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

June 14, 2024 updated by: Kyowa Kirin Co., Ltd.

A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study 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). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

29

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

  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H3G 1A6
        • Shriners Hospital for Children - Canada
  • United States
    • California
      • San Francisco, California, United States, 94143
        • University of California, San Francisco Medical Center
    • Connecticut
      • New Haven, Connecticut, United States, 06520
        • Yale University School Of Medicine
    • Indiana
      • Indianapolis, Indiana, United States, 46202-5250
        • 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

Description

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

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 4 doses)
Drug: KRN23
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.

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: 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.
On-Treatment: 6.5 months, 27 total visits

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluation of Effect of Repeated SC Injections of KRN23
Time Frame: 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.
On-Treatment: 6.5 months, 27 total visits

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluation of Effect of Repeated SC Injections of KRN23 in Bone Substudy
Time Frame: On-Treatment: 6.5 months, 27 total visits
Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density and bone quality.
On-Treatment: 6.5 months, 27 total visits

Collaborators and Investigators

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

Sponsor

Kyowa Kirin Co., Ltd.

Collaborators

Kyowa Kirin Co., Ltd.

Investigators

  • Study Director: Amy Zhang, PhD, Kyowa Hakko Kirin Pharma, Inc.

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.

General Publications

Helpful Links

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

April 1, 2011

Primary Completion (Actual)

May 1, 2013

Study Completion (Actual)

October 1, 2013

Study Registration Dates

First Submitted

April 15, 2011

First Submitted That Met QC Criteria

April 20, 2011

First Posted (Estimated)

April 22, 2011

Study Record Updates

Last Update Posted (Actual)

June 18, 2024

Last Update Submitted That Met QC Criteria

June 14, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KRN23-INT-001

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.

Clinical Trials on X-linked Hypophosphatemia

Clinical Trials on KRN23

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Romania

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe