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

January 29, 2021 updated by: Kyowa Hakko Kirin Pharma, Inc.

An Open-Label, Long-Term, Extension Study to Evaluate the Safety and Efficacy of KRN23 in Adult Subjects With X-Linked Hypophosphatemia

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).

Study Overview

Status

Completed

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

23

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Quebec
      • Montreal, Quebec, Canada, H3G 1A6
        • Shriners Hospital for Children - Canada
    • California
      • San Francisco, California, United States, 94143
        • University of California San Francisco
    • Connecticut
      • New Haven, Connecticut, United States, 06520
        • Yale University School of Medicine
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Clinical Research Center, Indiana University School of Medicine
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke Clinical Research Unit
    • Texas
      • Houston, Texas, United States, 77030
        • University of Texas Health Science Center at Houston

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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)
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and Efficacy of Repeated SC Injections of KRN23.
Time Frame: 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.
13.5 months,(50 visits)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluation of Effect of Repeated SC Injections of KRN23
Time Frame: 13.5 months, (50 visits)
Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters (serum phosphorus)
13.5 months, (50 visits)

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluation of Effect of Repeated SC Injections of KRN23 in Bone Substudy
Time Frame: 13.5 months,(50 visits)
Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density, bone quality and histomorphometric parameters.
13.5 months,(50 visits)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2012

Primary Completion (Actual)

September 1, 2013

Study Completion (Actual)

June 1, 2014

Study Registration Dates

First Submitted

February 23, 2012

First Submitted That Met QC Criteria

April 3, 2012

First Posted (Estimate)

April 5, 2012

Study Record Updates

Last Update Posted (Actual)

February 17, 2021

Last Update Submitted That Met QC Criteria

January 29, 2021

Last Verified

October 1, 2020

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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