Somatropin Treatment to Final Height in Turner Syndrome (GDCT)

Lilly

Status and phase: Completed; Phase 3

Conditions: Turner Syndrome

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT00191113

#4419 GDCT/1 Addenda

817/4419

#817 B9R-CA-GDCT Core study

Details and patient eligibility

About

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.

Full 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.

Enrollment: 154 patients

Sex: Female

Ages: 7 to 13 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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154 participants in 2 patient groups

Control

No Intervention group

Description:

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).

Treatment:

Drug: Ethinyl estradiol

Drug: Medroxyprogesterone acetate

Humatrope

Experimental group

Description:

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).

Treatment:

Drug: Ethinyl estradiol

Drug: Somatropin

Drug: Medroxyprogesterone acetate