Clinical trials: Effect Neonatal
Phenylketonuria
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
The study is a single-cohort, interventional, open-label trial to evaluate long-term neurocognitive (NC) outcomes in children aged 4 to 5 years with phenylketonuria (PKU) treated with Kuvan® and Phenylalanine-restricted diet over…
- Trial name
- KOGNITO (NCT01965912)
- Testing
- Kuvan®
- Where
- 12 sites in Germany, Italy and 2 more
- Volunteers
- 34 people
- Sponsor
- BioMarin Pharmaceutical
Prader-Willi Syndrome
A 5 Treatment Period Pharmacokinetic Study Evaluating Dose Proportionality and Food Effects of Diazoxide Choline Controlled-Release Tablet (DCCR)
Open label, parallel-group, single site, 5 treatment-period study with 4 dose levels of DCCR, 1 of which is administered both with and without food.
- Trial name
- NCT02893618
- Testing
- Diazoxide choline controlled-release tablet
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Essentialis, Inc.
IBS, Constipation Predominant
The Beneficial Effects on the Intestinal Function of a Food Supplement Based on an Extract of Green Tea and a Pool of Probiotics in Subjects With Irritable Bowel Syndrome With Predominance of Constipation
The study will aim to evaluate the effectiveness of the use of a food supplement, based on a green tea extract (Camellia sinensis (L.) Kuntze - folium) and a pool of probiotics (L. plantarum, L. rhamnosus and B. animalis subsp.
- Trial name
- NCT07094035
- Testing
- food supplement based on a mixture of green tea extract and a pool of probiotics.
- Where
- Italy
- Volunteers
- 64 people
- Sponsor
- Adamas Biotech S.r.l.
Sickle Cell Disease
Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects
The purpose of this study to evaluate the effect of concomitant administration of GBT440 on caffeine (a CYP1A2 probe substrate), S warfarin (a CYP2C9 probe substrate), omeprazole (a CYP2C19 probe substrate), and midazolam (a…
- Trial name
- NCT02567682
- Testing
- GBT440
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
Homozygous Familial Hypercholesterolemia
Study to Evaluate the Effects of MBX-8025 in Patients With HoFH
A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH).
- Trial name
- NCT02472535
- Testing
- MBX-8025 50 mg (Dose Escalation Period 1), MBX-8025 50 mg or 100 mg (Dose Escalation Period 2), MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)
- Where
- 5 sites in Canada, France and 2 more
- Volunteers
- 13 people
- Sponsor
- Gilead Sciences
Growth Failure, Congenital Heart Disease
A Study to Evaluate the Effects of a New Formula on the Growth, Safety and Tolerance of Infants With Growth Failure
This study is a prospective, open-label, multi-site, growth, safety and tolerance study to evaluate a NF (New Formula). A minimum of 45 evaluable infants with confirmed growth failure will be enrolled.
- Trial name
- NCT03563391
- Testing
- New Infant Formula
- Where
- 5 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nutricia North America
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Healthy Volunteer, Cystic Fibrosis
Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis
This trial will consist of two parts: Part 1 and Part 2. Part 1 will enroll adult healthy volunteers (HV) into four treatment groups.
- Trial name
- NCT03140527
- Testing
- PTI-801, PTI-808
- Where
- 38 sites in United States, Canada and 3 more
- Volunteers
- 171 people, healthy volunteers welcome
- Sponsor
- Proteostasis Therapeutics, Inc.
Healthy Adults
A Study to Learn if a Medicine That Reduces Stomach Acid Affects the Blood Level of Study Medicine PF-08049820 in Healthy Chinese Adults.
This clinical study is designed to evaluate a study medicine called PF-08049820. The main aim is to understand how the body processes this medicine and whether giving it with another medicine, rabeprazole, affects how it behaves.
- Trial name
- NCT07284173
- Testing
- PF-08049820, Rabeprazole
- Where
- 2 sites in China
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Pfizer
Diarrhea, Atopic Dermatitis
Cesarean Section Study
Effect, tolerance and safety of a supplementation with a probiotic on the risk of gastrointestinal infections, on growth, and on gut microbiota in healthy newborn term infants born by Cesarean section.
