Clinical trials: Genetics
Transthyretin (TTR) Amyloid Cardiomyopathy
Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy
This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or…
- Trial name
- ATTR-ACT (NCT01994889)
- Testing
- Tafamidis
- Where
- 80 sites in United States, Belgium and 11 more
- Volunteers
- 441 people
- Sponsor
- Pfizer
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study of NIDO-361 in Patients With SBMA
The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- PIONEER KD (NCT06411912)
- Testing
- NIDO-361
- Where
- 5 sites in Denmark, Italy and 2 more
- Volunteers
- 54 people
- Sponsor
- Nido Biosciences, Inc.
Cystic Fibrosis
Phase 3 Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy Regimen for the Treatment of Chronic Pseudomonas Aeruginosa Infection in Patients With CF
The primary objective of this study is to evaluate the safety and efficacy of a CAT regimen with aztreonam for inhalation solution (AZLI) and tobramycin inhalation solution (TIS) in adult and pediatric subjects with cystic…
- Trial name
- AZLI CAT (NCT01641822)
- Testing
- AZLI, Tobramycin inhalation solution
- Where
- 71 sites in United States
- Volunteers
- 107 people
- Sponsor
- Gilead Sciences
Epilepsy, Dravet Syndrome
An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Adults With Dravet or Lennox-Gastaut Syndromes
To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and adults with Dravet or Lennox-Gastaut syndromes.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- GWPCARE5 (NCT02224573)
- Testing
- GWP42003-P
- Volunteers
- 681 people
- Sponsor
- GW Research Ltd
Pulmonary Hypertension, Sickle Cell Disease
Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease…
- Conditions
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- Trial name
- NCT01048905
- Testing
- L-Glutamine
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Atopic Dermatitis
A Study to Learn About Abrocitinib Tablets in People With Atopic Dermatitis in India
The purpose of this clinical trial is to learn about the safety and how well the study medicine (called Abrocitinib) works for the potential treatment of moderate to severe Atopic Dermatitis (AD) in India.
- Trial name
- NCT05375929
- Testing
- Abrocitinib 100 mg, Abrocitinib 200 mg
- Where
- 15 sites in India
- Volunteers
- 200 people
- Sponsor
- Pfizer
Hemophilia
Weight-based Dosing in Hemophilia A
Hemophilia A is an inherited (genetic) disease where a protein, factor VIII (FVIII), which promotes blood clotting is missing or does not work properly. Individuals with hemophilia A are at risk for bleeding.
- Trial name
- NCT02586012
- Testing
- rFVIII
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Craig Seaman
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Huntington's Disease
A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease
This is a multicenter, multinational, randomized, parallel-group, double-blind, placebo-controlled, dose range finding study to compare the efficacy and safety of different doses of pridopidine versus placebo in the treatment of…
- Trial name
- NCT02006472
- Testing
- Pridopidine
- Where
- 58 sites in United States, Australia and 10 more
- Volunteers
- 408 people
- Sponsor
- Prilenia
Acute Gout
Targeted Dose Finding of Canakinumab (ACZ885) for Management of Acute Flare in Refractory or Contraindicated Gout Patients
This 8-week study is designed to determine the target dose of canakinumab (ACZ885) for the management of acute flare in gout patients who are contraindicated to Non-Steroidal anti-inflammatory drugs and/or colchicine.
- Trial name
- NCT00798369
- Testing
- Canakinumab, Triamcinolone acetonide
- Where
- 75 sites in United States, Argentina and 9 more
- Volunteers
- 200 people
- Sponsor
- Novartis
Hemophilia A, Hemophilia B
An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa
The goal of this low-interventional study is to describe the overall joint health in patients with haemophilia A or haemophilia B prophylactically treated with rFVIIIFc or rFIXFc.
