Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH

• Status: Active, not recruiting
• Trial end date: 2022-01-31
• Target enrollment: 0
• Participant gender: All

Summary

This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab (UX023-CL303 / UX023-CL304).

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

Details

• Lead Sponsor: Kyowa Kirin Pharmaceutical Development Ltd
• Treatments: Antibodies; Antibodies, Monoclonal

Criteria

Inclusion Criteria:

1. Subjects who provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures.

2. Subjects who participated in Study UX023-CL303 or UX023-CL304. Any subjects that did not complete Study UX023-CL303 or UX023-CL304 may be included on a case-by-case basis. Subjects' enrolment is not dependent on any response to Primary or Secondary endpoints in studies UX023-CL303 or UX023-CL304.

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

4. 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.

5. Females of child-bearing potential must have a negative urine pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not to be of child-bearing potential include those who have been in menopause for at least two years prior to Screening, or have had tubal ligation at least one year prior to Screening, or have had a total hysterectomy or bilateral salpingo-oophorectomy. If sexually active, male and female subjects must be willing to use one highly effective method of contraception for the duration of the study.

Exclusion Criteria:

1. Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age-adjusted normal limits and deemed as clinically significant in the opinion of the investigator.

2. Presence of a concurrent disease or condition that would interfere with study participation or affect safety in the opinion of the investigator or Sponsor.

3. Use of any investigational product other than burosumab or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.

4. Subjects with major protocol deviations in Study UX023-CL303 or UX023-CL304 which in the view of the investigator places the subject at high risk of poor treatment compliance or of not completing the study.

5. Subjects who discontinued treatment from Study UX023-CL303 or UX023-CL304 due to either a grade ≥3 treatment-related hypersensitivity reaction or a burosumab-related hypersensitivity reaction reported as a SAE.