Clinical Trials on Neutral Lipid Storage Disease With Myopathy in South Africa

Total 95 results

Pfizer

Completed

A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Gaucher Disease

Israel, Paraguay, South Africa
Pfizer

Completed

A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Gaucher Disease

Israel, South Africa, Paraguay
Pfizer

Completed

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Gaucher Disease

Chile, United Kingdom, Canada, Spain, Israel, United States, Australia, South Africa
Pfizer

Completed

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

Gaucher Disease

United States, Chile, United Kingdom, Israel, Canada, Spain, South Africa, Italy
Amicus Therapeutics

Completed

A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

Gaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher Disease

United Kingdom, Israel, South Africa, United States
ISU Abxis Co., Ltd.

Completed

A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers

Gaucher Disease

Australia
Genzyme, a Sanofi Company

Completed

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

Glycogen Storage Disease Type II | Pompe Disease Infantile-Onset

United States, France, South Africa
LIB Therapeutics LLC
Medpace, Inc.

Enrolling by invitation

Long-term Efficacy and Safety of OLE LIB003 in HoFH, HeFH, and High-risk CVD Patients Requiring Further LDL-C Reduction (LIBerate-OLE)

Familial Hypercholesterolemia | Elevated Cholesterol | Cardiovascular Disease With Mention of Arteriosclerosis

United States, Israel, Norway, South Africa, Turkey, India
Amicus Therapeutics

Completed

A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects

Fabry Disease

Australia
Minster Research Ltd

Completed

Efficacy and Tolerability of Tonabersat in the Prophylaxis of Migraine Headache

Migraine Without Aura | Migraine With Aura

United Kingdom, Denmark, Hungary, South Africa
Genzyme, a Sanofi Company

Withdrawn

A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.

Fabry Disease

United States
BioMarin Pharmaceutical

Terminated

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Late-onset Pompe Patients Untreated or Treated With rhGAA

Spain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
Mandos LLC

Terminated

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

Niemann-Pick Disease, Type C

Costa Rica
Idorsia Pharmaceuticals Ltd.

Active, not recruiting

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

Fabry Disease

United States, United Kingdom, Norway, Germany, Australia, Canada, Spain, Switzerland, Austria, Belgium, France, Netherlands, Poland
Genzyme, a Sanofi Company

Recruiting

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Gaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease

Hungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more
Idorsia Pharmaceuticals Ltd.

Completed

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (MODIFY)

Fabry Disease

United States, Spain, Belgium, Australia, Austria, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Switzerland, United Kingdom
Genzyme, a Sanofi Company

Recruiting

Fabry Disease Registry & Pregnancy Sub-registry

Fabry Disease

Croatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more
Genzyme, a Sanofi Company

Completed

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (ASCEND)

Sphingomyelin Lipidosis

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, United Kingdom
Medical University of South Carolina
Entera Health, Inc

Completed

Efficacy of Serum Bovine Immunoglobulin in Improving Nutritional Status in Advanced COPD (SBI for COPD)

Advanced COPD (GOLD Stage 3 or 4) With Cachexia

United States
Medtronic Spinal and Biologics

Terminated

An Investigation of the Metal Concentration in Patients Implanted With the PRESTIGE LP™ Cervical Disc at Two Contiguous Levels in the Cervical Spine

Radiculopathy | Myelopathy | Cervical Degenerative Disc Disease

United States
Medical University of South Carolina

Completed

The Effect of Music Periodicity on Interictal Epileptiform Discharges

Epilepsy | Seizures | Rolandic Epilepsy | Benign Childhood Epilepsy With Centrotemporal Spikes

United States
Medical University of South Carolina

Completed

Effects of Neuromodulation and Rehabilitation of the Locomotor Network in Freezing of Gait (TMS/FOG)

Parkinson Disease With Freezing of Gait

United States
BioMarin Pharmaceutical

Terminated

Extension Study for Patients Who Have Participated in a BMN 701 Study

Pompe Disease

United States, United Kingdom, Australia, France, Germany, New Zealand
Jazz Pharmaceuticals

Terminated

Efficacy and Safety of GWP42003-P Oral Solution in Children With Epilepsy With Myoclonic-atonic Seizures

Seizures Associated With EMAS

United States, Italy
NeurAxon Inc.

