Clinical trials: Effect Neonatal
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis
Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis
The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM.
- Conditions
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Trial name
- ASCEND-ATTR (NCT07695701)
- Testing
- Acoramidis (AG10) oral tablet
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Atopic Dermatitis
Study to Investigate the Effect of Rocatinlimab (AMG 451) on the Pharmacokinetics of Multiple Cytochrome P450 (CYP450) Substrates in Participants With Moderate to Severe Atopic Dermatitis
The primary objective of the study is to evaluate the pharmacokinetics (PK) of multiple cytochrome P450 (CYP450) substrates alone and in combination with rocatinlimab in participants with moderate to severe atopic dermatitis (AD).
- Trial name
- NCT05891119
- Testing
- Caffeine, Metoprolol, Midazolam
- Where
- 7 sites in United States
- Volunteers
- 21 people
- Sponsor
- Amgen
Sickle Cell Disease
Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD)
This is a pilot study of daily dosing of NUV001 as a dietary supplement in 12 sickle cell disease patients with 3 months of follow-up plus 1 month after supplementation.The present study is designed to evaluate, first, the safety…
- Trial name
- NCT05789355
- Testing
- NUV001
- Where
- France
- Volunteers
- 12 people
- Sponsor
- LGD
Sickle Cell Disease
Voxelotor Brain Oxygenation and Neurocognitive Study
This is an open label, single arm multicenter trial to evaluate the effect of voxelotor treatment on cerebral blood flow (CBF) and neurocognitive function in adolescent and young adult participants (12-30 years of age) with…
- Trial name
- NCT05228821
- Testing
- Voxelotor Oral Tablet
- Where
- 2 sites in United States
- Sponsor
- Pfizer
Fabry Disease
To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease
Primary Objective: To assess reduction of plasma lyso-GL3 level after switch to agalsidase beta from agalsidase alfa Secondary Objectives: To assess reduction of kidney podocyte GL3 content after switch to agalsidase beta from…
- Trial name
- BCLEAR1 (NCT04143958)
- Testing
- agalsidase beta (GZ419828), agalsidase alfa
- Sponsor
- Sanofi
Hematologic Disease, Hemophilia A
BAY14-2222 Prophylaxis and Joint Function Improvement (Adults)
The purpose of the study is to evaluate the effect of prophylactic treatment on the number of joint bleeds and quality of life in severe hemophilia A subjects compared to on-demand treatment in a one-group two-treatment schedule…
- Trial name
- NCT00586521
- Testing
- Kogenate (BAY14-2222)
- Where
- 11 sites in United States, France and 3 more
- Volunteers
- 20 people
- Sponsor
- Bayer
Wilson's Disease
A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.
- Trial name
- NCT01874028
- Testing
- trientine dihydrochloride
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Univar BV
Psoriasis, Atopic Dermatitis
Topical Application of BMX-010 in Subjects With Atopic Dermatitis and Plaque Psoriasis
This is a randomized, placebo-controlled Phase 2 trial consisting of up to 300 subjects with either psoriasis or atopic dermatitis. In this trial BMX-010 will be topically applied twice daily for up to 28 days.
- Trial name
- NCT03381625
- Testing
- BMX-010
- Where
- 10 sites in United States
- Volunteers
- 139 people
- Sponsor
- BioMimetix JV, LLC
Mixed Hyperlipidemia
Carotid B-mode Ultrasound Study to Compare Anti-Atherosclerotic Effect of Torcetrapib/Atorvastatin to Atorvastatin.
The Torcetrapib project was terminated on December 2, 2006 due to safety findings. To look at ultrasound images taken in the carotid arteries and to look at various lipids in the blood of people with mixed hyperlipidemia
- Trial name
- RADIANCE 2 (NCT00134238)
- Testing
- torcetrapib/atorvastatin, atorvastatin
- Where
- 51 sites in United States, Canada and 4 more
- Volunteers
- 755 people
- Sponsor
- Pfizer
Hereditary Angioedema
A Relative Bioavailability Study of Two Formulations of BCX7353
This is an open-label, randomized study to investigate the relative bioavailability of two formulations of BCX7353 and to determine if there is a food effect
- Trial name
- NCT03202784
- Testing
- BCX7353
- Where
- United Kingdom
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Pregnancy Related
Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy
The study will include participants who were exposed to at least one dose of elafibranor either within the three weeks before conception or at any time during pregnancy (based on estimated last menstrual period [LMP]).
