An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia

Phase 1

Completed

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

• Registration Number: NCT01571596
• 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).

Detailed Description

Not available

Study Timeline

• Study Start: 2012-02
• Study First Submitted: 2012-02-23
• Study First Submitted QC: 2012-04-03
• Study First Posted: 2012-04-05
• Primary Completion: 2013-09
• Study Completion: 2014-06
• Disposition First Submitted: 2015-01-12
• Disposition First Submitted QC: 2015-01-12
• Disposition First Posted: 2015-01-26
• Results First Submitted: 2020-10-08
• Results First Submitted QC: 2021-01-29
• Results First Posted: 2021-02-17
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-14
• Last Update Posted: 2024-06-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

Inclusion Criteria

1. Satisfactory completion of KKP's sponsored KRN23-INT-001 clinical trial
2. eGFR ≥ 60 mL/min
3. Corrected Ca < 10.8 mg/dL
4. For female of child-bearing potential, a negative serum pregnancy test
5. A willingness to utilize adequate contraception and not become pregnant [or to have their partner(s) become pregnant] during the study
6. Additional inclusion criteria apply

Exclusion Criteria

1. Subject experienced a safety-related event in the KRN23-INT-001 study
2. Pregnant or lactating female subject or pregnant or female planning to become pregnant during the study
3. Receipt of a live (attenuated) vaccine (except for influenza vaccines) during the course of the KRN23-INT-001 study and/or of this study
4. Condition which could present a concern for either the subject's safety or difficulty with data interpretation
5. 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 12 doses)

Primary Outcome Measures

Name	Time	Method
Safety and Efficacy of Repeated SC Injections of KRN23.	13.5 months,(50 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	13.5 months, (50 visits)	Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters (serum phosphorus)

Trial Locations

Locations (6)

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

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

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

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

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

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