X-linked HypophosphatemiaPhase 1/2NCT01340482

A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia

A Phase 1/2 study testing KRN23 in people with X-linked Hypophosphatemia, run by Kyowa Kirin Co., Ltd. at 6 sites in 2 countries.

Completed

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

Official record

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Trial summary

The primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality. 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?: No, everyone receives an active treatment
How many people: 29 participants
Where: 6 sitesin 2 countries
Study dates: Apr 2011to Oct 2013
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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

Main Inclusion Criteria:

18 years or older
Clinical diagnosis of XLH
TmP/GFR < 2.0 mg/dL
GFR ≥60 mL/min
Corrected Ca <10.8 mg/dL
A willingness to utilize adequate contraception and not become pregnant[or to have their partner(s) become pregnant] during the study
Additional inclusion criteria apply

Main Exclusion Criteria:

Have any sign of active infectious disease or have had an infection requiring treatment with antibiotics within 3-weeks of Screening
History of known immunodeficiency
Pregnant or lactating females subjects or female planning to be become pregnant during the study
Receipt of a live (attenuated) vaccine (except for influenza vaccines) within 3-months of Screening, and/or during the study
Use of pharmacologic vitamin D metabolite or its analog, phosphate, calcimimetics, and ingestion of aluminum hydroxide antacids within 10-days prior to Screening and/or dosing
Use of any supplement containing phosphate, calcium and/or vitamin D within 10-days prior to Screening and/or dosing
Condition which could present a concern for either the subject's safety or difficulty with data interpretation
Additional exclusion criteria apply

Trial locations and contacts

6 sites in 2 countries.

Country

City, postcode or hospital

6 sites in 2 countries

United States5 sites

Duke Clinical Research Unit
Durham, North Carolina, 27710
University of Texas Health Science Center at Houston
Houston, Texas, 77030
Clinical Research Center, Indiana University School of Medicine
Indianapolis, Indiana, 46202-5250
Yale University School of Medicine
New Haven, Connecticut, 06520
University of California, San Francisco Medical Center
San Francisco, California, 94143

Other trials in United States

Canada1 site

Shriners Hospital for Children - Canada
Montreal, Quebec, H3G 1A6

Other trials in Canada

About this study

Official title: A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia

Conditions: X-linked Hypophosphatemia
Being tested: 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.

KRN23Drug
Subjects will receive escalating doses of KRN23 administered by SC injections every 28-days (up to 4 doses) based on a dosing algorithm and discretion of the investigator and sponsor.

Study groups in NCT01340482
Study group	Role	What they receive
KRN23	Experimental	Escalating doses of KRN23 (0.05, 0.10, 0.30 and 0.60 mg/kg) will be administered SC every 28 days (up to 4 doses) Receives: Drug: KRN23

What is being measured

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

Safety and Efficacy of Repeated SC Injections of KRN23
Safety and efficacy of repeated SC injections of KRN23 from baseline as assessed by serum phosphorus levels,immunogenicity, adverse events and clinically significant changes in vital signs and laboratory testing.
Measured: On-Treatment: 6.5 months, 27 total visits

Secondary outcomes (1)

Evaluation of Effect of Repeated SC Injections of KRN23
Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters including serum phosphorus, sex hormone, bone biomarkers, quality of life assessments and population pharmacokinetics of KRN23 dose levels from cumulative dosing. Summary of serum phosphorus by visit/day is captured below.
Measured: On-Treatment: 6.5 months, 27 total visits

Timeline

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

Apr 2011Oct 2013

First posted22 Apr 2011 (estimated)
Study startApr 2011
Primary completionMay 2013
Study completionOct 2013

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)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 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 29 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01340482
Sponsor's protocol ID: KRN23-INT-001
Status last verified: Jun 2024
First submitted: 15 Apr 2011
Last updated: 18 Jun 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Zhang X, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey J, Glorieux FH, Portale AA, Insogna K, Carpenter TO, Peacock M. Pharmacokinetics and pharmacodynamics of a human monoclonal anti-FGF23 antibody (KRN23) in the first multiple ascending-dose trial treating adults with X-linked hypophosphatemia. J Clin Pharmacol. 2016 Feb;56(2):176-85. doi: 10.1002/jcph.570. Epub 2015 Aug 11.
PubMed 26073451
Imel EA, Zhang X, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey JS, Glorieux FH, Portale AA, Insogna K, Peacock M, Carpenter TO. Prolonged Correction of Serum Phosphorus in Adults With X-Linked Hypophosphatemia Using Monthly Doses of KRN23. J Clin Endocrinol Metab. 2015 Jul;100(7):2565-73. doi: 10.1210/jc.2015-1551. Epub 2015 Apr 28.
PubMed 25919461

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1/2
A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
X-linked Hypophosphatemia
Active, not recruitingObservational study
X-linked Hypophosphatemia Disease Monitoring Program
X-linked Hypophosphatemia, Hypophosphatemic Rickets
Recruiting
Phosphorus-31 Spectroscopy in Phosphate Diabetes
Phosphate Diabetes, X-linked Hypophosphatemia
RecruitingObservational study
Registry for Patients With X-Linked Hypophosphatemia
X-Linked Hypophosphatemia
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome

All X-linked Hypophosphatemia trials

Study at a glance

From the registry record, last updated by the sponsor on 18 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase I/II, Open-Label, Repeat-Dose, Dose-Escalation Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia
ClinicalTrials.gov ID: NCT01340482
Acronym / sponsor ID: KRN23-INT-001
Conditions: X-linked Hypophosphatemia
Keywords: XLH
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 29 participants (actual)
Lead sponsor: Kyowa Kirin Co., Ltd.Industry (pharma, biotech, devices)
Study start: Apr 2011
Primary completion: May 2013 (actual)
Study completion: Oct 2013 (actual)
First posted: 22 Apr 2011 (estimated)
Results: Posted 26 Oct 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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: KRN23Escalating doses of KRN23 (0.05, 0.10, 0.30 and 0.60 mg/kg) will be administered SC every 28 days (up to 4 doses)

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

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
KRN23Subjects will receive escalating doses of KRN23 administered by SC injections every 28-days (up to 4 doses) based on a dosing algorithm and discretion of the investigator and sponsor.	Drug	KRN23

Primary outcome

Safety and Efficacy of Repeated SC Injections of KRN23
Time frame: On-Treatment: 6.5 months, 27 total visits
Safety and efficacy of repeated SC injections of KRN23 from baseline as assessed by serum phosphorus levels,immunogenicity, adverse events and clinically significant changes in vital signs and laboratory testing.

1 secondary outcome

Evaluation of Effect of Repeated SC Injections of KRN23
Time frame: On-Treatment: 6.5 months, 27 total visits

Results and safety

Results posted to the registry on 26 Oct 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
KRN23	27	25
Bone Substudy KRN23	1	1
Bone Substudy Placebo	1	1

Primary outcome: Safety and Efficacy of Repeated SC Injections of KRN23

Time frame: On-Treatment: 6.5 months, 27 total visits. Count of participants. Unit: Participants

Measure	KRN23 n = 27	Bone Substudy KRN23 n = 1	Bone Substudy Placebo n = 1
Subjects reporting at least one TEAE	24	1	1
Subjects who died	0	0	0
Subjects reporting SAE other than death	0	0	0
Subjects reporting at least one DLT	0	0	0
Subjects reporting AE leading to discontinuation	1	0	0

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Subjects were monitored for any untoward medical occurrences from the time of signed ICF through 30 days postdose.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
KRN23	0 of 27 (0%)	24 of 27 (88.9%)	0 of 27
Bone Substudy KRN23	0 of 1 (0%)	1 of 1 (100%)	0 of 1
Bone Substudy Placebo	0 of 1 (0%)	1 of 1 (100%)	0 of 1

Limitations and caveats (from the sponsor): Only 2 subjects were randomized in and completed the bone substudy. Subsequently, the Sponsor terminated the bone substudy due to slow accrual.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 26073451Zhang X, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey J, Glorieux FH, Portale AA, Insogna K, Carpenter TO, Peacock M. Pharmacokinetics and pharmacodynamics of a human monoclonal anti-FGF23 antibody (KRN23) in the first multiple ascending-dose trial treating adults with X-linked hypophosphatemia. J Clin Pharmacol. 2016 Feb;56(2):176-85. doi: 10.1002/jcph.570. Epub 2015 Aug 11.
Derived from this study: PubMed 25919461Imel EA, Zhang X, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey JS, Glorieux FH, Portale AA, Insogna K, Peacock M, Carpenter TO. Prolonged Correction of Serum Phosphorus in Adults With X-Linked Hypophosphatemia Using Monthly Doses of KRN23. J Clin Endocrinol Metab. 2015 Jul;100(7):2565-73. doi: 10.1210/jc.2015-1551. Epub 2015 Apr 28.
KNR23-INT-002: An Extension Study of KRN23 in Adults With X-Linked HypophosphatemiaLink provided by the sponsor.

Related trials for X-linked Hypophosphatemia

RecruitingPhase 1/2
A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
X-linked Hypophosphatemia
Active, not recruitingObservational study
X-linked Hypophosphatemia Disease Monitoring Program
X-linked Hypophosphatemia, Hypophosphatemic Rickets
Recruiting
Phosphorus-31 Spectroscopy in Phosphate Diabetes
Phosphate Diabetes, X-linked Hypophosphatemia
RecruitingObservational study
Registry for Patients With X-Linked Hypophosphatemia
X-Linked Hypophosphatemia
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome

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.

Amy Zhang, PhD study director

Kyowa Hakko Kirin Pharma, Inc.

6 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing KRN23 in people with X-linked Hypophosphatemia, run by Kyowa Kirin Co., Ltd. at 6 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?

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 18 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 6 sites in United States, Canada. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Oct 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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 Efficacy of Repeated SC Injections of KRN23 (time frame: On-Treatment: 6.5 months, 27 total visits). 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 NCT01340482, which also lists 2 linked publications.

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 18 Jun 2024.