Efficacy and Safety of a High Dosage Compared to the Label Dosage of Somatropin in Early Pubertal Stage Children With Growth Hormone Deficiency

Phase 3

Completed

• Conditions: Growth Hormone Deficiency
• Interventions: Drug: Somatropin

• Registration Number: NCT00191165
• Lead Sponsor: Eli Lilly and Company

Brief Summary

Multi-center, randomized, controlled, open-label, phase III study comparing the effects of two different dosages of somatropin treatment (in-label or doubled) after 12 and 24 months of treatment, on height velocity in early pubertal children with growth hormone deficiency (GHD). The study will be conducted in Italy. Approximately 26 subjects will participate in this study, distributed as 13 in the in-label dosage group (group A) and 13 in the doubled dosage group (group B).

Detailed Description

Not available

Study Timeline

• Study Start: 2004-03
• Study First Submitted: 2005-09-12
• Study First Submitted QC: 2005-09-12
• Study First Posted: 2005-09-19
• Primary Completion: 2008-04
• Study Completion: 2008-04
• Results First Submitted: 2009-04-07
• Results First Submitted QC: 2009-04-07
• Status Verified: 2009-06
• Results First Posted: 2009-06-08
• Last Update Submitted: 2009-06-08
• Last Update Posted: 2009-06-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 27

Inclusion Criteria

• Height velocity (cm/y) less than 25th percentile at the time of diagnosis.
• diagnosis of GHD
• Replacement therapy at label dosage (from 0.025 to 0.035 mg/kg/day) must be started at least 1 year before the enrolment in this study, but at most 3 years before enrolment
• Bone age less than 12 years for girls and less than 14 years for boys. This assessment should be made by the central reader of bone ages, based on the x-ray taken at Visit 0.
• Informed consent obtained from either both of the subject's parents or legal representative.

Exclusion Criteria

• Pubertal clinical stage less than 2 and greater than 3 according to Tanner score (for male subjects a volume of testicle greater than 15 mL).
• Any evidence of active malignancy. In case of previous surgical removal of both diencephalic and hypophysial masses, an MRI, performed within six months before enrollment into the study, must exclude the growth of residual tumoral tissue.
• Any known chronic disease such as diabetes mellitus, hepatic disease (defined by elevated liver enzymes 3-fold the upper limit of normal ranges), renal disease with creatinine levels greater than 130 micromol/L, or congestive heart failure.
• Glucocorticoid therapy in supra physiological doses (inhaled corticoids will be accepted if below 3 puffs/day, for no more than 10 days consecutively).
• Current or previous therapy with any therapy that may directly influence growth, including growth hormone releasing hormone, estrogens and anabolic steroids.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
2	Somatropin	In-label dosage
1	Somatropin	Doubled dosage

Primary Outcome Measures

Name	Time	Method
Height Velocity Standard Deviation Score (SDS) at 12-Month Endpoint	12-Months	Height velocity (difference between 2 height measurements, divided by years elapsed between measurements) SDS was derived by subtracting age and gender-matched population mean height velocity from patient's height velocity (based on measurements 12 months apart) then dividing this value by age and gender-matched population height velocity SD.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline to 12-Month and 24-Month Endpoints in Height Standard Deviation Score (SDS)	Baseline, 12-Months, 24-Months	This was derived by subtracting the age-and-gender-matched population 50th percentile height from the patient's height and then dividing this value by the age-and-gender-matched population height SD.
Height Velocity Standard Deviation Score (SDS) at 24 Month Endpoint	24 Months	Height velocity (difference between 2 height measurements, divided by years elapsed between measurements) SDS was derived by subtracting age and gender-matched population mean height velocity from patient's height velocity (based on measurements 12 months apart) then dividing this value by age and gender-matched population height velocity SD.

Trial Locations

Locations (1)

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Monday-Friday from 9:00 AM to 5:00 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician; 🇮🇹 Verona, Italy