Clinical trials: Neonates
Gout, Hyperuricemia
SAD,MAD and Food Effect Study of SAP-001 Tablets in Chinese Subjects
To evaluate the pharmacokinetics (PK) profile of single or multiple oral doses of SAP-001 tablets in healthy Chinese subjects and gout patients with hyperuricemia.
- Trial name
- NCT06678581
- Testing
- SAP-001
- Where
- China
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Shanton Pharma Co., Ltd.
Sickle Cell Disease, Pharmacokinetics
Pharmacokinetics and Safety of Endari (L-glutamine) in Sickle Cell Disease Patients
L-glutamine has been approved in the US to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients 5 years of age and older.
- Trial name
- NCT04684381
- Testing
- L-glutamine
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Emmaus Medical, Inc.
Fabry Disease, Lysosomal Storage Diseases
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme.
- Trial name
- NCT04455230
- Testing
- FLT190
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Spur Therapeutics
Adenomatous Polyposis Coli
A Study of Guselkumab in Participants With Familial Adenomatous Polyposis
The purpose of this study is to determine the effect of treatment with guselkumab in participants with familial adenomatous polyposis (FAP) on rectal/pouch polyp burden.
- Trial name
- NCT03649971
- Testing
- Guselkumab
- Where
- 32 sites in United States, France and 7 more
- Volunteers
- 77 people
- Sponsor
- Janssen Research & Development, LLC
Hemophilia A or B
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
A study to evaluate the safety of Hympavzi under the actual use in patients with congenital hemophilia who do not have inhibitors.
- Trial name
- HIZ (NCT07161687)
- Testing
- Marstacimab
- Where
- Japan
- Volunteers
- 50 people
- Sponsor
- Pfizer
Hypercholesterolemia, Familial, Hyperlipidemia
Study To Evaluate The Safety And Efficacy Of Torcetrapib/Atorvastatin In Subjects With Familial Hypercholerolemia
The Torcetrapib project was terminated on December 2, 2006 due to safety findings. To evaluate the efficacy and safety of torcetrapib/atorvastatin compared to atorvastatin alone in patients with heterozygous familial…
- Trial name
- NCT00134485
- Testing
- torcetrapib/atorvastatin, atorvastatin
- Where
- 41 sites in United States, Australia and 7 more
- Volunteers
- 400 people
- Sponsor
- Pfizer
Metachromatic Leukodystrophy (MLD)
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to…
- Trial name
- EMBOLDEN (NCT03771898)
- Testing
- SHP611
- Where
- 30 sites in United States, Argentina and 12 more
- Volunteers
- 36 people
- Sponsor
- Shire
Myotonic Dystrophy Type 1 (DM1)
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).
- Trial name
- NCT06926621
- Testing
- VX-670
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Glycogen Storage Disease, Pompe Disease
Pompe Lactation Sub-Registry
The objective is to determine if alglucosidase alfa is present in breast milk from mothers with Pompe Disease being treated with alglucosidase alfa and to measure breast milk production and composition in women with Pompe Disease…
- Trial name
- NCT00566878
- Testing
- alglucosidase alfa
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Genzyme, a Sanofi Company
Von Willebrand Disease (VWD)
A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
The main aim of the study is to evaluate the effectiveness of prophylaxis with vonicog alfa (recombinant von Willebrand factor [rVWF]) in children.
- Trial name
- NCT05582993
- Testing
- Vonicog Alfa, ADVATE
- Where
- 23 sites in United States, France and 4 more
- Volunteers
- 24 people
- Sponsor
- Takeda
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Primary Knee Arthroplasty
ATTUNE® Revision- Complex Primary in Total Knee Arthroplasty Population
Five-year, Prospective, multi-center, non-randomized, non-controlled study of the ATTUNE® Revision total knee prostheses, utilizing the fixed bearing (FB) and rotating platform (RP) tibial component with the posterior stabilizing…
- Trial name
- NCT03153449
- Testing
- ATTUNE Revision Knee System in Total Knee Arthroplasty
- Where
- 34 sites in United States, Australia and 8 more
- Volunteers
- 400 people
- Sponsor
- DePuy Orthopaedics
Fabry Disease, Lysosomal Storage Diseases
A Fabry Disease Gene Therapy Study
This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.