Completed

Study of NXN 188 for the Treatment of Migraine With Aura

Migraine With Aura

United States
Mandos LLC

Completed

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

Niemann-Pick Disease, Type C

United States, Australia, United Kingdom, Germany, France, New Zealand, Singapore, Spain, Turkey
Mandos LLC

Terminated

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Niemann-Pick Disease, Type C

United States, Australia, United Kingdom, Turkey, Switzerland, Germany, New Zealand, Singapore, France
Amicus Therapeutics

Active, not recruiting

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Pompe Disease

United States, Netherlands, United Kingdom, Australia, Germany, New Zealand
Amicus Therapeutics

Recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Pompe Disease (Late-onset)

United States, Taiwan, Japan, Australia, Canada
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
Greenwood Genetic Center

Completed

Longitudinal Studies of the Glycoproteinoses

Galactosialidosis | Sialidosis | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Beta Mannosidosis | Mucolipidosis II | Mucolipidosis III | Schindler Disease

United States
Amicus Therapeutics

Completed

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (PROPEL)

Pompe Disease (Late-onset)

United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
Amicus Therapeutics

Active, not recruiting

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

Pompe Disease (Late-onset)

United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
Genzyme, a Sanofi Company

Completed

Late-Onset Treatment Study Extension Protocol

Glycogen Storage Disease Type II (GSD-II) | Pompe Disease (Late-Onset) | Glycogenesis Type II | Acid Maltase Deficiency (AMD)

United States, France, Canada, Netherlands, Australia
Genzyme, a Sanofi Company

Recruiting

Pompe Disease Registry Protocol

Glycogen Storage Disease Type II | Pompe Disease

Hungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more
Cynthia West

Completed

Stop Atherosclerosis in Native Diabetics Study (SANDS)

Hypertension | Cardiovascular Disease | Diabetes | Carotid Atherosclerosis | Hyperlipidemia

United States
Amgen

Completed

Safety, Tolerability and Efficacy on Low Density Lipoprotein Cholesterol (LDL-C) of Evolocumab in Participants With Human Immunodeficiency Virus (HIV) and Hyperlipidemia/Mixed Dyslipidemia

Subjects With Hyperlipidemia, Dyslipidemia and HIV Infection

Canada, France, United States, Belgium, Italy, Spain, Switzerland, United Kingdom, South Africa, Portugal, Australia, Greece, Brazil, Romania, Poland
Solvay Pharmaceuticals
Nektar Therapeutics

Completed

Study to Evaluate the Efficacy and Safety of Dronabinol Metered Dose Inhaler (MDI) in Acute Treatment of Migraine Headache

Migraine Without Aura | Migraine With Aura

United States
Pfizer

Completed

Safety and Efficacy Study in Adult Subjects With Acute Migraines

Migraine, With or Without Aura

United States
Genzyme, a Sanofi Company

Approved for marketing

Alglucosidase Alfa Temporary Access Program (ATAP)

Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease (Late-Onset)

United States
Medtronic Spinal and Biologics

Completed

Study of the Safety and Effectiveness of the Artificial Cervical Disc - Low Profile Device at Two Adjacent Levels

Radiculopathy | Myelopathy | Cervical Degenerative Disc Disease

United States
Medtronic Spinal and Biologics

Completed

Pivotal IDE Study of the BRYAN(R) Cervical Disc Prosthesis in the Treatment of DDD Versus ACDF

Radiculopathy | Myelopathy | Cervical Degenerative Disc Disease

United States
University of Pennsylvania
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Completed

Plasma Exchange and Glucocorticoids for Treatment of Anti-Neutrophil Cytoplasm Antibody (ANCA) - Associated Vasculitis (PEXIVAS)

Microscopic Polyangiitis (MPA) | Granulomatosis With Polyangiitis (Wegener's) (GPA)

United States, Belgium, France, United Kingdom, Denmark, Canada, Japan, Australia, New Zealand, Sweden, Czechia, Italy, Greece, Mexico, Norway
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
InflaRx GmbH
Iqvia Pty Ltd

Terminated

Safety and Efficacy Study of IFX-1 in add-on to Standard of Care in GPA and MPA

Granulomatosis With Polyangiitis (GPA) | Microscopic Polyangiitis (MPA)

United States, Canada
Pfizer

Completed

Trial in Adult Subjects With Acute Migraines

Migraine, With or Without Aura

United States
Eli Lilly and Company
CoLucid Pharmaceuticals

Completed

Three Doses of Lasmiditan (50 mg, 100 mg and 200 mg) Compared to Placebo in the Acute Treatment of Migraine (SPARTAN)

Migraine With or Without Aura

United States
Allergan

Completed

Efficacy, Safety, and Tolerability of Oral Ubrogepant in the Acute Treatment of Migraine (ACHIEVE II)

Migraine, With or Without Aura

United States
Satsuma Pharmaceuticals, Inc.

Completed

A Randomized, Double-Blind, Parallel Group, Placebo-Controlled Study to Assess STS101 in the Acute Treatment of Migraine (SUMMIT)

Migraine | Migraine Without Aura | Migraine With Aura

United States
University Hospitals Cleveland Medical Center
National Institute of Allergy and Infectious Diseases (NIAID); University of... and other collaborators

Recruiting

Misfolded Proteins in the Skin of People With Parkinson's Disease and Other Parkinsonism

Parkinson Disease | Parkinsonism | Multiple System Atrophy | Corticobasal Degeneration | Progressive Supranuclear Palsy | Dementia With Lewy Bodies

United States

Clinical Trials on Neutral Lipid Storage Disease With Myopathy in South Africa

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Gabon

Conditions

Rare Diseases

Drug Interventions

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