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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Kyowa Hakko Kirin Company, Limited
Information provided by (Responsible Party):
Kyowa Hakko Kirin Pharma, Inc.
ClinicalTrials.gov Identifier:
NCT01571596
First received: February 23, 2012
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, bone quality and histomorphometric parameters.


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: An Open-Label, Long-Term, Extension Study to Evaluate the Safety and Efficacy 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: 13.5 months,(50 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: 13.5 months, (50 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: 13.5 months,(50 visits) ] [ Designated as safety issue: No ]
    Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density, bone quality and histomorphometric parameters.


Enrollment: 23
Study Start Date: February 2012
Estimated Study Completion Date: June 2014
Primary Completion Date: September 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 injection every 28-days (up to 4 doses) based on a dosing algorithm and discretion of Investigator and Sponsor.

  Eligibility

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

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
  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: NCT01571596

Locations
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 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: NCT01571596     History of Changes
Other Study ID Numbers: KRN23-INT-002
Study First Received: February 23, 2012
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:
Familial Hypophosphatemic Rickets
Hypophosphatemia
Avitaminosis
Bone Diseases
Bone Diseases, Metabolic
Calcium Metabolism Disorders
Deficiency Diseases
Genetic Diseases, Inborn
Hypophosphatemia, Familial
Kidney Diseases
Malnutrition
Metabolic Diseases
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Musculoskeletal Diseases
Nutrition Disorders
Phosphorus Metabolism Disorders
Renal Tubular Transport, Inborn Errors
Rickets
Rickets, Hypophosphatemic
Urologic Diseases
Vitamin D Deficiency

ClinicalTrials.gov processed this record on October 23, 2014