Trial record 15 of 185 for:
"National Institute of Dental and Craniofacial Research (NIDCR)"
Cinacalcet for Fibroblast Growth Factor 23 (FGF23)-Mediated Hypophosphatemia (Hypophosphatemic Rickets)
This study is currently recruiting participants.
Verified October 2012 by National Institutes of Health Clinical Center (CC)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Dental and Craniofacial Research (NIDCR) )
ClinicalTrials.gov Identifier:
NCT01748812
First received: December 11, 2012
Last updated: NA
Last verified: October 2012
History: No changes posted
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Purpose
Background:
- 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.
Objectives:
- To see if cinacalcet can be a safe and effective treatment for people with low phosphorus conditions due to high FGF23.
Eligibility:
- Individuals between 18 and 70 years of age who have different forms of hypophosphatemic rickets and tumor-induced hypophosphatemia
Design:
- 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.
Condition | Intervention | Phase |
---|---|---|
Osteomalacia |
Drug: Osteomalacia |
Phase 1 |
Study Type: | Interventional |
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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
hereditary hypophosphatemic rickets
MedlinePlus related topics:
Rickets
Drug Information available for:
Cinacalcet
U.S. FDA Resources
Further study details as provided by National Institutes of Health Clinical Center (CC):
Primary Outcome Measures:
- Evaluate the tolerability of cinacalcet in individuals with FGF23-mediated hypophosphatemia [ Time Frame: 4 years ]
Secondary Outcome Measures:
- 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 ]
Estimated Enrollment: | 17 |
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) |
Intervention Details:
Show Detailed Description-
Drug: Osteomalacia
N/A
Eligibility
Ages Eligible for Study: | 18 Years to 70 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Criteria
INCLUSION CRITERIA:
- Chronological age: 18-70 years
Diagnosis of a genetic form of FGF23-mediated hypophosphatemia:
- X-linked hypophosphatemic rickets (XLH)
- Autosomal dominant hypophosphatemic rickets (ADHR)
- Autosomal recessive hypophosphatemic rickets (ARHR)
Or, diagnosis of a non-genetic form of FGF23-mediated hypophosphatemia, i.e. tumor-induced osteomalacia (TIO)
- Ability to understand and provide informed consent
- Ability to complete the protocol scheduled assessments and medication regimen
- Women of child-bearing potential (not surgically sterile via tubal ligation, bilateral oophorectomy or hysterectomy, or who are not postmenopausal for greater than or equal to 1 year) must agree to practice adequate contraception that may include, but is not limited to, abstinence, monogamous relationship with vasectomized partner, barrier methods such as condoms, diaphragms, spermicides, intrauterine devices, and licensed hormonal methods for the duration of the treatment portion of the study.
EXCLUSION CRITERIA:
- Chronic or recurrent hypocalcemia defined by a serum calcium < 8.4 mg/dL (2.1 mmol/L)
- Tertiary hyperparathyroidism as evidenced by concurrent PTH and calcium levels above the upper limit of normal
- History of parathyroid surgery and/or hypoparathyroidism
- Hypercalciuria as defined as > 4 mg/kg/day (0.1 mmol/kg/day) on optimized conventional therapy (as determined during SOC optimization phase)
- Moderate to severe hepatic insufficiency as defined by total bilirubin > 2 mg/dL and serum albumin < 3 g/dL and International Normalized Ratio (INR) > 2 OR presence of ascites or hepatic encephalopathy.
- A calculated eGFR < 50 mL/min/1.73 m(2), using the CKD-EPI equation
- History of a non-febrile seizure disorder
- History of a clinically significant cardiac arrhythmia
- History of chronic gastrointestinal disease
- Current therapy (at the time of informed consent) bisphosphonates, calcitonin, diuretics or medications that may have a significant drug interaction with cinacalcet
- Known hypersensitivity to cinacalcet or any of its constituents
- Positive pregnancy test or lactation
- Use of another investigational agent (i.e., in the context of a clinical trial, use of an investigational product that may have impact on the study) within the last 3 months
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01748812
Contacts
Contact: Diala M El-Maouche, M.D. | (301) 435-6280 | elmaouched@mail.nih.gov |
Contact: Rachel I Gafni, M.D. | (301) 594-9924 | gafnir@mail.nih.gov |
Locations
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 prpl@mail.cc.nih.gov |
Sponsors and Collaborators
Investigators
Principal Investigator: | Rachel I Gafni, M.D. | National Institute of Dental and Craniofacial Research (NIDCR) |
More Information
Additional Information:
Publications:
Keywords provided by National Institutes of Health Clinical Center (CC):
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on February 21, 2013
Additional Information:
Publications:
Responsible Party: | National Institutes of Health Clinical Center (CC) ( National Institute of Dental and Craniofacial Research (NIDCR) ) |
ClinicalTrials.gov Identifier: | NCT01748812 History of Changes |
Other Study ID Numbers: | 130025, 13-D-0025 |
Study First Received: | December 11, 2012 |
Last Updated: | December 11, 2012 |
Health Authority: | United States: Federal Government |
Keywords provided by National Institutes of Health Clinical Center (CC):
Tumor Induced Osteomalacia Hypophosphatemic Rickets |
Additional relevant MeSH terms:
Osteomalacia Hypophosphatemia Hypophosphatemic Rickets, X-Linked Dominant Bone Diseases, Metabolic Bone Diseases Musculoskeletal Diseases Calcium Metabolism Disorders Metabolic Diseases Vitamin D Deficiency Avitaminosis Deficiency Diseases Malnutrition Nutrition Disorders Phosphorus Metabolism Disorders |
Rickets Hypophosphatemia, Familial Renal Tubular Transport, Inborn Errors Kidney Diseases Urologic Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Metal Metabolism, Inborn Errors Metabolism, Inborn Errors Mitogens Mitosis Modulators Molecular Mechanisms of Pharmacological Action Pharmacologic Actions |
ClinicalTrials.gov processed this record on February 21, 2013