Clinical trials: X-linked Hypophosphatemia
Osteogenesis Imperfecta, Rare Bone Disorders
Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis
MOSAIC aims to determine whether oro-dental morphological anomalies, particularly palatal morphology, associated with rare bone and cartilage diseases can be precisely characterized using 3D digital models analysed through…
- Conditions
- Osteogenesis Imperfecta, Rare Bone Disorders, Hypophosphatemia, X-Linked
- Trial name
- MOSAIC (NCT07666269)
- Testing
- intra-oral 3D optical impression
- Where
- France
- Volunteers
- 240 people, healthy volunteers welcome
- Sponsor
- University Hospital, Bordeaux
X-linked Hypophosphatemic Rickets/Osteomalacia
A Study of KRN23 in Subjects With X-linked Hypophosphatemic Rickets/Osteomalacia
The objective of this study is to assess the safety and tolerability of KRN23 after a single subcutaneous (SC) administration in subjects with X-linked hypophosphatemic rickets/osteomalacia (XLH) in Japan or Korea.
- Trial name
- NCT02181764
- Testing
- KRN23
- Where
- 2 sites in Japan, South Korea
- Volunteers
- 15 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Hypophosphatemic Rickets, X-Linked Dominant
Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic Rickets
This study will measure the effect of cinacalcet (Sensipar) on parathyroid hormone (PTH) secretion in children and adolescents with hypophosphatemic rickets (XLH).
- Trial name
- NCT00195936
- Testing
- Cinacalcet
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Children's Mercy Hospital Kansas City
X Linked Hypophosphatemia
Burden of Disease and Functional Impairment in XLH
Observational study comprising prospective follow up as well as retrospective chart review in order to evaluate the longitudinal course of the disease in XLH patients with a specific focus on functional impairment, physical…
- Trial name
- IdeFIX (NCT04946409)
- Testing
- no intervention
- Where
- Germany
- Volunteers
- 60 people
- Sponsor
- Wuerzburg University Hospital
X-linked Hypophosphatemia (XLH)
Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia
The purpose of this study is to better understand how X-linked hypophosphatemia (XLH) affects the body and daily life. Phosphate levels are critical in managing XLH, we aim to study how these levels change in patients taking…
- Trial name
- IBIS XLH (NCT07607600)
- Testing
- No treatment given
- Where
- 23 sites in United States, Australia and 6 more
- Sponsor
- Novo Nordisk A/S
X-linked Hypophosphatemia, Hereditary Hypophosphatemia
Characterising Pain, QoL, Body Composition, Arterial Stiffness, Muscles and Bones in Adult Persons With XLH and Healthy Controls
Hereditary hypophosphatemia (XLH) is a rare, inherited disease. Loss-of-function mutation in the phosphate regulating gene with homologies to endopeptidases on the X-chromosome (PHEX) results in excess fibroblast growth factor 23…
- Trial name
- NCT04273490
- Where
- Denmark
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- University of Aarhus
Hypophosphatemic Rickets, Growth Disorders
Effects of GH on Body Proportions and Final Height in X-Linked Hypophosphatemic Rickets
X-linked hypophosphatemic rickets (XLH) is characterized by rickets, disproportionate short stature, impaired renal phosphate reabsorption and vitamin D metabolism.
- Conditions
- Hypophosphatemic Rickets, Growth Disorders, Somatropin Therapy
- Trial name
- NCT00473187
- Testing
- somatropin
- Where
- Germany
- Sponsor
- University of Rostock
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH
The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in pediatric Chinese patients with XLH
- Trial name
- NCT04842032
- Testing
- KRN23
- Where
- 5 sites in China
- Volunteers
- 28 people
- Sponsor
- Kyowa Kirin Co., Ltd.
X-linked Hypophosphatemia
A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia
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).
- Trial name
- NCT01340482
- Testing
- KRN23
- Where
- 6 sites in United States, Canada
- Volunteers
- 29 people
- Sponsor
- Kyowa Kirin Co., Ltd.
