Clinical Trials on Dominantly Inherited Stargardt's Disease (STGD3) in South Africa

Total 138 results

Catalyst Biosciences

Completed

Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B

Hemophilia B

South Africa
Baxalta now part of Shire

Terminated

Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A

Hemophilia A

United States, South Africa, United Kingdom
Bayer

Completed

Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors (MATCHBOX)

Hemophilia A | Hemophilia B

South Africa, Poland, United Kingdom
Novo Nordisk A/S

Completed

Epidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries (HAEMOcare)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia B

India, Morocco, South Africa, Oman
Vega Therapeutics, Inc

Recruiting

Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease

Von Willebrand Diseases

United States, Austria, South Africa, United Kingdom
Belite Bio, Inc

Active, not recruiting

Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1 (DRAGON)

Stargardt Disease 1

United States, Australia, Belgium, China, France, Germany, Hong Kong, Netherlands, Switzerland, Taiwan, United Kingdom
The University of Queensland

Recruiting

Implementation of Metformin theraPy to Ease Decline of Kidney Function in Polycystic Kidney Disease (IMPEDE-PKD)

Autosomal Dominant Polycystic Kidney Disease

Australia
Kastle Therapeutics, LLC
Ionis Pharmaceuticals, Inc.

Completed

Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia (RADICHOL 1)

Metabolic Diseases | Congenital Abnormalities | Infant, Newborn, Diseases | Genetic Diseases, Inborn | Dyslipidemias | Metabolism, Inborn Errors | Lipid Metabolism Disorders | Hypercholesterolemia | Hyperlipidemias | Hyperlipoproteinemias | Lipid Metabolism, Inborn Errors | Hyperlipoproteinemia Type II | Metabolic... and other conditions

United States, Taiwan, Brazil, Canada, South Africa, United Kingdom, Singapore
BioMarin Pharmaceutical

Active, not recruiting

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 (GENEr8-AAV5+)

Hemophilia A | Gene Therapy | Blood Disorder | Clotting Disorders

Taiwan, Korea, Republic of, United Kingdom, South Africa
Catalyst Biosciences

Completed

Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B

Hemophilia A With Inhibitor | Hemophilia B With Inhibitor

Armenia, Georgia, South Africa, Poland, Russian Federation
Erasmus Medical Center

Recruiting

Treatment of Vascular Stiffness in ADPKD (TRAMPOLINE)

Autosomal Dominant Polycystic Kidney

Netherlands
Pfizer

Completed

PF-06741086 Multiple Dose Study in Severe Hemophilia

Hemophilia A or B

United States, Chile, Croatia, Poland, South Africa, Switzerland
Laboratoire français de Fractionnement et de Biotechnologies
LFB USA, Inc.

Completed

LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures (PerSept3)

Hemophilia

United States, Ukraine, Mexico, Russian Federation, South Africa, Spain
Western Sydney Local Health District
Westmead Institute for Medical Research

Completed

Effect of Beetroot Juice on Reducing Hypertension in Autosomal Dominant Polycystic Kidney Disease (BEET-PKD)

Hypertension | Endothelial Dysfunction | Autosomal Dominant Polycystic Kidney

Australia
IVERIC bio, Inc.

Active, not recruiting

Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

Stargardt Disease 1

Italy, United States, Israel, United Kingdom, Germany, Spain, Hungary, France
Medexus Pharma, Inc.

Completed

Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B

Hemophilia B

Turkey, Brazil, Georgia, Moldova, Republic of, South Africa, Ukraine
Laboratoire français de Fractionnement et de Biotechnologies
LFB USA, Inc.

Completed

A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (PERSEPT2)

Hemophilia A With Inhibitors | Hemophilia B With Inhibitors

Bulgaria, Ukraine, Czechia, United States, Georgia, South Africa
RBP4 Pty Ltd
Belite Bio, Inc

Completed

This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease

Stargardt Disease

Taiwan, Australia
Pfizer

Completed

PF-06741086 Long-term Treatment in Severe Hemophilia

Hemophilia A or B

Poland, Switzerland, South Africa, United States, Croatia, Brazil, Chile
CSL Behring

Terminated

Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors

Hemophilia A With Inhibitors | Hemophilia B With Inhibitors

Georgia, Italy, Malaysia, Russian Federation, South Africa, Spain, Thailand, Ukraine, United Kingdom
Christian Medical College, Vellore, India

