A Study of KRN23 in Pediatric Patients With X-linked Hypophosphatemic Rickets/Osteomalacia

• Status: Completed
• Trial end date: 2020-02-17
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of the study is to assess the efficacy and safety of KRN23 administered subcutaneously once every 2 weeks in children with X-linked hypophosphatemic rickets/osteomalacia(XLH)

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Kyowa Hakko Kirin Co., Ltd; Kyowa Kirin Co., Ltd.

Criteria

Inclusion Criteria:

1. Personally submitted voluntary written informed consent by a legally authorized representative.If appropriate, written or verbal assent to participate in the study should be obtained from patients.

2. Aged ≥ 1 and ≤12 years

3. Patients who have open growth plate

4. Willing to perform a self-administration of KRN23 and available to perform a self-administration

5. Diagnosis of XLH, and meeting any of the followings;

1. phosphate-regulating gene with homologies to endopeptidases on the X chromosome(PHEX) mutation in either the patient or in a directly related family member with appropriate X-linked inheritance

2. Serum intact FGF23 level at screening ≥ 30 pg/mL

6. Finding evidence of rickets or clinical symptoms

7. Meeting all of following criteria for laboratory test related to XLH;

1. Serum P: < 3.0 mg/dL

2. Serum Cr: Within the age-adjusted normal limits

3. Serum 25(OH)D: ≥ 16 ng/mL

8. For female patients who have reached menarche with child bearing potential; a negative urine pregnancy test at screening

9. For female patients with child baring potential, or male patients with reproductive capacity; willingness to use an acceptable method of contraception while participating in the study

10. Willingness to provide access to prior medical records to determine eligibility including data on imaging tests, blood chemistry, diagnosis, medication, and surgical history

11. Willingness and ability to cooperatively complete all study procedures, adhere to the visit schedule and follow the investigator's instructions, as considered by the investigator or subinvestigator

Exclusion Criteria:

1. Height percentile > 50% based on age-adjusted Japanese norms at screening

2. Use of aluminum hydroxide antacids, systemic corticosteroids, acetazolamide, and thiazides within 7 days prior to screening

3. Current or prior use of leuprorelin, triptorelin, goserelin, or other drugs known to delay puberty

4. Use of growth hormone therapy within 12 months before screening

5. Use of medication to suppress parathyroid hormone within 60 days prior to screening

6. Serum calcium levels outside the age-adjusted normal limits

7. Intact parathyroid hormone(iPTH) levels ≥ 163 pg/mL

8. Presence of nephrocalcinosis on renal ultrasound grade 4 based on the following scale:

"0" = Normal "1" = Faint hyperechogenic rim around the medullary pyramids "2" = More intense echogenic rim with echoes faintly filling the entire pyramid "3" = Uniformly intense echoes throughout the pyramid "4" = Stone formation: solitary focus of echoes at the tip of the pyramid

9. Planned or recommended orthopedic surgery

10. Blood or blood product transfusion within 60 days prior to screening

11. History of malignancy within 5 years prior to registration

12. History of being positive for human immunodeficiency virus antibody, hepatitis B antigen and/or hepatitis C virus antibody

13. Predisposition to infection, or history of recurrent infection or known immunodeficiency

14. Use of any investigational product or investigational medical device within 4 months prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments

15. Receiving investigational agent in the UX023-CL301 study

16. Use of a therapeutic monoclonal antibody other than KRN23 within 90 days prior to screening

17. History of allergic or anaphylactic reactions to KRN23, any of the KRN23 ingredients, or any other monoclonal antibodies

18. Anyone otherwise considered unsuitable participation in the study by the investigator or subinvestigator