A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia

Phase 1

Completed

• Conditions: X-linked Hypophosphatemia
• Interventions: Drug: KRN23

• Registration Number: NCT01340482
• Lead Sponsor: Kyowa Kirin Co., Ltd.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-04
• Study First Submitted: 2011-04-15
• Study First Submitted QC: 2011-04-20
• Study First Posted: 2011-04-22
• Primary Completion: 2013-05
• Study Completion: 2013-10
• Disposition First Submitted: 2015-01-12
• Disposition First Submitted QC: 2015-01-12
• Disposition First Posted: 2015-01-26
• Results First Submitted: 2020-09-30
• Results First Submitted QC: 2020-09-30
• Results First Posted: 2020-10-26
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-14
• Last Update Posted: 2024-06-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 29

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
KRN23	KRN23	Escalating doses of KRN23 (0.05, 0.10, 0.30 and 0.60 mg/kg) will be administered SC every 28 days (up to 4 doses)

Primary Outcome Measures

Name	Time	Method
Safety and Efficacy of Repeated SC Injections of KRN23	On-Treatment: 6.5 months, 27 total visits	Safety and efficacy of repeated SC injections of KRN23 from baseline as assessed by serum phosphorus levels,immunogenicity, adverse events and clinically significant changes in vital signs and laboratory testing.

Secondary Outcome Measures

Name	Time	Method
Evaluation of Effect of Repeated SC Injections of KRN23	On-Treatment: 6.5 months, 27 total visits	Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters including serum phosphorus, sex hormone, bone biomarkers, quality of life assessments and population pharmacokinetics of KRN23 dose levels from cumulative dosing. Summary of serum phosphorus by visit/day is captured below.

Trial Locations

Locations (6)

University of California, San Francisco Medical Center; 🇺🇸 San Francisco, California, United States

Clinical Research Center, Indiana University School of Medicine; 🇺🇸 Indianapolis, Indiana, United States

Yale University School of Medicine; 🇺🇸 New Haven, Connecticut, United States

Shriners Hospital for Children - Canada; 🇨🇦 Montreal, Quebec, Canada

Duke Clinical Research Unit; 🇺🇸 Durham, North Carolina, United States

University of Texas Health Science Center at Houston; 🇺🇸 Houston, Texas, United States