A Phase 2 interventional study of Burosumab in X-Linked Hypophosphatemia, sponsored by Kyowa Kirin, Inc.. Completed at 3 sites in United States. Open to participants aged 1 Year to 4 Years. Per ClinicalTrials.gov, last updated 2024-05-06.
Sponsored by Kyowa Kirin, Inc. · Phase 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 17th largest by enrollment, is one of 2 that started in 2016, is one of 3 in phase 2, and was the 9th 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: 1 Year to 4 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. 2 started in 2016.
This study's enrollment of 13 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:
This study is registered under Kyowa Kirin, Inc., one of the names counted as Kyowa Kirin Co., Ltd.. Under that name alone:
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.
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 16th largest by enrollment of the 16 interventional ones that give a count, is one of 2 that started in 2016, is one of 3 in phase 2, and was the 9th 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:
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The primary objectives of the study are to:
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 ONE or more of the following
Biochemical findings associated with XLH including:
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.
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.
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.
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.
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.
Burosumab subcutaneous (SC) injections every 2 weeks (Q2W) for a total of 160 weeks.
Biological: Burosumab
solution for subcutaneous injection
Also known as: KRN23, Crysvita®, UX023
Change From Baseline at Week 40 in Serum Phosphorus
The Generalized Estimation Equation (GEE) model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure.
Time frame: Baseline, Week 40
Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation
An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. A serious AE was defined as an AE that at any dose, in the view of either the Investigator or Sponsor, results in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or disability, a congenital anomaly/birth defect, or other important medical events (according to the investigator). An AE was considered a TEAE if it occurred on or after the first dose and was not present prior to the first dose, or it was present prior to the first dose but increased in severity during the study. Events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0: grade 1 (mild), grade 2 (moderate), grade 3 (severe), grade 4 (life-threatening), grade 5 (death).
Time frame: From first dose of study drug through the end of the study (Week 160). Maximum duration of exposure to study drug was 160 weeks.
Radiographic Global Impression of Change (RGI-C) Score at Week 40
Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The Analysis of Covariance (ANCOVA) model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.
Time frame: Week 40
RGI-C Score at Week 64
Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.
Time frame: Week 64
Change From Baseline in Rickets at Week 40 as Assessed by the RSS Total Score
The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity.The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.
Time frame: Baseline, Week 40
Change From Baseline in Rickets at Week 64 as Assessed by the RSS Total Score
The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. The GEE model includes the change from baseline in RSS as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.
Time frame: Baseline, Week 64
RGI-C Lower Limb Deformity Score at Week 40
Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.
Time frame: Week 40
RGI-C Lower Limb Deformity Score at Week 64
Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.
Time frame: Week 64
Change From Baseline Over Time in Recumbent Length/Standing Height
Time frame: Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160
Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Height-for-Age Z-Scores
Recumbent length/Standing height z scores are measures of height adjusted for a child's age and sex. The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome.
Time frame: Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160
Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Percentiles
Time frame: Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160
Change From Baseline Over Time in Serum Alkaline Phosphatase (ALP)
The GEE model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure.
Time frame: Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160
Percent Change From Baseline Over Time in Serum ALP
Time frame: Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160
| Milestone | Burosumab Q2W |
|---|---|
| Started | 13 |
| Completed week 40 | 13 |
| Completed week 64 | 13 |
| Completed | 12 |
| Not completed | 1 |
| Withdrew: Withdrawal by subject | 1 |
The Generalized Estimation Equation (GEE) model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure.
| mg/dL | Burosumab Q2W |
|---|---|
| Change From Baseline at Week 40 in Serum Phosphorus | 0.96 ± 0.117 |
An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. A serious AE was defined as an AE that at any dose, in the view of either the Investigator or Sponsor, results in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or disability, a congenital anomaly/birth defect, or other important medical events (according to the investigator). An AE was considered a TEAE if it occurred on or after the first dose and was not present prior to the first dose, or it was present prior to the first dose but increased in severity during the study. Events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0: grade 1 (mild), grade 2 (moderate), grade 3 (severe), grade 4 (life-threatening), grade 5 (death).
| Participants | Burosumab Q2W |
|---|---|
| Adverse Event Starting during Screening Period | 5 |
| TEAEs | 13 |
| Related TEAEs | 5 |
| Serious TEAEs | 1 |
| Serious Related TEAEs | 0 |
| Grade 3 or 4 TEAE | 2 |
| TEAE Leading to Study Discontinuation | 0 |
| TEAE Leading to Treatment Discontinuation | 0 |
| TEAE Leading to Death | 0 |
Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The Analysis of Covariance (ANCOVA) model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.
| units on a scale | Burosumab Q2W |
|---|---|
| Radiographic Global Impression of Change (RGI-C) Score at Week 40 | 2.21 ± 0.071 |
Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.
| units on a scale | Burosumab Q2W |
|---|---|
| RGI-C Score at Week 64 | 2.23 ± 0.111 |
The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity.The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.
| units on a scale | Burosumab Q2W |
|---|---|
| Change From Baseline in Rickets at Week 40 as Assessed by the RSS Total Score | -1.75 ± 0.116 |
The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. The GEE model includes the change from baseline in RSS as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.
| units on a scale | Burosumab Q2W |
|---|---|
| Change From Baseline in Rickets at Week 64 as Assessed by the RSS Total Score | -2.02 ± 0.115 |
Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.
| units on a scale | Burosumab Q2W |
|---|---|
| RGI-C Lower Limb Deformity Score at Week 40 | 1.21 ± 0.155 |
Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.
