Calcitriol Monotherapy for X-Linked Hypophosphatemia

Phase 0 Completed 16 enrolled Massachusetts General Hospital National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) · collab

Non-randomizedParallel-groupOpen-labelTreatment

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.

Timeline

Start: 2019-03-28
Primary completion: 2025-03-01
Completion: 2026-02-26

Publications

Background Liu ES, Martins JS, Raimann A, Chae BT, Brooks DJ, Jorgetti V, Bouxsein ML, Demay MB. 1,25-Dihydroxyvitamin D Alone Improves Skeletal Growth, Microarchitecture, and Strength in a Murine Model of XLH, Despite Enhanced FGF23 Expression. J Bone Miner Res. 2016 May;31(5):929-39. doi: 10.1002/jbmr.2783. Epub 2016 Feb 2.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Calcitriol	Small molecule	—	Oral

Calcitriol Monotherapy for X-Linked Hypophosphatemia

Summary

Timeline

Publications

Drugs

Indications