Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan Syndrome

• Status: Recruiting
• Trial end date: 2022-10-01
• Target enrollment: 0
• Participant gender: All

Summary

This study evaluate the efficacy of 3-hydroxy-3-methylglutaryl coenzyme A (HMG-COA) reductase inhibitors, also known as "statins" in the treatment of growth and skeletal abnormalities in children with Noonan syndrome. Half of patients will receive simvastatin while the other half will receive a placebo.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University Hospital, Toulouse
• Treatments: Hydroxymethylglutaryl-CoA Reductase Inhibitors; Simvastatin

Criteria

Inclusion Criteria:

• Genetically confirmed Noonan syndrome

• Female child between 6 to 15 years, without menses, with bone age < 13 years

• Male child between 6 to 16 years, with bone age < 14 years

• Decreased growth velocity (< -1 SDS) and/or short stature (height < -2 SDS or -1,5 SDS under target height)

• Informed consent obtained from child and parents

Exclusion Criteria:

• Contraindication to simvastatin treatment :

• Progressive liver disease, increased serum levels of alanine aminotransferase (ALT) (> 1,5 uper limit of normal (ULN)), aspartate aminotransferase (> 1,5 ULN)

• Known hypersensitivity to simvastatin

• Pregnancy

• Treatment with CYP3A4 inhibitors (erythromycin, clarithromycin, ketoconazole, or itraconazole)

• Growth promoting therapies such as recombinant human Growth Hormone (GH) or IGF-1 treatment