Clinical Trials on Hereditary Hypophosphatemic Rickets With Hypercalciuria in United States

Total 77 results

Yale University
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Completed

Calcitonin for Treating X-linked Hypophosphatemia

Hypophosphatemic Rickets, X Linked Dominant

United States
Indiana University

Completed

Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot Project.

Autosomal Dominant Hypophosphatemic Rickets

United States
Massachusetts General Hospital
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Recruiting

Calcitriol Monotherapy for X-Linked Hypophosphatemia

X-linked Hypophosphatemia | Hypophosphatemic Rickets | Hypophosphatemic Rickets, X-Linked Dominant

United States
Inozyme Pharma
Engage Health Inc.; GACI Global

Completed

Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 Deficiency

Generalized Arterial Calcification in Infancy | Autosomal Recessive Hypophosphatemic Rickets Type 2

United States
Children's Mercy Hospital Kansas City

Completed

Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic Rickets

Hypophosphatemic Rickets, X-Linked Dominant

United States
Yale University
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Completed

Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic Rickets

Hyperparathyroidism | Hypophosphatemia, Familial

United States
Children's Mercy Hospital Kansas City

Withdrawn

Calcimimetics in Hypophosphatemic Rickets (CHR)

Familial Hypophosphatemic Rickets

United States
Redwood Dermatology Sciences
Ultragenyx Pharmaceutical Inc

Completed

Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets

Pain, Chronic | Hypophosphatemia | Hypophosphatemic Rickets

United States
Ultragenyx Pharmaceutical Inc
Kyowa Kirin Co., Ltd.

Active, not recruiting

X-linked Hypophosphatemia Disease Monitoring Program

X-linked Hypophosphatemia | Hypophosphatemic Rickets

United States, Brazil, Argentina, Canada, Chile, Colombia
Kyowa Kirin Pharmaceutical Development Ltd

Completed

A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH

X-Linked Hypophosphataemia

United Kingdom
Inozyme Pharma

Recruiting

The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

Generalized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency

United States, United Kingdom, Canada
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Efficacy and Safety of Burosumab (KRN23) Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH)

X-Linked Hypophosphatemia

Korea, Republic of, Canada, United States, Australia, Japan, United Kingdom, Sweden
Kyowa Hakko Kirin Pharma, Inc.
Kyowa Kirin Co., Ltd.

Completed

A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia

X-linked Hypophosphatemia

United States, Canada
Kyowa Hakko Kirin Pharma, Inc.

Completed

A Study of KRN23 in X-linked Hypophosphatemia

X-linked Hypophosphatemia

United States
Kyowa Kirin Pharmaceutical Development Ltd

Recruiting

A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

X-Linked Hypophosphatemia

France, Netherlands, United Kingdom
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)

X-Linked Hypophosphatemia

United States
Kyowa Kirin Pharmaceutical Development Ltd

Active, not recruiting

Registry for Patients With X-Linked Hypophosphatemia (XLH Registry)

X-Linked Hypophosphatemia

Spain, Italy, United Kingdom, France, Belgium, Denmark, Germany, Hungary, Portugal, Norway, Switzerland, Israel, Netherlands, Czechia, Bulgaria, Sweden, Slovenia, Ireland, Latvia, Slovakia
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

X-linked Hypophosphatemia

United States, Korea, Republic of, Japan, Ireland, United Kingdom, France, Italy
Inozyme Pharma

Recruiting

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency (ENERGY)

Generalized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

United States, United Kingdom
Inozyme Pharma

Active, not recruiting

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

Generalized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

United States, Canada, Germany, France, United Kingdom
Kyowa Hakko Kirin Pharma, Inc.
Kyowa Kirin Co., Ltd.

Completed

An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia

X-linked Hypophosphatemia

United States, Canada
Kyowa Kirin, Inc.

Completed

Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)

X-Linked Hypophosphatemia

United States
Kyowa Kirin Pharmaceutical Development Ltd

Completed

Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH

X-linked Hypophosphatemia

United Kingdom, France, Ireland, Italy
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)

X-linked Hypophosphatemia

United States, Korea, Republic of, Canada, Japan, France, Denmark
Yale University

Completed

Examining the Effect of Burosumab on Muscle Function

X-linked Hypophosphatemia

United States
National Human Genome Research Institute (NHGRI)

Completed

Study of People With Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)

Generalized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets Type2

United States
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)

X-linked Hypophosphatemia

Netherlands, United States, United Kingdom, France
Kyowa Kirin Pharmaceutical Development Ltd

Completed

Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

X-linked Hypophosphatemia (XLH)

Sweden, Spain, United Kingdom, Italy, Austria, France
Wake Forest University Health Sciences

Completed

FGF23 and Angiotensin-(1-7) in Hypophosphatemia (GAP) (GAP)

Hypophosphatemia | X-linked Hypophosphatemia | Left Ventricular Hypertrophy | Renin-angiotensin System

United States
Medialis Ltd.
Metabolic Support UK

Completed

Retrospective and Prospective Disease Progression and Quality of Life in XLH

X-linked Hypophosphatemia (XLH)

United Kingdom
National Institute of Dental and Craniofacial Research...