| units on a scale | Burosumab Q2W |
|---|---|
| RGI-C Lower Limb Deformity Score at Week 64 | 1.51 ± 0.123 |
| cm | Burosumab Q2W |
|---|---|
| Week 12 | 1.44 ± 2.009 |
| Week 24 | 2.52 ± 1.518 |
| Week 40 | 4.29 ± 2.451 |
| Week 64 | 7.22 ± 3.157 |
| Week 88 | 10.30 ± 3.400 |
| Week 112 | 13.25 ± 3.724 |
| Week 136 | 15.41 ± 3.699 |
| Week 160 | 18.96 ± 4.206 |
Recumbent length/Standing height z scores are measures of height adjusted for a child's age and sex. The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome.
| Z score | Burosumab Q2W |
|---|---|
| Week 12 | -0.082 ± 0.4964 |
| Week 24 | -0.208 ± 0.4540 |
| Week 40 | -0.276 ± 0.6647 |
| Week 64 | -0.264 ± 0.8755 |
| Week 88 | -0.212 ± 0.9115 |
| Week 112 | -0.174 ± 0.9273 |
| Week 136 | -0.321 ± 0.9123 |
| Week 160 | -0.172 ± 0.9048 |
| percentiles | Burosumab Q2W |
|---|---|
| Week 12 | -0.006 ± 11.6149 |
| Week 24 | -4.940 ± 13.1323 |
| Week 40 | -5.283 ± 20.1675 |
| Week 64 | -4.980 ± 23.4412 |
| Week 88 | -4.155 ± 23.9126 |
| Week 112 | -3.000 ± 24.7569 |
| Week 136 | -7.026 ± 24.8583 |
| Week 160 | -3.628 ± 25.5113 |
The GEE model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure.
| U/L | Burosumab Q2W |
|---|---|
| Week 4 | -82.91 ± 23.348 |
| Week 12 | -83.84 ± 45.263 |
| Week 20 | -161.38 ± 12.924 |
| Week 40 | -214.99 ± 13.628 |
| Week 48 | -226.58 ± 11.491 |
| Week 56 | -216.45 ± 16.165 |
| Week 64 | -216.76 ± 12.705 |
| Week 76 | -231.22 ± 15.964 |
| Week 88 | -237.78 ± 12.837 |
| Week 100 | -218.14 ± 15.340 |
| Week 112 | -233.91 ± 11.098 |
| Week 124 | -252.22 ± 8.312 |
| Week 136 | -267.89 ± 13.124 |
| Week 148 | -248.05 ± 12.653 |
| Week 160 | -248.47 ± 10.990 |
| percent change | Burosumab Q2W |
|---|---|
| Week 4 | -12.47 ± 16.620 |
| Week 12 | -5.00 ± 60.124 |
| Week 20 | -24.76 ± 18.883 |
| Week 40 | -36.25 ± 12.787 |
| Week 48 | -37.29 ± 13.936 |
| Week 56 | -35.80 ± 16.568 |
| Week 64 | -35.95 ± 14.851 |
| Week 76 | -38.36 ± 15.621 |
| Week 88 | -39.08 ± 12.987 |
| Week 100 | -37.42 ± 12.466 |
| Week 112 | -39.05 ± 13.808 |
| Week 124 | -43.11 ± 12.493 |
| Week 136 | -47.01 ± 11.752 |
| Week 148 | -43.47 ± 11.321 |
| Week 160 | -42.35 ± 13.574 |
Collected over From first dose of study drug through the end of the study (at Week 160). Maximum duration of exposure to study drug was 160 weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Burosumab Q2W | 0/13 (0%) | 1/13 (7.7%) | 13/13 (100%) |
| Event | Burosumab Q2W |
|---|---|
| Tooth AbscessInfections and infestations | 1/13 |
| Event | Burosumab Q2W |
|---|---|
| PyrexiaGeneral disorders | 11/13 |
| CoughRespiratory, thoracic and mediastinal disorders | 11/13 |
| Tooth AbscessInfections and infestations | 10/13 |
| Upper Respiratory Tract InfectionInfections and infestations | 9/13 |
| Pain In ExtremityMusculoskeletal and connective tissue disorders | 9/13 |
| Pharyngitis StreptococcalInfections and infestations | 8/13 |
| Nasal CongestionRespiratory, thoracic and mediastinal disorders | 8/13 |
| VomitingGastrointestinal disorders | 7/13 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 7/13 |
| DiarrhoeaGastrointestinal disorders | 6/13 |
| Age, Continuous(years) | Burosumab Q2W |
|---|---|
| Mean | 2.94 ± 1.146 |
| Sex: Female, Male(Participants) | Burosumab Q2W |
|---|---|
| Female | 4 |
| Male | 9 |
| Ethnicity (NIH/OMB)(Participants) | Burosumab Q2W |
|---|---|
| Hispanic or Latino | 2 |
| Not Hispanic or Latino | 11 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(Participants) | Burosumab Q2W |
|---|---|
| White | 12 |
| Black or African American | 1 |
| Serum Phosphorus(mg/dL) | Burosumab Q2W |
|---|---|
| Mean | 2.51 ± 0.284 |
| Rickets Severity Score (RSS) Total Score(units on a scale) | Burosumab Q2W |
|---|---|
| Mean | 2.92 ± 1.367 |
| Recumbent length/Standing height(cm) | Burosumab Q2W |
|---|---|
| Mean | 89.15 ± 7.597 |
| Recumbent length/Standing height (Z score)(Z score) | Burosumab Q2W |
|---|---|
| Mean | -1.378 ± 1.1947 |
2 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from May 2016 to primary completion in Apr 2017, 11 months.
Results were first posted in Jun 2018, 1 year 1 month after primary completion.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Burosumab
Familial Hypophosphatemic Rickets →