- Trial name
- MARVEL1 (NCT04040049)
- Testing
- FLT190
- Where
- 13 sites in United States, Austria and 5 more
- Volunteers
- 3 people
- Sponsor
- Spur Therapeutics
Biliary Tract Cancer
Futibatinib (TAS-120) in Patients With Advanced Biliary Tract Cancer
To compare overall survival (OS) of patients in Futibatinib and Zimberelimab in Combination with Gemcitabine plus Cisplatin versus Durvalumab or Pembrolizumab in Combination with Gemcitabine plus Cisplatin for patients with…
- Trial name
- FOENIX-BTC (NCT07710885)
- Testing
- Futibatinib, Zimberelimab, Durvalumab
- Where
- 21 sites in Australia, Japan and 2 more
- Volunteers
- 784 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Gangliosidoses, GM2, Gangliosidosis, GM1
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2…
- Trial name
- NCT07082543
- Testing
- AZ-3102
- Where
- 27 sites in United States, Argentina and 12 more
- Volunteers
- 75 people
- Sponsor
- Azafaros B.V.
Meningococcal Disease
Safety and Immunogenicity of Novartis Meningococcal B Vaccine When Administered to Immunocompromised Children and Adolescents Compared to Healthy Subjects.
The study aims at evaluating the safety and immunogenicity of rMenB+OMV NZ when administered to subjects from 2 to 17 years of age with increased risk of meningococcal disease because either of primary or secondary complement…
- Trial name
- NCT02141516
- Testing
- rMenB+OMV
- Where
- 20 sites in Italy, Poland and 3 more
- Volunteers
- 239 people, healthy volunteers welcome
- Sponsor
- Novartis
Hereditary Angioedema
EASSI - Evaluation of the Safety of Self-Administration With Icatibant
This study is being conducted to explore the clinical safety, local tolerability, convenience and effectiveness of self-treatment of hereditary angioedema (HAE) attacks with subcutaneous injections of icatibant.
- Trial name
- EASSI (NCT00997204)
- Testing
- Icatibant
- Where
- 26 sites in Argentina, Austria and 8 more
- Volunteers
- 151 people
- Sponsor
- Shire
Osteoarthritis, Musculoskeletal Abnormalities
Prospective Evaluation Assessing U2 MB™ Total Knee System
The goal of this study is to evaluate the safety and effectiveness of the United Orthopaedics Mobile Bearing Total Knee System (U2 MB™ Knee) in patients undergoing total knee replacement surgery due to end-stage knee…
- Trial name
- NCT06996158
- Volunteers
- 150 people
- Sponsor
- MSK Doctors & Associates Ltd
Congenital Heart Disease
ABL90 FP MC Neonatal Arterial, Venous, Capillary Blood
The goal of this clinical trial is to validate performance claims for method comparison for ABL90 FLEX PLUS in heparinized neonatal arterial, venous, and capillary whole blood for ctBil and FHbF in a Point of Care (POC) setting.
- Trial name
- NCT06078943
- Testing
- method comparison
- Where
- United States
- Volunteers
- 612 people, healthy volunteers welcome
- Sponsor
- Radiometer Medical ApS
Retinopathy of Prematurity
Study to Assess the Efficacy, Safety, and Tolerability of Intravitreal Aflibercept Compared to Laser Photocoagulation in Patients With Retinopathy of Prematurity
The primary objective of the study is to assess the efficacy of aflibercept compared to laser in patients diagnosed with retinopathy of prematurity (ROP).
- Trial name
- BUTTERFLEYE (NCT04101721)
- Testing
- aflibercept, laser photocoagulation
- Where
- 52 sites in United States, Bulgaria and 11 more
- Volunteers
- 127 people
- Sponsor
- Regeneron Pharmaceuticals