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 8th largest by enrollment, is the only one that started in 2011, is one of 3 in phase 1/2, and was the 2nd 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 2011.
This study's enrollment of 29 is close to 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 7th largest by enrollment of the 16 interventional ones that give a count, is the only one that started in 2011, is one of 4 in phase 1/2, and was the 2nd 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). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality.
Main Inclusion Criteria:
Main 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.
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.
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 4 doses)
Drug: KRN23
Subjects will receive escalating doses of KRN23 administered by SC injections every 28-days (up to 4 doses) based on a dosing algorithm and discretion of the 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: On-Treatment: 6.5 months, 27 total visits
Evaluation of Effect of Repeated SC Injections of KRN23
Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters including serum phosphorus, sex hormone, bone biomarkers, quality of life assessments and population pharmacokinetics of KRN23 dose levels from cumulative dosing. Summary of serum phosphorus by visit/day is captured below.
Time frame: On-Treatment: 6.5 months, 27 total 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 and bone quality.
Time frame: On-Treatment: 6.5 months, 27 total visits
| Milestone | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo |
|---|---|---|---|
| Started | 27 | 1 | 1 |
| Completed | 25 | 1 | 1 |
| Not completed | 2 | 0 | 0 |
| Withdrew: Adverse event | 2 | 0 | 0 |
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 |
|---|---|---|---|
| Subjects reporting at least one TEAE | 24 | 1 | 1 |
| Subjects who died | 0 | 0 | 0 |
| Subjects reporting SAE other than death | 0 | 0 | 0 |
| Subjects reporting at least one DLT | 0 | 0 | 0 |
| Subjects reporting AE leading to discontinuation | 1 | 0 | 0 |
Effect of repeated SC injections of KRN23, from baseline, on pharmacodynamic parameters including serum phosphorus, sex hormone, bone biomarkers, quality of life assessments and population pharmacokinetics of KRN23 dose levels from cumulative dosing. Summary of serum phosphorus by visit/day is captured below.
| mg/dL | KRN23 |
|---|---|
| Visit 2 (D0)/0 | 1.89 (1.2 to 2.8) |
| Visit 3 (D3)/3 | 2.18 (1.5 to 2.9) |
| Visit 4 (D7)/7 | 2.20 (1.7 to 3.2) |
| Visit 5 (D12)/12 | 2.14 (1.7 to 2.7) |
| Visit 6 (D18)/18 | 2.15 (1.7 to 3.0) |
| Visit 7 (D26)/26 | 1.96 (1.4 to 2.7) |
| Visit 8 (D28)/0 | 1.93 (1.3 to 2.6) |
| Visit 9 (D31)/3 | 2.35 (1.5 to 3.3) |
| Visit 10 (D35)/7 | 2.45 (1.9 to 3.1) |
| Visit 11 (D40)/12 | 2.31 (1.6 to 3.2) |
| Visit 12 (D46)/18 | 2.28 (1.3 to 3.0) |
| Visit 13 (D54)/26 | 2.15 (1.7 to 3.0) |
| Visit 14 (D56)/0 | 2.12 (1.5 to 2.7) |
| Visit 15 (D59)/3 | 2.57 (1.7 to 3.8) |
| Visit 16 (D63)/7 | 2.85 (1.9 to 3.5) |
| Visit 17 (D68)/12 | 2.83 (1.9 to 3.9) |
| Visit 18 (D74)/18 | 2.60 (1.6 to 3.5) |
| Visit 19 (D82)/26 | 2.33 (1.7 to 3.0) |
| Visit 20 (D84)/0 | 2.26 (1.8 to 3.0) |
| Visit 21 (D87)/3 | 2.64 (1.9 to 3.6) |
| Visit 22 (D91)/7 | 3.03 (2.3 to 3.7) |
| Visit 23 (D96)/12 | 2.94 (2.2 to 3.5) |
| Visit 24 (D102)/18 | 2.82 (1.7 to 4.0) |
| Visit 25 (D110)/26 | 2.54 (1.9 to 3.3) |
| End of Study - Visit 26 (D120) | 2.22 (1.6 to 2.9) |
| Early Withdrawal | 1.30 (1.3 to 1.3) |
Evaluation of effect of repeated SC injections of KRN23, compared to Placebo on bone mineral density and bone quality.
| 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 Subjects were monitored for any untoward medical occurrences from the time of signed ICF through 30 days postdose.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| KRN23 | 0/27 (0%) | 0/27 (0%) | 24/27 (88.9%) |
| 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 |
|---|---|---|---|
| Ear painEar and labyrinth disorders | 0/27 | 1/1 | 0/1 |
| DyspepsiaGastrointestinal disorders | 2/27 | 1/1 | 0/1 |
| Mouth ulcerationGastrointestinal disorders | 0/27 | 1/1 | 0/1 |
| Lip ulcerationGastrointestinal disorders | 0/27 | 1/1 | 0/1 |
| PyrexiaGeneral disorders | 0/27 | 0/1 | 1/1 |
| Injection site painGeneral disorders | 0/27 | 1/1 | 0/1 |
| Influenza like illnessGeneral disorders | 0/27 | 1/1 | 0/1 |
| InfluenzaInfections and infestations | 1/27 | 0/1 | 1/1 |
| Urinary tract infectionInfections and infestations | 2/27 | 1/1 | 0/1 |
| NasopharyngitisInfections and infestations | 7/27 | 1/1 | 1/1 |
| Age, Categorical(Participants) | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo | Total |
|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 26 | 1 | 1 | 28 |
| >=65 years | 1 | 0 | 0 | 1 |
| Age, Continuous(years) | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo | Total |
|---|---|---|---|---|
| Median | 42 (19 to 66) | 21 (21 to 21) | 28 (28 to 28) | 41 (19 to 66) |
| Sex: Female, Male(Participants) | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo | Total |
|---|---|---|---|---|
| Female | 18 | 1 | 1 | 20 |
| Male | 9 | 0 | 0 | 9 |
| Race/Ethnicity, Customized(Participants) | KRN23 | Bone Substudy KRN23 | Bone Substudy Placebo | Total |
|---|---|---|---|---|
| White/Caucasian | 26 | 1 | 1 | 28 |
| 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 | 27 | 0 | 0 | 27 |
The record gives a start of Apr 2011, a primary completion of May 2013 and a completion of Oct 2013. It does not say whether these dates are actual or estimated.
Results were first posted in Oct 2020, 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 →