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Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia

NCT05419050 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2026-01-20

No results posted yet for this study

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.

Conditions

  • Fibrous Dysplasia

Interventions

DRUG

denosumab

monoclonal antibody to receptor activator of nuclear kappa-B ligand (RANKL), a protein involved in regulating osteoclastogenesis

Sponsors & Collaborators

  • National Institute of Dental and Craniofacial Research (NIDCR)

    lead NIH

Principal Investigators

  • Alison M Boyce, M.D. · National Institute of Dental and Craniofacial Research (NIDCR)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
14 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-12
Primary Completion
2026-01-09
Completion
2026-01-09
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05419050 on ClinicalTrials.gov