Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia (NCT05419050) | Clinical Trial Compass
CompletedPhase 2
Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia
United States15 participantsStarted 2022-10-12
Plain-language summary
Background:
Fibrous dysplasia (FD) is a disease that affects the bones. It causes bone lesions that can become weak and lead to fractures, deformity, and nerve injuries. FD bone lesions begin to develop soon after birth and grow during childhood. The lesions stop growing in adults but can still cause disability. Researchers want to find ways to stop the growth of FD bone lesions.
Objective:
To test a study drug (denosumab) in children with FD.
Eligibility:
Children aged 4 to 14 years with FD and who are also enrolled in the Screening and Natural History protocol (98-D-0145).
Design:
Participants will have a screening visit at the NIH clinic or by telehealth. Their medical history will be reviewed.
Participants will stay overnight in the hospital 4 times in 76 weeks. Each stay will last 5 to 7 nights.
Participants will also visit a local lab for blood and urine tests every 4 weeks during the study.
Participants will receive denosumab once every 4 weeks for 48 weeks. The medication is given as a shot injected under the skin using a small needle. Some injections may be performed at home by a caregiver. The caregiver will receive training for this procedure.
Participants will undergo many tests that may be repeated throughout the study. They will have a dental exam. They will have tests of their strength and ability to move freely. They will have x-rays and other scans to get pictures of their bones.
Participants will be given another medicine that is administered through a needle in the arm over 30 minutes.
Who can participate
Age range
4 Years – 14 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
* INCLUSION CRITERIA:
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
* Confirmed diagnosis of fibrous dysplasia
* Age 4 to 14 years
* Concurrent enrollment in the companion Screening and Natural History protocol 98-D-0145
* Provision of signed and dated informed consent form
* Stated willingness of guardian/Legally Authorized Representative (LAR) to comply with all study procedures and availability for the duration of the study
* Ability of guardian/LAR to understand and the willingness to sign a written informed consent document
* For females of reproductive potential: agreement to use highly effective contraception for during study participation. Highly effective contraception methods include:
* Total abstinence. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
* Combination of the following (a+b or a+c, or b+c):
* Use of oral, injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate \<1%), for example hormone vaginal ring or transdermal hormone contraception
* Placement of an intrauterine device (IUD) or intrauterine system (IUS)
* Barrier methods of contraception: Condom or Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository
* For males of reproductive potenti…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Skeletal Burden Score
Timeframe: 48 weeks
Trial details
NCT IDNCT05419050
SponsorNational Institute of Dental and Craniofacial Research (NIDCR)