Study of the Safety, Pharmacodynamics and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)

• Status: Active, not recruiting
• Trial end date: 0000-00-00
• Target enrollment: 13
• Participant gender: Both

Summary

UX023-CL205 is a multicenter, open-label, Phase 2 study in children from 1 to 4 years old with XLH to assess the safety, PD, and efficacy of KRN23 administered via subcutaneous (SC) injections every 2 weeks (Q2W) for a total of 64 weeks.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ultragenyx Pharmaceutical Inc
• Last Updated: 2016-08-30

Criteria

Inclusion Criteria:

1. Male or female, aged ≥1 year and <5 years

2. Diagnosis of XLH supported by ONE or more of the following

• Confirmed PHEX mutation in the patient or a directly related family member with appropriate X-linked inheritance

• Serum FGF23 level > 30 pg/mL by Kainos assay

3. Biochemical findings associated with XLH including:

• Serum phosphorus < 3.0 mg/dL (0.97 mmol/L)

• Serum creatinine within age-adjusted normal range

4. Radiographic evidence of rickets

5. Willing to provide access to prior medical records for the collection of historical growth, biochemical, and radiographic data and disease history

6. Provide written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures

7. Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, adhere to the study visit schedule, and comply with the assessments

Exclusion Criteria:

1. Unwilling to stop treatment with oral phosphate and/or pharmacologic vitamin D metabolite or analog (e.g. calcitriol, alfacalcidol) during the screening period and for the duration of the study

2. Planned or recommended orthopedic surgery including staples, 8-plates or osteotomy, within the clinical trial period

3. Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age-adjusted normal limits

4. Presence or history of any condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study

5. Presence of a concurrent disease or condition that would interfere with study participation or affect safety

6. History of recurrent infection or predisposition to infection, or of known immunodeficiency

7. Use of any investigational product or investigational medical device within 30 days prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments