Offered by: Nemours
Location: Delaware Valley
Evaluate Efficacy, Safety and Pharmacokinetics of Denosumab in Children With Osteogenesis Imperfecta (OI)
What is the trial about?
The purpose of this study is to find if denosumab is effective and safe in children with osteogenesis imperfecta. Denosumab is still experimental and has been approved by the FDA and EMS for people with osteoporosis at high risk of fracture.
Who can participate?
Male or female children ages 2-17yrs old, who have osteogenesis imperfecta are being asked to participate in the study.
What is involved?
Participation in the study will last 36 months or 3 years. Each clinic visit should last approximately 2 hours. If your child qualifies, your child will receive an injection of study medication once every 6 months. In addition, your child will have to take daily supplements of calcium and vitamin D which will be supplied by your child’s study doctor.
- Screening - 1 time
- Day 1 - 1 time
- Day 10-30 - 2 times
- Month 3 - 1 time
- Months 6 - 36 - every 6 months
What types of tests or procedures will be involved with this study?
If you decide to allow your child to participate in the study, some tests including HIV and Hepatitis B and C testing, will be done to see if your child is eligible for this study. If the test results show that your child meets the study requirements, then your child will be able to start the study. Some tests may be repeated to see if your child meets the study requirements the second time. If the test results show that your child does not meet the study requirements, your child will not be able to start the study. The study doctor will discuss other options with you and/or refer your child back to their regular doctor.
As part of your participation in this study, you will have tests or procedures at each visit as shown in the table below:
|Test/Procedure||Prestudy||During Study - Day 1||During Study - Day 10-Day 30||During Study - Months 3-30||End of Study - Month 36|
|Medical and Medication History||1 time||1 time||2 times||6 times||1 time|
|Physical Examination||1 time||1 time||6 times||1 time|
|Height||1 time||2 times||1 time|
|Weight||1 time||5 times||1 time|
|Vital Signs||1 time||1 time||2 times||6 times||1 time|
|Armspan||1 time||2 times||1 time|
|Blood Test||1 time||1 time||2 times||6 times||1 time|
|Pregnancy Test (in girls of childbearing potential)||1 time||1 time||5 times|
|DXA of Spine||1 time||4 times||1 time|
|DXA of Hip||1 time||4 times||1 time|
|X-ray of Spine||1 time||2 times||1 time|
|X-ray of Knees||1 time||5 times||1 time|
|Dental X-ray||1 time||2 times||1 time|
|Visual Inspection of Mouth||2 times||1 time|
|Administration of Questionnaires||1 time||2 times||1 time|
|Administration of Questionnaire to Parent||1 time||2 times||1 time|
|Dispensation of Calcium and Vitamin D||1 time||5 times|
|Administration of Study Drug||1 time||5 times|
A baseline EKG (electrocardiogram) will be done – This test is a painless way of recording your child’s heart function. The test takes about 15 minutes to complete.
DXA scan of spin and hip:
DXA scans will be used to measure the density (amount of mineral such as calcium) of your spine and hip bones. This will be done with a DXA scanner. This procedure involves lying on a table while the scan is being taken. The DXA scan is painless and will take 10 to 30 minutes.
X-rays of spine:
You child will have x-rays taken of their spine. Your child will be asked to lie down on an x-ray table or stand. The radiology technologist will position your child to obtain different x-ray views. Your child will be asked to hold his/her breath briefly when the x-rays are being taken. The x-rays are used to identify any fractures, dislocations, thinning of the bone or deformities in the curvature of the spine. There is low radiation exposure for the x-ray series. The amount of radiation received is approximately half of the annual naturally occurring background radiation from natural sources (sun, soil, food and water) to which a person is exposed.
X-ray of the knees:
Your child will have x-rays taken of their knees. Your child will be asked to lie down on a x-ray table and the radiology technician will position your child to obtain X-ray views. The x-rays are used to determine how the bones in the legs are growing. The amount of radiation is very low compared to annual naturally occurring background radiation.
X-rays of the face:
Your child will have x-rays taken of their face. Your child will be asked to stand or sit. The radiology technician will position your child to obtain different x-ray views. These x-rays will show features of the skull, jaws and teeth. Additional X-rays also may be obtained to see how the molar teeth are erupting. The amount of radiation is very low compared to annual naturally occurring background radiation.
All of the x-rays in this study were reviewed and approved by the radiation safety committee. The radiation safety committee deemed that the radiation exposure that your son or daughter will be exposed to by having the x-rays in this study is very low compared to the annual naturally occurring background radiation.
Visual inspection of the mouth
Your child will have his/her mouth inspected to evaluate presence of molar teeth.
At any study visit, about 0.7 teaspoons/0.2 tablespoons (3.4 mL) to 1.5 teaspoons/0.5 tablespoons (7.5 mL) of blood may be collected from your child. If your child completes the entire study, the total amount of blood collected from your child will be approximately 12 teaspoons/4 tablespoons (58 mL) over 3 years.
Contact Nemours Clinical Trials
Denosumab in Children With Osteogenesis Imperfecta