Clinical trials: Metabolic Abnormalities
Gout, Hyperuricemia
Tigulixostat, Phase 3 Study, Allopurinol Controlled in Gout Patients
The aim of this 12-month randomized multi-regional double-blind parallel group allopurinol and placebo-controlled phase 3 study is to assess the efficacy and safety of three different doses of Tigulixostat in gout patients with…
- Conditions
- Gout, Hyperuricemia, Gout Flare, Tophi
- Trial name
- EURELIA2 (NCT05586971)
- Testing
- Tigulixostat, Allopurinol
- Where
- 279 sites in United States, Argentina and 19 more
- Volunteers
- 2,202 people
- Sponsor
- LG Chem
Urea Cycle Disorders, Crigler Najjar Syndrome
Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.
- Trial name
- NCT02051049
- Where
- Belgium
- Volunteers
- 9 people
- Sponsor
- Cellaion SA
Friedreich Ataxia
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
The purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity.
- Trial name
- NCT03214588
- Testing
- TAK-831
- Where
- 6 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurocrine Biosciences
Haemophilia A, Haemophilia B, Obesity
Awareness, Care, and Treatment In Obesity Management of Patients With Hemophilia (ACTION) to Inform Hemophilia Obesity Patient Empowerment (HOPE)
An online web-based cross-sectional survey in which participants will complete a distinct questionnaire for patients with hemophilia and obesity (PwHO) and/or their carers/spouses/partners, or a distinct questionnaire for…
- Trial name
- ACTION to HOPE (NCT03430154)
- Testing
- No treatment given
- Where
- United States
- Volunteers
- 317 people
- Sponsor
- Novo Nordisk A/S
Arthritis, Gouty
The MAD Study of SSGJ-613 in Healthy Subjects
To evaluate the safety and tolerability of SSGJ-613 after multiple subcutaneous injections in healthy subjects.
- Trial name
- NCT05966701
- Testing
- SSGJ-613
- Where
- China
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd.
Phenylketonuria
GenSci144 Tablets Phase I Clinical Trial
This study is a Phase I single ascending dose clinical trial in which healthy adult volunteers were administered single oral doses of GenSci144 tablets at different levels.
- Trial name
- NCT07685210
- Testing
- GenSci144 tablets
- Where
- China
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Fibrodysplasia Ossificans Progressiva, FOP
A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP.
- Trial name
- OPTIMA-2 (NCT07559513)
- Testing
- garetosmab
- Volunteers
- 18 people
- Sponsor
- Regeneron Pharmaceuticals
Fabry Disease
A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
The purpose of this study is to observe the safety of agalsidase alfa in Canadian patients with Fabry disease.
- Trial name
- NCT01298141
- Testing
- agalsidase alfa
- Where
- 12 sites in Canada
- Volunteers
- 171 people
- Sponsor
- Shire
Arginase I Deficiency, Hyperargininemia
A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D.
- Trial name
- NCT05676853
- Testing
- Pegzilarginase
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Aeglea Biotherapeutics
Gout
Lesinurad and Allopurinol Combination Extension Study in Gout
This study will assess the serum uric acid lowering effects and safety of lesinurad in combination with allopurinol over a long-term timeframe.
- Trial name
- NCT01808131
- Testing
- Lesinurad
- Where
- 207 sites in United States, Australia and 9 more
- Volunteers
- 717 people
- Sponsor
- Ardea Biosciences, Inc.
Hyperlipoproteinemia Type I
Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other Studies
LPL (Lipoprotein Lipase) is an enzyme which plays an important role in the elimination of triglycerides (fat) and the clearance of dietary fat particles known as chylomicrons (CM) in the blood.
- Trial name
- NCT01447901
- Where
- Canada
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Amsterdam Molecular Therapeutics
Pyruvate Dehydrogenase Complex Deficiency
Expanded Access Treatment Protocol With DCA for Patients With PDCD
Expanded Access (EA) will provide a transition to continue therapy for those patients who are currently in the open label extension of the Phase III study, SL 1009-01, while also allowing new patients diagnosed with PDCD who meet…
- Trial name
- NCT06931262
- Testing
- Dichloroacetate (DCA)
- Sponsor
- Saol Therapeutics Inc
Inherited Metabolic Disorders IMD
Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
This study is designed to assess the safety and exploratory efficacy of using HSC835 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT02715505
- Testing
- Umbilical cord blood transplantation with HSC835
- Sponsor
- Novartis Pharmaceuticals
Phenylketonuria
A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria
The main goal of this study is to assess the safety, and tolerability of multiple doses of mRNA-3210 in participants with phenylketonuria (PKU).
- Trial name
- NCT06147856
- Testing
- mRNA-3210
- Sponsor
- ModernaTX, Inc.
Gaucher Disease
Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
The purpose of this study is to learn more about Gaucher disease. The information we collect from medical histories and a blood sample from people with Gaucher disease may help us pinpoint certain things that are different…
- Trial name
- NCT00351156
- Testing
- Blood sample
- Where
- 7 sites in United States
- Volunteers
- 50 people
- Sponsor
- Amicus Therapeutics
Lipoprotein Lipase Deficiency
Study to Re-assess and Re-confirm Data Previously Recorded About the Incidence and Severity of Acute Abdominal "Pancreatitis" Episodes in Lipoprotein Lipase Deficient (LPLD) Subjects Previously Enrolled on AMT Clinical Studies
Lipoprotein lipase deficiency (LPLD) is an autosomal recessive inherited condition caused by homozygosity or compound heterozygosity for mutations within the LPL gene.
- Trial name
- NCT01448577
- Where
- 2 sites in Canada
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Amsterdam Molecular Therapeutics
Hypercholesterolemia, Hyperlipoproteinemia Type II
Safety Study of AEM-28 to Treat Refractory Hypercholesterolemia
The purpose of the first part of this study is to determine the safety and tolerability of a single dose of AEM-28, an apolipoprotein E mimetic, in subjects with high total cholesterol who are otherwise healthy subjects.
- Trial name
- NCT02100839
- Testing
- AEM-28, Normal Saline
- Where
- Australia
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- LipimetiX Development, LLC
Ornithine Transcarbamylase Deficiency
Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency
This Phase 1/2, first-in-human study will evaluate the safety and tolerability of single escalating doses of MRT5201 administered intravenously to subjects with OTC Deficiency (OTCD).
- Trial name
- STEP-OTC (NCT03767270)
- Testing
- MRT5201
- Sponsor
- Translate Bio, Inc.
Prader-Willi Syndrome, Obesity
Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome
The purpose of this study is to evaluate efficacy and safety of ZGN-440 (beloranib) in obese adolescent and adult subjects with Prader-Willi Syndrome.
- Trial name
- bestPWS (NCT02179151)
- Testing
- ZGN-440 for Injectable Suspension
- Where
- 15 sites in United States
- Volunteers
- 108 people
- Sponsor
- Zafgen, Inc.
Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency
Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
Kuvan® is a synthetic copy of a body's own substance called tetrahydrobiopterin (BH4). BH4 is required by the body to use an amino acid called phenylalanine in order to build another substance called tyrosine.
- Trial name
- KAMPER (NCT01016392)
- Where
- 69 sites in Austria, France and 7 more
- Volunteers
- 627 people
- Sponsor
- BioMarin Pharmaceutical