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A Study of KRN23 in Subjects With Tumor-Induced Osteomalacia or Epidermal Nevus Syndrome

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ClinicalTrials.gov Identifier: NCT02722798
Recruitment Status : Active, not recruiting
First Posted : March 30, 2016
Last Update Posted : July 31, 2019
Sponsor:
Information provided by (Responsible Party):
Kyowa Kirin Co., Ltd.

Brief Summary:
The purpose of the study is to evaluate the efficacy and safety of KRN23 after its 48-week once every 4 weeks repeated subcutaneous administration to Japanese and Korean patients with Tumor-Induced Osteomalacia or Epidermal Nevus Syndrome by a multicenter, open-label, intraindividual dose adjustment study

Condition or disease Intervention/treatment Phase
Tumor-Induced Osteomalacia or Epidermal Nevus Syndrome Drug: KRN23 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Open-Label Trial to Assess the Efficacy and Safety of KRN23 in Patients With Tumor-Induced Osteomalacia or Epidermal Nevus Syndrome
Study Start Date : April 2016
Actual Primary Completion Date : July 2017
Estimated Study Completion Date : December 2020


Arm Intervention/treatment
Experimental: KRN23
Subjects will receive subcutaneous injections of KRN23 every 4 weeks from Week 0 through Week 44
Drug: KRN23
Doses may be titrated to achieve the target peak serum phosphorus range




Primary Outcome Measures :
  1. serum phosphorus concentration at each test time point [ Time Frame: up to week 48 ]

Secondary Outcome Measures :
  1. change from baseline in alkaline phosphatase [ Time Frame: up to week 48 ]
  2. change from baseline in 1,25(OH)2D [ Time Frame: up to week 48 ]
  3. change from baseline in urine P [ Time Frame: up to week 48 ]
  4. change from baseline in tubular reabsorption of phosphate [ Time Frame: up to week 48 ]
  5. change from baseline in ratio of renal tubular maximum phosphate reabsorption rate to glomerular filtration rate [ Time Frame: up to week 48 ]
  6. change from baseline in skeletal disease/osteomalacia through trans-iliac crest bone biopsy [ Time Frame: up to week 48 ]
  7. Effect to Sit to Stand (STS) test [ Time Frame: up to week 48 ]
  8. Effect to Hand Held Dynamometry (HHD) [ Time Frame: up to week 48 ]
  9. Effect to Weighted Arm Lift (WAL) test [ Time Frame: up to week 48 ]
  10. Effect to 6 minute walking test (6MWT) [ Time Frame: up to week 48 ]
  11. Effect to patient reported outcomes [ Time Frame: up to week 48 ]
  12. maximum concentration (Cmax) of KRN23 [ Time Frame: up to week 48 ]
  13. area under the curve (AUC) of KRN23 [ Time Frame: up to week 48 ]
  14. half-life (t1/2) of KRN23 [ Time Frame: up to week 48 ]

Other Outcome Measures:
  1. Number and types of adverse events [ Time Frame: up to week 48 ]


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
Criteria

Inclusion Criteria:

  1. Aged ≥ 18 years
  2. Diagnosis of Tumor-Induced Osteomalacia(TIO) or Epidermal Nevus Syndrome(ENS) and not amenable to receive surgical excision of the offending tumor/lesion
  3. Serum phosphorus level < 2.5 mg/dL
  4. Serum FGF23 level ≥ 100 pg/mL
  5. Ratio of renal tubular maximum phosphate reabsorption rate to glomerular filtration rate< 2.5 mg/dL
  6. Estimated glomerular filtration rate (eGFR) at screening ≥ 60 mL/min/1.73 m2, or eGFR ≥ 30 and < 60 mL/min/1.73 m2 with an evidence of no renal failure related to nephrocalcinosis
  7. Corrected serum calcium level < 10.8 mg/dL
  8. For female subjects of childbearing potential; negative urine pregnancy test and willingness to undergo additional pregnancy tests during the study
  9. Willingness to use an acceptable method of contraception while participating in the study
  10. Willingness to provide access to prior medical records to determine eligibility including data on imaging tests, blood chemistry, diagnosis, medication, and surgical history
  11. Willingness and ability to cooperatively complete all study procedures, adhere to the visit schedule and follow the investigator's instructions, as considered by the investigator or subinvestigator

Exclusion Criteria:

  1. Use of the following drugs within 14 days prior to screening: pharmacologic vitamin D metabolites or analogs, or drugs for treating TIO/ENS including oral phosphate, aluminum hydroxide antacids, acetazolamide, or thiazide diuretics
  2. Medication to suppress parathyroid hormone (PTH) within 60 days prior to screening
  3. Blood or blood product transfusion within 60 days prior to screening
  4. Chemotherapy for TIO or other malignant tumors within 4 months prior to screening
  5. History of being positive for human immunodeficiency virus antibody, hepatitis B antigen and/or hepatitis C virus antibody
  6. Predisposition to infection, or history of recurrent infection or known immunodeficiency
  7. Pregnant or breastfeeding at screening or intention to become pregnant during the study; for male subjects, the partner's intention to become pregnant during the study
  8. Use of an investigational product or device within 4 months prior to screening, or planning to receive other investigational product before completing all assessments in this study
  9. Use of therapeutic monoclonal antibodies including KRN23 within 90 days prior to screening
  10. History of allergic or anaphylactic reactions to KRN23, any of the KRN23 ingredients, or any other monoclonal antibodies
  11. Anyone otherwise considered unsuitable participation in the study by the investigator or subinvestigator

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): NCT02722798


Locations
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Japan
Osaka, Japan
Tokyo, Japan
Korea, Republic of
Seoul, Korea, Republic of
Sponsors and Collaborators
Kyowa Kirin Co., Ltd.

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Responsible Party: Kyowa Kirin Co., Ltd.
ClinicalTrials.gov Identifier: NCT02722798     History of Changes
Other Study ID Numbers: KRN23-002
First Posted: March 30, 2016    Key Record Dates
Last Update Posted: July 31, 2019
Last Verified: October 2018

Additional relevant MeSH terms:
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Syndrome
Nevus
Osteomalacia
Neoplasms, Connective Tissue
Nevus, Sebaceous of Jadassohn
Disease
Pathologic Processes
Nevi and Melanomas
Neoplasms by Histologic Type
Neoplasms
Rickets
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Metabolic Diseases
Calcium Metabolism Disorders
Vitamin D Deficiency
Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders
Neoplasms, Connective and Soft Tissue
Connective Tissue Diseases
Neurocutaneous Syndromes
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities