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NCT Number: NCT01340482

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

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.

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Key information

Conditions

X-linked Hypophosphatemia Avitaminosis Bone Diseases Bone Diseases, Metabolic Calcium Metabolism Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Deficiency Diseases Familial Hypophosphatemic Rickets Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hypophosphatemia Hypophosphatemia, Familial Kidney Diseases Male Urogenital Diseases Malnutrition Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Musculoskeletal Diseases Nutrition Disorders Nutritional and Metabolic Diseases Phosphorus Metabolism Disorders Renal Tubular Transport, Inborn Errors Rickets Rickets, Hypophosphatemic Urogenital Diseases Urologic Diseases Vitamin D Deficiency

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Shriners Hospital for Children - Canada, Montreal, Quebec, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

KRN23

Drug

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.

Primary outcomes

  1. 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 outcomes

  1. 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. Summary of serum phosphorus by visit/day is captured below.

Other outcomes

  1. 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.

Sponsors and collaborators

Lead sponsor

Kyowa Kirin Co., Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2011
Primary completion
2013
Study completion
2013
First posted
Apr 22, 2011
Registry last updated
Jun 18, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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