Familial Hypophosphatemic Rickets clinical trials
The registry lists 49 studies for Familial Hypophosphatemic Rickets across more than 10 countries. 8 are recruiting or about to start. 43 of them are mainly about Familial Hypophosphatemic Rickets (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06.
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Who runs them
Kyowa Kirin Co., Ltd. (10) · Kyowa Kirin, Inc. (6) · Kyowa Kirin Pharmaceutical Development Ltd (5) · Hospices Civils de Lyon (3) · Yale University (3) · Bicetre Hospital (2) · Assistance Publique - Hôpitaux de Paris (2) · Wuerzburg University Hospital (2)
What is being tested here
Drugs under test in Familial Hypophosphatemic Rickets studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Burosumab | 0 | 6 | 18 |
| Calcitriol | 0 | 0 | 2 |
| Cinacalcet | 0 | 0 | 2 |
| Paricalcitol | 0 | 1 | 1 |
| Salmon calcitonin | 0 | 0 | 1 |
| Growth Hormone | 0 | 0 | 1 |
| Human Growth Hormone | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The study
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
Who these studies take
Of the 4 Familial Hypophosphatemic Rickets studies recruiting now, 2 (50%) set no upper age limit and 2 (50%) stop at 75 or younger. 3 (75%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (25%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 3 (75%) | 3 (75%) |
| 18 | 4 (100%) | 4 (100%) |
| 30 | 3 (75%) | 3 (75%) |
| 40 | 3 (75%) | 3 (75%) |
| 50 | 3 (75%) | 3 (75%) |
| 65 | 3 (75%) | 3 (75%) |
| 75 | 2 (50%) | 2 (50%) |
| 85 | 2 (50%) | 2 (50%) |
Across all 49 studies, recruiting or not: 57% stop at 75 or younger and 53% at 65; 63% take children; 0% are for women only and 0% for men only; 14% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 49 of 49. search them.
- A First-in-human Study of KK8123 in Adults With X-linked HypophosphatemiaRecruitingNCT06525636Phase 1/25 countries
- Phosphorus-31 Spectroscopy in Phosphate DiabetesRecruitingNCT06921720Not applicable1 country
- Registry for Patients With X-Linked HypophosphatemiaRecruitingNCT0319347620 countries
- Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical DiagnosisNot yet recruitingNCT07666269Not applicable1 country
- Effective Dosing of Burosumab in XLHRecruitingNCT071835791 country
- Expanded Access to BurosumabAvailableNCT03775187
- Effect of Burosumab on the Inflammatory Profile of Patients With X-linked Hypophosphatemic Rickets FLAM-XLHNot yet recruitingNCT06248632
- Prevention of Spontaneous Dental Abscesses in Children With X-linked Hypophosphatemia : a RCTNot yet recruitingNCT04872907Phase 3
- Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked HypophosphatemiaWithdrawnNCT076076008 countries
- X-linked Hypophosphatemia Disease Monitoring ProgramActive, not recruitingNCT036515056 countries
- A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal GrowthCompletedNCT051818395 countries
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Calcitriol Monotherapy for X-Linked HypophosphatemiaCompletedNCT03748966Early Phase 11 country
- Study of Longitudinal Observation for Patient With X-linked Hypophosphatemic Rickets/Osteomalacia in Collaboration With Asian PartnersActive, not recruitingNCT037455211 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Burden of Disease and Functional Impairment in XLHActive, not recruitingNCT049464091 country
- Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of AgeCompletedNCT04188964Phase 1/26 countries
- Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLHCompletedNCT02915705Phase 37 countries
- A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLHCompletedNCT048194901 country
- Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With XLHCompletedNCT04842019Phase 41 country
- Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLHCompletedNCT04842032Phase 41 country
- Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)CompletedNCT02537431Phase 36 countries
- Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)CompletedNCT02526160Phase 37 countries
- A Repeated Study of KRN23 in Adults With X-Linked HypophosphatemiaCompletedNCT01340482Phase 1/22 countries
- An Extension Study of KRN23 in Adults With X-Linked HypophosphatemiaCompletedNCT01571596Phase 1/22 countries
- A Study of KRN23 in X-linked HypophosphatemiaCompletedNCT00830674Phase 11 country
- Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)CompletedNCT02750618Phase 21 country
- Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)CompletedNCT02312687Phase 21 country
- Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)CompletedNCT02163577Phase 24 countries
- Characterising Pain, QoL, Body Composition, Arterial Stiffness, Muscles and Bones in Adult Persons With XLH and Healthy ControlsCompletedNCT042734901 country
- Retrospective and Prospective Disease Progression and Quality of Life in XLHCompletedNCT040498771 country
- Foot Disorders in X-linked HypophosphatemiaCompletedNCT060679321 country
- Examining the Effect of Burosumab on Muscle FunctionCompletedNCT04146935Phase 41 country
- Anti-FGF23 (Burosumab) in Adult Patients With XLHCompletedNCT04695860Phase 31 country
- A Study of KRN23 in Pediatric Patients With X-linked Hypophosphatemic Rickets/OsteomalaciaCompletedNCT03233126Phase 31 country
- FGF23 and Angiotensin-(1-7) in Hypophosphatemia (GAP)CompletedNCT034899931 country
- Dental Implants in Patients With X-linked HypophosphatemiaStatus unknownNCT038799151 country
- Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLHCompletedNCT03920072Phase 34 countries
- Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic RicketsCompletedNCT00195936Phase 11 country
- Calcimimetics in Hypophosphatemic RicketsWithdrawnNCT00844740Not applicable1 country
- X-linked Hypophosphatemia and FGF21CompletedNCT035965541 country
- Burosumab in Children and Adolescents With X-linked HypophosphatemiaStatus unknownNCT04419363Phase 41 country
- Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic RicketsCompletedNCT00417612Phase 31 country
- Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked HypophosphatemiaStatus unknownNCT03820518Phase 41 country
- Calcitonin for Treating X-linked HypophosphatemiaCompletedNCT01652573Not applicable1 country
- A Study of KRN23 in Subjects With X-linked Hypophosphatemic Rickets/OsteomalaciaCompletedNCT02181764Phase 12 countries
- Growth Hormone Treatment in Children With Hypophosphatemic RicketsCompletedNCT02720770Phase 1/2
- Assessment Of Vitamin D Role In The Pathogenesis Of Asthma In Vitamin D Resistent PatientsCompletedNCT01578824Not applicable1 country
- Effects of GH on Body Proportions and Final Height in X-Linked Hypophosphatemic RicketsStatus unknownNCT00473187Phase 11 country