Clinical Trials on Lysosome Alteration in South Africa

Total 49 results

United States Army Research Institute of Environmental...
United States Department of Defense

Completed

Efficacy of a Once Daily Calcium and Vitamin D Fortified Food Product to Improve Bone Microarchitecture

Dietary Modification | Bone Alteration

United States
BioMed Valley Discoveries, Inc

Terminated

Study of Ulixertinib for Patients With Advanced Malignancies Harboring MEK or Atypical BRAF Alterations

Advanced Solid Tumor | MAP2K1 Gene Mutation | BRAF Gene Mutation | MEK Mutation | BRAF Gene Alteration | MEK Alteration | MAP2K1 Gene Alteration | MAP2K2 Gene Mutation | MAP2K2 Gene Alteration

United States
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
PLx Pharma

Completed

Evaluation of Upper GI Ulceration Induced by PL2200 Versus Aspirin in At-Risk Subjects

Gastroduodenal Ulceration

United States
TransThera Sciences (Nanjing), Inc.

Completed

Study to Evaluate the Efficacy and Safety of TT-00420 (Tinengotinib) in Cholangiocarcinoma

Cholangiocarcinoma | FGFR2 Gene Mutation | FGFR2 Fusion | FGFR1 Alteration | FGFR3 Alteration

United States
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
Sonderborg Hospital
Aase and Ejnar Danielsens Foundation; The Augustinus Foundation, Denmark.; The...

Completed

Acupuncture for Pain Relief During Perineal Repair After Childbirth

Pain | Lacerations | Birth Injuries

Denmark
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
Pfizer

Completed

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Gaucher Disease

Chile, United Kingdom, Canada, Spain, Israel, United States, Australia, South Africa
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
Nacuity Pharmaceuticals, Inc.

Recruiting

PK and PD Study of NPI-001 and Cysteamine Bitartrate (INCA)

Cystinosis

Australia
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
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
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
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
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
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
Greenwood Genetic Center
Shriners Hospitals for Children; BioMarin Pharmaceutical

Completed

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

MPS IVA | Maroteaux Lamy Syndrome | MPS VI | Morquio Syndrome A

United States
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Enrolling by invitation

A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

Mucopolysaccharidosis III-A

United States, Australia, Spain
Tyra Biosciences, Inc

Recruiting

Safety and Preliminary Anti-Tumor Activity of TYRA-300 in Advanced Urothelial Carcinoma and Other Solid Tumors With FGFR3 Gene Alterations (SURF301)

Solid Tumor | Bladder Cancer | Advanced Solid Tumor | Urothelial Carcinoma | Metastatic Urothelial Carcinoma | Solid Tumor, Adult | FGFR3 Gene Mutation | Urinary Tract Cancer | Locally Advanced Urothelial Carcinoma | Advanced Urothelial Carcinoma | Non-muscle-invasive Bladder Cancer | FGFR3 Gene Alteration | Urinary... and other conditions

Australia, United States, France, Spain
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Terminated

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Mucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo A

United States, Spain, Australia
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
BioMarin Pharmaceutical

Completed

BMN 110 Phase 3B in Australian Patients (MOR-AUS)

Mucopolysaccharidosis IVA (Morquio A Syndrome)

Australia
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Active, not recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Mucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo A

United States, Australia, Spain
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
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
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

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
Loxo Oncology, Inc.
Eli Lilly and Company

Available

Expanded Access for the Treatment of Cancers With Rearranged During Transfection (RET) Activation

Breast Cancer | Pancreatic Cancer | Non Small Cell Lung Cancer | Colon Cancer | Medullary Thyroid Cancer | Papillary Thyroid Cancer | Other Solid Tumors With Evidence of Activating RET Alteration

Spain, United States, Australia, France, Germany, Israel, Italy, Japan, New Zealand, Poland, Singapore, Switzerland
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

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
Denali Therapeutics Inc.

Completed

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Mucopolysaccharidosis II

United Kingdom, United States, Italy, Netherlands
Denali Therapeutics Inc.

Active, not recruiting

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Mucopolysaccharidosis II

United States, Netherlands, Canada, United Kingdom
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
BioMarin Pharmaceutical

Completed

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, 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

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
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
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
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
BioMarin Pharmaceutical
ICON plc

Completed

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (MARS)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
Apollomics Inc.

Recruiting

APL-101 Study of Subjects With NSCLC With c-Met EXON 14 Skip Mutations and c-Met Dysregulation Advanced Solid Tumors (SPARTA)

Gastric Cancer | Pancreatic Cancer | NSCLC | Lung Cancer | Brain Tumor | Glioblastoma Multiforme | Advanced Cancer | Colon Cancer | Solid Tumors | Thyroid Cancer | Renal Cancer | Gastroesophageal Junction Adenocarcinoma | EGFR Gene Mutation | MET Amplification | HGF | MET Alteration | MET Fusion | Exon 14 Skipping

United States, Spain, France, United Kingdom, Taiwan, Australia, Finland, Italy, Singapore, Hungary, Canada, Puerto Rico, Russian Federation
Genzyme, a Sanofi Company

Recruiting

Mucopolysaccharidosis I (MPS I) Registry

Mucopolysaccharidosis I (MPS I)

Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more
Takeda

Available

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Hunter Syndrome

United States, Australia, Mexico, Spain, United Kingdom
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

Clinical Trials on Lysosome Alteration in South Africa

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Liberia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations