Overview

A Phase 2 Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia

Status:
Not yet recruiting

Trial end date:
2024-06-01

Target enrollment:

Participant gender:

Summary

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: Evaluate the effect of denosumab on FD lesion progression in children. Secondary Objectives: - Evaluate the effects of denosumab on FD lesion activity. - Evaluate the effect of denosumab on strength and mobility. - Evaluate the effect of denosumab on pain and quality of life. - Evaluate the safety and tolerability of denosumab in children with FD. Endpoints: Primary Endpoint: Change in Skeletal Burden Score from baseline to 48 weeks Secondary Endpoints: - Percent change in serum bone turnover markers from baseline to 48 weeks: Procollagen 1 Intact N-Terminal Propeptide (P1NP, formation marker), C- telopeptides (CTX, resorption marker), osteocalcin, and bone-specific alkaline phosphatase - Change in 18F-NaF PET/CT total lesion activity from baseline to 48 weeks - Change in 18F-NaF PET/CT sentinel lesion intensity (SUVmax) from baseline to 48 weeks - Change in functional parameters from baseline to 48 weeks, including muscle strength, range-of-motion, and walking speed - Change in patient-reported outcome scales evaluating pain and quality of life from baseline to 48 weeks, including PROMIS Pediatric measures of Pain Intensity, Pain Interference, Mobility, and Fatigue.

Phase:

Phase 2

Details

Lead Sponsor:

National Institute of Dental and Craniofacial Research (NIDCR)

Treatments:

Denosumab