Clinical trials: Effect Neonatal
Cystic Fibrosis, Pulmonary
Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of single doses of ARO-ENaC in healthy adult volunteers; and to evaluate the safety, tolerability, PK and efficacy of multiple doses of…
- Trial name
- NCT04375514
- Testing
- ARO-ENaC
- Where
- 7 sites in Australia, New Zealand
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Cystic Fibrosis
Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation
The purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D-CFTR mutation on at least 1 allele.
- Trial name
- NCT01262352
- Testing
- Ivacaftor
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Gout
A Clinical Trial of HEC93077 in Chinese Healthy Subjects and HUA Patients With Single or Multiple Dosing
A phase 1 clinical trial of HEC93077,to evaluate the safety ,PK,PD and food effect in Chinese healthy subjects and HUA patients after single or multiple dosing.
- Trial name
- NCT05127772
- Testing
- HEC93077
- Where
- China
- Volunteers
- 126 people, healthy volunteers welcome
- Sponsor
- Sunshine Lake Pharma Co., Ltd.
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Hypertrophic, Familial
Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure
The purpose of this study is to evaluate the effect of perhexiline on exercise performance (efficacy) and safety in patients with hypertrophic cardiomyopathy and moderate-to-severe heart failure following dosing for 16 weeks.
- Trial name
- NCT02862600
- Testing
- Perhexiline, Use of bioanalytical assay to monitor plasma levels of perhexiline
- Where
- 11 sites in United States
- Volunteers
- 35 people
- Sponsor
- Heart Metabolics Limited
Irritable Bowel Syndrome
A Phase 1, Randomized, Placebo-Controlled, Ascending Cohort, Dose Escalation Study in Normal Healthy Volunteers
GSK3179106 is a potent and relatively selective inhibitor of RET kinase which has been designed to be a safe and effective therapy for irritable bowel syndrome (IBS) patients .
- Trial name
- NCT02727283
- Testing
- GSK3179106
- Where
- Australia
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Infantile Colic
Effect of Probiotic Limosilactobacillus Reuteri (L. Reuteri) on Crying and Fussing Time in Infants With Colic
This is a double-blind, randomized, placebo-controlled, parallel-group study in infants with colic with the primary objective to evaluate crying and fussing time.
- Trial name
- NCT06462651
- Testing
- L. reuteri
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- BioGaia AB
Romano-Ward Syndrome, Long QT Syndrome
Evaluation of the Effects of KCNQ1 Mutation on Insulin Tolerance and Obsessive Compulsive Features in Long QT Romano-Ward Syndrome Patients.
The objectives of the study are to investigate if KCNQ1 mutation in Romano-Ward long QT patients can be associated with changes in insulin regulation and with psychological features of compulsivity, impulsivity and behavioural…
- Conditions
- Romano-Ward Syndrome, Long QT Syndrome, Compulsive Behavior, Healthy Individuals
- Trial name
- PRIME (NCT04715256)
- Testing
- Cognitive assessment of obsessive-compulsive and impulsive behaviours, Genomic analysis, Glucoregulation assessment
- Where
- 2 sites in France
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Biotrial
Sickle Cell Disease
A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
- Trial name
- NCT05036512
- Testing
- GBT021601
- Where
- 4 sites in United States, Australia
- Volunteers
- 129 people, healthy volunteers welcome
- Sponsor
- Pfizer
Infantile Colic, Constipation
Randomized Placebo-controlled Study of L. Reuteri NCIMB 30351 in GI Functional Disorders and Food Allergy in Newborns
This study is a randomized, placebo-controlled, single-masked (blinded), post-marketing clinical study of a drug Lactobacillus Reuteri NCIMB 30351 drops in functional disorders of gastrointestinal tract and skin symptoms of food…
- Conditions
- Infantile Colic, Constipation, Infantile Diarrhea, Gastro Esophageal Reflux
- Trial name
- NCT04262648
- Testing
- Lactobacillus Reuteri
- Where
- 2 sites in Russia
- Volunteers
- 90 people
- Sponsor
- NovoNatum Ltd
Fabry Disease
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat)…
- Trial name
- CARAT (NCT05280548)
- Testing
- Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828)
- Where
- 54 sites in United States, Austria and 17 more
- Volunteers
- 104 people
- Sponsor
- Sanofi
Dyslipidemias, High Cholesterol
Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.
