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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 Co., Ltd
Information provided by (Responsible Party):
Kyowa Kirin Pharmaceutical Development, Inc. ( Kyowa Hakko Kirin Pharma, Inc. )
ClinicalTrials.gov Identifier:
NCT01340482
First received: April 15, 2011
Last updated: June 10, 2016
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: KRN23 | Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | 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:
Genetics Home Reference related topics:
hereditary hypophosphatemic rickets
Genetic and Rare Diseases Information Center resources:
X-linked Hypophosphatemia
Hypophosphatemic Rickets
Rickets
U.S. FDA Resources
Further study details as provided by Kyowa Kirin Pharmaceutical Development, Inc. ( 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 ]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 ]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 ]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: KRN23
Escalating 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)
|
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.
|
Eligibility| Ages Eligible for Study: | 18 Years and older (Adult, Senior) |
| Sexes Eligible for Study: | All |
| 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
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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 Co., Ltd
Investigators
| Study Director: | Amy Zhang, PhD | Kyowa Hakko Kirin Pharma, Inc. |
More Information
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| 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: | June 10, 2016 |
Keywords provided by Kyowa Kirin Pharmaceutical Development, Inc. ( Kyowa Hakko Kirin Pharma, Inc. ):
|
XLH |
Additional relevant MeSH terms:
|
Hypophosphatemia Familial Hypophosphatemic Rickets Phosphorus Metabolism Disorders Metabolic Diseases Rickets, Hypophosphatemic Rickets Bone Diseases, Metabolic Bone Diseases Musculoskeletal Diseases Hypophosphatemia, Familial Renal Tubular Transport, Inborn Errors |
Kidney Diseases Urologic Diseases Metal Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Calcium Metabolism Disorders Vitamin D Deficiency Avitaminosis Deficiency Diseases Malnutrition Nutrition Disorders |
ClinicalTrials.gov processed this record on June 23, 2017