denosumab for Disease Progression

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
National Institutes of Health Clinical Center, Bethesda, MD
Disease Progression+2 More
denosumab - Drug
Eligibility
< 18
All Sexes
What conditions do you have?
Select

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.

Eligible Conditions

  • Disease Progression
  • Fibrous Dysplasia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Disease Progression

Study Objectives

1 Primary · 6 Secondary · Reporting Duration: 76 weeks

48 weeks
Change in 18F-NaF PET/CT sentinel lesion intensity (SUVmax)
Change in 18F-NaF PET/CT total lesion activity from baseline to 48 weeks
Change in Skeletal Burden Score
Change in functional parameters: - Muscle strength - Range-of-motion - Walking speed (6-minute walk)
Change in patient-reported outcome scales: - SF10 - Brief Pain Inventory - Brief Fatigue Inventory
Percent change in serum bone turnover markers from baseline to 48 weeks: procollagen 1 propeptide (P1NP, formation marker), beta crosslaps telopeptides (CTX, resorption marker), osteocalcin, and bone-specific alkaline phosphatase
76 weeks
Adverse events

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Disease Progression

Side Effects for

Standard Dose Teriparatide
5%wrist fracture
5%breast cancer
3%shoulder dislocation
3%pancreatitis
3%fibula avulsion fracture
3%vertebral fracture
3%clavicle fracture
3%kidney cancer
3%rib fracture
3%humeral fracture
3%throat cancer
0%anal cancer
0%back pain
This histogram enumerates side effects from a completed 2020 Phase 4 trial (NCT02176382) in the Standard Dose Teriparatide ARM group. Side effects include: wrist fracture with 5%, breast cancer with 5%, shoulder dislocation with 3%, pancreatitis with 3%, fibula avulsion fracture with 3%.

Trial Design

1 Treatment Group

treatment
1 of 1
Experimental Treatment

15 Total Participants · 1 Treatment Group

Primary Treatment: denosumab · No Placebo Group · Phase 2

treatment
Drug
Experimental Group · 1 Intervention: denosumab · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
denosumab
2014
Completed Phase 4
~4250

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 76 weeks
Closest Location: National Institutes of Health Clinical Center · Bethesda, MD
Photo of maryland 1Photo of maryland 2Photo of maryland 3
2016First Recorded Clinical Trial
3 TrialsResearching Disease Progression
276 CompletedClinical Trials

Who is running the clinical trial?

National Institute of Dental and Craniofacial Research (NIDCR)Lead Sponsor
275 Previous Clinical Trials
804,812 Total Patients Enrolled
1 Trials studying Disease Progression
157 Patients Enrolled for Disease Progression
Alison M Boyce, M.D.Principal InvestigatorNational Institute of Dental and Craniofacial Research (NIDCR)
3 Previous Clinical Trials
524 Total Patients Enrolled

Eligibility Criteria

Age < 18 · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have an IUD or IUS in place.
You have a confirmed diagnosis of fibrous dysplasia.
You are enrolled in the companion Screening and Natural History protocol 98-D-0145.
You are willing to comply with all study procedures and available for the duration of the study.
You are able to understand the information provided in the consent form and are willing to sign the form.
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.
You are using barrier methods of contraception.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.