Clinical Trials on Monocarboxylate Transporter 8 Deficiency in Australia

Total 7 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

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
Orthopedic Hospital Vienna Speising

Completed

Foot Disorders in X-linked Hypophosphatemia

X-linked Hypophosphatemia

Austria
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
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
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
University Hospital Freiburg

Completed

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Myelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Pearson Marrow-pancreas Syndrome | Shwachman-diamond Syndrome

Spain, Germany, Switzerland, Austria, Netherlands, Italy, Czech Republic, Belgium, Denmark, Ireland

Clinical Trials on Monocarboxylate Transporter 8 Deficiency in Australia

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Brazil

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Rare Diseases

Drug Interventions

Dietary Supplements

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