X-linked Hypophosphatemia
Dental Implants in Patients With X-linked Hypophosphatemia
X-linked hypophosphatemia (XLH) is a rare genetic skeletal disease where increased phosphate wasting in the kidney leads to hypophosphatemia and prevents normal mineralization of bone and dentin, with osteomalacia as a principal…
- Trial name
- IMPLANTS-XLH (NCT03879915)
- Testing
- Dental Implant placement
- Where
- France
- Volunteers
- 36 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
X-Linked Hypophosphatemic Rickets
Effect of Burosumab on the Inflammatory Profile of Patients With X-linked Hypophosphatemic Rickets FLAM-XLH
X-linked hypophosphataemia (XLH) is a rare genetic disorder associated with increased circulating levels of the hormone FGF23, most commonly through mutation of the PHEX gene.
- Trial name
- FLAM-XLH (NCT06248632)
- Testing
- Expression of inflammatory markers (Il6, Il8, Il1β, CXCL1, CCL2, CXCR3, Il1R, Il6R)
- Volunteers
- 20 people
- Sponsor
- Hospices Civils de Lyon
X-Linked Hypophosphatemia
Study of Longitudinal Observation for Patient With X-linked Hypophosphatemic Rickets/Osteomalacia in Collaboration With Asian Partners
Through observation of patients with X-linked hypophosphatemic rickets/osteomalacia (XLH) for up to 10 years, the study intends to collect data that allow achievement of the following objectives: 1.
- Trial name
- SUNFLOWER (NCT03745521)
- Testing
- no intervention
- Where
- Japan
- Volunteers
- 226 people
- Sponsor
- Kyowa Kirin Co., Ltd.
X Linked Hypophosphatemic Rickets
Growth Hormone Treatment in Children With Hypophosphatemic Rickets
This study evaluates the effect on height of a two year treatment with growth hormone in 19 children with X linked hypophosphatemic rickets.
- Trial name
- GH-XLH (NCT02720770)
- Testing
- norditropine simplex
- Volunteers
- 19 people
- Sponsor
- Bicetre Hospital
X-Linked Hypophosphatemia
Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)
The primary objectives of this study are to: Assess the long-term safety of KRN23 subcutaneous (SC) administration in adult subjects with XLH Assess the proportion of subjects achieving serum phosphorus levels in the normal range…
- Trial name
- NCT02312687
- Testing
- KRN23
- Where
- 5 sites in United States
- Volunteers
- 20 people
- Sponsor
- Kyowa Kirin, Inc.
Familial Hypophosphatemic Rickets
Calcimimetics in Hypophosphatemic Rickets
Currently, large oral doses of phosphate and 1,25(OH)2D (calcitriol) are the standard treatment of patients with familial hypophosphatemic rickets (XLH).
- Trial name
- CHR (NCT00844740)
- Testing
- Cinacalcet
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Children's Mercy Hospital Kansas City
X-linked Hypophosphatemia
Anti-FGF23 (Burosumab) in Adult Patients With XLH
X-linked hypophosphatemia (XLH) rare genetic disorder due by inactivating mutations in the PHEX gene leading to increased levels in FGF-23.
- Trial name
- BurGER (NCT04695860)
- Testing
- Burosumab
- Where
- Germany
- Volunteers
- 36 people
- Sponsor
- Wuerzburg University Hospital
X-linked Hypophosphatemic Rickets/Osteomalacia
A Study of KRN23 in Pediatric Patients With X-linked Hypophosphatemic Rickets/Osteomalacia
Before switching to the post-marketing study: To evaluate the efficacy and safety of KRN23 administered subcutaneously once every 2 weeks in children with X-linked hypophosphatemic rickets/osteomalacia(XLH).
- Trial name
- NCT03233126
- Testing
- KRN23
- Where
- 4 sites in Japan
- Volunteers
- 16 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
X-linked Hypophosphatemia
An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia
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).
- Trial name
- NCT01571596
- Testing
- KRN23
- Where
- 6 sites in United States, Canada
- Volunteers
- 23 people
- Sponsor
- Kyowa Kirin Co., Ltd.
X-Linked Hypophosphataemia
A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH
This study aims to provide Health-related Quality of Life (HRQoL) data from children and adolescents with growing skeletons in the United Kingdom (UK), including those treated with burosumab or alternative XLH treatment, as part…
- Trial name
- NCT04819490
- Where
- 6 sites in United Kingdom
- Volunteers
- 32 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd