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A Repeated 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
Kyowa Hakko Kirin Pharma, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for X-linked Hypophosphatemia focused on measuring XLH

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of California, San Francisco Medical Center
  • 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 4 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 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.

Full Information

First Posted
April 15, 2011
Last Updated
September 30, 2020
Sponsor
Kyowa Hakko Kirin Pharma, Inc.
Collaborators
Kyowa Kirin Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT01340482
Brief Title
A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia
Official Title
A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia
Study Type
Interventional

2. Study Status

Record Verification Date
September 2020
Overall Recruitment Status
Completed
Study Start Date
April 2011 (undefined)
Primary Completion Date
May 2013 (Actual)
Study Completion Date
October 2013 (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). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality.

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
29 (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 4 doses)
Intervention Type
Drug
Intervention Name(s)
KRN23
Intervention Description
Subjects will receive escalating doses of KRN23 administered by SC injections every 28-days (up to 4 doses) based on a dosing algorithm and discretion of the 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
On-Treatment: 6.5 months, 27 total 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 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.
Time Frame
On-Treatment: 6.5 months, 27 total 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 and bone quality.
Time Frame
On-Treatment: 6.5 months, 27 total visits

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Main Inclusion Criteria: 18 years or older Clinical diagnosis of XLH TmP/GFR < 2.0 mg/dL GFR ≥60 mL/min Corrected Ca <10.8 mg/dL 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 Main Exclusion Criteria: Have any sign of active infectious disease or have had an infection requiring treatment with antibiotics within 3-weeks of Screening History of known immunodeficiency Pregnant or lactating females subjects or female planning to be become pregnant during the study Receipt of a live (attenuated) vaccine (except for influenza vaccines) within 3-months of Screening, and/or during the study 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 Use of any supplement containing phosphate, calcium and/or vitamin D within 10-days prior to Screening and/or dosing 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 Medical Center
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
46202-5250
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
26073451
Citation
Zhang X, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey J, Glorieux FH, Portale AA, Insogna K, Carpenter TO, Peacock M. Pharmacokinetics and pharmacodynamics of a human monoclonal anti-FGF23 antibody (KRN23) in the first multiple ascending-dose trial treating adults with X-linked hypophosphatemia. J Clin Pharmacol. 2016 Feb;56(2):176-85. doi: 10.1002/jcph.570. Epub 2015 Aug 11.
Results Reference
derived
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
https://clinicaltrials.gov/ct2/show/NCT01571596?term=KRN23-INT-002&draw=2&rank=1
Description
KNR23-INT-002: An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia

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

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