X-linked Hypophosphatemic Rickets/OsteomalaciaPhase 1NCT02181764
A Study of KRN23 in Subjects With X-linked Hypophosphatemic Rickets/Osteomalacia
A Phase 1 study testing KRN23 in people with X-linked Hypophosphatemic Rickets/Osteomalacia, run by Kyowa Kirin Co., Ltd. at 2 sites in 2 countries.
Trial summary
The objective of this study is to assess the safety and tolerability of KRN23 after a single subcutaneous (SC) administration in subjects with X-linked hypophosphatemic rickets/osteomalacia (XLH) in Japan or Korea. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- X-linked Hypophosphatemic Rickets/Osteomalacia
- What is being tested?
- KRN23
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Jul 2014to Jun 2015
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18 years or older
- Patients with XLH
Who cannot join
- Have an active infection or chronic inflammatory disease
- Have uncontrolled hypertension
- Have uncontrolled diabetes mellitus
- History of known immunodeficiency
- Use of a pharmacologic vitamin D metabolite or its analogs within 21 days prior to screening and after screening
- Use of phosphate, calcium preparation, calcimimetics, aluminum hydroxide antacids, thiazide diuretic, acetazolamide, or phosphate, calcium, and/or vitamin D-containing supplements within 10 days prior to screening and after screening
- Pregnant or lactating females, women who are possibly pregnant or patients who have no intention of utilizing adequate contraception
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Phase 1, Multicenter, Open-label, Sequential Dose-escalation, Single-dose Study to Assess the Safety and Tolerability of KRN23 in Subjects With X-linked Hypophosphatemic Rickets/Osteomalacia.
The objective of this study is to assess the safety and tolerability of KRN23 after a single subcutaneous (SC) administration in subjects with X-linked hypophosphatemic rickets/osteomalacia (XLH) in Japan or Korea.
- Being tested
- KRN23
- Keywords
- XLH
- Medical subject headings
- Familial Hypophosphatemic Rickets, Osteomalacia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KRN23Drug
Single SC administration on day 1: 0.3, 0.6 and 1.0 mg/kg
| Study group | Role | What they receive |
|---|---|---|
| KRN23 | Experimental | Single SC administration on day 1 Receives: Drug: KRN23 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and types of adverse events
The safety of KRN23 assessed by number and types of adverse events, laboratory tests
Measured: Up to 7 weeks after dosing
Secondary outcomes (1)
Profiles of pharmacokinetics
Measured: Pre-dose, 24, 48, 96, 168, 336, 504, 672, 840, 1176 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jul 2014 (estimated)
- Study startJul 2014
- Primary completionJun 2015
- Study completionJun 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for X-linked Hypophosphatemic Rickets/OsteomalaciaStudies of the same condition, open and under way.
- More trials run by Kyowa Kirin Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02181764
- Sponsor's protocol ID
- KRN23-001
- Status last verified
- Feb 2017
- First submitted
- 2 Jul 2014
- Last updated
- 3 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Portale AA, Ward L, Dahir K, Florenzano P, Ing SW, Jan de Beur SM, Martin RM, Meza-Martinez AI, Paloian N, Ashraf A, Dixon BP, Khan A, Langman C, Chen A, Wang C, Roberts MS, Tandon PK, Bedrosian C, Imel EA. Nephrocalcinosis and kidney function in children and adults with X-linked hypophosphatemia: baseline results from a large longitudinal study. J Bone Miner Res. 2024 Sep 26;39(10):1493-1502. doi: 10.1093/jbmr/zjae127.
PubMed 39151033
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Multicenter, Open-label, Sequential Dose-escalation, Single-dose Study to Assess the Safety and Tolerability of KRN23 in Subjects With X-linked Hypophosphatemic Rickets/Osteomalacia.
- ClinicalTrials.gov ID
- NCT02181764
- Acronym / sponsor ID
- KRN23-001
- Keywords
- XLH
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Kyowa Kirin Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Jul 2014 (actual)
- Primary completion
- Jun 2015 (actual)
- Study completion
- Jun 2015 (actual)
- First posted
- 4 Jul 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to assess the safety and tolerability of KRN23 after a single subcutaneous (SC) administration in subjects with X-linked hypophosphatemic rickets/osteomalacia (XLH) in Japan or Korea.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| KRN23 | Experimental | Drug: KRN23Single SC administration on day 1 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 18 years or older
- Patients with XLH
Key exclusion criteria (7 listed)
- Have an active infection or chronic inflammatory disease
- Have uncontrolled hypertension
- Have uncontrolled diabetes mellitus
- History of known immunodeficiency
- Use of a pharmacologic vitamin D metabolite or its analogs within 21 days prior to screening and after screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KRN23Single SC administration on day 1: 0.3, 0.6 and 1.0 mg/kg | Drug | KRN23 |
Primary outcome
Number and types of adverse events
Time frame: Up to 7 weeks after dosing
The safety of KRN23 assessed by number and types of adverse events, laboratory tests
1 secondary outcome
Profiles of pharmacokinetics
Time frame: Pre-dose, 24, 48, 96, 168, 336, 504, 672, 840, 1176 hours post-dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2017.
- Derived from this study: PubMed 39151033Portale AA, Ward L, Dahir K, Florenzano P, Ing SW, Jan de Beur SM, Martin RM, Meza-Martinez AI, Paloian N, Ashraf A, Dixon BP, Khan A, Langman C, Chen A, Wang C, Roberts MS, Tandon PK, Bedrosian C, Imel EA. Nephrocalcinosis and kidney function in children and adults with X-linked hypophosphatemia: baseline results from a large longitudinal study. J Bone Miner Res. 2024 Sep 26;39(10):1493-1502. doi: 10.1093/jbmr/zjae127.
Related trials for X-linked Hypophosphatemic Rickets/Osteomalacia
Active, not recruitingObservational study
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X-Linked Hypophosphatemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing KRN23 in people with X-linked Hypophosphatemic Rickets/Osteomalacia, run by Kyowa Kirin Co., Ltd. at 2 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 2 inclusion and 7 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 3 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 and types of adverse events (time frame: Up to 7 weeks after dosing). 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 NCT02181764, 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 3 Mar 2017.