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

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ClinicalTrials.gov Identifier: NCT01340482
Recruitment Status : Completed
First Posted : April 22, 2011
Last Update Posted : June 14, 2016
Information provided by (Responsible Party):

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.

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

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 29 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia
Study Start Date : April 2011
Primary Completion Date : May 2013
Study Completion Date : October 2013

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

Other Outcome Measures:
  1. 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.

Eligibility Criteria

Information from the National Library of Medicine

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

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
Contacts and Locations

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 ClinicalTrials.gov identifier (NCT number): NCT01340482

United States, California
University of California, San Francisco Medical Center
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, 46202-5250
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 Hakko Kirin Co., Ltd
Study Director: Amy Zhang, PhD Kyowa Hakko Kirin Pharma, Inc.
More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Kyowa Hakko Kirin Pharma, Inc.
ClinicalTrials.gov Identifier: NCT01340482     History of Changes
Other Study ID Numbers: KRN23-INT-001
First Posted: April 22, 2011    Key Record Dates
Last Update Posted: June 14, 2016
Last Verified: January 2015

Keywords provided by Kyowa Kirin Pharmaceutical Development, Inc. ( Kyowa Hakko Kirin Pharma, Inc. ):

Additional relevant MeSH terms:
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