Clinical Trials / Research Studies

Clinical Trials / Research Studies

What will happen in the study?

This study includes 3 study visits (lasting 6 to 10 hrs.) over 12 months. You, as the parent or guardian, will be asked to complete at least four check-in calls during the 12 month period as well.

Your child will be treated with a single dose regimen of daily growth hormone for a period of 12 months to look at the effects on growth response. The dose that will be used is 50 micrograms/kg/day. This is a dose that is already approved for children diagnosed with Turner and Noonan syndrome, which both result in short stature not caused by growth hormone deficiency. Your child’s weight will be used to calculate the volume of their daily dose.

These are the things that will happen at the study visits.

We will:

• Complete a routine physical exam, including to see if your child has started puberty
• Complete a detailed joint exam, including to check for curvature of the spine
• Complete a detailed musculoskeletal evaluation which looks at knee and back health and includes an assessment of how your child walks
• Collect vital signs, height and weight measurements
• Collect blood samples
• Take photographs
• Ask you questions about your child’s family and medical history that focus on growth and joint issues
• Ask you questions about your child’s quality of life and physical activity
• Take an x-ray of your child’s hand and knees
• Take a bone density scan (DXA) of your child
• Take MRI pictures of your child’s knees (to look for early signs of joint disease)

These are the things that will happen during the check-in calls.

We will:

• Discuss any issues with medication administration and compliance
• Record any adverse events not previously reported
• Confirm that new supplies/medication will be shipped to the participant (upon completion of the check-in call)
• Ask you about the blood sample which will need to be drawn at a local facility (i.e. lab, doctor’s office) to monitor IGF-I levels at the time of the 3 month check in call. This will be subject to your insurance company and the study is not able to reimburse the cost.

Parents or guardians interested in having their child participate will be given a consent form that thoroughly explains all of the details of the study. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.