A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia
This study has been completed.
Sponsor:
Kyowa Hakko Kirin Pharma, Inc.
Collaborator:
Kyowa Hakko Kirin Company, Limited
Information provided by (Responsible Party):
Kyowa Hakko Kirin Pharma, Inc.
ClinicalTrials.gov Identifier:
NCT01340482
First received: April 15, 2011
Last updated: January 12, 2015
Last verified: January 2015
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
X-linked Hypophosphatemia |
Drug: Biological KRN23 |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia |
Resource links provided by NLM:
Further study details as provided by Kyowa Hakko Kirin Pharma, Inc.:
Primary Outcome Measures:
- Safety and Efficacy of repeated SC injections of KRN23 [ Time Frame: On-Treatment: 6.5 months, 27 total visits ] [ Designated as safety issue: Yes ]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.
Secondary Outcome Measures:
- Evaluation of effect of repeated SC injections of KRN23 [ Time Frame: On-Treatment: 6.5 months, 27 total visits ] [ Designated as safety issue: No ]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.
Other Outcome Measures:
- Evaluation of effect of repeated SC injections of KRN23 in Bone Substudy [ Time Frame: On-Treatment: 6.5 months, 27 total visits ] [ Designated as safety issue: No ]Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density and bone quality.
| Enrollment: | 29 |
| Study Start Date: | April 2011 |
| Study Completion Date: | October 2013 |
| Primary Completion Date: | May 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Open-Label KRN23; Single-blind KRN23 vs Placebo |
Drug: Biological 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.
|
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Main Inclusion Criteria:
- 18 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
- Additional inclusion criteria apply
Main Exclusion Criteria:
- 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
- Additional exclusion criteria apply
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01340482
Please refer to this study by its ClinicalTrials.gov identifier: NCT01340482
Locations
| United States, California | |
| University of California, San Francisco Medical Center | |
| San Francisco, California, United States, 94143 | |
| United States, Connecticut | |
| Yale University School of Medicine | |
| New Haven, Connecticut, United States, 06520 | |
| United States, Indiana | |
| Clinical Research Center, Indiana University School of Medicine | |
| Indianapolis, Indiana, United States, 46202-5250 | |
| United States, North Carolina | |
| Duke Clinical Research Unit | |
| Durham, North Carolina, United States, 27710 | |
| United States, Texas | |
| University of Texas Health Science Center at Houston | |
| Houston, Texas, United States, 77030 | |
| Canada, Quebec | |
| Shriners Hospital for Children - Canada | |
| Montreal, Quebec, Canada, H3G 1A6 | |
Sponsors and Collaborators
Kyowa Hakko Kirin Pharma, Inc.
Kyowa Hakko Kirin Company, Limited
Investigators
| Study Director: | Amy Zhang, PhD | Kyowa Hakko Kirin Pharma |
More Information
Additional Information:
No publications provided
| Responsible Party: | Kyowa Hakko Kirin Pharma, Inc. |
| ClinicalTrials.gov Identifier: | NCT01340482 History of Changes |
| Other Study ID Numbers: | KRN23-INT-001 |
| Study First Received: | April 15, 2011 |
| Last Updated: | January 12, 2015 |
| Health Authority: | United States: Food and Drug Administration Canada: Health Canada |
Keywords provided by Kyowa Hakko Kirin Pharma, Inc.:
|
XLH |
Additional relevant MeSH terms:
|
Familial Hypophosphatemic Rickets Hypophosphatemia Avitaminosis Bone Diseases Bone Diseases, Metabolic Calcium Metabolism Disorders Deficiency Diseases Genetic Diseases, Inborn Hypophosphatemia, Familial Kidney Diseases Malnutrition |
Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Musculoskeletal Diseases Nutrition Disorders Phosphorus Metabolism Disorders Renal Tubular Transport, Inborn Errors Rickets Rickets, Hypophosphatemic Urologic Diseases Vitamin D Deficiency |
ClinicalTrials.gov processed this record on February 27, 2015