Clinical trials: Effect Neonatal
ADPKD (Autosomal Dominant Polycystic Kidney Disease)
A Phase 1 Study of Single and Multiple Ascending Doses of GV-100
This is a first-in-human, multi-part clinical study designed to evaluate the safety, tolerability, pharmacokinetics (PK), food effect, and drug-drug interaction (DDI) potential of GV-100 following oral administration in healthy…
- Trial name
- NCT07788014
- Testing
- GV-100
- Where
- Australia
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Gilva Therapeutics Inc.
Healthy
A Study of the Cardiac Effects of ALXN1840 in Healthy Adults
This study will evaluate the effect of a supratherapeutic dose of ALXN1840 on the heart rate (HR)-corrected QT interval (QTc) in healthy adult participants. Moxifloxacin will be used as the active control.
- Trial name
- NCT04560816
- Testing
- ALXN1840, Moxifloxacin
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Anemia, Sickle Cell
Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease
The purpose of the study is to determine the maximum tolerated dose, safety and effect on induction of fetal hemoglobin of pomalidomide in patients with Sickle Cell Disease.
- Trial name
- SCD-001 (NCT01522547)
- Testing
- pomalidomide
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Celgene
Hypertriglyceridemia, Mixed Hyperlipidemia
A Pilot Study to Evaluate the Lipid Effects of TRIA-662
The purpose of this pilot study is to learn what study factors are important in designing a large, full-scale study of the effects of TRIA-662 on serum triglycerides (TG) and high-density lipoprotein cholesterol (HDL-C) levels.
- Trial name
- NCT02008084
- Testing
- TRIA-662
- Where
- 17 sites in Canada
- Volunteers
- 71 people
- Sponsor
- Cortria Corporation
Myotonic Dystrophy 1
Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1
The purpose of this study is to determine whether MYODM (formulated composition containing Theobroma cacao supplemented with caffeine (caffeine/theobromine ratio1/1.85, w/w) is effective in the treatment of excessive daytime…
- Trial name
- NCT04634682
- Testing
- MYODM
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- Myogem Health Company, S.L.
Ventricular Ejection Fraction, LVF
AI-Enabled Direct-from-ECG Ejection Fraction (EF) Severity Assessment Using COR ECG Wearable Monitor
This prospective, multicenter, cluster-randomized controlled study aims to evaluate the accuracy of an investigational artificial intelligence (AI) Software as a Medical Device (SaMD) designed to compute ejection fraction (EF)…
- Conditions
- Ventricular Ejection Fraction, LVF, LV Dysfunction, Atrial Enlargement
- Trial name
- EFACT (NCT06699056)
- Testing
- 15-minutes of sitting during COR ECG Acquistion
- Where
- 8 sites in United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Peerbridge Health, Inc
Cystic Fibrosis
Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation
Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic…
- Trial name
- NCT02564354
- Testing
- QR-010
- Where
- 5 sites in United States, Belgium and 1 more
- Volunteers
- 18 people
- Sponsor
- ProQR Therapeutics
Achondroplasia
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
- Trial name
- NCT03197766
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 121 people
- Sponsor
- BioMarin Pharmaceutical
Healthy Volunteers, Sickle Cell Disease
A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants
Novo Nordisk is developing a new study medicine, Etavopivat, to treat individuals with sickle cell disease (SCD). The purpose of the study is to determine the effect of Etavopivat on the electrical activity of the heart in…
- Conditions
- Healthy Volunteers, Sickle Cell Disease, Thalassemia
- Trial name
- NCT07023029
- Testing
- Etavopivat, Moxifloxacin
- Where
- United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Dyslipidemias, Familial Hypercholesterolemia
Study of ARO-ANG3 in Healthy Volunteers and in Dyslipidemic Patients
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetcs and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial…
- Conditions
- Dyslipidemias, Familial Hypercholesterolemia, Hypertriglyceridemia
- Trial name
- NCT03747224
- Testing
- ARO-ANG3, sterile normal saline (0.9% NaCl)
- Where
- 6 sites in Australia, New Zealand
- Volunteers
- 93 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Cystic Fibrosis
A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation
This is a Phase 4, randomized, double-blind, placebo-controlled, parallel-group study in subjects aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation.
