Clinical trials: X-linked Hypophosphatemia
X-Linked Hypophosphatemia
A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth
An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.
- Trial name
- NCT05181839
- Testing
- Burosumab
- Where
- 14 sites in France, Germany and 3 more
- Volunteers
- 25 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Muscle Weakness, XLH
Muscle Abnormalities in Children With XLH
XLH rickets is a rare disease with muscle weakness. Fat parameters such as IMAT and intraMAT could be increased in this disease.
- Trial name
- MDmuscleXLH (NCT05312086)
- Testing
- MRI
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
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 (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
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.
XLH
A Study of KRN23 in Adult and Pediatric Patients With X-linked Hypophosphatemic Rickets/Osteomalacia
Before switching to the post-marketing study: Assess the efficacy and safety of KRN23 administered subcutaneously once every 4 or 2 weeks in adult or children with XLH After switching to the post-marketing study: To evaluate the…
- Trial name
- NCT04308096
- Testing
- KRN23
- Where
- 10 sites in Japan, South Korea
- Volunteers
- 27 people
- Sponsor
- Kyowa Kirin Co., Ltd.
X-Linked Hypophosphatemia
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
The primary objectives of the study are to: Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers…
- Trial name
- NCT02750618
- Testing
- Burosumab
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin, Inc.
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.
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 (XLH)
Prevention of Spontaneous Dental Abscesses in Children With X-linked Hypophosphatemia : a RCT
This study is a prospective, randomized, single-blind, split-mouth, national multicenter trial, comparing the efficacy of a self-etch adhesive system combined (SAM) with a flowable composite to that of a fluoride varnish for the…
- Trial name
- PRADEX (NCT04872907)
- Testing
- adhesive system, fluoride varnish, flowable composite
- Volunteers
- 60 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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, Hypophosphatemic Rickets
Calcitriol Monotherapy for X-Linked Hypophosphatemia
Children and adults with XLH recruited will be treated with calcitriol alone (without phosphate supplementation) for one year, during which the calcitriol dose will be escalated during the first 3 months of therapy.
- Conditions
- X-linked Hypophosphatemia, Hypophosphatemic Rickets, Hypophosphatemic Rickets, X-Linked Dominant
- Trial name
- NCT03748966
- Testing
- Calcitriol
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Massachusetts General Hospital
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
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association
Rare Diseases, X-linked Hypophosphatemia
Burosumab in Children and Adolescents With X-linked Hypophosphatemia
In this prospective longitudinal cohort study we studied the efficacy and safety of burosumab in real-clinical practice for <13- and >13-years old children affected with X-linked hypophosphatemia.
- Trial name
- NCT04419363
- Testing
- Burosumab Injection
- Where
- France
- Volunteers
- 57 people
- Sponsor
- Bicetre Hospital
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
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
The objectives of the study are to: Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants Establish the safety profile of burosumab for the treatment of…
- Trial name
- NCT02163577
- Testing
- burosumab
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 52 people
- Sponsor
- Kyowa Kirin, Inc.
Hypophosphatemia, Familial, Hyperparathyroidism
Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic Rickets
The purpose of this study is to determine the effectiveness of paricalcitol, a form of synthetic vitamin D, in lowering parathyroid hormone (PTH) levels and reducing disease symptoms in children and adults with X-linked…
- Trial name
- NCT00417612
- Testing
- Paricalcitol
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Yale University
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