A Phase 3 interventional study of burosumab in X-linked Hypophosphatemia, sponsored by Kyowa Kirin, Inc.. Completed at 14 sites in 6 countries. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2024-06-18.
Sponsored by Kyowa Kirin, Inc. · Phase 3, Interventional, and Treatment
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).
49 studies on the registry are indexed under Familial Hypophosphatemic Rickets; 8 are open to participants now.
This study's enrollment of 14 is below the median of 28 across 29 interventional studies indexed under Familial Hypophosphatemic Rickets.
Browse Familial Hypophosphatemic Rickets studies →Kyowa Kirin, Inc. is the lead sponsor of 36 studies on the registry; 2 are open to participants now.
Of its 7 completed or terminated interventional studies of FDA-regulated products, 3 (43%) have results posted.
Counted across the registry records on this site, refreshed daily.
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 one of the following at Screening:
Biochemical findings consistent with XLH based on overnight fasting (min. 8 hours):
Exclusion Criteria:
Use of 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.
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.
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.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
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.
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.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
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.
1.0 mg/kg burosumab monthly (Q4W), calculated based on baseline weight and up to a maximum dose of 90 mg.
Biological: burosumab
solution for subcutaneous injection
Also known as: KRN23, UX023, Crysvita
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).
Time frame: Baseline, 48 weeks
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.
Time frame: 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.
Time frame: Baseline, 48 weeks
Percent Change From Baseline in OS/BS at Week 48
OS/Bs: percent of bone surface covered in osteoid.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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 \[bone formation rate over the entire osteoid surface\]).
Time frame: Baseline, 48 weeks
Change From Baseline in BFR/BV at Week 48
BFR/BV: equivalent to bone turnover rate.
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
The LLN was defined as 2.5 mg/dL (0.81 mmol/L). The 95% CI was calculated using Wilson score method.
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
TmP/GFR: ratio of renal tubular maximum reabsorption rate of phosphate to glomerular filtration rate.
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
TRP: tubular reabsorption of phosphate.
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
P1NP: procollagen type 1 N-propeptide.
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
CTx: carboxy-terminal cross-linked telopeptide of type I collagen.
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
CTx: carboxy-terminal cross-linked telopeptide of type I collagen.
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
BALP: bone-specific alkaline phosphatase.
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
BALP: bone-specific alkaline phosphatase.
Time frame: Baseline, Week 12, Week 24, Week 48, Week 72, Week 96, EOSII (up to Week 141)
| Milestone | Open-Label Burosumab Q4W |
|---|---|
| Started | 14 |
| Completed | 13 |
| Not completed | 1 |
| Withdrew: Withdrawal by subject | 1 |
| Milestone | Open-Label Burosumab Q4W |
|---|---|
| Started | 13 |
| Completed | 13 |
| Not completed | 0 |
| Milestone | Open-Label Burosumab Q4W |
|---|---|
| Started | 8 |
| Completed | 8 |
| Not completed | 0 |
OV/BV: percent of a given volume of bone tissue that consists of unmineralized bone (osteoid).
| percentage change of unmineralized bone | Open-Label Burosumab Q4W |
|---|---|
| Percent Change From Baseline in OV/BV at Week 48 | -54.18 ± 20.211 |
The LLN was defined as 2.5 mg/dL (0.81 mmol/L). The 95% confidence interval (CI) was calculated using Wilson score method.
| percentage of participants | Open-Label Burosumab Q4W |
|---|---|
| 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 | 92.9 (68.5 to 98.7) |
O.Th: mean thickness, given in micrometers, for osteoid seams.
| percentage change in thickness | Open-Label Burosumab Q4W |
|---|---|
| Percent Change From Baseline in O.Th at Week 48 | -32.21 ± 11.966 |
OS/Bs: percent of bone surface covered in osteoid.
| percent change of bone surface covered | Open-Label Burosumab Q4W |
|---|---|
| Percent Change From Baseline in OS/BS at Week 48 | -26.00 ± 15.012 |
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.
| percent change in average time interval | Open-Label Burosumab Q4W |
|---|---|
| Percent Change From Baseline in MLt at Week 48 | -52.24 ± 58.487 |
MAR: linear rate of new bone deposition; mean distance between the double labels, divided by the time interval between them.
| µm/day | Open-Label Burosumab Q4W |
|---|---|
| Change From Baseline in MAR at Week 48 | 0.04 ± 0.506 |
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.
| percent of mineralizing surface | Open-Label Burosumab Q4W |
|---|---|
| Change From Baseline in MS/BS at Week 48 | 1.32 ± 4.365 |
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.
| µm^3/µm^2/year | Open-Label Burosumab Q4W |
|---|---|
| Change From Baseline in BFR/BS at Week 48 | -4.90 ± 27.356 |
BFR/OS: bone formation rate to osteoid surface ratio, related to the Aj.AR (amount of new bone created \[bone formation rate over the entire osteoid surface\]).
| µm^3/µm^2/year | Open-Label Burosumab Q4W |
|---|---|
| Change From Baseline in BFR/OS at Week 48 | -1557.25 ± 3139.075 |
BFR/BV: equivalent to bone turnover rate.
| percentage of bone turnover/year | Open-Label Burosumab Q4W |
|---|---|
| Change From Baseline in BFR/BV at Week 48 | -13.50 ± 32.904 |
The LLN was defined as 2.5 mg/dL (0.81 mmol/L). The 95% CI was calculated using Wilson score method.
| percentage of participants | Open-Label Burosumab Q4W |
|---|---|
| Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the LLN at the End of the Dosing Cycle Between Baseline and Week 24 | 78.6 (52.4 to 92.4) |
| mg/dL | Open-Label Burosumab Q4W |
|---|---|
| Mean Change of Serum Phosphorus Levels at the Mid-Point of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | 1.07 ± 0.300 |
| percent change of serum phosphorus level | Open-Label Burosumab Q4W |
|---|---|
| Percent Change of Serum Phosphorus Levels at the Mid-Point of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | 50.39 ± 19.942 |
| mg/dL | Open-Label Burosumab Q4W |
|---|---|
| Mean Change of Serum Phosphorus Levels at the End of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | 0.46 ± 0.303 |
| percent change of serum phosphorus level | Open-Label Burosumab Q4W |
|---|---|
| Percentage Change of Serum Phosphorus Levels at the End of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | 23.32 ± 19.836 |
| mg/dL | Open-Label Burosumab Q4W |
|---|---|
| Time-Adjusted Area Under the Curve (AUC) of Serum Phosphorus Between Baseline and Week 24 | 3.023 ± 0.2716 |
| pg/mL | Open-Label Burosumab Q4W |
|---|---|
| Week 1 | 107.75 ± 15.964 |
| Week 2 | 48.41 ± 7.397 |
| Week 4 | 13.58 ± 3.435 |
| Week 20 | -1.34 ± 3.617 |
| Week 21 | 31.75 ± 5.233 |
| Week 22 | 11.50 ± 4.060 |
| Week 24 | -3.04 ± 4.891 |
| Week 48 | -1.72 ± 4.190 |
| Week 60 | -5.73 ± 3.395 |
| Week 70 | 3.36 ± 3.987 |
| Week 72 | -5.55 ± 2.895 |
| Week 84 | -5.55 ± 2.962 |
| Week 94 | 9.63 ± 4.239 |
| Week 96 | -6.09 ± 2.403 |
| Week 108 | 0.02 ± 4.714 |
| Week 120 | 1.45 ± 4.197 |
| Week 132 | -1.69 ± 1.993 |
| g/24 hours | Open-Label Burosumab Q4W |
|---|---|
| Week 12 | 0.10 ± 0.128 |
| Week 24 | -0.04 ± 0.075 |
| Week 36 | -0.01 ± 0.059 |
| Week 48 | -0.04 ± 0.076 |
| Week 72 | 0.00 ± 0.096 |
| Week 96 | -0.13 ± 0.082 |
| EOSII | -0.07 ± 0.171 |
TmP/GFR: ratio of renal tubular maximum reabsorption rate of phosphate to glomerular filtration rate.
| mg/dL | Open-Label Burosumab Q4W |
|---|---|
| Week 2 | 1.76 ± 0.136 |
| Week 4 | 0.78 ± 0.097 |
| Week 12 | 0.58 ± 0.122 |
| Week 22 | 0.87 ± 0.064 |
| Week 24 | 0.44 ± 0.101 |
| Week 48 | 0.20 ± 0.097 |
| Week 60 | 0.30 ± 0.171 |
| Week 72 | 0.28 ± 0.127 |
| Week 84 | 0.39 ± 0.136 |
| Week 96 | 0.29 ± 0.097 |
| EOSII | 0.21 ± 0.068 |
TRP: tubular reabsorption of phosphate.
| fraction of phosphorus reabsorbed | Open-Label Burosumab Q4W |
|---|---|
| Week 2 | 0.07 ± 0.005 |
| Week 4 | 0.03 ± 0.013 |
| Week 12 | 0.01 ± 0.013 |
| Week 22 | 0.04 ± 0.007 |
| Week 24 | 0.01 ± 0.015 |
| Week 48 | -0.00 ± 0.021 |
| Week 60 | 0.03 ± 0.016 |
| Week 72 | -0.02 ± 0.035 |
| Week 84 | 0.02 ± 0.019 |
| Week 96 | 0.02 ± 0.014 |
| EOSII | 0.01 ± 0.011 |
P1NP: procollagen type 1 N-propeptide.
