Status:
ACTIVE_NOT_RECRUITING
Calcitriol Monotherapy for X-Linked Hypophosphatemia
Lead Sponsor:
Massachusetts General Hospital
Collaborating Sponsors:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Conditions:
X-linked Hypophosphatemia
Hypophosphatemic Rickets
Eligibility:
All Genders
3-70 years
Phase:
EARLY_PHASE1
Brief Summary
Children and adults with XLH recruited will be treated with calcitriol alone (without phosphate supplementation) for one year, during which the calcitriol dose will be escalated during the first 3 mon...
Eligibility Criteria
Inclusion
- Clinical diagnosis of XLH with family history excluding male-to-male transmission, or positive genotype for PHEX mutation
- Serum PTH levels less than 1.5x the upper limit of normal
- Serum calcium levels less than 10.0 mg/dl
- eGFR \>= 60 mL/min/1.73m2
- 25(OH) vitamin D level \>= 20 ng/dL
Exclusion
- Known allergy to calcitriol
- Pregnancy or breast feeding
- Use of skeletally active agents such as bisphosphonates, teriparatide, SERMS, hormone replacement therapy and progesterone-only contraceptive agents (combination oral contraceptive use in premenopausal women is not an exclusion criterion).
- Unwilling or unable to stop therapy with calcitriol and phosphate therapy for two weeks prior to study
- Therapy with cinacalcet within the past two weeks
- Current use of growth hormone therapy
- Use of diuretics or medications that alter renal handling of mineral ions.
- Use of glucocorticoids for more than 14 days in the past 12 months with the exception of inhaled agents.
- History of malignancy except basal and squamous cell carcinoma of the skin.
- Significant history of psychiatric disease per DSM-5.
- Substance use disorder per DSM-5.
- Significant cardiopulmonary disease (unstable CAD or stage D ACC/AHA heart failure).
- Absence of laboratory values for serum calcium, phosphate and creatinine in the 24 months prior to enrollment.
Key Trial Info
Start Date :
March 28 2019
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2026
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT03748966
Start Date
March 28 2019
End Date
May 1 2026
Last Update
October 1 2025
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114