A Phase 1/2 interventional study of KRN23 in X-linked Hypophosphatemia, sponsored by Kyowa Kirin Co., Ltd.. Completed at 6 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-06-18.
Sponsored by Kyowa Kirin Co., Ltd. · Phase 1/2, Interventional, and Treatment
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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 10th largest by enrollment, is the only one that started in 2012, is one of 3 in phase 1/2, and was the 3rd 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 and older · 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. 1 started in 2012.
This study's enrollment of 23 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 9th largest by enrollment of the 16 interventional ones that give a count, is the only one that started in 2012, is one of 4 in phase 1/2, and was the 3rd 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 primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH).
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.
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.
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.
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.
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.
Escalating doses of KRN23 (0.05, 0.10, 0.30, and 0.60 mg/kg) will be administered SC every 28 days (up to 12 doses)
Drug: KRN23
Subjects will receive escalating doses of KRN23 administered by SC injection every 28-days (up to 12 doses) based on a dosing algorithm and discretion of Investigator and Sponsor.
Safety and Efficacy of Repeated SC Injections of KRN23.
Safety and efficacy of repeated SC injections of KRN23, from Baseline, as assessed by serum phosphorus levels, immunogenicity, adverse events and clinically significant changes in vital signs and laboratory testing.
Time frame: 13.5 months,(50 visits)
Evaluation of Effect of Repeated SC Injections of KRN23
Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters (serum phosphorus)
Time frame: 13.5 months, (50 visits)
Evaluation of Effect of Repeated SC Injections of KRN23 in Bone Substudy
Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density, bone quality and histomorphometric parameters.
Time frame: 13.5 months,(50 visits)
| Milestone | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo |
|---|---|---|---|
| Started | 21 | 1 | 1 |
| Completed | 19 | 0 | 0 |
| Not completed | 2 | 1 | 1 |
| Withdrew: Adverse event | 2 | 0 | 0 |
| Withdrew: Study terminated by the sponsor | 0 | 1 | 1 |
Safety and efficacy of repeated SC injections of KRN23, from Baseline, as assessed by serum phosphorus levels, immunogenicity, adverse events and clinically significant changes in vital signs and laboratory testing.
| Participants | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo |
|---|---|---|---|
| With Any TEAE | 21 | 1 | 1 |
| With Any Drug-related TEAE | 13 | 1 | 0 |
| With Any TEAE with an Outcome of Death | 0 | 0 | 0 |
| With Any Serious TEAE | 3 | 0 | 0 |
| With Any Drug-related Serious TEAE | 0 | 0 | 0 |
| With Any Serious TEAE with an Outcome of Death | 0 | 0 | 0 |
Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters (serum phosphorus)
| mg/dL | KRN23 |
|---|---|
| Baseline | 1.85 (1.2 to 2.3) |
| Ext Visit 1 /0 | 1.88 (1.3 to 2.5) |
| Ext Visit 2 /7 | 2.86 (2.1 to 3.6) |
| Ext Visit 3 /14 | 2.87 (2.0 to 3.5) |
| Ext Visit 4 /25 | 2.48 (2.0 to 3.0) |
| Ext Visit 5 /0 | 2.25 (1.8 to 3.1) |
| Ext Visit 6 /7 | 2.91 (2.1 to 3.9) |
| Ext Visit 7 /14 | 2.95 (2.3 to 3.8) |
| Ext Visit 8 /25 | 2.67 (1.9 to 3.3) |
| Ext Visit 9 /0 | 2.43 (1.6 to 2.9) |
| Ext Visit 10 /7 | 2.96 (2.2 to 3.8) |
| Ext Visit 11 /14 | 2.84 (2.2 to 3.5) |
| Ext Visit 12 /25 | 2.59 (2.1 to 3.5) |
| Ext Visit 13 /0 | 2.42 (1.7 to 3.1) |
| Ext Visit 14 /7 | 2.92 (1.9 to 3.8) |
| Ext Visit 15 /14 | 2.90 (2.1 to 3.6) |
| Ext Visit 16 /25 | 2.57 (1.9 to 3.4) |
| Ext Visit 17 /0 | 2.35 (1.7 to 3.4) |
| Ext Visit 18 /7 | 2.82 (2.1 to 3.5) |
| Ext Visit 19 /14 | 2.74 (2.0 to 3.5) |
| Ext Visit 20 /25 | 2.60 (1.8 to 3.5) |
| Ext Visit 21 /0 | 2.35 (1.7 to 3.0) |
| Ext Visit 22 /7 | 2.94 (2.3 to 3.8) |
| Ext Visit 23 /14 | 2.93 (2.0 to 3.8) |
| Ext Visit 24 /25 | 2.60 (1.8 to 3.5) |
