A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia
A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia
1 other identifier
interventional
29
2 countries
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). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Apr 2011
Typical duration for phase_1
6 active sites
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
April 1, 2011
CompletedFirst Submitted
Initial submission to the registry
April 15, 2011
CompletedFirst Posted
Study publicly available on registry
April 22, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2013
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2013
CompletedResults Posted
Study results publicly available
October 26, 2020
CompletedJune 18, 2024
June 1, 2024
2.1 years
April 15, 2011
September 30, 2020
June 14, 2024
Conditions
Keywords
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.
On-Treatment: 6.5 months, 27 total visits
Secondary Outcomes (1)
Evaluation of Effect of Repeated SC Injections of KRN23
On-Treatment: 6.5 months, 27 total visits
Other Outcomes (1)
Evaluation of Effect of Repeated SC Injections of KRN23 in Bone Substudy
On-Treatment: 6.5 months, 27 total visits
Study Arms (1)
KRN23
EXPERIMENTALEscalating doses of KRN23 (0.05, 0.10, 0.30 and 0.60 mg/kg) will be administered SC every 28 days (up to 4 doses)
Interventions
Subjects will receive escalating doses of KRN23 administered by SC injections every 28-days (up to 4 doses) based on a dosing algorithm and discretion of the investigator and sponsor.
Eligibility Criteria
You may qualify if:
- years or older
- Clinical diagnosis of XLH
- TmP/GFR \< 2.0 mg/dL
- GFR ≥60 mL/min
- Corrected Ca \<10.8 mg/dL
- 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:
- Have any sign of active infectious disease or have had an infection requiring treatment with antibiotics within 3-weeks of Screening
- History of known immunodeficiency
- Pregnant or lactating females subjects or female planning to be become pregnant during the study
- Receipt of a live (attenuated) vaccine (except for influenza vaccines) within 3-months of Screening, and/or during the study
- Use of pharmacologic vitamin D metabolite or its analog, phosphate, calcimimetics, and ingestion of aluminum hydroxide antacids within 10-days prior to Screening and/or dosing
- Use of any supplement containing phosphate, calcium and/or vitamin D within 10-days prior to Screening and/or dosing
- Condition which could present a concern for either the subject's safety or difficulty with data interpretation
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (6)
University of California, San Francisco Medical Center
San Francisco, California, 94143, United States
Yale University School of Medicine
New Haven, Connecticut, 06520, United States
Clinical Research Center, Indiana University School of Medicine
Indianapolis, Indiana, 46202-5250, United States
Duke Clinical Research Unit
Durham, North Carolina, 27710, United States
University of Texas Health Science Center at Houston
Houston, Texas, 77030, United States
Shriners Hospital for Children - Canada
Montreal, Quebec, H3G 1A6, Canada
Related Publications (2)
Zhang X, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey J, Glorieux FH, Portale AA, Insogna K, Carpenter TO, Peacock M. Pharmacokinetics and pharmacodynamics of a human monoclonal anti-FGF23 antibody (KRN23) in the first multiple ascending-dose trial treating adults with X-linked hypophosphatemia. J Clin Pharmacol. 2016 Feb;56(2):176-85. doi: 10.1002/jcph.570. Epub 2015 Aug 11.
PMID: 26073451DERIVEDImel 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: 25919461DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
Only 2 subjects were randomized in and completed the bone substudy. Subsequently, the Sponsor terminated the bone substudy due to slow accrual.
Results Point of Contact
- Title
- Kyowa Kirin Pharmaceutical Development
- Organization
- Kyowa Kirin Pharmaceutical Development
Study Officials
- STUDY DIRECTOR
Amy Zhang, PhD
Kyowa Hakko Kirin Pharma, Inc.
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
April 15, 2011
First Posted
April 22, 2011
Study Start
April 1, 2011
Primary Completion
May 1, 2013
Study Completion
October 1, 2013
Last Updated
June 18, 2024
Results First Posted
October 26, 2020
Record last verified: 2024-06