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Calcitriol Monotherapy for X-Linked Hypophosphatemia

NCT03748966 · Status: COMPLETED · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2026-05-07

Study results available
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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

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.

Sponsors & Collaborators

Principal Investigators

  • Eva Liu, MD · Massachusetts General Hospital and Brigham and Women's Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-03-28
Primary Completion
2025-03-01
Completion
2026-02-26
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03748966 on ClinicalTrials.gov