NCT03748966

Calcitriol Monotherapy for X-Linked Hypophosphatemia

Calcitriol Monotherapy for X-Linked Hypophosphatemia: Effects on Mineral Ions, Growth and Skeletal Parameters

• Status: Active Not Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Massachusetts General Hospital · Academic / Other
• Sex: All
• Age: 3 Years – 70 Years
• Healthy volunteers: Not accepted

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 months of therapy. The investigators hypothesize that treatment of adults and children with XLH alone will improve serum phosphate levels and skeletal mineralization without causing an increase in kidney calcifications. The study will also examine if calcitriol therapy will improve growth in children.

Conditions

• X-linked Hypophosphatemia
• Hypophosphatemic Rickets
• Hypophosphatemic Rickets, X-Linked Dominant

Interventions

Type	Name	Description
DRUG	Calcitriol	Adults and children (age 3-17) with X-linked hypophosphatemia will be treated with calcitriol therapy without phosphate supplementation. Doses of calcitriol will be escalated and optimized in the first three months of the study. Calcitriol is an oral medication taken once a day.

Timeline

• Start date: 2019-03-28
• Primary completion: 2025-03-01
• Completion: 2026-05-01
• First posted: 2018-11-21
• Last updated: 2025-10-01

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study