| Ext Visit 25 /0 | 2.37 (1.8 to 2.9) |
| Ext Visit 26 /7 | 2.83 (2.3 to 3.5) |
| Ext Visit 27 /14 | 2.79 (2.1 to 3.5) |
| Ext Visit 28 /25 | 2.59 (2.1 to 3.2) |
| Ext Visit 29 /0 | 2.41 (1.7 to 3.4) |
| Ext Visit 30 /7 | 2.87 (2.1 to 3.7) |
| Ext Visit 31 /14 | 2.81 (1.9 to 3.5) |
| Ext Visit 32 /25 | 2.55 (2.0 to 3.4) |
| Ext Visit 33 /0 | 2.42 (1.6 to 3.2) |
| Ext Visit 34 /7 | 2.79 (2.0 to 3.8) |
| Ext Visit 35 /14 | 2.75 (1.7 to 3.4) |
| Ext Visit 36 /25 | 2.53 (1.9 to 3.6) |
| Ext Visit 37 /0 | 2.52 (1.9 to 3.4) |
| Ext Visit 38 /7 | 2.85 (1.9 to 3.9) |
| Ext Visit 39 /14 | 2.69 (1.7 to 3.7) |
| Ext Visit 40 /25 | 2.48 (1.8 to 3.5) |
| Ext Visit 41 /0 | 2.29 (1.8 to 3.2) |
| Ext Visit 42 /7 | 2.71 (1.7 to 3.8) |
| Ext Visit 43 /14 | 2.70 (2.0 to 3.6) |
| Ext Visit 44 /25 | 2.43 (1.9 to 2.9) |
| Ext Visit 45 /0 | 2.25 (1.7 to 2.8) |
| Ext Visit 46 /7 | 2.73 (2.1 to 3.6) |
| Ext Visit 47 /14 | 2.61 (1.9 to 3.4) |
| Ext Visit 48 /25 | 2.43 (1.9 to 3.0) |
| Ext Visit 49 /38 | 2.07 (1.6 to 2.6) |
| Ext Visit 50 /81 | 1.78 (1.6 to 1.9) |
Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density, bone quality and histomorphometric parameters.
| units on a scale | Bone Substudy KRN23 | Bone Substudy Placebo |
|---|---|---|
| Evaluation of Effect of Repeated SC Injections of KRN23 in Bone Substudy | NA | NA |
Collected over After the first dose of study drug in study through the end of study, up to 13.5 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| KRN23 | 0/21 (0%) | 3/21 (14.3%) | 21/21 (100%) |
| Bone Substudy KRN23 | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Bone Substudy Placebo | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Event | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo |
|---|---|---|---|
| Cervical Spinal StenosisMusculoskeletal and connective tissue disorders | 1/21 | 0/1 | 0/1 |
| Breast CancerNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/21 | 0/1 | 0/1 |
| Hypertensive crisisVascular disorders | 1/21 | 0/1 | 0/1 |
| Event | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo |
|---|---|---|---|
| GINGIVAL ULCERATIONGastrointestinal disorders | 0/21 | 1/1 | 0/1 |
| GASTROOESOPHAGEAL REFLUX DISEASEGastrointestinal disorders | 0/21 | 1/1 | 0/1 |
| LIP ULCERATIONGastrointestinal disorders | 0/21 | 1/1 | 0/1 |
| INJECTION SITE HAEMATOMAGeneral disorders | 0/21 | 1/1 | 0/1 |
| INJECTION SITE PAINGeneral disorders | 1/21 | 1/1 | 0/1 |
| NASOPHARYNGITISInfections and infestations | 2/21 | 1/1 | 1/1 |
| GINGIVAL INJURYInjury, poisoning and procedural complications | 0/21 | 1/1 | 0/1 |
| MIGRAINENervous system disorders | 1/21 | 0/1 | 1/1 |
| HEADACHENervous system disorders | 2/21 | 1/1 | 0/1 |
| OROPHARYNGEAL PAINRespiratory, thoracic and mediastinal disorders | 0/21 | 1/1 | 0/1 |
| Age, Categorical(Participants) | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo | Total |
|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 20 | 1 | 1 | 22 |
| >=65 years | 1 | 0 | 0 | 1 |
| Age, Continuous(years) | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo | Total |
|---|---|---|---|---|
| Mean | 43.0 (19 to 66) | 22 (22 to 22) | 28 (28 to 28) | 41.1 (19 to 66) |
| Sex: Female, Male(Participants) | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo | Total |
|---|---|---|---|---|
| Female | 12 | 1 | 1 | 14 |
| Male | 9 | 0 | 0 | 9 |
| Race/Ethnicity, Customized(Participants) | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo | Total |
|---|---|---|---|---|
| White/Caucasian | 20 | 1 | 1 | 22 |
| Black/African American/African Caribbean | 1 | 0 | 0 | 1 |
| Region of Enrollment(participants) | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo | Total |
|---|---|---|---|---|
| Canada | 0 | 1 | 1 | 2 |
| United States | 21 | 0 | 0 | 21 |
The record gives a start of Feb 2012, a primary completion of Sep 2013 and a completion of Jun 2014. It does not say whether these dates are actual or estimated.
Results were first posted in Feb 2021, 7 years 5 months after primary completion. A results delay was requested in Jan 2015.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jun 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Burosumab
Familial Hypophosphatemic Rickets →