X-Linked HypophosphatemiaPhase 2NCT02312687

Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)

A Phase 2 study testing KRN23 in people with X-Linked Hypophosphatemia, run by Kyowa Kirin, Inc. at 5 sites in United States.

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

The primary objectives of this study are to: Assess the long-term safety of KRN23 subcutaneous (SC) administration in adult subjects with XLH Assess the proportion of subjects achieving serum phosphorus levels in the normal range (2.5-4.5 mg/dL) with long-term administration of KRN23 Assess long-term pharmacodynamics… Read more about this study

Who is it for?: Any age
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: 20 participants
Where: 5 sitesUnited States
Study dates: 30 Jan 2015to 30 Nov 2018
Phase: Phase 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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Have participated in Kyowa Hakko Kirin Pharma, Inc.'s KRN23-INT-001 (NCT01340482) or KRN23-INT-002 (NCT01571596) studies (received at least 2 doses of KRN23)
Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min or eGFR of 45 to < 60 mL/min at Screening with confirmation that the renal insufficiency was not due to nephrocalcinosis.
Sexually active subjects must be willing to use an acceptable method of contraception (e.g., double barrier method) while participating in the study and for 30 days after receiving the last dose of KRN23.

Who cannot join

Subject experienced a safety-related event in the KRN23-INT-001 or KRN23-INT-002 study that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
Presence of nephrocalcinosis on renal ultrasound that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
Participation in an investigational drug or device trial within 30 days of enrollment (other than KRN23-INT-001 or KRN23-INT-002).
Use of a pharmacologic vitamin D metabolite or analog (e.g., calcitriol, doxercalciferol, and paricalcitol), phosphate, or aluminum hydroxide antacids (e.g., Maalox® and Mylanta®) within 21 days prior to Screening or during the study.
Use of medication to suppress parathyroid hormone (PTH) (e.g., Sensipar®, cinacalcet, calcimimetics) within 2 months prior to Screening.

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

United States5 sites

Duke University
Durham, North Carolina, 27710
Houston Methodist Reasearch Institute
Houston, Texas, 77030
Indiana University Hospital
Indianapolis, Indiana, 46202
Yale University School of Medicine
New Haven, Connecticut, 06510
University California San Francisco Hospital
San Francisco, California, 94143

Other trials in United States

About this study

Official title: A Phase 2b, Open-Label, Long-Term Extension Study to Evaluate the Safety and Pharmacodynamics of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)

The primary objectives of this study are to:

Assess the long-term safety of KRN23 subcutaneous (SC) administration in adult subjects with XLH
Assess the proportion of subjects achieving serum phosphorus levels in the normal range (2.5-4.5 mg/dL) with long-term administration of KRN23
Assess long-term pharmacodynamics (PD) of KRN23 as measured by changes in the following: serum intact parathyroid hormone (iPTH); serum and urinary phosphorus; ratio of renal tubular maximum phosphate reabsorption rate to glomerular filtration rate (TmP/GFR) and tubular reabsorption of phosphate (TRP); serum 1,25-dihydroxy vitamin D (1,25[OH]2D); serum fibroblast growth factor 23 (FGF23); bone biomarkers: serum alkaline phosphatase (ALP), bone-specific ALP (BALP), carboxy terminal crosslinked telopeptide of type I collagen (CTx), and procollagen type 1 N-terminal propeptide (P1NP)
Assess long-term immunogenicity of KRN23 as measured by presence of anti-KRN23 antibody (ADA)

Conditions: X-Linked Hypophosphatemia
Being tested: KRN23
Keywords: XLH, FGF23, KRN23
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.

