6):944-9. doi: 10.1016/s0022-3476(86)80273-6.

    PubMed 3537250

  • Urban MD, Lee PA, Dorst JP, Plotnick LP, Migeon CJ. Oxandrolone therapy in patients with Turner syndrome. J Pediatr. 1979 May;94(5):823-7. doi: 10.1016/s0022-3476(79)80170-5.

    PubMed 448501

  • Quigley CA, Wan X, Garg S, Kowal K, Cutler GB Jr, Ross JL. Effects of low-dose estrogen replacement during childhood on pubertal development and gonadotropin concentrations in patients with Turner syndrome: results of a randomized, double-blind, placebo-controlled clinical trial. J Clin Endocrinol Metab. 2014 Sep;99(9):E1754-64. doi: 10.1210/jc.2013-4518. Epub 2014 Apr 24.

    PubMed 24762109

  • Ross JL, Quigley CA, Cao D, Feuillan P, Kowal K, Chipman JJ, Cutler GB Jr. Growth hormone plus childhood low-dose estrogen in Turner's syndrome. N Engl J Med. 2011 Mar 31;364(13):1230-42. doi: 10.1056/NEJMoa1005669.

    PubMed 21449786

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Growth Hormone (Humatrope) in people with Gonadal Dysgenesis and Turner's Syndrome, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.

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 8 inclusion and 13 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 4 Mar 2008. 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 United States. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00001221, which also lists 5 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 4 Mar 2008.

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