- Trial name
- NCT01992497
- Testing
- Probiotic
- Where
- Germany
- Volunteers
- 721 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Dermatitis, Atopic
A Study to Test the Effect of 2 Different Doses of Topical GW870086X on Atopic Dermatitis Also Including a Postive Control and a Placebo
This study is a randomised, double-blind, placebo-controlled study to assess the efficacy of GW870086X cream formulation in subjects with moderate to severe atopic dermatitis.
- Trial name
- NCT01299610
- Testing
- GW870086 2.0%, GW870086 0.2%, FP 0.05%
- Where
- Germany
- Volunteers
- 25 people
- Sponsor
- GlaxoSmithKline
Thalassemia (Transfusion Delendent)
Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
The CENTAurus trial is a prospective clinical study designed to address systematically some of the relevant endocrine complications in an iron overloaded thalassemic population, primary objective being the assessment of the…
- Trial name
- CENTAurus (NCT02069886)
- Testing
- deferasirox
- Sponsor
- Novartis Pharmaceuticals
Healthy Volunteers, Polycystic Kidney, Autosomal Dominant
Effect of Venglustat in Patients With Renal Impairment
Primary Objective: To study the effect of mild, moderate and severe renal impairment on the pharmacokinetics (PK) of Venglustat following a single dose.
- Trial name
- NCT03687554
- Testing
- Venglustat GZ/SAR402671
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Skin Abnormalities
Moisturizing Effect of Two Dexpanthenol Formulations
The study focuses to prove the equivalent moisturizing effect of two different Dexpanthenol formulations
- Trial name
- NCT00839280
- Testing
- Dexpanthenol foam spray, new formulation, Dexpanthenol foam spray, old formulation
- Where
- France
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Bayer
Tourette Syndrome, Chronic Motor Tic Disorder
A Study to Evaluate the Effects of ABX-1431 on Patients With Tourette Syndrome
The study will investigate the effects and the safety of a single-dose of ABX-1431 HCl on tics and other symptoms of Tourette Syndrome. During part 1 (periods 1 and 2) each patient will receive study drug once and placebo once.
- Trial name
- NCT03058562
- Testing
- ABX-1431
- Where
- Germany
- Volunteers
- 23 people
- Sponsor
- Abide Therapeutics
Healthy Volunteers, Atopic Diathesis and Atopic Dermatitis
Single Dose and Multiple Dose Study to Assess Safety and Tolerability of LOU064
This is a 6-part first-in-human study in up to approximately 184 participants. Parts 1 to 5 is in health volunteers and part 6 is in subjects with atopic dermatitis.
- Trial name
- NCT03918980
- Testing
- LOU064
- Where
- 2 sites in Germany, Netherlands
- Volunteers
- 185 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
The purpose of the study is to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease
- Trial name
- HESTIA3 (NCT03615924)
- Testing
- Ticagrelor
- Where
- 55 sites in United States, Belgium and 14 more
- Volunteers
- 193 people
- Sponsor
- AstraZeneca
Atopic Dermatitis, Atopic
A Phase 2b Study of the Effects of Camoteskimab in Adults With Moderate-to-Severe Atopic Dermatitis
This is a phase 2b, multicenter, randomized, double-blind, placebo-controlled study.
- Conditions
- Atopic Dermatitis, Atopic, Dermatitis, Dermatitis, Atopic
- Trial name
- NCT07599813
- Testing
- Camoteskimab
- Where
- 85 sites in United States, Bulgaria and 6 more
- Volunteers
- 280 people
- Sponsor
- Apollo Therapeutics Ltd
Tourette Syndrome, Tourette Syndrome in Adolescence
A Study to Explore the Effect of Sepranolone in Tourette Syndrome
The study was an open-label, randomized, multicenter, parallel, Phase 2a study in adolescents and adult patients with Tourette syndrome that aimed to explore the efficacy of Sepranolone as a treatment for Tourette syndrome, by…
- Trial name
- NCT05434546
- Testing
- Sepranolone
- Where
- 2 sites in Denmark
- Volunteers
- 26 people
- Sponsor
- Asarina Pharma
Healthy Volunteers, Cryopyrin Associated Periodic Syndrome
Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334
This is a first in human (FIH), single-centre, double -blind, randomised, cross-over, SAD followed by a MAD study of IZD334 conducted in healthy adult participants as well as an open-label cohort in adult patients with CAPS.
- Trial name
- NCT04086602
- Testing
- IZD334
- Where
- Australia
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Inflazome UK Ltd