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

This study has been completed.
Sponsor:
Collaborator:
Kyowa Hakko Kirin Company, Limited
Information provided by (Responsible Party):
Kyowa Hakko Kirin Pharma, Inc.
ClinicalTrials.gov Identifier:
NCT01340482
First received: April 15, 2011
Last updated: October 18, 2013
Last verified: October 2013
  Purpose

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 Intervention Phase
X-linked Hypophosphatemia
Drug: Biological KRN23
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Kyowa Hakko Kirin Pharma, Inc.:

Primary Outcome Measures:
  • Safety and Efficacy of repeated SC injections of KRN23 [ Time Frame: On-Treatment: 6.5 months, 27 total visits ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: On-Treatment: 6.5 months, 27 total visits ] [ Designated as safety issue: No ]
    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:
  • Evaluation of effect of repeated SC injections of KRN23 in Bone Substudy [ Time Frame: On-Treatment: 6.5 months, 27 total visits ] [ Designated as safety issue: No ]
    Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density and bone quality.


Enrollment: 29
Study Start Date: April 2011
Study Completion Date: October 2013
Primary Completion Date: May 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Open-Label KRN23; Single-blind KRN23 vs Placebo Drug: Biological 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01340482

Locations
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 Company, Limited
Investigators
Study Director: Amy Zhang, PhD Kyowa Hakko Kirin Pharma
  More Information

Additional Information:
No publications provided

Responsible Party: Kyowa Hakko Kirin Pharma, Inc.
ClinicalTrials.gov Identifier: NCT01340482     History of Changes
Other Study ID Numbers: KRN23-INT-001
Study First Received: April 15, 2011
Last Updated: October 18, 2013
Health Authority: United States: Food and Drug Administration
Canada: Health Canada

Keywords provided by Kyowa Hakko Kirin Pharma, Inc.:
XLH

Additional relevant MeSH terms:
Hypophosphatemia
Hypophosphatemic Rickets, X-Linked Dominant
Phosphorus Metabolism Disorders
Metabolic Diseases
Rickets
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Hypophosphatemia, Familial
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Calcium Metabolism Disorders
Vitamin D Deficiency
Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders

ClinicalTrials.gov processed this record on July 24, 2014