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Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)

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ClinicalTrials.gov Identifier: NCT02312687
Recruitment Status : Active, not recruiting
First Posted : December 9, 2014
Last Update Posted : September 20, 2018
Sponsor:
Collaborator:
Kyowa Hakko Kirin Co., Ltd
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Brief Summary:
UX023-CL203 is a Phase 2b, open-label, long-term extension study. The study will be conducted in adults to assess the safety and pharmacodynamics of KRN23 administered via subcutaneous injections monthly (every 4 weeks) for a total of 68 weeks. The study will enroll approximately 25 adults with XLH who participated in Kyowa Hakko Kirin Pharma, Inc.'s (KHK's) study KRN23-INT-001 or KRN23-INT-002. Subjects will need to discontinue oral phosphate and vitamin D metabolite therapy prior to enrollment and throughout the duration of the study.

Condition or disease Intervention/treatment Phase
X-Linked Hypophosphatemia Biological: KRN23 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2b, Open-Label, Long-Term Extension Study to Evaluate the Safety and Pharmacodynamics of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)
Actual Study Start Date : March 12, 2015
Estimated Primary Completion Date : November 24, 2018
Estimated Study Completion Date : November 24, 2018


Arm Intervention/treatment
Experimental: All Subjects
Subjects will receive sub-cutaneous injections of KRN23 every 4 weeks. Starting doses will be based on the subject's last dose in study KRN23-INT-001 or KRN23-INT-002. Doses may be titrated to achieve the target peak serum phosphorus range.
Biological: KRN23
KRN23 is a recombinant fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23).
Other Names:
  • burosumab
  • Crysvita®




Primary Outcome Measures :
  1. Incidence, frequency, and severity of adverse events and serious adverse events [ Time Frame: Up to 192 Weeks or November 2018 ]

Secondary Outcome Measures :
  1. Change from Baseline in serum FGF23 [ Time Frame: Up to 184 Weeks or September 2018 ]
  2. Change from Baseline in serum ALP [ Time Frame: Up to 184 Weeks or September 2018 ]
  3. Change from Baseline in serum 1,25(OH)2D [ Time Frame: Up to 184 Weeks or September 2018 ]
  4. Change from Baseline in serum iPTH [ Time Frame: Up to 184 Weeks or September 2018 ]
  5. Change from Baseline in serum and urine phosphorus [ Time Frame: Up to 184 Weeks or September 2018 ]
  6. Change from Baseline in serum TRP and TMP/GFR [ Time Frame: Up to 184 Weeks or September 2018 ]
  7. Change from Baseline in serum BALP [ Time Frame: Up to 184 Weeks or September 2018 ]
  8. Change from Baseline in serum CTx [ Time Frame: Up to 184 Weeks or September 2018 ]
  9. Change from Baseline in serum P1NP [ Time Frame: Up to 184 Weeks or September 2018 ]


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Have participated in KHK's KRN23-INT-001 or KRN23-INT-002 studies (received at least 2 doses of KRN23)
  2. Estimated glomerular filtration rate (eGFR) ≥60 mL/min at Screening.
  3. Sexually active subjects must be willing to use an acceptable method of contraception (e.g., double barrier method) while participating in the study and for 30 days after receiving the last dose of KRN23.

Exclusion Criteria:

  1. Subject experienced a safety-related event in the KRN23-INT-001 or KRN23-INT-002 study that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
  2. Presence of nephrocalcinosis on renal ultrasound that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
  3. Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
  4. Participation in an investigational drug or device trial within 30 days of enrollment (other than KRN23-INT-001 or KRN23-INT-002).
  5. Use of a pharmacologic vitamin D metabolite or analog (e.g., calcitriol, doxercalciferol, and paricalcitol), phosphate, or aluminum hydroxide antacids (e.g., Maalox® and Mylanta®) within 21 days prior to Screening or during the study.
  6. Use of medication to suppress PTH (e.g., Sensipar®, cinacalcet, calcimimetics) within 2 months prior to Screening.

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): NCT02312687


Locations
United States, California
University California San Francisco Hospital
San Francisco, California, United States, 94143
United States, Connecticut
Yale University School of Medicine
New Haven, Connecticut, United States, 06520-8064
United States, Indiana
Indiana University Hospital
Indianapolis, Indiana, United States, 46202
United States, North Carolina
Duke University
Durham, North Carolina, United States, 27705
United States, Texas
Houston Methodist Reasearch Institute
Houston, Texas, United States, 77030
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Kyowa Hakko Kirin Co., Ltd
Investigators
Study Director: Medical Director Ultragenyx Pharmaceutical Inc

Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT02312687     History of Changes
Other Study ID Numbers: UX023-CL203
First Posted: December 9, 2014    Key Record Dates
Last Update Posted: September 20, 2018
Last Verified: September 2018

Keywords provided by Ultragenyx Pharmaceutical Inc:
XLH
FGF23
KRN23

Additional relevant MeSH terms:
Hypophosphatemia
Familial Hypophosphatemic Rickets
Phosphorus Metabolism Disorders
Metabolic Diseases
Rickets, Hypophosphatemic
Rickets
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
Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders