Overview

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.