Ignite Creation Date:
2025-12-24 @ 5:19 PM
Ignite Modification Date:
2026-02-25 @ 9:40 PM
Study NCT ID:
NCT05419050
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-02-25
First Post:
2022-06-11
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia
Sponsor:
National Institute of Dental and Craniofacial Research (NIDCR)
Organization:
Study Overview
Official Title:
A Phase 2 Study of Denosumab for Prevention of Skeletal Disease Progression in Children With Fibrous Dysplasia
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-02-21
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 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.
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.
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:
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.
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.
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 000780-D | None | None | View |