Somatropin Treatment to Final Height in Turner Syndrome

NCT00191113 | Completed Phase 3 Results DMC

• 3 other identifiers: 817/4419#817 B9R-CA-GDCT Core study#4419 GDCT/1 Addenda
• Study Type: interventional
• Target: 154
• Locations: 1 country
• Sites: 14

Brief Summary

A randomized, controlled trial in girls with Turner syndrome at least 7 years old and younger than 13 at study entry, to determine the efficacy and safety of Humatrope (somatropin) treatment in promoting linear growth to final height.

Conditions

Turner Syndrome

Keywords

syndrome; Turner; Turner's; height; growth; growth hormone; somatropin; short stature; short; hearing; glucose metabolism

Outcome Measures

Primary Outcomes (2)

• Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Change From Baseline to Last Measurement, As Randomized Population

  Value analyzed is change from baseline to the most mature height measurement available. The terms Standard Deviation Score (SDS) and National Center for Health Statistics (NCHS) were defined in baseline characteristics. Greater height SDS values indicate greater height; positive values of change from baseline indicate increased height.

  Baseline, and end of 4-year addendum

• Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Last Measurement After Attainment of Final Height

  SDS report the number of standard deviations from the mean for age and sex for an individual measurement (normal range: -2 to +2 SDS). Height SDS \[NCHS\] uses the NCHS US general female population reference height values for age (Kuczmarski RJ et al. 2000) as the population mean and standard deviation. Calculation of Height SDS is provided in Height SDS \[Lyon\] description (Baseline). Since data reported by Kuczmarski RJ et al provides US general female population standards, values of Height SDS \[NCHS\] for untreated patients with Turner syndrome tend to be below zero e.g, -2.0 to -4.0 SDS.

  at completion of core study, or at end of 4-year addendum

Secondary Outcomes (16)

• Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Change From Baseline, As-Treated Population

  every 3 months during core study, and at start and end of 4-year addendum

• Height (Centimeters [cm])

  every 3 months during core study, and at start and end of 4-year addendum

• Number of Participants With an Abnormal Pure Tone Audiometry, Audiologist Assessment

  at completion of core study or beginning of addendum

• Number of Participants With Abnormal Speech Audiometry, Audiologist Assessment

  at completion of core study or beginning of addendum

• Number of Participants With Abnormal Impedance Tympanometry, Audiologist Assessment

  at completion of core study or beginning of addendum

Study Arms (2)

Control

NO INTERVENTION

Control arm; untreated with Humatrope. Ethinyl estradiol (escalating doses to 20 mcg daily) after age 13, and medroxyprogesterone acetate (10 mg tablets ten days monthly) after age 15. Subject continues until Core study completion criteria are met (protocol final height).

Drug: Ethinyl estradiol; Drug: Medroxyprogesterone acetate

Humatrope

EXPERIMENTAL

Humatrope (0.05 mg/kg/dose) by subcutaneous injection 6 times per week. Ethinyl estradiol (escalating doses to 20 mcg daily) after age 13, and medroxyprogesterone acetate (10 mg tablets ten days monthly) after age 15. Subject continues until Core study completion criteria are met (protocol final height).

Drug: Somatropin; Drug: Ethinyl estradiol; Drug: Medroxyprogesterone acetate

Interventions

Somatropin DRUG

0.05 mg/kg/dose by subcutaneous injection 6 times per week, until Core study completion criteria are met (protocol final height).

Also known as: Humatrope, Growth hormone

Humatrope

Ethinyl estradiol DRUG

escalating doses 2.5-20.0 mcg tablets daily after age 13 and at least one year on study, continuing until Core study completion criteria are met.

Control; Humatrope

Medroxyprogesterone acetate DRUG

10 mg tablets, ten days monthly, after age 15, continuing until Core study completion criteria are met.

