An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia
Primary Purpose
X-linked Hypophosphatemia
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
KRN23
Sponsored by
About this trial
This is an interventional treatment trial for X-linked Hypophosphatemia focused on measuring XLH
Eligibility Criteria
Inclusion Criteria:
- Satisfactory completion of KKP's sponsored KRN23-INT-001 clinical trial
- eGFR ≥ 60 mL/min
- Corrected Ca < 10.8 mg/dL
- For female of child-bearing potential, a negative serum pregnancy test
- A willingness to utilize adequate contraception and not become pregnant [or to have their partner(s) become pregnant] during the study
- Additional inclusion criteria apply
Exclusion Criteria:
- Subject experienced a safety-related event in the KRN23-INT-001 study
- Pregnant or lactating female subject or pregnant or female planning to become pregnant during the study
- Receipt of a live (attenuated) vaccine (except for influenza vaccines) during the course of the KRN23-INT-001 study and/or of this study
- Condition which could present a concern for either the subject's safety or difficulty with data interpretation
- Additional exclusion criteria apply
Sites / Locations
- University of California San Francisco
- Yale University School of Medicine
- Clinical Research Center, Indiana University School of Medicine
- Duke Clinical Research Unit
- University of Texas Health Science Center at Houston
- Shriners Hospital for Children - Canada
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
KRN23
Arm Description
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)
Outcomes
Primary Outcome Measures
Safety and Efficacy of Repeated SC Injections of KRN23.
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
Evaluation of Effect of Repeated SC Injections of KRN23
Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters (serum phosphorus)
Full Information
NCT ID
NCT01571596
First Posted
February 23, 2012
Last Updated
January 29, 2021
Sponsor
Kyowa Hakko Kirin Pharma, Inc.
Collaborators
Kyowa Kirin Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT01571596
Brief Title
An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia
Official Title
An Open-Label, Long-Term, Extension Study to Evaluate the Safety and Efficacy of KRN23 in Adult Subjects With X-Linked Hypophosphatemia
Study Type
Interventional
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
Completed
Study Start Date
February 2012 (undefined)
Primary Completion Date
September 2013 (Actual)
Study Completion Date
June 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Kyowa Hakko Kirin Pharma, Inc.
Collaborators
Kyowa Kirin Co., Ltd.
4. Oversight
Data Monitoring Committee
No
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
X-linked Hypophosphatemia
Keywords
XLH
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
23 (Actual)
8. Arms, Groups, and Interventions
Arm Title
KRN23
Arm Type
Experimental
Arm Description
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)
Intervention Type
Drug
Intervention Name(s)
KRN23
Intervention Description
Subjects will receive escalating doses of KRN23 administered by SC injection every 28-days (up to 12 doses) based on a dosing algorithm and discretion of Investigator and Sponsor.
Primary Outcome Measure Information:
Title
Safety and Efficacy of Repeated SC Injections of KRN23.
Description
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.
Time Frame
13.5 months,(50 visits)
Secondary Outcome Measure Information:
Title
Evaluation of Effect of Repeated SC Injections of KRN23
Description
Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters (serum phosphorus)
Time Frame
13.5 months, (50 visits)
Other Pre-specified Outcome Measures:
Title
Evaluation of Effect of Repeated SC Injections of KRN23 in Bone Substudy
Description
Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density, bone quality and histomorphometric parameters.
Time Frame
13.5 months,(50 visits)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Satisfactory completion of KKP's sponsored KRN23-INT-001 clinical trial
eGFR ≥ 60 mL/min
Corrected Ca < 10.8 mg/dL
For female of child-bearing potential, a negative serum pregnancy test
A willingness to utilize adequate contraception and not become pregnant [or to have their partner(s) become pregnant] during the study
Additional inclusion criteria apply
Exclusion Criteria:
Subject experienced a safety-related event in the KRN23-INT-001 study
Pregnant or lactating female subject or pregnant or female planning to become pregnant during the study
Receipt of a live (attenuated) vaccine (except for influenza vaccines) during the course of the KRN23-INT-001 study and/or of this study
Condition which could present a concern for either the subject's safety or difficulty with data interpretation
Additional exclusion criteria apply
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Amy Zhang, PhD
Organizational Affiliation
Kyowa Hakko Kirin Pharma, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of California San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Yale University School of Medicine
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06520
Country
United States
Facility Name
Clinical Research Center, Indiana University School of Medicine
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Duke Clinical Research Unit
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
University of Texas Health Science Center at Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Shriners Hospital for Children - Canada
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3G 1A6
Country
Canada
12. IPD Sharing Statement
Citations:
PubMed Identifier
25919461
Citation
Imel EA, Zhang X, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey JS, Glorieux FH, Portale AA, Insogna K, Peacock M, Carpenter TO. Prolonged Correction of Serum Phosphorus in Adults With X-Linked Hypophosphatemia Using Monthly Doses of KRN23. J Clin Endocrinol Metab. 2015 Jul;100(7):2565-73. doi: 10.1210/jc.2015-1551. Epub 2015 Apr 28. Erratum In: J Clin Endocrinol Metab. 2017 Jan 1;102(1):336.
Results Reference
derived
Links:
URL
http://www.clinicaltrials.gov/ct2/show/NCT00830674?term=XLH&rank=1
Description
Related Info
URL
http://www.clinicaltrials.gov/ct2/show/NCT01340482?term=KRN23&rank=1
Description
Related Info
Learn more about this trial
An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia
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