Plain-English translation of NCT03301038 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
This trial is testing whether , an antibiotic medication, can help treat a rare genetic condition called idiopathic infantile hypercalcemia. In this condition, the body accumulates too much calcium in the blood and urine because it cannot properly break down vitamin D. The study is exploring whether the medication can activate alternative pathways in the body to eliminate excess vitamin D, bringing calcium levels back to normal.
People with this genetic condition currently have very few treatment options, and high calcium levels can cause serious complications like kidney damage and bone problems. Researchers believe this medication might offer a new way to help by activating enzymes that can break down vitamin D through a different route than the faulty one caused by the genetic mutation.
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You would visit the research center for an 8-week observation period before starting treatment, then take the medication by mouth once daily for 16 weeks while having regular check-ups to monitor your calcium levels and how you're tolerating the medication. After treatment ends, you would have 8 more weeks of follow-up visits. Throughout the study, researchers will track your blood and urine calcium levels, study how your body processes the medication, and measure how much calcium your intestines are absorbing.
AI-generated summary from trial data · Oct 12, 2026 · Not medical advice
United States