X-linked HypophosphatemiaPhase 1NCT00830674
A Study of KRN23 in X-linked Hypophosphatemia
A Phase 1 study testing KRN23 in people with X-linked Hypophosphatemia, run by Kyowa Kirin Co., Ltd. at 4 sites in United States.
Trial summary
The primary objective of this study is to assess the safety and tolerability of KRN23 after a single intravenous (IV) and subcutaneous (SC) administration in XLH patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- X-linked Hypophosphatemia
- What is being tested?
- KRN23
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 42 participants
- Where
- 4 sitesUnited States
- Study dates
- Dec 2008to May 2013
- 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
- Clinical diagnosis of XLH
- TmP/GFR is less than 2.0 mg/dL
- GFR is 60 mL/min or above
Who cannot join
- Have any sign of active infectious disease or have had an infection requiring treatment with antibiotics within three weeks prior to screening
- History of known immunodeficiency
- Lactating females, female patients who are pregnant or planning to become pregnant during the study
- Use of a pharmacologic vitamin D metabolite or its analog, phosphate, calcimimetics, and ingestion of aluminum hydroxide antacids within 10 days prior to screening and dosing
- Use of any supplement contained phosphate, calcium and/or vitamin D within 10 days prior to screening and dosing
- Receipt of live (attenuated) vaccine within 3-months prior to screening
- Have any condition which, in the opinion of the Investigator, could present a concern for either patient safety or difficulty with data interpretation
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Duke Clinical Research Unit
Durham, North Carolina, 27710
University of Texas Health Science Center at Houston
Houston, Texas, 77030
General Clinical Research Center, Indiana University School of Medicine
Indianapolis, Indiana, 46202-5250
Yale University School of Medicine
New Haven, Connecticut, 06510
About this study
Official title: A Phase I, Double-blind, Randomized, Placebo-controlled, Single-dose, Dose-escalation Study of KRN23 in X-linked Hypophosphatemia
The primary objective of this study is to assess the safety and tolerability of KRN23 after a single intravenous (IV) and subcutaneous (SC) administration in XLH patients.
- Conditions
- X-linked Hypophosphatemia
- Being tested
- Placebo, KRN23
- Keywords
- XLH
- Medical subject headings
- Familial Hypophosphatemic Rickets
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
single dose IV or SC administration
KRN23Drug
Single IV administration on day 1: 0.003, 0.01, 0.03, 0.1 and 0.3 mg/kg and Single SC administration on day 1: 0.1, 0.3, 0.6 and 1.0 mg/kg
| Study group | Role | What they receive |
|---|---|---|
| KRN23 | Experimental | Single IV or SC administration on day 1 Receives: Drug: KRN23 |
| Placebo | Placebo comparator | Single IV or SC administration on day 1 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability
AEs, etc
Measured: Up to 7 weeks after dosing (maximally 11 weeks)
Secondary outcomes (1)
Effect to pharmacodynamic parameter
Change in Serum Phosphate
Measured: Up to 7 weeks after dosing (maximally 11 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2009 (estimated)
- Study startDec 2008
- Primary completionDec 2011
- Study completionMay 2013
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 HypophosphatemiaStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Kyowa Hakko Kirin Pharma, Inc.
- Responsible party
- Sponsor
- Investigators
- Thomas O. Carpenter, M.D., Principal investigator, Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00830674
- Sponsor's protocol ID
- KRN23-US-02
- Status last verified
- Jun 2024
- First submitted
- 27 Jan 2009
- Last updated
- 17 Jun 2024
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Carpenter TO, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Wooddell MM, Kawakami T, Ito T, Zhang X, Humphrey J, Insogna KL, Peacock M. Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia. J Clin Invest. 2014 Apr;124(4):1587-97. doi: 10.1172/JCI72829. Epub 2014 Feb 24.
PubMed 24569459
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Double-blind, Randomized, Placebo-controlled, Single-dose, Dose-escalation Study of KRN23 in X-linked Hypophosphatemia
- ClinicalTrials.gov ID
- NCT00830674
- Acronym / sponsor ID
- KRN23-US-02
- Conditions
- X-linked Hypophosphatemia
- Keywords
- XLH
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- Kyowa Kirin Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Kyowa Hakko Kirin Pharma, Inc.
- Study start
- Dec 2008
- Primary completion
- Dec 2011 (actual)
- Study completion
- May 2013 (actual)
- First posted
- 28 Jan 2009 (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 primary objective of this study is to assess the safety and tolerability of KRN23 after a single intravenous (IV) and subcutaneous (SC) administration in XLH patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| KRN23 | Experimental | Drug: KRN23Single IV or SC administration on day 1 |
| Placebo | Placebo comparator | Drug: PlaceboSingle IV or 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 (4 listed)
- 18 years or older
- Clinical diagnosis of XLH
- TmP/GFR is less than 2.0 mg/dL
- GFR is 60 mL/min or above
Key exclusion criteria (7 listed)
- Have any sign of active infectious disease or have had an infection requiring treatment with antibiotics within three weeks prior to screening
- History of known immunodeficiency
- Lactating females, female patients who are pregnant or planning to become pregnant during the study
- Use of a pharmacologic vitamin D metabolite or its analog, phosphate, calcimimetics, and ingestion of aluminum hydroxide antacids within 10 days prior to screening and dosing
- Use of any supplement contained phosphate, calcium and/or vitamin D within 10 days prior to screening and dosing
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 |
|---|---|---|
| Placebosingle dose IV or SC administration | Drug | Placebo |
| KRN23Single IV administration on day 1: 0.003, 0.01, 0.03, 0.1 and 0.3 mg/kg and Single SC administration on day 1: 0.1, 0.3, 0.6 and 1.0 mg/kg | Drug | KRN23 |
Primary outcome
Safety and tolerability
Time frame: Up to 7 weeks after dosing (maximally 11 weeks)
AEs, etc
1 secondary outcome
Effect to pharmacodynamic parameter
Time frame: Up to 7 weeks after dosing (maximally 11 weeks)
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 17 Jun 2024.
- Derived from this study: PubMed 24569459Carpenter TO, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Wooddell MM, Kawakami T, Ito T, Zhang X, Humphrey J, Insogna KL, Peacock M. Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia. J Clin Invest. 2014 Apr;124(4):1587-97. doi: 10.1172/JCI72829. Epub 2014 Feb 24.
- KRN23-INT-002 An Extension Study of KRN23 in Adults With X-Linked HypophosphatemiaLink provided by the sponsor.
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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.
Thomas O. Carpenter, M.D. principal investigator
Yale University
4 sites in 1 country: 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 Hypophosphatemia, run by Kyowa Kirin Co., Ltd. at 4 sites in United States.
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 4 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 17 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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. Some participants in this study may receive a placebo. 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?
Safety and tolerability (time frame: Up to 7 weeks after dosing (maximally 11 weeks)). 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 NCT00830674, 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 17 Jun 2024.