Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03581591
Recruitment Status : Active, not recruiting
First Posted : July 10, 2018
Last Update Posted : April 3, 2019
Sponsor:
Collaborator:
Ultragenyx Pharmaceutical Inc
Information provided by (Responsible Party):
Redwood Dermatology Sciences

Tracking Information
First Submitted Date  ICMJE October 27, 2017
First Posted Date  ICMJE July 10, 2018
Last Update Posted Date April 3, 2019
Actual Study Start Date  ICMJE January 31, 2018
Estimated Primary Completion Date June 30, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 1, 2019)
The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets [ Time Frame: 18 months ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 26, 2018)
The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets [ Time Frame: 1 year ]
Change History Complete list of historical versions of study NCT03581591 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets
Official Title  ICMJE An Open Label Trial to Assess the Safety and Efficacy of Burosumab (KRN23), an Investigational Antibody to FGF23, in a Single Pediatric Patient With Epidermal Nevus Syndrome(ENS) and Associated Hypophosphatemic Rickets
Brief Summary

A 52 week, 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

A 26 weeks extension to original study to monitor patient lab results for her safety.

Detailed Description

1.1 Primary Objective

The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets

1.2 Secondary Objectives

  1. The PD profile of KRN23 as assessed by changes from baseline over time 1,25(OH)2D, iPTH, Serum Calcium, TRP and TmP/GFR (the ratio of renal tubular maximum phosphate reabsorption rate to glomerular filtration rate)
  2. Changes in underlying skeletal disease/rickets as assessed by standard radiographs utilizing the Radiographic Global Impression of Change (RGI-C) rating scales
  3. Effects of KRN23 on biochemical markers of bone turnover that reflect rickets severity, alkaline phosphatase (ALP)
  4. Walking ability as assessed by 6-Minute Walk Test (6MWT)
  5. Patient/parent-Reported Outcomes as assessed by PROMIS and FPS-R rating scales

1.3 Exploratory Objective

1. Dual-energy X-ray absorptiometry (DXA)

1.4 Safety Objective Assess the safety of KRN23 administration in a single patient with ENS-associated hypophosphatemic rickets, based on adverse events (AEs), laboratory assessments, cardiac imaging and renal ultrasound.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Hypophosphatemia
  • Hypophosphatemic Rickets
  • Pain, Chronic
Intervention  ICMJE Biological: Burosumab
recombinant human IgG1 monoclonal antibody to fibroblast growth factor 23)
Study Arms  ICMJE Experimental: Primary; open label
Injection of Burosumab every two to three weeks based on Serum Phosphorus level of subject. Initial dose to be 0.3 mg/kg. Subsequent dosing will be titrated up or down depending on Serum Phosphorus level for that time period.
Intervention: Biological: Burosumab
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: June 26, 2018)
1
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 30, 2019
Estimated Primary Completion Date June 30, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Patient has confirmed ENS by physician diagnosis
  2. Patient has confirmed FGF23 elevations in the context of low serum phosphorous < 4.1 mg/dL
  3. Patient able to tolerate KRN23 treatment
  4. Have a corrected serum calcium level < 10.8mg/dL
  5. Have an eGFR >60 ml/min
  6. Must be willing in the opinion of the investigator, to comply with study procedures and schedule
  7. Provide written informed consent by a parent after the study has been explained and prior to any research related procedures begin

Exclusion Criteria:

  1. Concomitant use of active vitamin D (i.e calcitriol) and/or exogenous phosphate supplementation. Patient will be allowed OTC Vitamin D should levels drop below <20 ng/ml
  2. The use or enrollment in studies using other investigational therapies including other monoclonal antibodies
  3. Subject and their parent not willing or not able to give written informed consent
  4. In the Investigators opinion, the patient may not be able to meet all the requirements for study participation
  5. Patient has a history of hypersensitivity to KRN23 excipients that in the opinion of the investigator, places the patient at an increased risk of adverse effects
  6. Patient has a condition that in the opinion of the investigator could present a concern for subject safety or data interpretation.
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE up to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03581591
Other Study ID Numbers  ICMJE ENSKRN23.1
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Redwood Dermatology Sciences
Study Sponsor  ICMJE Redwood Dermatology Sciences
Collaborators  ICMJE Ultragenyx Pharmaceutical Inc
Investigators  ICMJE Not Provided
PRS Account Redwood Dermatology Sciences
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP