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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01571596
Recruitment Status : Completed
First Posted : April 5, 2012
Results First Posted : February 17, 2021
Last Update Posted : February 17, 2021
Kyowa Kirin Co., Ltd.
Information provided by (Responsible Party):
Kyowa Kirin, Inc. ( Kyowa Hakko Kirin Pharma, Inc. )

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).

Condition or disease Intervention/treatment Phase
X-linked Hypophosphatemia Drug: KRN23 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : February 2012
Actual Primary Completion Date : September 2013
Actual Study Completion Date : June 2014

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)
Drug: KRN23
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 Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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)

Other Outcome Measures:
  1. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01571596

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United States, California
University of California San Francisco
San Francisco, California, United States, 94143
United States, Connecticut
Yale University School of Medicine
New Haven, Connecticut, United States, 06520
United States, Indiana
Clinical Research Center, Indiana University School of Medicine
Indianapolis, Indiana, United States, 46202
United States, North Carolina
Duke Clinical Research Unit
Durham, North Carolina, United States, 27710
United States, Texas
University of Texas Health Science Center at Houston
Houston, Texas, United States, 77030
Canada, Quebec
Shriners Hospital for Children - Canada
Montreal, Quebec, Canada, H3G 1A6
Sponsors and Collaborators
Kyowa Hakko Kirin Pharma, Inc.
Kyowa Kirin Co., Ltd.
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Study Director: Amy Zhang, PhD Kyowa Hakko Kirin Pharma, Inc.
Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Kyowa Hakko Kirin Pharma, Inc. Identifier: NCT01571596    
Other Study ID Numbers: KRN23-INT-002
First Posted: April 5, 2012    Key Record Dates
Results First Posted: February 17, 2021
Last Update Posted: February 17, 2021
Last Verified: October 2020
Keywords provided by Kyowa Kirin, Inc. ( Kyowa Hakko Kirin Pharma, Inc. ):
Additional relevant MeSH terms:
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Familial Hypophosphatemic Rickets
Phosphorus Metabolism Disorders
Metabolic Diseases
Rickets, Hypophosphatemic
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Hypophosphatemia, Familial
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Calcium Metabolism Disorders
Vitamin D Deficiency
Deficiency Diseases
Nutrition Disorders