- Trial name
- NCT07556913
- Testing
- Elafibranor
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Ipsen
Neonatal Sepsis
Potential Protective Effect of a Formula Supplemented With Fermented Matrices on the Risk of Developing Neonatal Sepsis
This is a multicenter, randomized, double-blind, placebo controlled trial, with parallel groups and reference group. The aim of the study was to evaluate the hypothesis that an immunonutritional strategy, based on use of…
- Trial name
- NCT04742582
- Testing
- Preterm Infants - fed fermented formula, Preterm Infants - fed standard formula, Pre-term Infants - breastfed
- Where
- Italy
- Volunteers
- 876 people
- Sponsor
- Heinz Italia SpA
Healthy Volunteers
Evaluation Of The Potential Effect That The Administration Of Food Or Antacid Medication May Have In The Oral Absorption Of Dacomitinib (PF-00299804)
Evaluation of the potential effect that the administration of food or antacid medication may have in the oral absorption of dacomitinib relative to the administration of dacomitinib in absence of food or antacid medication
- Trial name
- NCT01702506
- Testing
- dacomitinib fasted, dacomitinib fed, dacomitinib+antacid
- Where
- Belgium
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Pfizer
Dermatitis Atopic
An Open-label, DDI Study to Investigate the Effects of Amlitelimab on the PK of Selected Cytochrome P450 Substrates
This is an open-label, single group, 2-period, Phase 1, single-sequence study. The study duration will be up to 342 days. The treatment period will be up to Week 29, where Week 29 is defined as End of Treatment (last amlitelimab…
- Trial name
- NCT06686628
- Testing
- Amlitelimab, Midazolam, Caffeine
- Where
- Germany
- Volunteers
- 23 people
- Sponsor
- Sanofi
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
Acute Ischemic Stroke
Research Into the Effect of a Clot-dissolving Agent and Its Inhibitor
Single-chain urokinase-type plasminogen activator (pro-urokinase) is a highly effective thrombolytic drug. At pharmacologic concentrations however, pro-urokinase is converted to urokinase - a non specific thrombolytic, limiting…
- Trial name
- NCT01694381
- Testing
- M5, C1-inhibitor
- Where
- Netherlands
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- TSI, LLC
Mucopolysaccharidosis (MPS), Hunter Syndrome
A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter…
- Trial name
- NCT05494593
- Testing
- ELAPRASE, Rituximab, Methotrexate
- Where
- 8 sites in United States
- Sponsor
- Takeda
Retinitis Pigmentosa (RP), Usher Syndrome
Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual Performance
Retinitis pigmentosa and similar degenerative diseases of the retina lead to progressive loss of vision. TES therapy with the CE-marked OkuStim® System is a treatment approved in the EU for slowing the progression of the disease.
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome, Cone Rod Dystrophy
- Trial name
- TES-STEP (NCT07548944)
- Testing
- Transcorneal Electrical Stimulation using the OkuStim System
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Okuvision GmbH
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
Alzheimer's Disease, Huntington's Disease
A Phase 1, Randomized, Open-Label, Two-Way Crossover Study To Evaluate The Steady-State Effect Of Dimebon (PF 01913539) On The Single-Dose Pharmacokinetics And Pharmacodynamics Of Warfarin In Healthy Subjects
This study will evaluate the potential drug-drug interaction of Dimebon with the FDA-recommended CYP2C9 substrate warfarin in healthy subjects.
- Trial name
- NCT00827034
- Testing
- Warfarin, Dimebon
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Pfizer