Cinacalcet for Fibroblast Growth Factor 23 (FGF23)-Mediated Hypophosphatemia (Hypophosphatemic Rickets)
- Hypophosphatemia is a condition where a person has low levels of phosphorus in the blood. Low blood phosphorus can cause muscle and bone weakness (such as rickets) and teeth problems. One cause of the condition is having too much fibroblast growth factor 23 (FGF23). FGF23 is a hormone that causes the kidney to get rid of phosphorus in the urine. It can also prevent the body from making vitamin D, which helps the body absorb phosphorus in food.
- Many people with low blood phosphorus take high doses of phosphorus and calcium medications. However, one side effect of these drugs is increased blood levels of parathyroid hormone (PTH). The drug cinacalcet can help lower PTH levels, which may decrease the amount of phosphorus lost in the urine and increase the phosphorus levels in the blood. Researchers want to see if cinacalcet can help blood phosphorus and decrease the amount of phosphorus supplements that people need to take.
- To see if cinacalcet can be a safe and effective treatment for people with low phosphorus conditions due to high FGF23.
- Individuals between 18 and 70 years of age who have different forms of hypophosphatemic rickets and tumor-induced hypophosphatemia
- Participants will have up to 25 study visits over about 28 weeks.
- Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected.
- Up to three more lab visits for blood and urine tests will be required before treatment. Imaging studies of the bones, spine, and kidneys will be performed.
- Participants will have a 3-night hospital stay to start treatment. They will take cinacalcet once a day. Treatment will be monitored with frequent blood tests and imaging studies.
- Participants will continue to take cinacalcet once a day for 3 weeks. They will have regular study visits to monitor the treatment.
- There will be up to two other overnight hospital stays (1 to 3 nights) to adjust cinacalcet doses. The dose will increase until the maximum dose is reached, or side effects develop.
- After the end of the cinacalcet study, participants will have several more followup visits to monitor the effects of treatment.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Open-label Dose-titration Study of the Tolerability and Efficacy of Cinacalcet to Treat Fibroblast Growth Factor 23 (FGF23)-Mediated Hypophosphatemia|
- Evaluate the tolerability of cinacalcet in individuals with FGF23-mediated hypophosphatemia [ Time Frame: 4 years ]
- To explore the effectiveness of cinacalcet at decreaing renal phosphate wasting [ Time Frame: 4 years ]
- Evaluate the pharmacodynamics of cinacalcet in this subject population. [ Time Frame: 4 years ]
|Study Start Date:||November 2012|
|Estimated Study Completion Date:||October 2015|
|Estimated Primary Completion Date:||October 2015 (Final data collection date for primary outcome measure)|
|Contact: Diala M El-Maouche, M.D.||(301) firstname.lastname@example.org|
|Contact: Rachel I Gafni, M.D.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL) 800-411-1222 ext TTY8664111010 firstname.lastname@example.org|
|Principal Investigator:||Rachel I Gafni, M.D.||National Institute of Dental and Craniofacial Research (NIDCR)|