Unknown

Musculoskeletal Function in Hemophilia

Hemophilia

India, Argentina, Brazil, Egypt, Iran, Islamic Republic of, Singapore, South Africa, Thailand, Venezuela
BioMarin Pharmaceutical

Recruiting

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors (GENEr8-INH)

Hemophilia A With Inhibitor | Hemophilia A With Anti Factor VIII

United States, United Kingdom, Taiwan, Israel, Korea, Republic of, South Africa, Brazil, Italy, Germany
Otsuka Pharmaceutical Development & Commercialization...

Completed

Tolvaptan Extension Study in Participants With ADPKD (TEMPO 4/4)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

United States, Australia, Belgium, Germany, Italy, Poland, Romania, United Kingdom, Argentina, Canada, France, Netherlands, Russian Federation
Hoffmann-La Roche
Chugai Pharmaceutical

Completed

A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors (HAVEN 2)

Hemophilia A

United States, France, Germany, Italy, South Africa, Spain, Turkey, Japan, United Kingdom, Costa Rica
Sanofi

Terminated

Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat (STAGED-PKD-EXT)

Congenital Cystic Kidney Disease

Germany, Spain, United States, Australia, Belgium, Japan, Korea, Republic of, Netherlands
Octapharma

Completed

Wilate in Subjects With Von Willebrand Disease Who Undergo Surgery (WONDERS)

Prevent Bleeding in Major Surgery

United States, Turkey, Romania, India, Bulgaria, Italy, Oman, Poland, South Africa
Kastle Therapeutics, LLC
Ionis Pharmaceuticals, Inc.

Completed

An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia

Metabolic Diseases | Congenital Abnormalities | Infant, Newborn, Diseases | Genetic Diseases, Inborn | Dyslipidemias | Metabolism, Inborn Errors | Lipid Metabolism Disorders | Hypercholesterolemia | Hyperlipidemias | Hyperlipoproteinemias | Lipid Metabolism, Inborn Errors | Hyperlipoproteinemia Type II | Metabolic... and other conditions

United States, Canada, Brazil, Taiwan, Singapore, South Africa, United Kingdom
Hoffmann-La Roche
Chugai Pharmaceutical

Completed

A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors (HAVEN 3)

Hemophilia A

Australia, Taiwan, Korea, Republic of, France, Germany, Italy, South Africa, Spain, United States, Japan, Ireland, Poland, United Kingdom, Costa Rica
Otsuka Pharmaceutical Development & Commercialization...

Completed

Efficacy and Safety of Tolvaptan in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease

Chronic Kidney Disease | Autosomal Dominant Polycystic Kidney Disease

Australia, Belgium, Italy, Spain, Sweden, United States, Canada, Netherlands, Czechia, Poland, Germany, Russian Federation, Hungary, Israel, Puerto Rico, Argentina, Denmark, South Africa, United Kingdom, Norway, France, Romania
Laboratoire français de Fractionnement et de Biotechnologies

Recruiting

A Phase 3 Study of the Safety and Efficacy of Coagulation Factor VIIa (Recombinant) for the Prevention of Excessive Bleeding in Patients With Congenital Hemophilia A or B With Inhibitors to Factor VIII or IX Undergoing Elective Major Surgical Procedures SCOPE HIM (SCOPE HIM)

Hemophilia

United States, Thailand, Malaysia, Mexico, Turkey, South Africa
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B (Kids B-LONG)

Hemophilia B

United States, United Kingdom, South Africa, Netherlands, Ireland, Australia
Otsuka Pharmaceutical Development & Commercialization...

Completed

8-Week Study of Tolvaptan Dose Forms in Autosomal Dominant Polycystic Kidney Disease (ADPKD) (NOCTURNE)

Autosomal Dominant Polycystic Kidney Disease

United States
Otsuka Pharmaceutical Development & Commercialization...
Otsuka Pharmaceutical Co., Ltd.

