31 completed, 6 unknown, 4 active not recruiting, 4 recruiting. 9 Phase 1, 8 Phase 2, 8 Phase 3, 5 Not applicable, 5 Phase 4.
Follow Familial Hypophosphatemic Rickets: new trials by email Literature Review of Familial Hypophosphatemic Rickets trials Research Report on Familial Hypophosphatemic Rickets
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis intra-oral 3D optical impression - Osteogenesis Imperfecta, Rare Bone Disorders | Not applicable | Not yet recruiting | University Hospital, Bordeaux | 240 | 2026-09-01 | 2026-06-24 |
| Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia No treatment given - X-linked Hypophosphatemia (XLH) | Withdrawn | Novo Nordisk A/S | 2026-07-07 | 2026-08-28 | ||
| Effective Dosing of Burosumab in XLH X-linked Hypophosphatemia (XLH) | Recruiting | University of Nottingham | 120 | 2025-11-03 | 2026-05-11 | |
| Phosphorus-31 Spectroscopy in Phosphate Diabetes intra-muscular ATP values in phosphate diabetes, intra-muscular phosphate values in phosphate diabetes - Phosphate Diabetes, X-linked Hypophosphatemia | Not applicable | Recruiting | Hospices Civils de Lyon | 65 | 2025-05-16 | 2026-07-23 |
| A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia KK8123 - X-linked Hypophosphatemia | Phase 1 / Phase 2 | Recruiting | Kyowa Kirin Co., Ltd. | 39 | 2024-10-09 | 2026-09-21 |
| Effect of Burosumab on the Inflammatory Profile of Patients With X-linked Hypophosphatemic Rickets FLAM-XLH Expression of inflammatory markers (Il6, Il8, Il1β, CXCL1, CCL2, CXCR3, Il1R, Il6R) - X-Linked Hypophosphatemic Rickets | Unknown | Hospices Civils de Lyon | 20 | 2024-04-01 | 2024-03-06 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth Burosumab - X-Linked Hypophosphatemia | Completed | Kyowa Kirin Pharmaceutical Development Ltd | 25 | 2021-11-24 | 2026-07-13 | |
| Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH KRN23 - X-linked Hypophosphatemia (XLH) | Phase 4 | Completed | Kyowa Kirin Co., Ltd. | 28 | 2021-11-01 | 2024-07-09 |
| Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With XLH KRN23 - X-linked Hypophosphatemia (XLH) | Phase 4 | Completed | Kyowa Kirin Co., Ltd. | 18 | 2021-09-28 | 2024-07-09 |
| Prevention of Spontaneous Dental Abscesses in Children With X-linked Hypophosphatemia : a RCT adhesive system, fluoride varnish, flowable composite - X-linked Hypophosphatemia (XLH) | Phase 3 | Unknown | Assistance Publique - Hôpitaux de Paris | 60 | 2021-06-01 | 2021-05-05 |
| A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH X-Linked Hypophosphataemia | Completed | Kyowa Kirin Pharmaceutical Development Ltd | 32 | 2021-04-01 | 2024-07-25 | |
| Anti-FGF23 (Burosumab) in Adult Patients With XLH Burosumab - X-linked Hypophosphatemia | Phase 3 | Completed | Wuerzburg University Hospital | 36 | 2021-01-07 | 2023-05-03 |
| Burden of Disease and Functional Impairment in XLH no intervention - X Linked Hypophosphatemia | Active not recruiting | Wuerzburg University Hospital | 60 | 2020-10-01 | 2025-07-20 | |
| Foot Disorders in X-linked Hypophosphatemia Foot X rays, Clinical examination + scores - X-linked Hypophosphatemia | Completed | Orthopedic Hospital Vienna Speising | 28 | 2020-07-01 | 2023-10-10 | |
| Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age Burosumab - X-linked Hypophosphatemia (XLH) | Phase 1 / Phase 2 | Completed | Kyowa Kirin Pharmaceutical Development Ltd | 16 | 2020-02-26 | 2024-11-07 |
| Characterising Pain, QoL, Body Composition, Arterial Stiffness, Muscles and Bones in Adult Persons With XLH and Healthy Controls X-linked Hypophosphatemia, Hereditary Hypophosphatemia | Completed | University of Aarhus | 92 | 2020-02-18 | 2023-11-29 | |
| Examining the Effect of Burosumab on Muscle Function Burosumab Injection [Crysvita] - X-linked Hypophosphatemia | Phase 4 | Completed | Yale University | 10 | 2019-11-13 | 2023-08-24 |
| Dental Implants in Patients With X-linked Hypophosphatemia Dental Implant placement - X-linked Hypophosphatemia | Unknown | Assistance Publique - Hôpitaux de Paris | 36 | 2019-09-27 | 2022-06-30 | |
| Retrospective and Prospective Disease Progression and Quality of Life in XLH None - Observational study - X-linked Hypophosphatemia (XLH) | Completed | Medialis Ltd. | 11 | 2019-07-07 | 2023-10-23 |
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Hypophosphatemia (8) · Rickets, Hypophosphatemic (5) · Rickets (4) · Hyperparathyroidism (3) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (2) · Acidemia, isovaleric (2) · Anemia, Sickle Cell (2) · Argininosuccinic Aciduria (2) · Ataxia with vitamin E deficiency (2) · Basal ganglia disease, biotin-responsive (2) · Beta ketothiolase deficiency (2) · Biotinidase Deficiency (2) · Bone Diseases (2) · Brown-Vialetto-Van Laere syndrome (2)
Avitaminosis (49) · Calcium Metabolism Disorders (49) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (49) · Deficiency Diseases (49) · Female Urogenital Diseases (49) · Female Urogenital Diseases and Pregnancy Complications (49) · Genetic Diseases, Inborn (49) · Male Urogenital Diseases (49) · Malnutrition (49) · Metabolism, Inborn Errors (49) · Metal Metabolism, Inborn Errors (49) · Nutrition Disorders (49) · Nutritional and Metabolic Diseases (49) · Phosphorus Metabolism Disorders (49) · Renal Tubular Transport, Inborn Errors (49)
burosumab (20) · Calcitriol (2) · Cinacalcet (2) · 1,25-dihydroxyvitamin D (1) · Growth Hormone (1) · Human Growth Hormone (1) · Interleukin-6 (1) · Interleukin-8 (1) · Physical Examination (1) · flowable hybrid composite (1) · paricalcitol (1) · salmon calcitonin (1)
Kyowa Kirin Co., Ltd. (10) · Kyowa Kirin, Inc. (6) · Kyowa Kirin Pharmaceutical Development Ltd (5) · Hospices Civils de Lyon (3) · Yale University (3) · Assistance Publique - Hôpitaux de Paris (2) · Bicetre Hospital (2) · Children's Mercy Hospital Kansas City (2) · Wuerzburg University Hospital (2) · Centre Hospitalier Universitaire de Liege (1) · Massachusetts General Hospital (1) · Medialis Ltd. (1) · Novo Nordisk A/S (1) · Orthopedic Hospital Vienna Speising (1) · Peking Union Medical College Hospital (1)
United States (20) · France (14) · United Kingdom (10) · Canada (7) · Germany (7) · Japan (7) · Italy (5) · Netherlands (4) · South Korea (4) · Spain (4) · China (3) · Denmark (3) · Ireland (3) · Sweden (3) · Australia (2)
Amy Zhang, PhD (2) · Andrew M South, MD MS (1) · Curt Scharfe, MD, PhD (1) · Dieter Haffner, MD (1) · Dirk Schnabel, MD (1) · Eva Liu, MD (1) · Gabriel Mindler, Dr (1) · Hagen Staude (1) · James Law, BMBS, PhD, MRCPCH (1) · Karl L Insogna, M.D. (1) · Karl L Insogna, MD (1)
PMID 39151033, PMID 32721016, PMID 40962182, PMID 25919461, PMID 36854566, PMID 24569459, PMID 25029424, PMID 26073451, PMID 28822957, PMID 29791829, PMID 29947083, PMID 30638856, PMID 31104833, PMID 31165191, PMID 31315600, PMID 32601114, PMID 33484279, PMID 34548383, PMID 34636899, PMID 35142618, PMID 35533340, PMID 35775373, PMID 35927518, PMID 36072994, PMID 36082134, PMID 36719566, PMID 37084401, PMID 37752558, PMID 40074938, PMID 41030383, PMID 41634723, PMID 41860923, PMID 41911946, PMID 42038817, PMID 42044650, PMID 42756680
2014 (1), P50AR054086 (1), R21AR061818 (1)