A Study of KRN23 in Adult and Pediatric Patients With X-linked Hypophosphatemic Rickets/Osteomalacia (NCT04308096) | Clinical Trial Compass
CompletedPhase 3
A Study of KRN23 in Adult and Pediatric Patients With X-linked Hypophosphatemic Rickets/Osteomalacia
Japan, South Korea27 participantsStarted 2018-01-09
Plain-language summary
Before switching to the post-marketing study:
Assess the efficacy and safety of KRN23 administered subcutaneously once every 4 or 2 weeks in adult or children with XLH
After switching to the post-marketing study:
To evaluate the safety and efficacy of KRN23, which was switched from the investigational product to the post-marketing investigational product, at the approved dose and dosing regimen in subjects who continued treatment
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Personally submitted voluntary written informed consent to participate in the study; For pediatric patients, personally submitted voluntary written informed consent by a legally authorized representative.
. Patients meeting any of the followings;
. For adult XLH patients, completion the final observation at Week 96 in UX023-CL303 or UX023-CL304
. For pediatric patients, completion the final observation at Week 64 in UX023-CL301
. For female patients; women of childbearing potential (except for females who have not reached menarche, permanently sterilized, postmenopausal \[12 months with no menses without an alternative medical cause\] or anatomically not of childbearing potential) with negative pregnancy test at pre-treatment assessment of Week 0
. For female patient with childbearing potential, or male patients with reproductive capacity; willingness to use acceptable methods of contraception while participating in the study
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of subjects for each adverse events
Timeframe: up to week 140
2
Effect to Body temperature
Timeframe: up to week 140
3
Effect to Pulse rate
Timeframe: up to week 140
4
Effect to Respiratory rate
Timeframe: up to week 140
5
Effect to Systolic blood pressure in sitting position
Timeframe: up to week 140
6
Effect to Diastolic blood pressure in sitting position
. Willingness and ability to cooperatively complete all study procedures, adhere to the visit schedule and follow the investigator's instructions, as considered by investigator or subinvestigator
Exclusion criteria
. Use of oral phosphate for treating XLH, pharmacologic vitamin D metabolites or analogs, aluminum hydroxide antacids, systemic corticosteroids, acetazolamide, and thiazides within 7 days prior to scheduled initial administration of investigational drug
. Planned or recommended orthopedic surgery (implantation or removal), including staples, 8 plates or osteotomy, during the study period
. Blood or blood product transfusion within 60 days prior to scheduled initial administration of investigational drug
. Use of growth hormone therapy within 12 months prior to scheduled initial administration of investigational drug
. Use of medication to suppress the secretion of parathyroid hormone (e.g., cinacalcet) within 60 days prior to scheduled initial administration of investigational drug
. Use of any investigational product (except for investigational product of the preceding study) or investigational medical device within 4 months prior to scheduled initial administration of investigational drug, or requirement for any investigational agent prior to completion of all scheduled study assessments
. Use of a therapeutic monoclonal antibody other than KRN23 within 90 days prior to scheduled initial administration of investigational drug
. History of being positive for HIV antibody, HBs antigen and/or HCV antibody