Clinical Trials on Monocarboxylate Transporter 8 Deficiency in New Zealand

Total 28 results

Ultragenyx Pharmaceutical Inc

Completed

Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

United States, Spain, Australia, France, Israel, Italy, United Kingdom
Ultragenyx Pharmaceutical Inc

Terminated

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

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

Recruiting

Study of Voicing My CHOiCES as a Tool for Advanced Care Planning in Young Adults With Cancer

Cancer | HIV | Chronic Granulomatous Disease | Dock 8 Deficiency

United States
Ultragenyx Pharmaceutical Inc

Terminated

Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome

United States, Spain, Denmark, United Kingdom, Australia
Yale University

Completed

Examining the Effect of Burosumab on Muscle Function

X-linked Hypophosphatemia

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
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
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 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
Memorial Sloan Kettering Cancer Center
National Cancer Institute (NCI); Miltenyi Biotec, Inc.

Completed

Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine

Fanconi Anemia

United States
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
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
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, 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
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, 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, 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
Mayo Clinic

Recruiting

Prospective Research Rare Kidney Stones (ProRKS) (ProRKS)

Cystinuria | Hyperoxaluria | Dent Disease | Lowe Syndrome | Adenine Phosphoribosyltransferase Deficiency

United States, Canada, Iceland, Israel
NYU Langone Health
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Completed

Health-related Quality of Life in Rare Kidney Stone

Cystinuria | Primary Hyperoxaluria | Dent Disease | Adenine Phosphoribosyl Transferase Deficiency

United States, Iceland
Foresee Pharmaceuticals Co., Ltd.

Recruiting

A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia

Fanconi Anemia

United States
Mayo Clinic
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Recruiting

Rare Kidney Stone Consortium Patient Registry (RKSC)

Cystinuria | Primary Hyperoxaluria | Dent Disease | APRT Deficiency

United States, Iceland
Memorial Sloan Kettering Cancer Center
Pediatric Brain Tumor Consortium

Terminated

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

Fanconi Anemia | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML)

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
Children's Hospital Medical Center, Cincinnati
Memorial Sloan Kettering Cancer Center; Fred Hutchinson Cancer Center

Recruiting

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA)

Fanconi Anemia | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Severe Marrow Failure

United States
Novartis Pharmaceuticals

Completed

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

Myelodysplastic Syndromes | Fanconi Syndrome | Anemia, Aplastic | Beta-thalassemia | Anemia, Diamond-Blackfan

United States
Gilead Sciences

Completed

Fanconi Syndrome Due to ARVs in HIV-Infected Persons

HIV Infections | Renal Impairment | Fanconi Syndrome | Kidney Disease

Canada, United States
Academic and Community Cancer Research United
National Cancer Institute (NCI)

Recruiting

Olaparib in Treating Patients With Metastatic Biliary Tract Cancer With Aberrant DNA Repair Gene Mutations

Bile Duct Adenocarcinoma | Fanconi Anemia Complementation Group Gene Mutation | Metastatic Bile Duct Carcinoma | PTEN Gene Deletion

United States

Clinical Trials on Monocarboxylate Transporter 8 Deficiency in New Zealand

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Guinea

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations