A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia

X-linked Hypophosphatemia Clinical Trial

Official title:

A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01340482
• Other study ID #: KRN23-INT-001
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: April 2011
• Est. completion date: October 2013

Study information

• Verified date: June 2024
• Source: Kyowa Kirin Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 29
• Est. completion date: October 2013
• Est. primary completion date: May 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Familial Hypophosphatemic Rickets
• Hypophosphatemia
• X-linked Hypophosphatemia

Intervention

Drug:
• KRN23
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.

Locations

Country	Name	City	State
Canada	Shriners Hospital for Children - Canada	Montreal	Quebec
United States	Duke Clinical Research Unit	Durham	North Carolina
United States	University of Texas Health Science Center at Houston	Houston	Texas
United States	Clinical Research Center, Indiana University School of Medicine	Indianapolis	Indiana
United States	Yale University School of Medicine	New Haven	Connecticut
United States	University of California, San Francisco Medical Center	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Kyowa Kirin Co., Ltd.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Evaluation of Effect of Repeated SC Injections of KRN23 in Bone Substudy	Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density and bone quality.	On-Treatment: 6.5 months, 27 total visits
Primary	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	Evaluation of Effect of Repeated SC Injections of KRN23	Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters including serum phosphorus, sex hormone, bone biomarkers, quality of life assessments and population pharmacokinetics of KRN23 dose levels from cumulative dosing. Summary of serum phosphorus by visit/day is captured below.	On-Treatment: 6.5 months, 27 total visits

External Links

•   KNR23-INT-002: An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia