Clinical trial NCT00191113: Somatropin Treatment to Final Height in Turner Syndrome
Official title: Humatrope Treatment to Final Height in Turner's Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Turner Syndrome
Keywords: syndrome
Turner
Turner's
height
growth
growth hormone
somatropin
short stature
short
hearing
glucose metabolism
Lead sponsor: Eli Lilly and Company
Sponsor class: INDUSTRY
First posted: 2005-09-19
Enrollment: 154
Sex: FEMALE
Minimum age (years): 7
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Detailed description: A randomized, controlled trial of Humatrope (somatropin) treatment in girls with Turner syndrome at least 7 years old and younger than 13 at study entry.

Core study objectives are to determine the efficacy of Humatrope in promoting linear growth to final height in girls with Turner syndrome, and to assess the safety of this treatment. Core study completion criteria (protocol final height) are that the patient has both a height velocity \< 2 cm per year and a bone age of 14 years or greater.

Addendum 1 provides the option of Humatrope treatment to patients who were randomized to the Control arm of the Core study and who discontinued from the study on or after December 19, 1997.

Addendum 2 objectives are: 1) to collect true final height data; 2) to evaluate hearing, tympanic membrane function and other specific areas of interest with respect to the safety of growth hormone therapy in Turner syndrome; 3) to evaluate pancreatic beta cell function (glucose metabolism) in patients previously enrolled in the Core study.

Addendum 3 objective is to determine the parental origin of the retained X chromosome of an appropriate subset of patients currently or previously enrolled in the Core study, and to determine whether this parental origin holds any predictive value for spontaneous growth or for response to growth hormone therapy.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

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

Primary outcomes:
measure: Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Change From Baseline to Last Measurement, As Randomized Population
timeFrame: Baseline, and end of 4-year addendum
measure: Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Last Measurement After Attainment of Final Height
timeFrame: at completion of core study, or at end of 4-year addendum
Secondary outcomes:
measure: Height Standard Deviation Score (SDS) (National Center for Health Statistics [NCHS]), Change From Baseline, As-Treated Population
timeFrame: every 3 months during core study, and at start and end of 4-year addendum
measure: Height (Centimeters [cm])
timeFrame: every 3 months during core study, and at start and end of 4-year addendum
measure: Number of Participants With an Abnormal Pure Tone Audiometry, Audiologist Assessment
timeFrame: at completion of core study or beginning of addendum
measure: Number of Participants With Abnormal Speech Audiometry, Audiologist Assessment
timeFrame: at completion of core study or beginning of addendum
measure: Number of Participants With Abnormal Impedance Tympanometry, Audiologist Assessment
timeFrame: at completion of core study or beginning of addendum
measure: Number of Participants With Hearing Loss, Audiologist Assessment
timeFrame: at completion of core study or beginning of addendum
measure: Fasting Glucose, Change From Baseline
timeFrame: At core study baseline, and at end of 4-year addendum
measure: Maximum Fasting Glucose Value
timeFrame: At start and through end of 4-year addendum (up to an additional 2 years)
measure: Number of Participants With Any Abnormal Fasting Glucose Value
timeFrame: At start and through end of 4-year addendum
measure: Maximum Fasting Insulin Values
timeFrame: At start and through end of 4-year addendum (up to an additional 2 years)
measure: Number of Participants With Any Abnormal Fasting Insulin Value
timeFrame: At start and through end of 4-year addendum
measure: Minimum Fasting Glucose/Insulin Ratio Values
timeFrame: At start and through end of 4-year addendum (up to an additional 2 years)
measure: Number of Participants With Any Abnormal Fasting Glucose/Insulin Ratio Value
timeFrame: At start and through end of 4-year addendum
measure: Glycosylated Hemoglobin, Change From Baseline
timeFrame: At core study baseline, and at end of 4-year addendum
measure: Maximum Glycosylated Hemoglobin
timeFrame: At start and through end of 4-year addendum (up to an additional 2 years)
measure: Number of Participants With Any Abnormal Glycosylated Hemoglobin (HbA1c) Value
timeFrame: At start and through end of 4-year addendum
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: 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.
state: Alberta
city: Edmonton
country: Canada
facility: 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.
state: Alberta
city: Vancouver
country: Canada
facility: 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.
state: British Columbia
city: Winnipeg
country: Canada
facility: 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.
state: Manitoba
city: Halifax
country: Canada
facility: 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.
state: Nova Scotia
city: Hamilton
country: Canada
facility: 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.
state: Ontario
city: Kingston
country: Canada
facility: 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.
state: Ontario
city: London
country: Canada
facility: 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.
state: Ontario
city: Ottawa
country: Canada
facility: 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.
state: Ontario
city: Toronto
country: Canada
facility: 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.
state: Ontario
city: Montreal
country: Canada
facility: 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.
state: Quebec
city: Montreal
country: Canada
facility: 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.
state: Quebec
city: Sainte-Foy
country: Canada
facility: 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.
state: Quebec
city: Sherbrooke
country: Canada
facility: 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.
state: Quebec
Study officials:
affiliation: Eli Lilly and Company
name: 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)
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.