Clinical trials: Hypophosphatemic Rickets
Asthma, Vitamin D Resistant Rickets
Assessment Of Vitamin D Role In The Pathogenesis Of Asthma In Vitamin D Resistent Patients
Research Title: Evaluation of airway reactivity, allergy and inflammatory mediators in exhaled breath condensate in vitamin D resistant rickets.
- Trial name
- NCT01578824
- Testing
- Methacholine Challenge Test
- Where
- Israel
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Rambam Health Care Campus
X-linked Hypophosphatemia, Hypophosphatemic Rickets
X-linked Hypophosphatemia Disease Monitoring Program
The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.
- Trial name
- NCT03651505
- Testing
- No intervention
- Where
- 38 sites in United States, Argentina and 4 more
- Volunteers
- 782 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
X-Linked Hypophosphatemia
Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH
The primary objective of this study is to evaluate the effect of KRN23 (burosumab) therapy in improving rickets in children with XLH compared with active control (oral phosphate/active vitamin D).
- Trial name
- NCT02915705
- Testing
- burosumab, Oral Phosphate Supplement, active vitamin D
- Where
- 16 sites in United States, Australia and 5 more
- Volunteers
- 61 people
- Sponsor
- Kyowa Kirin, Inc.
X-linked Hypophosphatemia
A Study of KRN23 in X-linked Hypophosphatemia
The primary objective of this study is to assess the safety and tolerability of KRN23 after a single intravenous (IV) and subcutaneous (SC) administration in XLH patients.
- Trial name
- NCT00830674
- Testing
- KRN23
- Where
- 4 sites in United States
- Volunteers
- 42 people
- Sponsor
- Kyowa Kirin Co., Ltd.
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With XLH
The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in adult Chinese patients with XLH
- Trial name
- NCT04842019
- Testing
- KRN23
- Where
- 5 sites in China
- Volunteers
- 18 people
- Sponsor
- Kyowa Kirin Co., Ltd.
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
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)
Effective Dosing of Burosumab in XLH
X-linked hypophosphataemia (XLH) is a rare, hereditary condition. The genetic defect leads to low blood phosphate levels and vitamin D suppression. Phosphate is required for strong bones and teeth and to store energy in cells.
- Trial name
- NCT07183579
- Where
- United Kingdom
- Volunteers
- 120 people
- Sponsor
- University of Nottingham
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency
The purpose of Study INZ701-108 (ENABLE) is to assess the safety and efficacy of INZ-701 in patients 1 year of age and older with ENPP1 Deficiency who have not previously received INZ-701 and are not eligible for existing…
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy 1
- Trial name
- NCT06739980
- Testing
- INZ-701
- Sponsor
- Inozyme Pharma
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
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age
A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Paediatric Patients from Birth to Less than 1 Year of Age with X-linked Hypophosphatemia…
- Trial name
- NCT04188964
- Testing
- Burosumab
- Where
- 9 sites in Austria, France and 4 more
- Volunteers
- 16 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
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
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 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
FGF23-related Hypophosphataemic Rickets and Osteomalacia
Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients
The objective of this post-marketing surveillance (PMS) study is to assess the safety and effectiveness of CRYSVITA injection 10, 20, and 30mg, equivalent to in routine clinical settings
- Trial name
- NCT06202027
- Testing
- FGF23-related hypophosphataemic rickets and osteomalacia
- Where
- 13 sites in South Korea
- Volunteers
- 100 people
- Sponsor
- Kyowa Kirin Korea Co., Ltd.
Hypophosphatemia, X-linked Hypophosphatemia
FGF23 and Angiotensin-(1-7) in Hypophosphatemia (GAP)
Hereditary hypophosphatemia encompasses rare genetic conditions characterized by renal phosphate wasting. Increased circulating levels of fibroblast growth factor 23 (FGF23), a key regulator of phosphorus metabolism, are critical…
- Conditions
- Hypophosphatemia, X-linked Hypophosphatemia, Renin-angiotensin System, Left Ventricular Hypertrophy
- Trial name
- GAP (NCT03489993)
- Testing
- Ang II and Ang-(1-7), FGF23 and klotho
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Wake Forest University Health Sciences
X-linked Hypophosphatemia
Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia
X-linked hypophosphatemia (XLH) is the most common form of heritable rickets. Current treatments include active vitamin D metabolites (e.g. calcitriol) and phosphate salts.
- Trial name
- NCT03820518
- Testing
- Calcitriol
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Peking Union Medical College 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.
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy 1
- Trial name
- NCT07473973
- Testing
- INZ-701
- Where
- 8 sites in Brazil, France and 6 more
- Volunteers
- 2 people
- Sponsor
- Inozyme Pharma
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International