Status of Growth Hormone/ Insulin-like Growth Factor-1 (GH/IGF-1) Axis and Growth Failure in Ataxia Telangiectasia (AT)

• Status: Unknown status
• Trial end date: 2012-09-01
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the status of the growth hormone/ insulin-like growth factor-1 (GH/IGF-1) axis in relation to growth failure, body weight and composition and neuroprotection in children with Ataxia telangiectasia (AT).

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Johann Wolfgang Goethe University Hospital
• Treatments: Clonidine; Estradiol; Estradiol 17 beta-cypionate; Estradiol 3-benzoate; Estradiol valerate; Hormones; Mitogens; Polyestradiol phosphate

Criteria

Inclusion Criteria:

• Have a diagnosis of AT

• Have no fusion of epiphyses/closed growth plates as determined by X-ray of left wrist and hand (special skeletal age film)

• Be between 3 years to 18 years old and have not completed puberty

• Consent to permit blood and/or tissue samples for storage

• Demonstrate growth failure: height below the 10th percentile for chronological age

• Have a primary care physician at home

• Demonstrate growth failure, defined as growth velocity (measured as linear growth) that is less than 5% to 10% of that expected for children of the same age group, over the past 12 months

• Willingness to remain hospitalized for several days

• Provide evidence of serum IGF-1 level performed within the preceding 6 months and the results fall below 25% range of normal limits for age

Exclusion Criteria:

• Have fusion of epiphyseal plates

• Be under the age of 3 years or have reached completion of puberty

• Have a serum IGF-1 level that is above the 25% range of normal limits for age

• Be above the 10th percentile height for chronological age

• Demonstrate any history of anaphylactic reaction or hypersensitivity to one of the GH formulation

• Have any active or suspected neoplasia

• Demonstrate signs of intracranial hypertension as evidenced by papilledema upon examination by fundoscopy

• Have any condition that, in the investigator's opinion, places the patient at undue risk by participating in the study

• Be unwilling to undergo testing or procedures associated with this protocol

• Have acute or chronic infections

• Have a hypersensitivity to one of the drugs: Clonidine hydrochlorid, Arginine hydrochlorid, Estradiol valerate, Somatropin

• Have a presence of bradycardia, cardiac arrhythmia, have symptoms of a sick sinus syndrome

• Suffer from depression

• Have acute or recurrent thrombosis

• Have acute liver diseases