This study will be a placebo-controlled, double-blind, randomized, phase 3 study to Evaluate the Efficacy, Safety, and Tolerability of Obicetrapib in Participants with a History of Heterozygous Familial Hypercholesterolemia…
- Conditions
- Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia
- Trial name
- BROOKLYN (NCT05425745)
- Testing
- Obicetrapib
- Where
- 96 sites in United States, Canada and 8 more
- Volunteers
- 354 people
- Sponsor
- NewAmsterdam Pharma
Eczema, Atopic Dermatitis
Study Testing Response Effect of KY1005 Against Moderate-to-Severe Atopic Dermatitis, The STREAM-AD Study
This is an interventional, randomized, parallel group, treatment, Phase IIb, double blind, 5-arm study to assess the effect of Anti-OX40L Monoclonal Antibody (KY1005) in adult participants with moderate to severe atopic…
- Trial name
- STREAM-AD (NCT05131477)
- Testing
- Amlitelimab
- Where
- 103 sites in United States, Australia and 10 more
- Volunteers
- 390 people
- Sponsor
- Kymab Limited
Pulmonary/Respiratory Diseases
A Study to Assess the Effect of AZD5634 on Mucociliary Clearance, Safety, Tolerability and Pharmacokinetic Parameters in Patients With Cystic Fibrosis
This study will assess the effect of inhaled AZD5634 on Mucociliary clearance (MCC) in patients with Cystic fibrosis (CF) after single-dose administration.
- Trial name
- NCT02950805
- Testing
- AZD5634
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- AstraZeneca
Breast Cancer, Solid Tumors, Adult
First-in-Human Study of Tersolisib (STX-478) as Monotherapy and in Combination With Other Antineoplastic Agents in Participants With Advanced Solid Tumors
Study STX-478-101 (LY4064809) is a multipart, open-label, phase 1/2 study evaluating the safety, tolerability, pharmacokinetics (PK), and preliminary antitumor activity of STX-478 (LY4064809) in participants with advanced solid…
- Trial name
- PIKALO-1 (NCT05768139)
- Testing
- STX-478, Fulvestrant, Ribociclib
- Where
- 65 sites in United States, Belgium and 7 more
- Volunteers
- 880 people
- Sponsor
- Eli Lilly and Company
Atopic Dermatitis
A Clinical Study of HRS-8797 in Healthy Participants
The purpose of this phase I study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of HRS-8797 in healthy participants.
- Trial name
- NCT07698756
- Testing
- HRS-8797
- Where
- China
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
Atopic Dermatitis
Vaccine Responses in Tralokinumab-Treated Atopic Dermatitis - ECZTRA 5 (ECZema TRAlokinumab Trial No. 5)
The purpose of this trial is to test if treatment with the trial drug, tralokinumab, can affect the body's immune response to vaccines.
- Trial name
- ECZTRA 5 (NCT03562377)
- Testing
- Tralokinumab, Tdap vaccine, Meningococcal vaccine
- Where
- 51 sites in United States, Canada
- Volunteers
- 215 people
- Sponsor
- LEO Pharma
Classical Homocystinuria
The Effect of Dietary Management and Cysteine Supplementation on Growth Parameters and Biochemical Control for Pediatric Qatari Patients Affected with Classical B6 Non-responsive Homocystinuria.
Classical homocystinuria (HCU) is an autosomal recessive disorder caused by the deficiency of an enzyme cystathionine β-synthase (CβS) that affects the catabolic pathway of the amino acid methionine (Met) which leads to an…
- Trial name
- NCT06622577
- Testing
- Cysteine
- Where
- Qatar
- Volunteers
- 50 people
- Sponsor
- Hamad Medical Corporation
Irritable Bowel Syndrome, Wheat Sensitivity
Effects of Wheat Breads on Symptoms of IBS, a Pilot Study
This is randomized pilot study comparing tolerability of yeast-baked wheat bread and sourdough wheat bread in irritable bowel syndrome.
- Trial name
- Wheat-IBS (NCT02572908)
- Testing
- Sourdough wheat bread, Regular yeast baked toast bread, Gluten-free diet
- Where
- Finland
- Volunteers
- 26 people
- Sponsor
- Oy Karl Fazer Ab
Infantile Colic
Evaluation of Safety and Efficacy of New Infant Formula in Infantile Colic
The study is conducted to examine the safety and efficacy of a new infant formula and its effects on the gastrointestinal tolerance in infants suffering from colic.
- Trial name
- Coco (NCT01721850)
- Testing
- control formula, intervention formula 1, intervention formula 2
- Where
- 18 sites in Germany, Italy
- Volunteers
- 169 people, healthy volunteers welcome
- Sponsor
- HiPP GmbH & Co. Vertrieb KG
Gout
D-0120 Safety and PK/PD, Food Effect Study in Healthy Volunteers
This initial clinical study in the US will be a randomized, double-blind, placebo-controlled, single-dose, dose-escalation, and sequential cohort study to evaluate the safety, tolerability, PK and PD of D-0120-NA in fasting…
- Trial name
- NCT03291782
- Testing
- D-0120
- Where
- United States
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- InventisBio Co., Ltd