NCT02181764

Brief Summary

The objective of this study is to assess the safety and tolerability of KRN23 after a single subcutaneous (SC) administration in subjects with X-linked hypophosphatemic rickets/osteomalacia (XLH) in Japan or Korea.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jul 2014

Geographic Reach
2 countries

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2014

Completed
1 day until next milestone

First Submitted

Initial submission to the registry

July 2, 2014

Completed
2 days until next milestone

First Posted

Study publicly available on registry

July 4, 2014

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2015

Completed
Last Updated

March 3, 2017

Status Verified

February 1, 2017

Enrollment Period

11 months

First QC Date

July 2, 2014

Last Update Submit

February 28, 2017

Conditions

X-linked Hypophosphatemic Rickets/Osteomalacia

Keywords

XLH

Outcome Measures

Primary Outcomes (1)

  • Number and types of adverse events

    The safety of KRN23 assessed by number and types of adverse events, laboratory tests

    Up to 7 weeks after dosing

Secondary Outcomes (1)

  • Profiles of pharmacokinetics

    Pre-dose, 24, 48, 96, 168, 336, 504, 672, 840, 1176 hours post-dose

Study Arms (1)

KRN23

EXPERIMENTAL

Single SC administration on day 1

Drug: KRN23

Interventions

KRN23DRUG

Single SC administration on day 1: 0.3, 0.6 and 1.0 mg/kg

KRN23

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • years or older
  • Patients with XLH

You may not qualify if:

  • Have an active infection or chronic inflammatory disease
  • Have uncontrolled hypertension
  • Have uncontrolled diabetes mellitus
  • History of known immunodeficiency
  • Use of a pharmacologic vitamin D metabolite or its analogs within 21 days prior to screening and after screening
  • Use of phosphate, calcium preparation, calcimimetics, aluminum hydroxide antacids, thiazide diuretic, acetazolamide, or phosphate, calcium, and/or vitamin D-containing supplements within 10 days prior to screening and after screening
  • Pregnant or lactating females, women who are possibly pregnant or patients who have no intention of utilizing adequate contraception

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Unknown Facility

Tokyo, Japan

Location

Unknown Facility

Seoul, South Korea

Location

MeSH Terms

Conditions

Familial Hypophosphatemic RicketsOsteomalacia

Interventions

burosumab

Condition Hierarchy (Ancestors)

Rickets, HypophosphatemicRicketsBone Diseases, MetabolicBone DiseasesMusculoskeletal DiseasesHypophosphatemia, FamilialRenal Tubular Transport, Inborn ErrorsKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesMetal Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesCalcium Metabolism DisordersHypophosphatemiaPhosphorus Metabolism DisordersVitamin D DeficiencyAvitaminosisDeficiency DiseasesMalnutritionNutrition Disorders

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 2, 2014

First Posted

July 4, 2014

Study Start

July 1, 2014

Primary Completion

June 1, 2015

Study Completion

June 1, 2015

Last Updated

March 3, 2017

Record last verified: 2017-02

Locations

KR(1)JP(1)