A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia

KRN23-INT-001

The primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality.

2011-04-01

2013-05-01

2013-10-01

Completed

Early phase I

29

Main Inclusion Criteria: 1. 18 years or older 2. Clinical diagnosis of XLH 3. TmP/GFR \< 2.0 mg/dL 4. GFR ≥60 mL/min 5. Corrected Ca \<10.8 mg/dL 6. A willingness to utilize adequate contraception and not become pregnant\[or to have their partner(s) become pregnant\] during the study 7. Additional inclusion criteria apply Main Exclusion Criteria: 1. Have any sign of active infectious disease or have had an infection requiring treatment with antibiotics within 3-weeks of Screening 2. History of known immunodeficiency 3. Pregnant or lactating females subjects or female planning to be become pregnant during the study 4. Receipt of a live (attenuated) vaccine (except for influenza vaccines) within 3-months of Screening, and/or during the study 5. Use of pharmacologic vitamin D metabolite or its analog, phosphate, calcimimetics, and ingestion of aluminum hydroxide antacids within 10-days prior to Screening and/or dosing 6. Use of any supplement containing phosphate, calcium and/or vitamin D within 10-days prior to Screening and/or dosing 7. Condition which could present a concern for either the subject's safety or difficulty with data interpretation 8. Additional exclusion criteria apply

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 29, 'type': 'ACTUAL'}}

2024-06-18