- Trial name
- JOIN-us (NCT05856266)
- Testing
- Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US), Haemophilia Joint Health Score (HJHS)
- Where
- 56 sites in Bulgaria, Croatia and 9 more
- Volunteers
- 10 people
- Sponsor
- Swedish Orphan Biovitrum
Atherosclerotic Cardiovascular Disease, Symptomatic Atherosclerosis
An Extension Trial of Inclisiran in Participants With Cardiovascular Disease and High Cholesterol
This clinical study was designed to assess the efficacy, safety, and tolerability of long-term dosing of inclisiran and evolocumab given as subcutaneous injections in participants with high cardiovascular risk and elevated…
- Conditions
- Atherosclerotic Cardiovascular Disease, Symptomatic Atherosclerosis, Type2 Diabetes, Familial Hypercholesterolemia
- Trial name
- ORION-3 (NCT03060577)
- Testing
- Inclisiran, Evolocumab
- Where
- 52 sites in United States, Canada and 3 more
- Volunteers
- 382 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis
Tralokinumab in Combination With Topical Corticosteroids for Moderate to Severe Atopic Dermatitis - ECZTRA 3
Primary objective: To demonstrate that tralokinumab in combination with topical corticosteroids (TCS) is superior to placebo in combination with TCS in treating moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT03363854
- Testing
- Tralokinumab
- Where
- 66 sites in United States, Belgium and 6 more
- Volunteers
- 380 people
- Sponsor
- LEO Pharma
Hemophilia A, Hemophilia B
Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen
The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.
- Trial name
- NCT01757405
- Testing
- Recombinant Factor VIIa BI (rFVIIa BI)
- Where
- 16 sites in United States, Japan and 7 more
- Volunteers
- 40 people
- Sponsor
- Baxalta now part of Shire
Cystic Fibrosis
Comparative Analysis of CO2 Monitoring Methods in Patients With CF Undergoing General Anesthesia
Four methods are routinely used to monitor CO2 in patients. End tidal CO2 (EtCO2) is monitored through the endotracheal tube during general anesthesia.
- Trial name
- NCT03734822
- Testing
- End tidal CO2 (EtCO2), Transcutaneous CO2 (TCO2), Capillary CO2 (CapCO2)
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy
Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients
The specific aim of this Phase I/II study is to assess the safety of intravenous administered Morpholino oligomer directed against exon 51 (AVI-4658 PMO).
- Trial name
- NCT00844597
- Testing
- AVI-4658 for Injection
- Where
- 2 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Sarepta Therapeutics, Inc.
Poliomyelitis
Phase 1 Novel Live Attenuated Serotype 2 Oral Polio Vaccine Study in Inactivated Polio Vaccine (IPV) Primed Adults
This first-in-human (FIH) phase 1 study is designed to evaluate in contained conditions the safety, immunogenicity, shedding, and genetic stability of two novel oral polio vaccine type 2 (nOPV2) vaccine candidates in IPV-primed…
- Trial name
- nOPV2M4a (NCT03430349)
- Testing
- Novel OPV2 candidate 1, Novel OPV2 candidate 2
- Where
- Belgium
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Pierre Van Damme
Hypercholesterolemia, Hypercholesterolemia, Familial
Evaluating the Efficacy of Pediatric Lipid Screening Alerts
The purpose of the study is to evaluate prospectively the impact of different system alerts on the prescription of lipid panels to pediatric Geisinger patients (9-11 years old), as per the now-universal guidelines.
- Conditions
- Hypercholesterolemia, Hypercholesterolemia, Familial, Hypercholesteremia in Children, Hyperlipidemia in Children
- Trial name
- NCT04118348
- Testing
- Best Practice Alert, Health Maintenance Topic
- Where
- United States
- Volunteers
- 13,340 people
- Sponsor
- Geisinger Clinic
Tourette Syndrome
Safety and Tolerability Study of NBI-98854 for the Treatment of Subjects With Tourette Syndrome
Phase 2, open-label, fixed-dose titration study to evaluate the safety and tolerability of NBI-98854 administered once daily for a total of 24 weeks in children, adolescents, and adults with Tourette Syndrome (TS).
- Trial name
- NCT02879578
- Testing
- Valbenazine
- Where
- 13 sites in United States
- Volunteers
- 155 people
- Sponsor
- Neurocrine Biosciences
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome
A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid beta-glucosidase, also known as glucocerebrosidase.
- Conditions
- Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00358150
- Testing
- Eliglustat tartrate
- Where
- 17 sites in United States, Argentina and 4 more
- Volunteers
- 26 people
- Sponsor
- Genzyme, a Sanofi Company