- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04320316
A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome (ENS) (ENS)
An Open Label Trial to Assess the Safety and Efficacy of KRN23, an Investigational Antibody to FGF23, in a Single Pediatric Patient With Epidermal Nevus Syndrome (ENS) and Associated Hypophosphatemic Rickets
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 4
Contacts and Locations
Study Locations
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Alabama
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Birmingham, Alabama, United States, 35233
- University of Alabama at Birmingham
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Patient has confirmed ENS by physician diagnosis
- Patient has confirmed FGF23 elevations in the context of low serum phosphorous < 4.1 mg/dL
- Patient able to tolerate KRN23 treatment
- Have a corrected serum calcium level < 10.8mg/dL
- Have an eGFR >60 ml/min
- Must be willing in the opinion of the investigator, to comply with study procedures and schedule
- Provide written informed consent by a parent after
Exclusion Criteria:
- Patient should not use CRYSVITA with Oral phosphate or active Vitamin D analogs.
- Patient and investigator should not initiate CRYSVITA if Phosphorus level is within or above normal.
- CRYSVITA is contraindicated in patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism.
- The use or enrollment in studies using other investigational therapies including other monoclonal antibodies
- Subject and their Parent not willing or not able to give written informed consent
- In the Investigators opinion, the subject may not be able to meet all the requirements for study participation
- Subject has a history of hypersensitivity to KRN23 excipients that in the opinion of the investigator, places the subject at an increased risk of adverse effects
- Subject has a condition that in the opinion of the investigator could present a concern for subject safety or data interpretation.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Crysvita (burosumab-twza) Treatment
The starting dose will be 0.3 mg/kg to be given every 2 weeks. If required dose may be titrated with increments of 0.1 mg/kg/dose every 4 weeks up to a maximum of dose of 2.0mg/kg (not to exceed 90mg per dose) until phosphorus level is WNL. Patient will receive study drug via SC injection to the abdomen, upper arms, thighs, or buttocks; the injection site will be rotated with each injection. If the dose level exceeds 1.5 mL in volume, the dose should be administered at two injection sites. Duration of treatment is 52 weeks. Subjects that complete treatment through week 52 may have the option to continue KRN23 treatment. If this is warranted based on preliminary efficacy, the current protocol will be amended to allow for an extension. |
KRN23 is a fully human IgG1monoclonal antibody that binds to and inhibits the activity of FGF23, leading to an increase in serum phosphorus levels.
It is a potential therapeutic candidate for the treatment of XLH, Tumor-Induced Osteomalacia (TIO), and the rickets/osteomalacia resulting from Epidermal Nevus Syndrome (ENS).
All of these conditions are diseases of bone hypomineralization, caused by urinary phosphate wasting due to elevated levels of FGF23.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
The Participant Will Achieve Normal Age-adjusted Phosphorous Levels as Tested by Fasting Serum Lab Values
Time Frame: every 2 week, from baseline to 52 weeks.
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Checking PO4 levels every two weeks and adjusting doses every 4 weeks in blood
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every 2 week, from baseline to 52 weeks.
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Participant Will Achieve Improving Vitamin D Levels as Measured by Serum Blood Tests.
Time Frame: 1 year
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check Vitamin D 1,25 in blood every 3 months
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1 year
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Participant Will Achieve Improving iPTH Levels as Measured by Serum Blood Tests.
Time Frame: every 3 months, From Baseline to 52 weeks
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measure PTH levels approximately every 3 months
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every 3 months, From Baseline to 52 weeks
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Participant Will Achieve Improving Calcium Levels as Measured by Serum Blood Tests.
Time Frame: every 3 months, From Baseline to 52 weeks
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measure Calcium level every 3 months
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every 3 months, From Baseline to 52 weeks
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Participant Will Achieve Improvement of Underlying Skeletal Disease/Rickets as Assessed by Standard Radiographs.
Time Frame: baseline scans prior to drug administration
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DEXA (dual energy X-ray Absorbometry) scans and whole body x-rays will be taken at baseline.
A lower Z score is indicative of poor results.
Z score compares the standard deviations of the reading with matched aged persons.
The normal range is +2 to - 2 Standard deviations and those are what we call Z scores.
A Z score of 0 is the population mean.
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baseline scans prior to drug administration
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Participant Will Achieve Improving Levels of Alkaline Phosphatase (ALP)
Time Frame: every 3 months, From baseline to 52 weeks
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obtain Alkaline phosphatase in blood every 3 months
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every 3 months, From baseline to 52 weeks
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Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: Hussein Abdul-Latif, MD, University of Alabama at Birmingham
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- IRB-300004900
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Epidermal Nevus Syndrome
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Albert ChiouNFlection Therapeutics, Inc.Not yet recruitingEpidermal Nevi | Nevus SebaceusUnited States
-
Kyowa Kirin, Inc.CompletedTumor Induced Osteomalacia (TIO) | Epidermal Nevus Syndrome (ENS)United States
-
Kyowa Kirin Co., Ltd.CompletedTumor-Induced Osteomalacia or Epidermal Nevus SyndromeJapan, Korea, Republic of
-
Laura TosiChildren's National Research Institute; Ultragenyx Pharmaceutical IncActive, not recruitingCutaneous Skeletal Hypophosphatemia Syndrome (CSHS) | Epidermal Nevus SyndromeUnited States
-
Massachusetts General HospitalNot yet recruitingCongenital Melanocytic Nevus
-
University of California, DavisRecruiting
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Peking Union Medical College HospitalCancer Institute and Hospital, Chinese Academy of Medical Sciences; Air Force...UnknownBlue Rubber Bleb Nevus Syndrome | Venous MalformationChina
-
Ascend Biopharmaceuticals LtdSuspendedBasal Cell Carcinoma in Basal Cell Nevus SyndromeUnited States
-
Russian Academy of Medical SciencesBlokhin's Russian Cancer Research CenterRecruitingMelanoma | Nevus | Nevus, Pigmented | Melanoma (Skin) | Mucosal Melanoma | Moles | Dysplastic Nevus Syndrome | Nevus, Blue | Nevus, Spitz | Nevi, Spindle Cell | Nevi, Dysplastic | Mucosal MelanosisRussian Federation
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Mahidol UniversityUnknownFocus on Laser Treatment of Nevus of Ota in Thai PatientsThailand
Clinical Trials on Crysvita (burosumab-twza) Treatment
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Ultragenyx Pharmaceutical IncKyowa Kirin Co., Ltd.AvailableX-linked Hypophosphatemia | Tumor-Induced Osteomalacia
-
Laura TosiChildren's National Research Institute; Ultragenyx Pharmaceutical IncActive, not recruitingCutaneous Skeletal Hypophosphatemia Syndrome (CSHS) | Epidermal Nevus SyndromeUnited States
-
Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaUnited States
-
Kyowa Kirin Pharmaceutical Development LtdCompletedX-linked HypophosphatemiaUnited Kingdom, France, Ireland, Italy
-
Kyowa Kirin Pharmaceutical Development LtdActive, not recruitingX-linked Hypophosphatemia (XLH)Sweden, Spain, United Kingdom, Italy, Austria, France
-
Kyowa Kirin Co., Ltd.Active, not recruitingTumor-Induced Osteomalacia (TIO)China
-
Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Korea, Republic of, Canada, Japan, France, Denmark
-
Yale UniversityCompleted
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Korea, Republic of, Japan, Ireland, United Kingdom, France, Italy
-
Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaNetherlands, United States, United Kingdom, France