KRN23Biological
Also called burosumab, Crysvita®
solution for SC injection

Study groups in NCT02312687
Study group	Role	What they receive
KRN23	Experimental	KRN23 subcutaneous (SC) injections every 4 weeks. Starting doses will be based on the subject's last dose in study KRN23-INT-001 (NCT02312687) or KRN23-INT-002 (NCT01571596). Doses may be titrated to achieve the target peak serum phosphorus range. Receives: Biological: KRN23

What is being measured

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

Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious AEs (SAEs), and AEs Leading to Discontinuation or Death
An AE is defined as any untoward medical occurrence, whether or not considered drug related. An SAE or serious suspected adverse reaction is an AE or suspected adverse reaction that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect; an important medical event. A TEAE is an AE that occurred on or after the first burosumab dose. AEs were graded as 1 (mild), 2 (moderate), 3 (severe), 4 (life-threatening), 5 (death). AEs were classified by the Investigator as possibly related, probably related, or definitely related.
Measured: Screening through the end of study plus 4-8 weeks. The mean duration of burosumab exposure was 165.6 weeks (range: 68-184 weeks).
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Clinically significant changes from baseline reported as adverse events are presented.
Measured: Through Week 184
Number of Participants With Clinically Significant Changes From Baseline in Laboratory Values, by Category
Clinically significant changes from baseline reported as adverse events are presented.
Measured: Through Week 184
Number of Participants With Clinically Significant Changes From Baseline in Physical Exams, by Category
Clinically significant changes from baseline reported as adverse events are presented.
Measured: Through Week 184
Number of Participants With Clinically Significant Changes From Baseline in Echocardiogram (ECHO) Tests
Clinically significant changes from baseline reported as adverse events are presented.
Measured: Through Week 184
Number of Participants With Clinically Significant Changes From Baseline in ECGs
Clinically significant changes from baseline reported as adverse events are presented.
Measured: Through Week 184
Number of Participants With Clinically Significant Changes From Baseline in Renal Ultrasound, by Category
Clinically significant changes from baseline reported as adverse events are presented.
Measured: Through Week 184
Number of Participants Positive for Anti-KRN23 Antibodies and Neutralizing Antibodies at Baseline and Anytime Post-Baseline
Measured: Through Week 184
Percentage of Participants Reaching Serum Phosphorus Normal Range at Baseline and Any Time After Dosing
Measured: Through Week 184
Change From Baseline Over Time in Serum Phosphorus
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Serum iPTH
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Serum Total FGF23
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Serum Free FGF23
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Serum 1,25(OH)2D
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Ovr Time in 2-hour Urine TmP/GFR
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in in 2-hour Urine TRP
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in FEP
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in 24-hour Urine Phosphorus
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in 24-Hour Urine Calcium
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in 24-Hour Urine Creatinine
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in 24-Hour Urine Calcium/Creatinine Ratio
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Total ALP
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in BALP
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in CTx
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in P1NP
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144

Timeline

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

30 Jan 201530 Nov 2018

First posted9 Dec 2014 (estimated)
Study start30 Jan 2015
Primary completion30 Nov 2018
Study completion30 Nov 2018

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 (1)Papers the sponsor has linked to this study.
Other trials for X-Linked HypophosphatemiaStudies of the same condition, open and under way.
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 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 20 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02312687
Sponsor's protocol ID: UX023-CL203
Status last verified: May 2024
First submitted: 3 Dec 2014
Last updated: 6 May 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Expanded access: Available

Publications

Weber TJ, Imel EA, Carpenter TO, Peacock M, Portale AA, Hetzer J, Merritt JL, Insogna K. Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia. J Clin Endocrinol Metab. 2022 Dec 17;108(1):155-165. doi: 10.1210/clinem/dgac518.
PubMed 36072994

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 6 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2b, Open-Label, Long-Term Extension Study to Evaluate the Safety and Pharmacodynamics of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)
ClinicalTrials.gov ID: NCT02312687
Acronym / sponsor ID: UX023-CL203
Conditions: X-Linked Hypophosphatemia
Keywords: XLH, FGF23, KRN23
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 20 participants (actual)
Lead sponsor: Kyowa Kirin, Inc.Industry (pharma, biotech, devices)
Collaborators: Kyowa Kirin Co., Ltd.
Study start: 30 Jan 2015 (actual)
Primary completion: 30 Nov 2018 (actual)
Study completion: 30 Nov 2018 (actual)
First posted: 9 Dec 2014 (estimated)
Results: Posted 16 Dec 2019

Clinical summary

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

The primary objectives of this study are to:

Assess the long-term safety of KRN23 subcutaneous (SC) administration in adult subjects with XLH
Assess the proportion of subjects achieving serum phosphorus levels in the normal range (2.5-4.5 mg/dL) with long-term administration of KRN23
Assess long-term pharmacodynamics (PD) of KRN23 as measured by changes in the following: serum intact parathyroid hormone (iPTH); serum and urinary phosphorus; ratio of renal tubular maximum phosphate reabsorption rate to glomerular filtration rate (TmP/GFR) and tubular reabsorption of phosphate (TRP); serum 1,25-dihydroxy vitamin D (1,25[OH]2D); serum fibroblast growth factor 23 (FGF23); bone biomarkers: serum alkaline phosphatase (ALP), bone-specific ALP (BALP), carboxy terminal crosslinked telopeptide of type I collagen (CTx), and procollagen type 1 N-terminal propeptide (P1NP)
Assess long-term immunogenicity of KRN23 as measured by presence of anti-KRN23 antibody (ADA)

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	Biological: KRN23KRN23 subcutaneous (SC) injections every 4 weeks. Starting doses will be based on the subject's last dose in study KRN23-INT-001 (NCT02312687) or KRN23-INT-002 (NCT01571596). Doses may be titrated to achieve the target peak serum phosphorus range.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

Have participated in Kyowa Hakko Kirin Pharma, Inc.'s KRN23-INT-001 (NCT01340482) or KRN23-INT-002 (NCT01571596) studies (received at least 2 doses of KRN23)
Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min or eGFR of 45 to < 60 mL/min at Screening with confirmation that the renal insufficiency was not due to nephrocalcinosis.
Sexually active subjects must be willing to use an acceptable method of contraception (e.g., double barrier method) while participating in the study and for 30 days after receiving the last dose of KRN23.

Key exclusion criteria (6 listed)

Subject experienced a safety-related event in the KRN23-INT-001 or KRN23-INT-002 study that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
Presence of nephrocalcinosis on renal ultrasound that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
Participation in an investigational drug or device trial within 30 days of enrollment (other than KRN23-INT-001 or KRN23-INT-002).
Use of a pharmacologic vitamin D metabolite or analog (e.g., calcitriol, doxercalciferol, and paricalcitol), phosphate, or aluminum hydroxide antacids (e.g., Maalox® and Mylanta®) within 21 days prior to Screening or during the study.

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
KRN23Also: burosumab, Crysvita®solution for SC injection	Biological	KRN23

Primary outcome

Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious AEs (SAEs), and AEs Leading to Discontinuation or Death
Time frame: Screening through the end of study plus 4-8 weeks. The mean duration of burosumab exposure was 165.6 weeks (range: 68-184 weeks).
An AE is defined as any untoward medical occurrence, whether or not considered drug related. An SAE or serious suspected adverse reaction is an AE or suspected adverse reaction that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect; an important medical event. A TEAE is an AE that occurred on or after the first burosumab dose. AEs were graded as 1 (mild), 2 (moderate), 3 (severe), 4 (life-threatening), 5 (death). AEs were classified by the Investigator as possibly related, probably related, or definitely related.
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Time frame: Through Week 184
Clinically significant changes from baseline reported as adverse events are presented.
Number of Participants With Clinically Significant Changes From Baseline in Laboratory Values, by Category
Time frame: Through Week 184
Clinically significant changes from baseline reported as adverse events are presented.
Number of Participants With Clinically Significant Changes From Baseline in Physical Exams, by Category
Time frame: Through Week 184
Clinically significant changes from baseline reported as adverse events are presented.
Number of Participants With Clinically Significant Changes From Baseline in Echocardiogram (ECHO) Tests
Time frame: Through Week 184
Clinically significant changes from baseline reported as adverse events are presented.
Number of Participants With Clinically Significant Changes From Baseline in ECGs
Time frame: Through Week 184
Clinically significant changes from baseline reported as adverse events are presented.
Number of Participants With Clinically Significant Changes From Baseline in Renal Ultrasound, by Category
Time frame: Through Week 184
Clinically significant changes from baseline reported as adverse events are presented.
Number of Participants Positive for Anti-KRN23 Antibodies and Neutralizing Antibodies at Baseline and Anytime Post-Baseline
Time frame: Through Week 184
Percentage of Participants Reaching Serum Phosphorus Normal Range at Baseline and Any Time After Dosing
Time frame: Through Week 184
Change From Baseline Over Time in Serum Phosphorus
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Serum iPTH
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Serum Total FGF23
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Serum Free FGF23
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Serum 1,25(OH)2D
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Ovr Time in 2-hour Urine TmP/GFR
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in in 2-hour Urine TRP
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in FEP
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in 24-hour Urine Phosphorus
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in 24-Hour Urine Calcium
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in 24-Hour Urine Creatinine
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in 24-Hour Urine Calcium/Creatinine Ratio
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in Total ALP
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in BALP
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in CTx
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144
Change From Baseline Over Time in P1NP
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, 144

Results and safety

Results posted to the registry on 16 Dec 2019, 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	20	19

Primary outcome: Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious AEs (SAEs), and AEs Leading to Discontinuation or Death

Time frame: Screening through the end of study plus 4-8 weeks. The mean duration of burosumab exposure was 165.6 weeks (range: 68-184 weeks).. Count of participants. Unit: Participants

Measure	KRN23 n = 20
All AEs	20
TEAEs	20
Related AEs	12
SAEs	9
Related SAEs	1
Grade 3 and 4 AEs	10
AEs Leading to Discontinuation	0
AEs Leading to Death	0

Population analysed: Safety Analysis Set

Primary outcome: Number of Participants With Clinically Significant Changes From Baseline in Vital Signs

Time frame: Through Week 184. Count of participants. Unit: Participants

Measure	KRN23 n = 20
Hypertension	2
Decreased blood pressure	1

Primary outcome: Number of Participants With Clinically Significant Changes From Baseline in Laboratory Values, by Category

Time frame: Through Week 184. Count of participants. Unit: Participants

Measure	KRN23 n = 20
Elevated eosinophils	1
Hypercholesterolemia	1
Hyperkalemia	1
Hyperglycemia	1
Elevated alanine aminotransferase	1
Elevated aspartate aminotransferase	1
Elevated gamma-glutamyl transferase	2
Proteinuria	2

Primary outcome: Number of Participants With Clinically Significant Changes From Baseline in Physical Exams, by Category

Time frame: Through Week 184. Count of participants. Unit: Participants

Measure	KRN23 n = 20
Bone pain	1
Conjunctival haemorrhage	1
Hypoacusis	1
Dermal cyst	1
Osteoarthritis	1
Gravitational oedema	1
Hypoaesthesia	1
Neuropathy peripheral	2

Adverse events

Time frame: Screening through the end of study plus 4-8 weeks. The mean duration of burosumab exposure was 165.6 weeks (range: 68-184 weeks).. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
KRN23	9 of 20 (45%)	20 of 20 (100%)	0 of 20

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 36072994Weber TJ, Imel EA, Carpenter TO, Peacock M, Portale AA, Hetzer J, Merritt JL, Insogna K. Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia. J Clin Endocrinol Metab. 2022 Dec 17;108(1):155-165. doi: 10.1210/clinem/dgac518.

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
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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.

Medical Director study director

Ultragenyx Pharmaceutical Inc

5 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing KRN23 in people with X-Linked Hypophosphatemia, run by Kyowa Kirin, Inc. at 5 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 3 inclusion and 6 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 May 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 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. 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 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 16 Dec 2019). 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?

Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious AEs (SAEs), and AEs Leading to Discontinuation or Death (time frame: Screening through the end of study plus 4-8 weeks. The mean duration of burosumab exposure was 165.6 weeks (range: 68-184 weeks).). The study lists 25 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 NCT02312687, 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 May 2024.