AzTrials
AzTrials

XLHPhase 3NCT04308096

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

A Phase 3 study testing KRN23 in people with XLH, run by Kyowa Kirin Co., Ltd. at 10 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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… Read more about this study

Who is it for?
Any age
Conditions the study is for
XLH
What is being tested?
KRN23
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
27 participants
Where
10 sitesin 2 countries
Study dates
9 Jan 2018to 4 Dec 2020
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.
  • If appropriate, written or verbal assent to participate in the study should be obtained from patients.
  • 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
  • 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

Who cannot join

  • 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
Show all 13Show fewer
  • 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
  • Anyone otherwise considered unsuitable for the study by the investigator or subinvestigator
  • At the time of switching to the post-marketing clinical study:
  • Subjects eligible for enrollment in the post-marketing clinical study must have met both of the following criteria:
  • Personally submitted voluntary written informed consent to participate in the postmarketing clinical study. For pediatric patients, personally submitted voluntary written informed consent by a legally authorized representative. If appropriate, written or verbal assent to participate in the post-marketing clinical study was to be obtained from subjects.
  • Switching to the post-marketing clinical study was necessary and appropriate for the subject from the viewpoint of efficacy and safety, as judged by the investigator or subinvestigator

Trial locations and contacts

10 sites in 2 countries.

10 sites in 2 countries

Japan8 sites

  • The University of Tokyo Hospital

    Bunkyō-Ku, Tokyo

  • Toranomon Hospital

    Minato-Ku, Tokyo

  • Okayama Saiseikai General Hospital

    Okayama

  • Japan Community Health Care Organization Osaka Hospital

    Osaka

  • Osaka City University Hospital

    Osaka

  • Hokkaido University Hospital

    Sapporo, Hokkaido

  • National University Corporation Osaka University

    Suita, Osaka

  • Kanagawa Prefectural Hospital Organization Kanagawa Children's Medical Center

    Yokohama, Kanagawa

Other trials in Japan

South Korea2 sites

  • Asan Medical Center

    Seoul, Korea

  • Seoul National University hospital

    Seoul, Korea

Other trials in South Korea

About this study

Official title: A Phase 3 Long-term Extension Study of KRN23 in Patients With X-linked Hypophosphatemic Rickets/Osteomalacia and a Post-marketing Study of KRN23 Switched From the Phase 3 Long-term Extension Study

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

Conditions
XLH
Being tested
KRN23

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • KRN23Drug

    The starting dose of KRN23 will be the dose used for the last administration in the preceding studies. The dose may be modified subsequently in accordance with the criteria for dose and dose adjustment.

Study groups in NCT04308096
Study groupRoleWhat they receive
KRN23ExperimentalSubjects will receive subcutaneous injections of KRN23 every 4 weeks (adult) or 2 weeks (pediatric) from Week 0 through Week 140.
Receives: Drug: KRN23

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of subjects for each adverse events

    Measured: up to week 140

  2. Effect to Body temperature

    Measured: up to week 140

  3. Effect to Pulse rate

    Measured: up to week 140

  4. Effect to Respiratory rate

    Measured: up to week 140

  5. Effect to Systolic blood pressure in sitting position

    Measured: up to week 140

  6. Effect to Diastolic blood pressure in sitting position

    Measured: up to week 140

  7. Effect to 12-lead electrocardiogram (ECG)

    The presence of abnormality in the electrocardiogram

    Measured: up to week 140

  8. Effect to renal ultrasound

    The evaluation to nephrocalcinosis in five grades by renal ultrasound

    Measured: up to week 140

  9. Effect to Echocardiogram

    The presence of ectopic calcification in the heart by Echocardiogram

    Measured: up to week 140

Secondary outcomes (14)

  1. Concentration of serum phosphorus

    Measured: up to week 140

  2. Concentration of serum 1,25(OH)2D

    Measured: up to week 140

  3. Concentration of urinary phosphorus

    Measured: up to week 140

  4. Concentration of tubular resorption of phosphorus（TRP）

    Measured: up to week 140

  5. Concentration of maximum tubular reabsorption of phosphate/glomerular filtration rate (TmP/GFR)

    Measured: up to week 140

  6. concentration of Carboxy terminal cross-linked telopeptide of type 1 collagen (CTx) (Adult patients with XLH)

    Measured: up to week 140

  7. concentration of Procollagen type 1 N-propeptide (P1NP) (Adult patients with XLH)

    Measured: up to week 140

  8. concentration of Bone-specific alkaline phosphatase (BALP)(Adult patients with XLH)

    Measured: up to week 140

  9. Concentration of serum alkaline phosphatase (ALP) (Pediatric patients with XLH)

    Measured: up to week 140

  10. Motor functions (6 minutes walk test (6MWT))

    Measured: up to week 140

  11. Radiographic findings of fracture and enthesopathy (Adult patients with XLH)

    The presence of radiographic fracture and enthesopathy assessed by X-ray (Adult patients with XLH)

    Measured: up to week 140

  12. Rickets Severity Score (RSS) (Pediatric patients with XLH)

    Measured: up to week 140

  13. Radiographic Global Impression of Change (RGI-C)(Pediatric patients with XLH)

    Measured: up to week 140

  14. Z score of height (LMS method) (Pediatric patients with XLH)

    Measured: up to week 140

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Mar 2020
  2. Study start9 Jan 2018
  3. Primary completion4 Dec 2020
  4. Study completion4 Dec 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
27 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04308096
Sponsor's protocol ID
KRN23-004
Status last verified
Sep 2022
First submitted
26 Feb 2020
Last updated
6 Sep 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Kubota T, Namba N, Tanaka H, Muroya K, Imanishi Y, Takeuchi Y, Kanematsu M, Sun W, Seino Y, Ozono K. Self-Administration of Burosumab in Children and Adults with X-Linked Hypophosphataemia in Two Open-Label, Single-Arm Clinical Studies. Adv Ther. 2023 Apr;40(4):1530-1545. doi: 10.1007/s12325-022-02412-x. Epub 2023 Jan 31.

    PubMed 36719566

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing KRN23 in people with XLH, run by Kyowa Kirin Co., Ltd. at 10 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 6 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in Japan, South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of subjects for each adverse events (time frame: up to week 140). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04308096, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Sep 2022.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in