| ng/mL | Open-Label Burosumab Q4W |
|---|---|
| Week 12 | 99.18 ± 11.403 |
| Week 24 | 104.33 ± 11.153 |
| Week 48 | 52.49 ± 11.554 |
| Week 72 | 37.29 ± 12.294 |
| Week 96 | 29.29 ± 13.321 |
| EOSII | 2.14 ± 10.105 |
| percent change in P1NP | Open-Label Burosumab Q4W |
|---|---|
| Week 12 | 133.08 ± 13.680 |
| Week 24 | 137.80 ± 15.741 |
| Week 48 | 76.86 ± 14.114 |
| Week 72 | 50.46 ± 13.659 |
| Week 96 | 41.36 ± 11.566 |
| EOSII | 26.20 ± 9.919 |
CTx: carboxy-terminal cross-linked telopeptide of type I collagen.
| pg/mL | Open-Label Burosumab Q4W |
|---|---|
| Week 12 | 464.84 ± 61.696 |
| Week 24 | 404.13 ± 55.948 |
| Week 48 | 175.13 ± 44.022 |
| Week 72 | 143.11 ± 92.503 |
| Week 96 | 76.80 ± 57.357 |
| EOSII | -41.32 ± 83.144 |
CTx: carboxy-terminal cross-linked telopeptide of type I collagen.
| percent change in CTx | Open-Label Burosumab Q4W |
|---|---|
| Week 12 | 89.68 ± 13.316 |
| Week 24 | 70.17 ± 10.341 |
| Week 48 | 35.86 ± 7.396 |
| Week 72 | 34.00 ± 14.016 |
| Week 96 | 25.86 ± 8.461 |
| EOSII | 17.88 ± 9.284 |
BALP: bone-specific alkaline phosphatase.
| μg/L | Open-Label Burosumab Q4W |
|---|---|
| Week 12 | 10.93 ± 3.547 |
| Week 24 | 5.82 ± 2.980 |
| Week 48 | 4.50 ± 3.990 |
| Week 72 | 3.13 ± 2.435 |
| Week 96 | 1.14 ± 2.028 |
| EOSII | -5.80 ± 3.396 |
BALP: bone-specific alkaline phosphatase.
| percent change in BALP | Open-Label Burosumab Q4W |
|---|---|
| Week 12 | 52.54 ± 15.999 |
| Week 24 | 31.37 ± 11.808 |
| Week 48 | 24.35 ± 17.630 |
| Week 72 | 15.13 ± 13.431 |
| Week 96 | 6.92 ± 10.387 |
| EOSII | -27.30 ± 12.767 |
Collected over Week 0 to EOSII (up to Week 141). Mean (SE) duration of exposure to study drug was 716 (35.1) days. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Open-Label Burosumab Q4W | 0/14 (0%) | 4/14 (28.6%) | 14/14 (100%) |
| Event | Open-Label Burosumab Q4W |
|---|---|
| Atrial FibrillationCardiac disorders | 1/14 |
| Obstruction GastricGastrointestinal disorders | 1/14 |
| Splenic RuptureInjury, poisoning and procedural complications | 1/14 |
| MigraineNervous system disorders | 1/14 |
| ParaesthesiaNervous system disorders | 1/14 |
| Event | Open-Label Burosumab Q4W |
|---|---|
| Tooth AbscessInfections and infestations | 7/14 |
| Procedural PainInjury, poisoning and procedural complications | 7/14 |
| Back PainMusculoskeletal and connective tissue disorders | 6/14 |
| PainGeneral disorders | 5/14 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 5/14 |
| ToothacheGastrointestinal disorders | 4/14 |
| InfluenzaInfections and infestations | 4/14 |
| NasopharyngitisInfections and infestations | 4/14 |
| Muscle SpasmsMusculoskeletal and connective tissue disorders | 4/14 |
| OsteoarthritisMusculoskeletal and connective tissue disorders | 4/14 |
| Age, Continuous(years) | Open-Label Burosumab Q4W |
|---|---|
| Mean | 40.13 ± 8.725 |
| Sex: Female, Male(Participants) | Open-Label Burosumab Q4W |
|---|---|
| Female | 8 |
| Male | 6 |
| Ethnicity (NIH/OMB)(Participants) | Open-Label Burosumab Q4W |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 13 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Open-Label Burosumab Q4W |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 4 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 9 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Osteoid Volume/Bone Volume (OV/BV)(percentage of osteoid volume) | Open-Label Burosumab Q4W |
|---|---|
| Mean | 26.12 ± 12.357 |
| Serum Phosphorus(mg/dL) | Open-Label Burosumab Q4W |
|---|---|
| Mean | 2.24 ± 0.396 |
| Osteoid Thickness (O.Th)(µm) | Open-Label Burosumab Q4W |
|---|---|
| Mean | 17.21 ± 4.105 |
| Osteoid Surface/Bone Surface (OS/BS)(percentage of osteoid surface) | Open-Label Burosumab Q4W |
|---|---|
| Mean | 91.73 ± 3.438 |
14 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Burosumab
Familial Hypophosphatemic Rickets →