Clinical trials: Hypophosphatemic Rickets
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
X-linked Hypophosphatemia
Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)
The primary efficacy objective of this study is to establish the effect of burosumab treatment compared with placebo on increasing serum phosphorus levels in adults with XLH.
- Trial name
- NCT02526160
- Testing
- burosumab
- Where
- 25 sites in United States, France and 5 more
- Volunteers
- 134 people
- Sponsor
- Kyowa Kirin, Inc.
Generalized Arterial Calcification of Infancy, Autosomal Recessive Hypophosphatemic Rickets Type2
Study of People With Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)
Background: Generalized Arterial Calcification of Infancy (GACI) is a very rare disorder. It can be fatal before birth or by age 6 months. Anumber of people with GACI survive into adulthood.
- Trial name
- NCT03478839
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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, Tumor-Induced Osteomalacia
Expanded Access to Burosumab
Individual patient expanded access requests may be considered for patients who have no other treatment options
- Trial name
- NCT03775187
- Testing
- Burosumab
- Sponsor
- Kyowa Kirin Co., Ltd.
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency
- Trial name
- ENERGY (NCT05734196)
- Testing
- INZ-701
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Inozyme Pharma
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
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.
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.
Hypophosphatemia, Familial, Rickets
Hypophosphatemic Rickets in Norway
The purpose of the study is to do a follow-up survey of all individuals with hereditary hypophosphatemia in Norway, focusing on manifestations in childhood and adolescence.
- Conditions
- Hypophosphatemia, Familial, Rickets, Hyperphosphatemia
- Trial name
- NCT01057186
- Testing
- Alfacalcidol; phosphate., Sevelamer
- Where
- Norway
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Haukeland University Hospital
X-linked Hypophosphatemia
Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH
This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab…
- Trial name
- NCT03920072
- Testing
- Burosumab
- Where
- 10 sites in France, Ireland and 2 more
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Hypophosphatemic Rickets
Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts
Fibroblast growth factor 23 (FGF23) is the cornerstone of phosphate / calcium / vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a Phosphating agent, inhibitor of dihydroxyvitamin D, and inhibitor of…
- Trial name
- HYPO-CLASTE (NCT04184661)
- Testing
- blood sample
- Where
- 3 sites in France
- Volunteers
- 52 people
- Sponsor
- Hospices Civils de Lyon
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
Craniosynostoses
Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts
FGF23 is the cornerstone of phosphate / calcium / vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a phosphaturizing agent, inhibitor of dihydroxyvitamin D, and inhibitor of synthesis and secretion of PTH…
- Trial name
- HYPO-BLASTES (NCT04159675)
- Testing
- osteoblast biology study
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Hospices Civils de Lyon
Generalized Arterial Calcification in Infancy, Autosomal Recessive Hypophosphatemic Rickets Type 2
Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 Deficiency
To date, the investigators lack characterization of, the burden of, and the systemic progression of disease in ENPP1 deficiency and ABCC6 deficiency from a patient and/or parent perspective.
- Trial name
- NCT04372446
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Inozyme Pharma