Trial Results Posted: Calcitriol Monotherapy for X-Linked Hypophosphatemia (NCT03748966)
WHY IT MATTERS
This completed trial tested a simpler treatment approach for XLH using calcitriol monotherapy, which could potentially reduce the pill burden for patients who typically need both calcitriol and phosphate supplements.
Researchers at Massachusetts General Hospital completed a small study testing whether a vitamin D medication called calcitriol could help people with X-linked hypophosphatemia (XLH), a rare genetic condition that causes weak bones and low phosphate levels in the blood. Sixteen children and adults took calcitriol alone for one year to see if it could improve their phosphate levels and bone strength without causing kidney problems. The study also looked at whether the treatment helped children grow taller.
NCT ID: NCT03748966 Status: COMPLETED Conditions: X-linked Hypophosphatemia, Hypophosphatemic Rickets, Hypophosphatemic Rickets, X-Linked Dominant Phase: EARLY_PHASE1 Enrollment: 16 Sponsor: Massachusetts General Hospital 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.
ASK YOUR DOCTOR
If you or your child has XLH, ask your doctor whether the results from this trial might apply to your current treatment plan or if there are other ongoing trials you could join.