- Trial name
- NCT02875366
- Testing
- LUM/IVA
- Where
- 12 sites in Australia, United Kingdom
- Volunteers
- 70 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Healthy
Study of PF 04965842 Effect on MATE1/2K Activity in Healthy Participants
This is a Phase 1, randomized, 2 way crossover, open label study of the effect of PF-04965842 on metformin (a probe for MATE1/2K activity) PK in healthy adult participants.
- Trial name
- NCT03796182
- Testing
- Metformin, PF-04965842
- Where
- Belgium
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Pfizer
Spinocerebellar Ataxia
Effects of Oral Administration of Antrodia Cinnamomea Products for Clinical Symptoms in Spinocerebellar Ataxia Patients
This study aims to assess the effect of Antrodia cinnamomea on clinical symptoms in spinocerebellar ataxia patients. To investigate the advancements in neurodegenerative diseases.
- Trial name
- NCT06633003
- Testing
- Antrodia cinnamomea Products
- Where
- Taiwan
- Volunteers
- 60 people
- Sponsor
- ALPS Biotech CO. LTD
Hereditary Angioedema
A Study of the Relative Bioavailability of a New Formulation of BCX4161 and the Effect of Food on BCX4161
The purpose of the study is to compare how the body takes up and then eliminates the study drug BCX4161 when it is taken orally as a hard gelatin capsule and as a soft gelatin capsule.
- Trial name
- NCT02125162
- Testing
- BCX4161
- Where
- United Kingdom
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Hemophilia A, Prophylaxis of Bleeding
An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A.
This is an observational study in which data from people with hemophilia A who decide on their own or by recommendation of their doctors to take Jivi are collected and studied.
- Trial name
- JOIHA (NCT05643560)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- Italy
- Volunteers
- 33 people
- Sponsor
- Bayer
Atopic Dermatitis
Cutivate Lotion HPA Axis Pediatric Study
A multi-center, open-label, Phase IV, unblinded study using Cutivate (fluticasone propionate, 0.05%)lotion and it's possible effects on the HPA axis of infants diagnosed with atopic dermatitis.
- Trial name
- NCT00546000
- Testing
- Fluticasone propionate 0.05% lotion
- Where
- 10 sites in United States
- Volunteers
- 56 people
- Sponsor
- Fougera Pharmaceuticals Inc.
Atopic Dermatitis (Eczema)
The Effects of a Botanical Moisturizer, an Oral Supplement, and an Unscented Soap on Eczema and Itch
The purpose of this study is to examine the effects of a botanical moisturizer, an oral supplement, and an unscented soap on eczema severity, itch, mood, and various skin measures (such as skin hydration) in those with mild to…
- Trial name
- NCT06819709
- Testing
- Botanical Moisturizer + Oral Supplement + Unscented Soap
- Where
- Poland
- Volunteers
- 35 people
- Sponsor
- Codex Labs Corporation
Healthy Volunteers
Single and Multiple Dose Study to Evaluate Safety and Pharmacokinetics of BMS-986533 in Healthy Participants and Assessments of Food and pH Effects on Relative Bioavailability, and Drug-Drug Interaction Potential in Healthy Participants
The purpose of this study is to evaluate the safety and pharmacokinetics of BMS-986533 in healthy participants receiving single and multiple doses, to assess food and pH effects on the relative bioavailability of BMS-986533, and…
- Trial name
- NCT07629999
- Testing
- BMS-986533, Famotidine, Dabigatran Etexilate
- Where
- United States
- Volunteers
- 136 people, healthy volunteers welcome
- Sponsor
- Bristol-Myers Squibb
Pregnancy Related
Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation
The participants of this study will be of any age who are exposed to at least 1 dose of odevixibat at any time during pregnancy (from 1 day prior to conception to pregnancy outcome) and/or at any time during lactation (up to 12…
- Trial name
- NCT06258902
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Ipsen
Atopic Dermatitis
The Purpose of the Study is to Evaluate the Effect of Ruxolitinib Cream on Itch in Participants With Atopic Dermatitis
The purpose of the study is to evaluate the effect of ruxolitinib cream on itch in participants with Atopic Dermatitis.
- Trial name
- SCRATCH-AD (NCT04839380)
- Testing
- ruxolitinib cream
- Where
- 2 sites in Canada
- Volunteers
- 49 people
- Sponsor
- Incyte Corporation