A Study of KRN23 in Subjects With X-linked Hypophosphatemic Rickets/Osteomalacia

A Phase 1, Multicenter, Open-label, Sequential Dose-escalation, Single-dose Study to Assess the Safety and Tolerability of KRN23 in Subjects With X-linked Hypophosphatemic Rickets/Osteomalacia.

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.

Study Overview

• Status: Completed
• Conditions: X-linked Hypophosphatemic Rickets/Osteomalacia
• Intervention / Treatment: Drug: KRN23

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 1

Contacts and Locations

Study Locations

• Japan
  • Tokyo, Japan
• Korea, Republic of
  • Seoul, Korea, Republic of

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. 18 years or older
2. Patients with XLH

Exclusion Criteria:

1. Have an active infection or chronic inflammatory disease
2. Have uncontrolled hypertension
3. Have uncontrolled diabetes mellitus
4. History of known immunodeficiency
5. Use of a pharmacologic vitamin D metabolite or its analogs within 21 days prior to screening and after screening
6. 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
7. Pregnant or lactating females, women who are possibly pregnant or patients who have no intention of utilizing adequate contraception

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: KRN23; Single SC administration on day 1	Drug: KRN23; Single SC administration on day 1: 0.3, 0.6 and 1.0 mg/kg

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
Profiles of pharmacokinetics	Pre-dose, 24, 48, 96, 168, 336, 504, 672, 840, 1176 hours post-dose

Collaborators and Investigators

• Sponsor: Kyowa Kirin Co., Ltd.

Study record dates

Study Major Dates

• Study Start (ACTUAL): July 1, 2014
• Primary Completion (ACTUAL): June 1, 2015
• Study Completion (ACTUAL): June 1, 2015

Study Registration Dates

• First Submitted: July 2, 2014
• First Submitted That Met QC Criteria: July 2, 2014
• First Posted (ESTIMATE): July 4, 2014

Study Record Updates

• Last Update Posted (ACTUAL): March 3, 2017
• Last Update Submitted That Met QC Criteria: February 28, 2017
• Last Verified: February 1, 2017

More Information

Terms related to this study

• Keywords: XLH
• Additional Relevant MeSH Terms: Metabolic Diseases; Kidney Diseases; Urologic Diseases; Nutrition Disorders; Genetic Diseases, Inborn; Musculoskeletal Diseases; Avitaminosis; Deficiency Diseases; Malnutrition; Bone Diseases; Metabolism, Inborn Errors; Bone Diseases, Metabolic; Renal Tubular Transport, Inborn Errors; Calcium Metabolism Disorders; Metal Metabolism, Inborn Errors; Phosphorus Metabolism Disorders; Vitamin D Deficiency; Hypophosphatemia, Familial; Hypophosphatemia; Rickets; Familial Hypophosphatemic Rickets; Rickets, Hypophosphatemic; Osteomalacia
• Other Study ID Numbers: KRN23-001