X-linked HypophosphatemiaPhase 3NCT02537431
Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)
A Phase 3 study testing burosumab in people with X-linked Hypophosphatemia, run by Kyowa Kirin, Inc. at 14 sites in 6 countries.
Trial summary
The primary objective of this study is to establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- X-linked Hypophosphatemia
- What is being tested?
- burosumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 14 sitesin 6 countries
- Study dates
- 23 Dec 2015to 13 Dec 2018
- Phase
- Phase 3
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female, aged 18 - 65 years, inclusive
- Diagnosis of XLH supported by classic clinical features of adult XLH (such as short stature or bowed legs), and at least one of the following at Screening:
- Documented phosphate regulating gene with homology to endopeptidases located on the X chromosome (PHEX) PHEX mutation in either the patient or in a directly related family member with appropriate X-linked inheritance
- Serum intact FGF23 (iFGF23) level > 30 pg/mL by Kainos assay
- Biochemical findings consistent with XLH based on overnight fasting (min. 8 hours):
- Serum phosphorus < 2.5 mg/dL at Screening
Show all 14Show fewer
- Ratio of renal tubular maximum phosphate reabsorption rate to glomerular filtration rate (TmP/GFR) < 2.5 mg/dL at Screening
- Presence of skeletal pain attributed to XLH/osteomalacia, as defined by a score of ≥ 4 on the Brief Pain Inventory question 3 (BPI-Q3, Worst Pain) at Screening. (Skeletal pain that, in the opinion of the investigator, is attributed solely to causes other than XLH/osteomalacia-for example, back pain or joint pain in the presence of severe osteoarthritis by radiograph in that anatomical location-in the absence of any skeletal pain likely attributed to XLH/osteomalacia should not be considered for eligibility)
- Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min (using the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation) or estimated glomerular filtration rate (eGFR) eGFR of 45 to <60 mL/min at Screening with confirmation that the renal insufficiency is not due to nephrocalcinosis
- Provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures. If the subject in a minor, provide written assent and have a legally authorized representative willing and able to provide written informed consent, after the nature of the study has been explained, and prior to any research-related procedures
- Willing to provide access to prior medical records for the collection of biochemical and radiographic data and disease history
- Females of child-bearing potential must have a negative urine pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not to be of childbearing potential include those who have been in menopause for at least two years prior to Screening, or have had tubal ligation at least one year prior to Screening, or have had a total hysterectomy or bilateral salpingo-oophorectomy.
- Participants of child-bearing potential or with partners of child-bearing potential who have not undergone a total hysterectomy or a bilateral salpingo-oophorectomy and are sexually active must consent to use two effective methods of contraception as determined by the site investigator (i.e. oral hormonal contraceptives, patch hormonal contraceptives, vaginal ring, intrauterine device, physical double-barrier methods, surgical hysterectomy, vasectomy, tubal ligation, or true abstinence) from the period following the signing of the informed consent through 12 weeks after last dose of study drug
- Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, adhere to the study visit schedule and comply with the assessments
Who cannot join
- Use of any pharmacologic vitamin D metabolite or analog (e.g. calcitriol, doxercalciferol, and paricalcitol) within the 2 years before Screening
- Use of oral phosphate within 2 years before Screening
- Use of aluminum hydroxide antacids, acetazolamides, and thiazides within 7 days prior to Screening
- Use of bisphosphonates in the 2 years prior to Screening
- Use of denosumab in the 6 months prior to Screening
- Use of teriparatide in the 2 months prior to Screening
Show all 25Show fewer
- Chronic use of systemic corticosteroids defined as more than 10 days in the 2 months prior to Screening
- Corrected serum calcium level ≥ 10.8 mg/dL (2.7 mmol/L) at Screening
- Serum intact parathyroid hormone (iPTH) ≥ 2.5 times the upper limit of normal (ULN) at Screening
- Use of medication to suppress parathyroid hormone (PTH) (cinacalcet for example) within 60 days prior to Screening
- Prothrombin time/Partial thromboplastin time (PT/PTT) outside the normal range at Screening
- Evidence of any disease or use of anticoagulant medication (such as warfarin, heparin, direct thrombin inhibitors, or Xa inhibitors (xabans) that, in the opinion of the investigator, cannot be discontinued) that may increase the risk of bleeding during the biopsy procedure
- Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study
- Unable or unwilling to withhold prohibited medications throughout the study
- Documented dependence on narcotics
- Use of KRN23, or any other therapeutic monoclonal antibody within 90 days prior to Screening
- Use of investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
- OR, in Japan, use of any investigational product or investigational medical device within 4 months prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
- Presence or history of any hypersensitivity, allergic or anaphylactic reactions to any monoclonal antibody or KRN23 excipients that, in the judgment of the investigator, places the subject at increased risk for adverse effects
- History of allergic reaction or adverse reactions to tetracycline or demeclocycline
- Prior history of positive test for human immunodeficiency virus antibody, hepatitis B surface antigen, and/or hepatitis C antibody
- History of recurrent infection (other than dental abscesses, which are known to be associated with XLH) or predisposition to infection, or of known immunodeficiency
- Presence of malignant neoplasm (except basal cell carcinoma)
- Presence of a concurrent disease or condition that would interfere with study participation or affect safety
- Presence or history of any condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study
Trial locations and contacts
14 sites in 6 countries.
14 sites in 6 countries
United States5 sites
Duke University Medical Center
Durham, North Carolina, 27710
Houston Methodist Hospital
Houston, Texas, 77030
Indiana University Department of Medicine University Hospital
Indianapolis, Indiana, 46202
Yale University School of Medicine - Yale New-Haven Hospital/Yale Center for Clinical Investigation
New Haven, Connecticut, 06510
UCSF Medical Center at Mission
San Francisco, California, 94158
Japan3 sites
Osaka University Hospital
Osaka, 565-0871
Hokkaido University Hospital
Sapporo, 060-8648
The University of Tokyo Hospital
Tokyo, 113-8655
Canada2 sites
Shriners Hospital for Children
Montreal, Quebec, H3G 1A6
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
France2 sites
CHU de Bicêtre, Service d'Endocrinologie et des Maladies de la Reproduction
Le Kremlin-Bicêtre, 94275
CHU Paris Centre - Hôpital Cochin
Paris, 75014
Denmark1 site
Aarhus University Hospital-Dept of Endocrinology and Internal Medicine
Aarhus, 8000
About this study
Official title: An Open-Label, Single-Arm, Phase 3 Study to Evaluate the Effects of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)
The primary objective of this study is to establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV).
- Conditions
- X-linked Hypophosphatemia
- Being tested
- burosumab
- Keywords
- KRN23, Fibroblast growth factor 23 (FGF23), XLH, X-linked Hypophosphatemia
- 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.
burosumabBiological
Also called KRN23, UX023, Crysvita
solution for subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Open-Label Burosumab Q4W | Experimental | 1.0 mg/kg burosumab monthly (Q4W), calculated based on baseline weight and up to a maximum dose of 90 mg. Receives: Biological: burosumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline in OV/BV at Week 48
OV/BV: percent of a given volume of bone tissue that consists of unmineralized bone (osteoid).
Measured: Baseline, 48 weeks
Secondary outcomes (25)
Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the Lower Limit of Normal (LLN) at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24
The LLN was defined as 2.5 mg/dL (0.81 mmol/L). The 95% confidence interval (CI) was calculated using Wilson score method.
Measured: Baseline, up to 24 weeks
Percent Change From Baseline in O.Th at Week 48
O.Th: mean thickness, given in micrometers, for osteoid seams.
Measured: Baseline, 48 weeks
Percent Change From Baseline in OS/BS at Week 48
OS/Bs: percent of bone surface covered in osteoid.
Measured: Baseline, 48 weeks
Percent Change From Baseline in MLt at Week 48
MLt: average time interval between osteoid formation and its subsequent mineralization; calculated by dividing the osteoid thickness by the adjusted apposition rate (O.Th/Aj.AR). Aj.AR; amount of new bone created (bone formation rate over the entire osteoid surface). Based on imputed MLt values.
Measured: Baseline, 48 weeks
Change From Baseline in MAR at Week 48
MAR: linear rate of new bone deposition; mean distance between the double labels, divided by the time interval between them.
Measured: Baseline, 48 weeks
Change From Baseline in MS/BS at Week 48
MS/BS: percent of bone surface that displays a tetracycline label reflecting active mineralization; calculated as the double-labeled surface plus one half of the single-labeled surface and is expressed as a function of total bone surface ([dLS + sLS/2]/BS). It is a measure of the proportion of bone surface upon which new mineralized bone was being deposited during the period of tetracycline labeling.
Measured: Baseline, 48 weeks
Change From Baseline in BFR/BS at Week 48
BFR/BS: amount of new bone formed in unit time per unit of bone surface; calculated by multiplying MS/BS by the MAR.
MS/BS: percent of bone surface that displays a tetracycline label reflecting active mineralization; calculated as the double-labeled surface plus one half of the single-labeled surface and is expressed as a function of total bone surface ([dLS + sLS/2]/BS). It is a measure of the proportion of bone surface upon which new mineralized bone was being deposited during the period of tetracycline labeling. MAR: linear rate of new bone deposition; mean distance between the double labels, divided by the time interval between them.
Measured: Baseline, 48 weeks
Change From Baseline in BFR/OS at Week 48
BFR/OS: bone formation rate to osteoid surface ratio, related to the Aj.AR (amount of new bone created [bone formation rate over the entire osteoid surface]).
Measured: Baseline, 48 weeks
Change From Baseline in BFR/BV at Week 48
BFR/BV: equivalent to bone turnover rate.
Measured: Baseline, 48 weeks
Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the LLN at the End of the Dosing Cycle Between Baseline and Week 24
The LLN was defined as 2.5 mg/dL (0.81 mmol/L). The 95% CI was calculated using Wilson score method.
Measured: Baseline, up to 24 weeks
Mean Change of Serum Phosphorus Levels at the Mid-Point of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24
Measured: Baseline, up to 24 weeks
Percent Change of Serum Phosphorus Levels at the Mid-Point of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24
Measured: Baseline, up to 24 weeks
Mean Change of Serum Phosphorus Levels at the End of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24
Measured: Baseline, up to 24 weeks
Percentage Change of Serum Phosphorus Levels at the End of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24
Measured: Baseline, up to 24 weeks
Time-Adjusted Area Under the Curve (AUC) of Serum Phosphorus Between Baseline and Week 24
Measured: Baseline, up to 24 weeks
Change From Baseline Over Time in Serum 1,25(OH)2D
Measured: Baseline, Week 1, Week 2, Week 4, Week 20, Week 21, Week 22, Week 24, Week 48, Week 60, Week 70, Week 72, Week 84, Week 94, Week 96, Week 108, Week 120, Week 132
Change From Baseline Over Time in 24-Hour Urinary Phosphorus
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, Week 72, Week 96, End of Study II (EOSII) (up to Week 141)
Change From Baseline Over Time in TmP/GFR
TmP/GFR: ratio of renal tubular maximum reabsorption rate of phosphate to glomerular filtration rate.
Measured: Baseline, Week 2, Week 4, Week 12, Week 22, Week 24, Week 48, Week 60, Week 72, Week 84, Week 96, EOSII (up to Week 141)
Change From Baseline Over Time in TRP
TRP: tubular reabsorption of phosphate.
Measured: Baseline, Week 2, Week 4, Week 12, Week 22, Week 24, Week 48, Week 60, Week 72, Week 84, Week 96, EOSII (up to Week 141)
Change From Baseline Over Time in P1NP
P1NP: procollagen type 1 N-propeptide.
Measured: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Percent Change From Baseline Over Time in P1NP
Measured: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Change From Baseline Over Time in CTx
CTx: carboxy-terminal cross-linked telopeptide of type I collagen.
Measured: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Percent Change From Baseline Over Time in CTx
CTx: carboxy-terminal cross-linked telopeptide of type I collagen.
Measured: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Change From Baseline Over Time in BALP
BALP: bone-specific alkaline phosphatase.
Measured: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Percent Change From Baseline Over Time in BALP
BALP: bone-specific alkaline phosphatase.
Measured: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2015 (estimated)
- Study start23 Dec 2015
- Primary completion30 Aug 2017
- Study completion13 Dec 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 (2)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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyowa Kirin, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Kyowa Kirin Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02537431
- Sponsor's protocol ID
- UX023-CL304
- Status last verified
- Jun 2024
- First submitted
- 25 Aug 2015
- Last updated
- 18 Jun 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- Expanded access
- Available
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 39151033Fratzl-Zelman N, Hartmann MA, Gamsjaeger S, Rokidi S, Paschalis EP, Blouin S, Zwerina J. Bone Matrix Mineralization and Response to Burosumab in Adult Patients With X-Linked Hypophosphatemia: Results From the Phase 3, Single-Arm International Trial. J Bone Miner Res. 2022 Sep;37(9):1665-1678. doi: 10.1002/jbmr.4641. Epub 2022 Aug 10.
PubMed 35775373
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
- An Open-Label, Single-Arm, Phase 3 Study to Evaluate the Effects of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)
- ClinicalTrials.gov ID
- NCT02537431
- Acronym / sponsor ID
- UX023-CL304
- Conditions
- X-linked Hypophosphatemia
- Keywords
- KRN23, Fibroblast growth factor 23 (FGF23), XLH, X-linked Hypophosphatemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Kyowa Kirin, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Kyowa Kirin Co., Ltd.
- Study start
- 23 Dec 2015 (actual)
- Primary completion
- 30 Aug 2017 (actual)
- Study completion
- 13 Dec 2018 (actual)
- First posted
- 1 Sep 2015 (estimated)
- Results
- Posted 25 Sep 2018
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 establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open-Label Burosumab Q4W | Experimental | Biological: burosumab1.0 mg/kg burosumab monthly (Q4W), calculated based on baseline weight and up to a maximum dose of 90 mg. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Male or female, aged 18 - 65 years, inclusive
- Diagnosis of XLH supported by classic clinical features of adult XLH (such as short stature or bowed legs), and at least one of the following at Screening:
- Biochemical findings consistent with XLH based on overnight fasting (min. 8 hours):
- Presence of skeletal pain attributed to XLH/osteomalacia, as defined by a score of ≥ 4 on the Brief Pain Inventory question 3 (BPI-Q3, Worst Pain) at Screening. (Skeletal pain that, in the opinion of the investigator, is attributed solely to causes other than XLH/osteomalacia-for example, back pain or joint pain in the presence of severe osteoarthritis by radiograph in that anatomical location-in the absence of any skeletal pain likely attributed to XLH/osteomalacia should not be considered for eligibility)
- Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min (using the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation) or estimated glomerular filtration rate (eGFR) eGFR of 45 to <60 mL/min at Screening with confirmation that the renal insufficiency is not due to nephrocalcinosis
Key exclusion criteria (25 listed)
- Use of any pharmacologic vitamin D metabolite or analog (e.g. calcitriol, doxercalciferol, and paricalcitol) within the 2 years before Screening
- Use of oral phosphate within 2 years before Screening
- Use of aluminum hydroxide antacids, acetazolamides, and thiazides within 7 days prior to Screening
- Use of bisphosphonates in the 2 years prior to Screening
- Use of denosumab in the 6 months prior to 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 |
|---|---|---|
| burosumabAlso: KRN23, UX023, Crysvitasolution for subcutaneous injection | Biological | Open-Label Burosumab Q4W |
Primary outcome
Percent Change From Baseline in OV/BV at Week 48
Time frame: Baseline, 48 weeks
OV/BV: percent of a given volume of bone tissue that consists of unmineralized bone (osteoid).
25 secondary outcomes
Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the Lower Limit of Normal (LLN) at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24
Time frame: Baseline, up to 24 weeks
Percent Change From Baseline in O.Th at Week 48
Time frame: Baseline, 48 weeks
Percent Change From Baseline in OS/BS at Week 48
Time frame: Baseline, 48 weeks
Percent Change From Baseline in MLt at Week 48
Time frame: Baseline, 48 weeks
Change From Baseline in MAR at Week 48
Time frame: Baseline, 48 weeks
Change From Baseline in MS/BS at Week 48
Time frame: Baseline, 48 weeks
Change From Baseline in BFR/BS at Week 48
Time frame: Baseline, 48 weeks
Change From Baseline in BFR/OS at Week 48
Time frame: Baseline, 48 weeks
Change From Baseline in BFR/BV at Week 48
Time frame: Baseline, 48 weeks
Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the LLN at the End of the Dosing Cycle Between Baseline and Week 24
Time frame: Baseline, up to 24 weeks
Mean Change of Serum Phosphorus Levels at the Mid-Point of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24
Time frame: Baseline, up to 24 weeks
Percent Change of Serum Phosphorus Levels at the Mid-Point of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24
Time frame: Baseline, up to 24 weeks
Mean Change of Serum Phosphorus Levels at the End of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24
Time frame: Baseline, up to 24 weeks
Percentage Change of Serum Phosphorus Levels at the End of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24
Time frame: Baseline, up to 24 weeks
Time-Adjusted Area Under the Curve (AUC) of Serum Phosphorus Between Baseline and Week 24
Time frame: Baseline, up to 24 weeks
Change From Baseline Over Time in Serum 1,25(OH)2D
Time frame: Baseline, Week 1, Week 2, Week 4, Week 20, Week 21, Week 22, Week 24, Week 48, Week 60, Week 70, Week 72, Week 84, Week 94, Week 96, Week 108, Week 120, Week 132
Change From Baseline Over Time in 24-Hour Urinary Phosphorus
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, Week 72, Week 96, End of Study II (EOSII) (up to Week 141)
Change From Baseline Over Time in TmP/GFR
Time frame: Baseline, Week 2, Week 4, Week 12, Week 22, Week 24, Week 48, Week 60, Week 72, Week 84, Week 96, EOSII (up to Week 141)
Change From Baseline Over Time in TRP
Time frame: Baseline, Week 2, Week 4, Week 12, Week 22, Week 24, Week 48, Week 60, Week 72, Week 84, Week 96, EOSII (up to Week 141)
Change From Baseline Over Time in P1NP
Time frame: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Percent Change From Baseline Over Time in P1NP
Time frame: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Change From Baseline Over Time in CTx
Time frame: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Percent Change From Baseline Over Time in CTx
Time frame: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Change From Baseline Over Time in BALP
Time frame: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Percent Change From Baseline Over Time in BALP
Time frame: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
Results and safety
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 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.
- Derived from this study: PubMed 35775373Fratzl-Zelman N, Hartmann MA, Gamsjaeger S, Rokidi S, Paschalis EP, Blouin S, Zwerina J. Bone Matrix Mineralization and Response to Burosumab in Adult Patients With X-Linked Hypophosphatemia: Results From the Phase 3, Single-Arm International Trial. J Bone Miner Res. 2022 Sep;37(9):1665-1678. doi: 10.1002/jbmr.4641. Epub 2022 Aug 10.
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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.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing burosumab in people with X-linked Hypophosphatemia, run by Kyowa Kirin, Inc. at 14 sites in 6 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 14 inclusion and 25 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 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 14 sites in United States, Japan, Canada, France, Denmark and 1 more country. 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?
Yes. Results are posted in the registry (first posted 25 Sep 2018). 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?
Percent Change From Baseline in OV/BV at Week 48 (time frame: Baseline, 48 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 NCT02537431, 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.