Burosumab for Fibrous Dysplasia

Overseen By Olivia J de Jong, C.R.N.P.

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: National Institute of Dental and Craniofacial Research (NIDCR)

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

Background: Fibrous dysplasia (FD) is a disorder that affects bone growth. Affected bone tissue is weakened, and people with FD are prone to deformities, fractures, and other problems. People with FD may also have low blood phosphate levels. This can make bones even weaker. Better treatments are needed. Objective: To test a study drug (burosumab) in people with FD who have low blood phosphate levels. Eligibility: People aged 1 year or older who have FD and low blood phosphate levels. Design: Participants will visit the NIH 3 times in 48 weeks. Each visit will last 5 to 7 days. Participants will self-inject burosumab under the skin in their belly, upper arm, or thigh. They (or a caregiver) will do this at home 1 or 2 times a month. They will be trained in person on how to inject the drug. Home injections will be guided via telehealth. During NIH visits, participants will have a physical exam with blood and urine tests. They will have x-rays of different parts of their body. They will have a radioactive tracer injected into their vein; then they will have a bone scan. They will have tests to assess their strength, walking, and movement. They will complete questionnaires about their pain, mobility, and fatigue levels. Adult participants may have bone biopsies. These will be done under anesthesia with sedation. Small samples of FD-affected bone will be removed for study. Between NIH visits, participants will go to a local laboratory for blood and urine tests. Child participants will have an additional follow-up visit 2 weeks after the final NIH visit.

Research Team

Alison M Boyce, M.D.

Principal Investigator

National Institute of Dental and Craniofacial Research (NIDCR)

Eligibility Criteria

This trial is for people of any age over 1 year with fibrous dysplasia (FD) and low blood phosphate levels. Participants must be able to self-inject the study drug, burosumab, agree to use effective contraception if of reproductive potential, and have a minimum body weight of 7.5 kg. Those who are pregnant or lactating, have severe kidney issues, or have used burosumab or other investigational drugs recently cannot join.

Inclusion Criteria

I have been diagnosed with fibrous dysplasia.

I am a male sterilized at least 6 months ago, or my only partner is a vasectomized male.

-- Placement of an intrauterine device (IUD) or intrauterine system (IUS)

Exclusion Criteria

Have any condition which in the opinion of the PI could present a concern for subject safety or difficulty with data interpretation

I have severe kidney problems or am in the final stage of kidney disease.

Pregnancy or lactation

Treatment Details

Interventions

• Burosumab (Monoclonal Antibodies)

Trial Overview The trial tests burosumab in individuals with FD who suffer from weak bones due to low phosphate levels. Over 48 weeks, participants will visit NIH three times for extensive testing including physical exams and bone scans while self-administering injections at home regularly under telehealth guidance.

Participant Groups

Group I: Treatment Experimental Treatment 1 Intervention

Patients receiving treatment

Who Is Running the Clinical Trial?

National Institute of Dental and Craniofacial Research (NIDCR)

Lead Sponsor

Trials

312

Recruited

853,000+