Control; Humatrope

Eligibility Criteria

• Age: 7 Years - 13 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• girl with Turner syndrome
• prepubertal, Tanner stage I breast
• height velocity less than 6 cm/year and height less than or equal to the tenth percentile for sex and age in general population
• at least 6 months (preferably 12 months) of accurate height measurements available for calculation of pre-study height velocity
• if thyroxine deficient, to have received replacement therapy, and for six months prior to enrollment have had normal thyroid function tests

You may not qualify if:

• prior treatment with growth hormone
• presence of a Y component in karyotype with gonads in situ
• diabetes mellitus

Sponsors & Collaborators

• Eli Lilly and Company: lead

Study Sites (14)

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Calgary, Alberta, T2T 5C7, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Edmonton, Alberta, T6G 2B7, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Vancouver, British Columbia, V6H 3V4, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Winnipeg, Manitoba, R3E 0Z2, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Halifax, Nova Scotia, B3J 3G9, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Hamilton, Ontario, L8S 3Z5, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Kingston, Ontario, K7L 3N6, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

London, Ontario, K7L 3N6, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Ottawa, Ontario, K1H 8L1, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Toronto, Ontario, M5G 1X8, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Montreal, Quebec, H3H 1P3, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Montreal, Quebec, H3T 1C5, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Sainte-Foy, Quebec, G1V 4G2, Canada

Location

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Sherbrooke, Quebec, J1G 2E8, Canada

Location

Related Publications (5)

• Lyon AJ, Preece MA, Grant DB. Growth curve for girls with Turner syndrome. Arch Dis Child. 1985 Oct;60(10):932-5. doi: 10.1136/adc.60.10.932.

  PMID: 4062345 BACKGROUND

• Kuczmarski RJ, Ogden CL, Grummer-Strawn LM, Flegal KM, Guo SS, Wei R, Mei Z, Curtin LR, Roche AF, Johnson CL. CDC growth charts: United States. Adv Data. 2000 Jun 8;(314):1-27.

  PMID: 11183293 BACKGROUND

• Hamelin CE, Anglin G, Quigley CA, Deal CL. Genomic imprinting in Turner syndrome: effects on response to growth hormone and on risk of sensorineural hearing loss. J Clin Endocrinol Metab. 2006 Aug;91(8):3002-10. doi: 10.1210/jc.2006-0490. Epub 2006 Jun 6.

  PMID: 16757526 RESULT

• Stephure DK; Canadian Growth Hormone Advisory Committee. Impact of growth hormone supplementation on adult height in turner syndrome: results of the Canadian randomized controlled trial. J Clin Endocrinol Metab. 2005 Jun;90(6):3360-6. doi: 10.1210/jc.2004-2187. Epub 2005 Mar 22.

  PMID: 15784709 RESULT

• Taback SP, Van Vliet G. Health-related quality of life of young adults with Turner syndrome following a long-term randomized controlled trial of recombinant human growth hormone. BMC Pediatr. 2011 May 29;11:49. doi: 10.1186/1471-2431-11-49.

  PMID: 21619701 DERIVED

Related Links

• Lilly Clinical Trial Registry

MeSH Terms

Conditions

Turner Syndrome; Syndrome; Dwarfism

Interventions

Human Growth Hormone; Growth Hormone; Ethinyl Estradiol; Medroxyprogesterone Acetate

Condition Hierarchy (Ancestors)

Gonadal Dysgenesis; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Sex Chromosome Disorders of Sex Development; Male Urogenital Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Sex Chromosome Disorders; Chromosome Disorders; Genetic Diseases, Inborn; Gonadal Disorders; Endocrine System Diseases; Disease; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins; Norpregnatrienes; Norpregnanes; Norsteroids; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Estrogenic Steroids, Alkylated; Estradiol Congeners; Gonadal Steroid Hormones; Gonadal Hormones; Medroxyprogesterone; Hydroxyprogesterones; Progesterone; Pregnenediones; Pregnenes; Pregnanes

Results Point of Contact

• Title: Chief Medical Officer
• Organization: Eli Lilly and Company

1-800-545-5979

Study Officials

• Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 am - 5 pm Eastern Time (UTC/GMT - 5 hours, EST)

  Eli Lilly and Company

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: September 12, 2005
• First Posted: September 19, 2005
• Study Start: February 1, 1989
• Primary Completion: December 1, 2007
• Study Completion: December 1, 2007
• Last Updated: January 27, 2010
• Results First Posted: January 27, 2010

Record last verified: 2009-12

Locations

CA (14)