Terminated

Cinacalcet for Fibroblast Growth Factor 23 (FGF23)-Mediated Hypophosphatemia (Hypophosphatemic Rickets)

Osteomalacia

United States
Yale University
National Center for Advancing Translational Sciences (NCATS)

Suspended

The Impact of Phosphate Metabolism on Healthy Aging

Rickets | Hypophosphatemia | Hypercalciuria | XLH | HHRH

United States
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
University of Wisconsin, Madison

Completed

Does Treatment of Hypovitaminosis D Increase Calcium Absorption?

Vitamin D Deficiency | Osteoporosis | Osteopenia | Hypoparathyroidism | Hypercalciuria | Hypercalcemia

United States
Washington University School of Medicine

Completed

Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy (RVCL)

Vasculopathy, Retinal, With Cerebral Leukodystrophy | Cerebroretinal Vasculopathy, Hereditary

United States
Akcea Therapeutics

Recruiting

TEGSEDI Pregnancy Surveillance Program

Pregnancy | Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy

United States
Pharvaris Netherlands B.V.

Active, not recruiting

Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II (HAE CHAPTER-1)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

United States, Poland, Germany, Austria, Bulgaria, Canada, Ireland, Italy, United Kingdom
Pharvaris Netherlands B.V.

Completed

Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (RAPIDe-1)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

Bulgaria, United States, Spain, Israel, Germany, Canada, Czechia, France, Hungary, Italy, Netherlands, Poland, United Kingdom
Pharvaris Netherlands B.V.

Recruiting

Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema (RAPIDe-3)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

United States, Puerto Rico
Pharvaris Netherlands B.V.

Recruiting

Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (RAPIDe-2)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

United States, Bulgaria, Czechia, Hungary, Spain, France, Germany, Poland, Canada, Israel
Akcea Therapeutics

Recruiting

A Clinical Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Patients With hATTR-PN

Hereditary Transthyretin Amyloidosis With Polyneuropthy

United States, Canada
Columbia University
Ludwig-Maximilians - University of Munich; Moorfields Eye Hospital NHS Foundation... and other collaborators

Recruiting

CNGB1 and Allied Disorders

Retinitis Pigmentosa Associated With CNGB1 Mutations

United States, France, Germany, United Kingdom
National Cancer Institute (NCI)

Completed

Relationship Stressors in Parents of Children With Cancer or Neurofibromatosis Type 1 (NF1)

Parents of Children With Cancer | Parents of Children With NF1 | Parents of Children With Neurofibromatosis Type I

United States, Canada
Akcea Therapeutics
United BioSource, LLC

Recruiting

A Non-interventional Cohort Safety Study of Patients With hATTR-PN

Hereditary Transthyretin Amyloidosis With Polyneuropthy

United States, France, Greece, Italy, Spain, Canada, Germany, Portugal
Ultragenyx Pharmaceutical Inc
Newcastle University

Completed

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Nonaka Disease | Quadriceps Sparing Myopathy (QSM) | Distal Myopathy With Rimmed Vacuoles (DMRV)

United States, Bulgaria, Canada, France, United Kingdom
Ultragenyx Pharmaceutical Inc

Terminated

A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

Hereditary Inclusion Body Myopathy | GNE Myopathy | Inclusion Body Myopathy 2 | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

Canada, United States, Bulgaria
NYU Langone Health
Bristol-Myers Squibb

Completed

A Pilot Study of the Safety and Efficacy of Abatacept Injections in the Treatment of Behcet's Syndrome

Female Patients With Behcet's Syndrome

United States
Ultragenyx Pharmaceutical Inc

Completed

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (GNEM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Ultragenyx Pharmaceutical Inc

Terminated

Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Jazz Pharmaceuticals

Recruiting

Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

Seizure in Participants With Tuberous Sclerosis Complex | Seizure in Participants With Dravet Syndrome | Seizure in Participants With Lennox-Gastaut Syndrome

United States

Clinical Trials on Hereditary Hypophosphatemic Rickets With Hypercalciuria in United States

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