Status of the growth hormone/ insulin-like growth factor-1 (GH/IGF-1) axis in relation to growth failure, body weight and neuroprotection in children with Ataxia telangiectasia (AT) - Growth hormone in AT (GHAT)

• Conditions: 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)

• Registration Number: EUCTR2009-015739-34-DE
• Lead Sponsor: Goethe-Universität Frankfurt/Main

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-09-14
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• diagnosis of AT
• no fusion of epiphyses/closed growth plates determined by X-ray of left wrist and hand (special skeletal age film)
• age between 3 years to 18 years and no completed puberty
• growth failure: height below the 10th percentile for chronological age
• growth failure, defined as growth velocity (measured as linear growth) less than 5% to 10% of that expected for children of the same age group, over the past 12 months
•provide evidence of serum IGF-1 level performed within the preceding 6 months and the results fall below normal limits for age

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• fusion of epiphyseal plates
• age under 3 years or completion of puberty
• serum IGF-1 level that is within normal limits for age
• be above the 10th percentile height for chronological age
• history of anaphylactic reaction or hypersensitivity to one of the GH formulation
• any active or suspected neoplasia
• signs of intracranial hypertension as evidenced by papilledema

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Primary objective: Primary efficacy endpoint: The primary objective is to evaluate the IGF1/GH increase after Arginine provocation test ;Secondary Objective: Secondary objective(s): <br>The GH increase after Clonidine Provocation Test<br>To evaluate the safety and efficacy of the IgF-1 generation test.<br>To correlate GH/IGF-1 deficiency to BMI<br>To correlate GH/IGF-1 deficiency to MRI findings<br>;Primary end point(s): Primary efficacy endpoint: The primary objective is to evaluate the IGF1/GH increase after Arginine provocation test