Completed

Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease (OVERTURE)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

United States, Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, France, Germany, Italy, Japan, Netherlands, Norway, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
Catalyst Biosciences

Completed

Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia

Hemophilia A

United States, Belgium, Hungary, Italy, New Zealand, South Africa, Turkey, United Kingdom
Kubota Vision Inc.
Food and Drug Administration (FDA)

Completed

Safety and Efficacy of Emixustat in Stargardt Disease (SeaSTAR)

Stargardt Disease

United States, United Kingdom, Germany, Spain, Brazil, Canada, Denmark, France, Italy, Netherlands, South Africa
Hoffmann-La Roche
Chugai Pharmaceutical

Completed

A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors (HAVEN 1)

Hemophilia A

Korea, Republic of, United States, France, Germany, Italy, South Africa, Spain, Japan, United Kingdom, Poland, Taiwan, Australia, Costa Rica, New Zealand
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire

Terminated

BAX 802 in CHA With Inhibitors (CHAWI)

Hemophilia A

Netherlands, Spain, Italy, Turkey, Bulgaria, United States, Canada, Germany, Norway, Poland, Russian Federation, South Africa
Otsuka Pharmaceutical Development & Commercialization...
Otsuka Pharmaceutical Co., Ltd.

Completed

Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD) (TEMPO3:4)

Polycystic Kidney Disease, Autosomal Dominant

United States, Belgium, United Kingdom, Russian Federation, France, Australia, Netherlands, Italy, Japan, Denmark, Romania, Poland, Canada, Argentina, Germany
Novo Nordisk A/S

Completed

Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B (Explorer 1)

Healthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Austria, Sweden, Spain, Switzerland, United Kingdom, Germany, Malaysia, Denmark, South Africa, Thailand, Italy
Hoffmann-La Roche

Completed

A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment

Hemophilia A

United States, Korea, Republic of, Italy, South Africa, Spain, China, Japan, Poland, Australia, Costa Rica, Germany, Taiwan
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A (Kids ALONG)

Hemophilia A

United States, Poland, United Kingdom, Netherlands, South Africa, Hong Kong, Australia, Ireland
Palladio Biosciences
Centessa Pharmaceuticals plc

Terminated

Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease (ACTION)

Autosomal Dominant Polycystic Kidney | ADPKD

United States, Spain, Hungary, United Kingdom, Australia, Bulgaria, Poland, Turkey
Novo Nordisk A/S

Completed

A Research Study Investigating Mim8 in People With Haemophilia A (FRONTIER1)

Healthy Volunteers | Haemophilia A With or Without Inhibitors

United States, Germany, Spain, Austria, Bulgaria, Italy, Japan, South Africa, Switzerland, Turkey, United Kingdom, Poland
Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors (HAVEN 7)

Severe Hemophilia A

Canada, United States, United Kingdom, Belgium, Australia, Austria, Brazil, France, Germany, Israel, Italy, South Africa, Spain, Turkey
Novo Nordisk A/S

Active, not recruiting

A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5) (FRONTIER 5)

Haemophilia A With Inhibitors | Haemophilia A

Spain, Korea, Republic of, Canada, United Kingdom, United States, Germany, South Africa, Italy, Austria, Belgium, France, Japan
Otsuka Pharmaceutical Development & Commercialization...

Completed

Long Term Safety of Immediate-release Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease

Polycystic Kidney, Autosomal Dominant

Belgium, Germany, Italy, Spain, Sweden, United States, Canada, Australia, Netherlands, Czechia, Poland, Denmark, Israel, Hungary, Russian Federation, South Africa, United Kingdom, Romania, Argentina, Norway
Freeline Therapeutics

Terminated

A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy

Hemophilia B

United Kingdom
Spark Therapeutics
Children's Hospital of Philadelphia; University of Pittsburgh; St. James's Hospital... and other collaborators

Terminated

Hemophilia B Gene Therapy With AAV8 Vector

Hemophilia B

United States, Australia
Reata, a wholly owned subsidiary of Biogen

Terminated

A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON (FALCON)

Autosomal Dominant Polycystic Kidney | ADPKD

United States, Spain, United Kingdom, Australia, France, Japan, Germany, Belgium, Czechia, Italy
Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors (HAVEN 6)

Hemophilia A | Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor | Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor

United States, Netherlands, Belgium, France, Germany, South Africa, Spain, Canada, United Kingdom, Poland

Clinical Trials on Dominantly Inherited Stargardt's Disease (STGD3) in South Africa

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CROs in Netherlands

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