Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia
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ClinicalTrials.gov Identifier: NCT05509595 |
Recruitment Status :
Active, not recruiting
First Posted : August 22, 2022
Last Update Posted : March 19, 2024
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Tracking Information | |||||
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First Submitted Date ICMJE | August 19, 2022 | ||||
First Posted Date ICMJE | August 22, 2022 | ||||
Last Update Posted Date | March 19, 2024 | ||||
Actual Study Start Date ICMJE | December 7, 2022 | ||||
Estimated Primary Completion Date | September 30, 2030 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
The proportion of subjects achieving serum phosphate levels within the target range (Z-score -1 to +2) at Week 48 [ Time Frame: 48 weeks ] Serum phosphate is the primary driver of skeletal complications in patients with FGF23-mediated hypophosphatemia and has been correlated with poor clinical outcomes in patients with FD.
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Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Same as current | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia | ||||
Official Title ICMJE | A Phase 2 Study of Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia | ||||
Brief Summary | 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. |
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Detailed Description | Study Description: This will be a phase 2, open-label, single-arm study to evaluate the safety and efficacy of burosumab to normalize serum phosphate levels in subjects with fibrous dysplasia (FD) and fibroblast growth factor 23 (FGF23)-mediated hypophosphatemia. Objectives: Primary Objective: -Evaluate the efficacy of burosumab to normalize serum phosphate levels in subjects with FD and FGF23-mediated hypophosphatemia at 48 weeks. Secondary Objectives:
Endpoints: Primary Endpoint: -The proportion of subjects achieving serum phosphate levels within the target range (Z-score -1 to +2) at Week 48. Secondary Endpoints:
Change from baseline to 48 weeks in patient reported outcomes measures:
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 2 | ||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Fibrous Dysplasia Of Bone | ||||
Intervention ICMJE | Drug: Burosumab
Human recombinant monoclonal antibody to fibroblast growth factor-23 (FGF23)
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Study Arms ICMJE | Experimental: Treatment
Patients receiving treatment
Intervention: Drug: Burosumab
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Estimated Enrollment ICMJE |
15 | ||||
Original Estimated Enrollment ICMJE | Same as current | ||||
Estimated Study Completion Date ICMJE | September 30, 2030 | ||||
Estimated Primary Completion Date | September 30, 2030 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE |
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
EXCLUSION CRITERIA: An individual who meets any of the following criteria will be excluded from participation in this study:
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Sex/Gender ICMJE |
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Ages ICMJE | 1 Year to 99 Years (Child, Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT05509595 | ||||
Other Study ID Numbers ICMJE | 10000798 000798-D |
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Has Data Monitoring Committee | Not Provided | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | National Institutes of Health Clinical Center (CC) ( National Institute of Dental and Craniofacial Research (NIDCR) ) | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | National Institute of Dental and Craniofacial Research (NIDCR) | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | National Institutes of Health Clinical Center (CC) | ||||
Verification Date | March 15, 2024 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |