A Phase 4 interventional study of KRN23 in X-linked Hypophosphatemia (XLH), sponsored by Kyowa Kirin Co., Ltd.. Completed at 5 sites in China. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2024-07-09.
Sponsored by Kyowa Kirin Co., Ltd. · Phase 4, Interventional, and Treatment
Help build ClinicalTrials.gg — short surveys, remote interviews or testing, in your language. Take part →
Among 18 studies testing burosumab in Familial Hypophosphatemic Rickets — none open to participants now, none under way, none of them phase 3. burosumab · Familial Hypophosphatemic Rickets.
Among the 18 studies testing burosumab in Familial Hypophosphatemic Rickets, this one is the 12th largest by enrollment, is one of 3 that started in 2021, is one of 4 in phase 4, and was the 17th registered.
49 studies on the registry are indexed under Familial Hypophosphatemic Rickets; 8 are open to participants now.
Browse Familial Hypophosphatemic Rickets studies →None of the 18 studies testing burosumab in Familial Hypophosphatemic Rickets is open now. The open studies under Familial Hypophosphatemic Rickets, anywhere →
burosumab: first FDA approval 2018 (CRYSVITA), first EMA approval 2018. What it is approved for, and how studies give it, is on its page →
Of the 4 Familial Hypophosphatemic Rickets studies recruiting now, 2 set no upper age limit and 2 stop at 75 or younger; 3 take children under 18; 1 takes healthy volunteers.
This study: 18 Years to 65 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
18 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 5 · Phase 2 6 · Phase 3 6 · Phase 4 4
First in 2008; this study’s year is marked. 3 started in 2021.
This study's enrollment of 18 is below the median of 28 across 29 interventional studies indexed under Familial Hypophosphatemic Rickets.
Median enrollment 26 across the 18 interventional studies. Of the 17 that completed or were terminated, 9 (53%) have results posted.
This tab as a report to print or save as PDF → With an account.
15 of 18 have an industry lead sponsor. The 6 most frequent:
Kyowa Kirin Co., Ltd. is the lead sponsor of 159 studies on the registry; 8 are open to participants now.
21 studies in Familial Hypophosphatemic Rickets by Kyowa Kirin Co., Ltd.: 2 open now, 3 under way, none of them phase 3. Of 17 finished, 8 (47%) have results posted.
Among the 21 studies in Familial Hypophosphatemic Rickets by Kyowa Kirin Co., Ltd., this one is the 11th largest by enrollment of the 16 interventional ones that give a count, is one of 4 that started in 2021, is one of 2 in phase 4, and was the 18th registered.
Kyowa Kirin Co., Ltd.’s Familial Hypophosphatemic Rickets studies by the year they started, since 2008.
None of the studies under way reaches primary completion within a year.
Among the 18 studies testing burosumab in Familial Hypophosphatemic Rickets, from their registry updates:
Nothing recorded among them since 2026-09-25.
Among Kyowa Kirin Co., Ltd.’s 21 in Familial Hypophosphatemic Rickets:
This tab as a report to print or save as PDF → With an account.
The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in adult Chinese patients with XLH
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Diagnosis of XLH supported by classic clinical features of adult XLH (such as short stature or bowed legs) and at least either of the following at screening:
Biochemical findings consistent with XLH following overnight fasting (≥8 hours) at Screening:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
KRN23 1 mg/kg administered subcutaneously (SC) every 4 weeks for 48 weeks. Before KRN23 treatment, all patients will receive oral phosphate and vitamin D analogs for 12 weeks of Run-in period.
Drug: KRN23
KRN23 is a sterile clear colourless and preservative free solution supplied in single use 5 mL vials containing 1 mL of KRN23 at a concentration of 30mg/mL
Also known as: Burosumab, Crysvita
Change from Baseline (CFB) in mean serum phosphorus level at the end of the dose cycle
Time frame: Weeks 4, 8, 12, 16, 20, 24, 36, and 48
Change in Brief Pain Inventory (BPI) Worst Pain score over time
Change from baseline to post-baseline visits in BPI-Q3 (Worst Pain) score as averaged from daily diary scores recorded over 1 week and the study visit score. The BPI evaluates the condition of all pain over the previous 24 hours. Two dimensions are measured: pain severity (worst, least, average, and now) and the impact of pain on functioning (pain interference with general activity, walking, work, mood, enjoyment of life, relations with others, and sleep). Question 3 of the short-form BPI (BPI-Q3) asks subjects to rate their pain at its worst in the last 24 hours on a scale of 0 (no pain) to 10 (pain as bad as you can imagine).
Time frame: Weeks 0, 12, 24, 36, and 48
Change in BPI Pain Severity score and Pain Interference score over time
Change from baseline to post-baseline visits in BPI pain severity score as averaged from daily diary scores recorded over 1 week and the study visit score. The BPI evaluates the condition of all pain over the previous 24 hours. Two dimensions are measured: pain severity (worst, least, average, and now) and the impact of pain on functioning (pain interference with general activity, walking, work, mood, enjoyment of life, relations with others, and sleep). The severity of pain in the last 24 hours is rated on a scale of 0 (no pain) to 10 (pain as bad as you can imagine).
Time frame: Weeks 0, 12, 24, 36, and 48
Change in BPI Pain Interference score over time
Change from baseline to post-baseline visits in BPI pain interference score as recorded on the day of the study visit. The BPI evaluates the condition of all pain over the previous 24 hours. Two dimensions are measured: pain severity (worst, least, average, and now) and the impact of pain on functioning (pain interference with general activity, walking, work, mood, enjoyment of life, relations with others, and sleep). Pain interference in the last 24 hours is rated on a scale of 0 (does not interfere) to 10 (completely interferes).
Time frame: Weeks 0, 12, 24, 36, and 48
Change in the Stiffness and Physical Function domains of the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) over time
The WOMAC is a 24-item participant-reported questionnaire with two domains, Stiffness (2 questions) and Physical Function (17 questions) over the previous 48 hours. The WOMAC is administered in a 5-point Likert-scale format using descriptors of none, mild, moderate, severe, and extreme corresponding to an ordinal scale of 0-4. Higher scores on the WOMAC indicate worse stiffness and functional limitations. Scores are normalized to a 0-100 metric where 0 was the best health state and 100 the worst.
Time frame: Weeks 0, 12, 24, 36, and 48
Change in the health related QOL assessment by Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function over time
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013), (NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Physical Function Domain, increases indicate greater mobility.
Time frame: Weeks 0, 12, 24, 36, and 48
The proportion of subjects achieving mean serum phosphorus levels above the lower limit of normal (LLN; 2.5 mg/dL [0.81 mmol/L]) at the end of the dosing cycle
Time frame: Weeks 4, 8, 12, 16, 20, 24, 36, and 48
The proportion of subjects achieving mean serum phosphorus levels above the LLN (2.5 mg/dL [0.81 mmol/L]) at the mid-point of dosing cycle
Time frame: Weeks 2, 6, 10, 14, 18 and 22
CFB in mean serum phosphorus level at the mid-time point of dosing cycle
Time frame: Weeks 2, 6, 10, 14, 18 and 22
Mean percent CFB in serum phosphorus levels averaged at the end of dose cycles
Time frame: Baseline to Week 48
Cumulative exposure: area under curve (AUC) of serum phosphorus
Time frame: Baseline to Week 48
Change in serum 1,25(OH)2D over time
Time frame: Baseline to Week 48
Change in urinary phosphorus over time
Time frame: Baseline to Week 48
Change in ratio of renal tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR; algorithm method) over time
Time frame: Baseline to Week 48
Radiologic healing or resolution of pre-existing fractures and/or pseudofractures, as defined by skeletal survey at Baseline and subsequent targeted radiography
Time frame: Weeks 0, 12, 24, 36, and 48
Change in enthesopathy spur at the calcaneus measured by lateral foot X-rays
Time frame: Weeks 0, 24 and 48
Change in 6-minute walking test (6MWT) over time
Time frame: Weeks 0, 12, 24, 36, and 48
Safety of KRN23 by studying the number, severity and relatedness of Adverse Events (including laboratory and imaging assessments)
Incidence, frequency, and severity of AEs and SAEs, including clinically significant changes in laboratory assessments as well as ECHO, ECG, ultrasound, vital sign, X-Ray images and Anti-KRN23 antibody.
Time frame: Week 0 to Week 56
Pharmacokinetics: KRN23 concentrations
KRN23 concentration in the Mid and end of dose cycles.
Time frame: Week 0, 1, 2, 4, 21, 22, 24, 36, 48 and 56
Change from Week -14 to Baseline in serum phosphorus over time during Run-in Period
Time frame: Week -14, -10 and -4
Percent change from Week -14 to Baseline in serum phosphorus over time during Run-in Period
Time frame: Week -14, -10 and -4
Change from Week -14 to Baseline in serum 1,25(OH)2D over time during Run-in Period
Time frame: Week -14, -10 and -4
Percent change from Week -14 to Baseline in serum 1,25(OH)2D over time during Run-in Period
Time frame: Week -14, -10 and -4
Change from Week -14 to Baseline in plamas iPTH over time during Run-in Period
Time frame: Week -14, -10 and -4
Percent change from Week -14 to Baseline in plamas iPTH over time during Run-in Period
Time frame: Week -14, -10 and -4
Change from Week -14 to Baseline in serum calcium over time during Run-in Period
Time frame: Week -14, -10 and -4
Percent change from Week -14 to Baseline in serum calcium over time during Run-in Period
Time frame: Week -14, -10 and -4
Change from Week -14 to Baseline in urinary phosphorus over time during Run-in Period
Time frame: Week -14, -10 and -4
Percent change from Week -14 to Baseline in urinary phosphorus over time during Run-in Period
Time frame: Week -14, -10 and -4
Change from Week -14 to Baseline in TmP/GFR over time during Run-in Period
Time frame: Week -14, -10 and -4
Percent change from Week -14 to Baseline in TmP/GFR over time during Run-in Period
Time frame: Week -14, -10 and -4
Safety of conventional therapy by studying the number, severity and relatedness of Adverse Events
Incidence, frequency, and severity of AEs and SAEs, including clinically significant changes in laboratory assessments during Run-in period
Time frame: Week -14 to Week 0
Plan to share: No
No publications or documents are linked to this record.
Ran from Sep 2021 to primary completion in Aug 2023, 22 months.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jul 2024. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Burosumab
Familial Hypophosphatemic Rickets →