An Open Label Phase 2 Study to Assess the Safety Pharmacodynamics and Efficacy of KRN23 in Children from 1 to 4 years Old with X-linked Hypophosphatemia (XLH)
Study on Investigational Medication for Phosphate Metabolism in Children with XLH
Brief description of study.
The purpose of this study is to provide information about the safety profile, dosing, and effect of KRN23 on phosphate metabolism in children with XLH and confirmed evidence of rickets.
Detailed description of study
The purpose of this study is to provide information about the safety profile, dosing, and effect of KRN23 on phosphate metabolism in children with XLH and confirmed evidence of rickets.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: X-linked Hypophosphatemia
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Age: 100 years or below
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Gender: All
The purpose of this study is to investigate the safety, dosing, and effects of an investigational medication on phosphate metabolism in children diagnosed with X-linked hypophosphatemia (XLH) and showing signs of rickets. X-linked hypophosphatemia is a genetic disorder that affects bones, causing them to become soft and weak, often leading to rickets, a condition characterized by softening and weakening of bones in children.
Participants in the study will receive either the investigational medication or a placebo. A placebo is an inactive substance that looks like the investigational medicine but does not contain any medicine. The study will monitor the effects of the treatment on phosphate levels and bone health.
- Who can participate: Children aged 5-12 diagnosed with X-linked hypophosphatemia (XLH) and confirmed evidence of rickets are eligible to participate.
- Study details: Participants will receive the investigational medication or a placebo. A placebo is an inactive substance that looks like the investigational medicine but does not contain any medicine.
Interested in the study?
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