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

NCT00001221 | Completed Phase 2

• 2 other identifiers: 87015287-CH-0152
• Study Type: interventional
• Target: 160
• Locations: 1 country
• Sites: 1

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

Conditions

Gonadal Dysgenesis; Turner's Syndrome

Keywords

Growth; Gonadal Dysgenesis; Estrogen; Somatotropin; Turner's Syndrome; Growth Hormone

Interventions

Growth Hormone (Humatrope) DRUG

Eligibility Criteria

• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): lead

Study Sites (1)

National Institute of Child Health and Human Development (NICHD)

Bethesda, Maryland, 20892, United States

Location

Related Publications (5)

• 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.

  PMID: 3537249 BACKGROUND

• 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.

  PMID: 3537250 BACKGROUND

• 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.

  PMID: 448501 BACKGROUND

• 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.

  PMID: 24762109 DERIVED

• 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.

  PMID: 21449786 DERIVED

MeSH Terms

Conditions

Gonadal Dysgenesis; Turner Syndrome

Interventions

Growth Hormone; Human Growth Hormone

Condition Hierarchy (Ancestors)

Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Gonadal Disorders; Endocrine System Diseases; Sex Chromosome Disorders of Sex Development; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Sex Chromosome Disorders; Chromosome Disorders; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Design

• Study Type: interventional
• Phase: phase 2
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: November 3, 1999
• First Posted: November 4, 1999
• Study Start: September 1, 1987
• Study Completion: September 1, 2003
• Last Updated: March 4, 2008

Record last verified: 2003-09

Locations

US (1)