An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia

NCT01571596 | Completed Phase 1 Results

• 1 other identifier: KRN23-INT-002
• Study Type: interventional
• Target: 23
• Locations: 2 countries
• Sites: 6

Brief Summary

The primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH).

Conditions

X-linked Hypophosphatemia

Keywords

XLH

Outcome Measures

Primary Outcomes (1)

• Safety and Efficacy of Repeated SC Injections of KRN23.

  Safety and efficacy of repeated SC injections of KRN23, from Baseline, as assessed by serum phosphorus levels, immunogenicity, adverse events and clinically significant changes in vital signs and laboratory testing.

  13.5 months,(50 visits)

Secondary Outcomes (1)

• Evaluation of Effect of Repeated SC Injections of KRN23

  13.5 months, (50 visits)

Other Outcomes (1)

• Evaluation of Effect of Repeated SC Injections of KRN23 in Bone Substudy

  13.5 months,(50 visits)

Study Arms (1)

KRN23

EXPERIMENTAL

Escalating doses of KRN23 (0.05, 0.10, 0.30, and 0.60 mg/kg) will be administered SC every 28 days (up to 12 doses)

Drug: KRN23

Interventions

KRN23 DRUG

Subjects will receive escalating doses of KRN23 administered by SC injection every 28-days (up to 12 doses) based on a dosing algorithm and discretion of Investigator and Sponsor.

KRN23

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Satisfactory completion of KKP's sponsored KRN23-INT-001 clinical trial
• eGFR ≥ 60 mL/min
• Corrected Ca \< 10.8 mg/dL
• For female of child-bearing potential, a negative serum pregnancy test
• A willingness to utilize adequate contraception and not become pregnant \[or to have their partner(s) become pregnant\] during the study

You may not qualify if:

• Subject experienced a safety-related event in the KRN23-INT-001 study
• Pregnant or lactating female subject or pregnant or female planning to become pregnant during the study
• Receipt of a live (attenuated) vaccine (except for influenza vaccines) during the course of the KRN23-INT-001 study and/or of this study
• Condition which could present a concern for either the subject's safety or difficulty with data interpretation

Sponsors & Collaborators

• Kyowa Kirin Co., Ltd.: lead
• Kyowa Hakko Kirin Pharma, Inc.: collaborator

Study Sites (6)

University of California San Francisco

San Francisco, California, 94143, United States

Location

Yale University School of Medicine

New Haven, Connecticut, 06520, United States

Location

Clinical Research Center, Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

Duke Clinical Research Unit

Durham, North Carolina, 27710, United States

Location

University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

Shriners Hospital for Children - Canada

Montreal, Quebec, H3G 1A6, Canada

Location

Related Publications (1)

• Imel EA, Zhang X, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey JS, Glorieux FH, Portale AA, Insogna K, Peacock M, Carpenter TO. Prolonged Correction of Serum Phosphorus in Adults With X-Linked Hypophosphatemia Using Monthly Doses of KRN23. J Clin Endocrinol Metab. 2015 Jul;100(7):2565-73. doi: 10.1210/jc.2015-1551. Epub 2015 Apr 28.

  PMID: 25919461 DERIVED

Related Links

• Related Info
• Related Info

MeSH Terms

Conditions

Familial Hypophosphatemic Rickets

Interventions

burosumab

Condition Hierarchy (Ancestors)

Rickets, Hypophosphatemic; Rickets; Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Hypophosphatemia, Familial; Renal Tubular Transport, Inborn Errors; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Calcium Metabolism Disorders; Hypophosphatemia; Phosphorus Metabolism Disorders; Vitamin D Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders

Limitations and Caveats

Due to the lack of enrollment, the bone substudy was terminated early.

Results Point of Contact

• Title: Kyowa Kirin Pharmaceutical Development
• Organization: Kyowa Kirin Pharmaceutical Development

kkd.clintrial.82@kyowakirin.com

609-919-1100

Study Officials

• Amy Zhang, PhD

  Kyowa Hakko Kirin Pharma, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

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: February 23, 2012
• First Posted: April 5, 2012
• Study Start: February 1, 2012
• Primary Completion: September 1, 2013
• Study Completion: June 1, 2014
• Last Updated: June 18, 2024
• Results First Posted: February 17, 2021

Record last verified: 2024-06

Locations

CA (1); US (5)