Clinical trial NCT01340482: A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia
Official title: A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: X-linked Hypophosphatemia
Keywords: XLH
Lead sponsor: Kyowa Kirin Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2011-04-22
Enrollment: 29
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Main Inclusion Criteria:

1. 18 years or older
2. Clinical diagnosis of XLH
3. TmP/GFR \< 2.0 mg/dL
4. GFR ≥60 mL/min
5. Corrected Ca \<10.8 mg/dL
6. A willingness to utilize adequate contraception and not become pregnant\[or to have their partner(s) become pregnant\] during the study
7. Additional inclusion criteria apply

Main Exclusion Criteria:

1. Have any sign of active infectious disease or have had an infection requiring treatment with antibiotics within 3-weeks of Screening
2. History of known immunodeficiency
3. Pregnant or lactating females subjects or female planning to be become pregnant during the study
4. Receipt of a live (attenuated) vaccine (except for influenza vaccines) within 3-months of Screening, and/or during the study
5. 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
6. Use of any supplement containing phosphate, calcium and/or vitamin D within 10-days prior to Screening and/or dosing
7. Condition which could present a concern for either the subject's safety or difficulty with data interpretation
8. Additional exclusion criteria apply

Primary outcomes:
measure: Safety and Efficacy of Repeated SC Injections of KRN23
timeFrame: On-Treatment: 6.5 months, 27 total visits
Secondary outcomes:
measure: Evaluation of Effect of Repeated SC Injections of KRN23
timeFrame: On-Treatment: 6.5 months, 27 total visits
Other outcomes:
measure: Evaluation of Effect of Repeated SC Injections of KRN23 in Bone Substudy
timeFrame: On-Treatment: 6.5 months, 27 total visits
Locations:
city: San Francisco
country: United States
facility: University of California, San Francisco Medical Center
state: California
city: New Haven
country: United States
facility: Yale University School of Medicine
state: Connecticut
city: Indianapolis
country: United States
facility: Clinical Research Center, Indiana University School of Medicine
state: Indiana
city: Durham
country: United States
facility: Duke Clinical Research Unit
state: North Carolina
city: Houston
country: United States
facility: University of Texas Health Science Center at Houston
state: Texas
city: Montreal
country: Canada
facility: Shriners Hospital for Children - Canada
state: Quebec
Study officials:
affiliation: Kyowa Hakko Kirin Pharma, Inc.
name: Amy Zhang, PhD
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.