Status:
ACTIVE_NOT_RECRUITING
Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia
Lead Sponsor:
National Institute of Dental and Craniofacial Research (NIDCR)
Conditions:
Fibrous Dysplasia
Eligibility:
All Genders
4-14 years
Phase:
PHASE2
Brief 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 deve...
Detailed Description
Study Description: This will be a phase 2, open label, single arm study of denosumab treatment to prevent fibrous dysplasia (FD) lesion progression in children. Objectives: Primary Objective: Eval...
Eligibility Criteria
Inclusion
- 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 potential: use of condoms or other methods to ensure effective contraception with partner
- Minimum body weight of 12 kilograms
- EXCLUSION CRITERIA:
- An individual who meets any of the following criteria will be excluded from participation in this study:
- Pregnancy or lactation
- Known allergic reactions to denosumab
- Prior history, or current evidence, of osteomyelitis/osteonecrosis of the jaw
- Planned invasive dental procedure for the course of the study
- Presence of non-healed dental or oral surgery
- Orthopedic procedure performed less than 6-weeks prior to first day of the denosumab administration (Day 0)
- Acute fracture less than 6-weeks prior to first day of the denosumab administration (Day 0)
- Serum calcium or albumin-adjusted serum calcium below the normal range for the NIH laboratory (patients will be eligible for re-screening after a repletion period lasting up to 6 months)
- 25-hydroxyvitamin D level than 20 ng/mL (patients will be eligible for re screening after a repletion period lasting up to 6 months)
- Untreated or inadequately treated hypophosphatemia as determined by the principal investigator (patients will be eligible for re-screening after initiation or optimization of phosphorus replacement no longer than 6 months)
- Inability to comply with a non-sedated 18F-NaF PET/CT (subjects will be eligible for re- screening after 6 months)
- Use of another investigational agent within the last 3 months prior to the first day of the denosumab administration (Day 0)
- Have any condition which in the opinion of the PI could present a concern for subject safety or difficulty with data interpretation.
Exclusion
Key Trial Info
Start Date :
October 12 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 9 2026
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT05419050
Start Date
October 12 2022
End Date
January 9 2026
Last Update
February 25 2025
Active Locations (1)
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1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892