Clinical Trials on Autosomal Recessive Hypophosphatemic Rickets in United States

Total 59 results

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
Indiana University

Completed

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

Autosomal Dominant Hypophosphatemic Rickets

United States
Inozyme Pharma

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, United Kingdom, Canada, France, Germany
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
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
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
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
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
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
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
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
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.

Completed

A Study of KRN23 in X-linked Hypophosphatemia

X-linked Hypophosphatemia

United States
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 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, 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

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

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

X-linked Hypophosphatemia

United States, Korea, Republic of, Japan, Ireland, United Kingdom, France, Italy
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 Pharmaceutical Development Ltd

Completed

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

X-linked Hypophosphatemia

United Kingdom, France, Ireland, Italy
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.
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
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
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Hereditary Parkinson s Disease Natural History Protocol

Parkinson Disease 6, Early-Onset | Parkinson Disease (Autosomal Recessive, Early Onset) 7, Human | Parkinson Disease Autosomal Recessive, Early Onset | Parkinson Disease, Autosomal Recessive Early-Onset, Digenic, Pink1/Dj1

United States
Wayne State University

Unknown

Prenatal Computer-Aided Genetics Education Module (CAGEM)

Aneuploidy | Autosomal Recessive Disorder

United States
Otsuka Pharmaceutical Development & Commercialization...

Recruiting

A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Autosomal Recessive Polycystic Kidney (ARPKD)

United States, United Kingdom, Belgium, Poland, France, Germany, Italy
Kadmon Corporation, LLC

Completed

A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Polycystic Kidney, Autosomal Recessive

United States
Otsuka Pharmaceutical Development & Commercialization...

Recruiting

A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Autosomal Recessive Polycystic Kidney Disease (ARPKD)

United States, Belgium, Spain, Germany, United Kingdom, Poland
Newcastle-upon-Tyne Hospitals NHS Trust

Recruiting

Global Registry for COL6-related Dystrophies

Bethlem Myopathy | Ullrich Congenital Muscular Dystrophy 1, Digenic, Col6A1/Col6A2 | Ullrich Congenital Muscular Dystrophy 1, Autosomal Recessive | Ullrich Congenital Muscular Dystrophy 1, Autosomal Dominant | Bethlem Myopathy 1, Autosomal Recessive | UCMD | BTHLM1

United Kingdom
Origin Biosciences

Completed

Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

Molybdenum Cofactor Deficiency (MoCD) | Rare Autosomal Recessive Disorder | Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)

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
Alkeus Pharmaceuticals, Inc.

Enrolling by invitation

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

Stargardt Disease | Stargardt Macular Degeneration | Stargardt Macular Dystrophy | Autosomal Recessive Stargardt Disease 1 (ABCA4-related)

United States
Krystal Biotech, Inc.

Not yet recruiting

Topical KB105 for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

Autosomal Recessive Ichthyosis

United States
Clinic for Special Children

Recruiting

WiTNNess - TNNT1 Myopathy Natural History Study (WiTNNess)

Myopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANM

United States
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
Krystal Biotech, Inc.

Active, not recruiting

Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

TGM-1 Related Autosomal Recessive Congenital Ichthyosis

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
Alkeus Pharmaceuticals, Inc.

Recruiting

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

Stargardt Disease | Stargardt Macular Degeneration | Stargardt Macular Dystrophy | Autosomal Recessive Stargardt Disease 1 (ABCA4-related)

United States
Juan Pascual
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

Treatment Development of Triheptanoin (G1D)

Glucose Metabolism Disorders | Epilepsy | Glucose Transporter Type 1 Deficiency Syndrome | Glut1 Deficiency Syndrome 1, Autosomal Recessive | Glucose Transporter Protein Type 1 Deficiency Syndrome | Glucose Transport Defect | GLUT1DS1

United States
University of Texas Southwestern Medical Center
National Institute of Neurological Disorders and Stroke (NINDS)

Active, not recruiting

Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)

Glucose Metabolism Disorders | Epilepsy | Glucose Transporter Type 1 Deficiency Syndrome | Glut1 Deficiency Syndrome 1, Autosomal Recessive | Glucose Transporter Protein Type 1 Deficiency Syndrome | Glucose Transport Defect | GLUT1DS1

United States
CSL Behring

Completed

Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy

Common Variable Immunodeficiency | X-linked Agammaglobulinemia | Autosomal Recessive Agammaglobulinemia

Germany, United Kingdom, France, Italy, Poland, Romania, Spain, Sweden, Switzerland
National Institute of Allergy and Infectious Diseases...

Enrolling by invitation

pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

Chronic Granulomatous Disease | p47 | Autosomal Recessive

United States

Clinical Trials on Autosomal Recessive Hypophosphatemic Rickets in United States

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CROs in Mauritius

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Rare Diseases

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