Clinical trials: Newborn Infant
Cystic Fibrosis (CF), Non Cystic Fibrosis Bronchiectasis
Feasibility and Acceptance of SonoHeal for Airway Clearance and Preliminary Comparison With the Standard of Care
The proposed study is to gauge the early feasibility and acceptance of the SonoHeal device. The study will evaluate the usability of the SonoHeal prototype using well-established Human Factors Engineering methods recommended by…
- Conditions
- Cystic Fibrosis (CF), Non Cystic Fibrosis Bronchiectasis, Chronic Obstructive Pulmonary Disease (COPD)
- Trial name
- NCT07568119
- Testing
- Airway clearance therapy through acoustic oscillations
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Cognita Labs LLC
Mucopolysaccharidosis II
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II.
- Trial name
- NCT00069641
- Testing
- Iduronate-2-sulfatase enzyme replacement therapy
- Where
- 9 sites in United States, Brazil and 2 more
- Volunteers
- 96 people
- Sponsor
- Shire
Cystic Fibrosis
Drug-Drug Interaction Study of VX-770 and VX-809 in Healthy Subjects
The purpose of this study is to assess the pharmacokinetics and safety of VX-809 and VX-770 alone and in combination in healthy subjects.
- Trial name
- NCT01216046
- Testing
- VX-809, VX-770
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Study of Safety, Tolerability & Efficacy in Cystic Fibrosis Patients With Abnormal Glucose Tolerance
This is a Phase 2, multi-center, double blind, placebo controlled study to evaluate the safety and tolerability of PBI-4050, and its effects on the pancreatic, pulmonary functions and on various biomarkers in Cystic Fibrosis…
- Trial name
- NCT02955888
- Testing
- PBI4050
- Where
- 7 sites in Canada
- Volunteers
- 11 people
- Sponsor
- Liminal BioSciences Ltd.
Glaucoma, Ocular Hypertension
Pharmacokinetic and Safety Study of Travoprost 0.004% in Pediatric Glaucoma Patients
The purpose of this study was to assess the safety and describe the steady-state plasma pharmacokinetic (PK) profiles of Travoprost ophthalmic solution, 0.004% (new formulation) following a once daily administration for 7 days in…
- Trial name
- NCT01658839
- Testing
- Travoprost ophthalmic solution, 0.004% (new formulation)
- Volunteers
- 25 people
- Sponsor
- Alcon Research
Cystic Fibrosis
Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
The study population is comprised of adult subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation and are currently receiving background treatment with tezacaftor/ivacaftor for a minimum of 1 month prior…
- Trial name
- NCT03591094
- Testing
- PTI-428
- Where
- 26 sites in United States
- Volunteers
- 40 people
- Sponsor
- Proteostasis Therapeutics, Inc.
Primary Immune Deficiency
Retrospective Chart Review of Subcutaneous IgG Use in Infants
This study is a retrospective chart review of the use of subcutaneous IgG (SCIG) in infants less than two years old.
- Trial name
- NCT01166074
- Testing
- Subcutaneous IgG (SCIG)
- Where
- 5 sites in United States
- Volunteers
- 27 people
- Sponsor
- CSL Behring
Infant, Newborn
Comparative Gastrointestinal Tolerance of Various Infant Formulas in Healthy Term Infants
The objective of this study is to assess the comparative gastrointestinal (GI) tolerance of normal term infants to two experimental soy-based formulas, relative to a commercially available soy-based formula.
- Trial name
- NCT00798382
- Testing
- soy protein formula, Commercially available soy formula, Soy formula experimental #2
- Where
- 17 sites in United States
- Volunteers
- 154 people, healthy volunteers welcome
- Sponsor
- Abbott Nutrition
ADA-SCID, Adenosine Deaminase Deficiency
EZN-2279 in Patients With ADA-SCID
The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated with Adagen.
- Conditions
- ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
- Trial name
- NCT01420627
- Testing
- EZN-2279, Adagen
- Where
- 6 sites in United States
- Volunteers
- 7 people
- Sponsor
- Leadiant Biosciences, Inc.
Infantile Colic
Study to Evaluate the Efficacy of a Probiotic in Infantile Colic Symptoms Relief
This study evaluates the efficacy of single strain probiotic administered in a form of a sachet in the treatment of infant colic in infants 3-12 weeks old.
- Trial name
- PIXAR (NCT05432479)
- Testing
- Probiotic
- Where
- 2 sites in United States, India
- Volunteers
- 101 people
- Sponsor
- The Archer-Daniels-Midland Company
Hemophilia A
Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A
Most transient inhibitor formation, if any, will develop within the first 4 weeks. The study is to further monitor whether participants with severe Hemophilia A will develop inhibitors or antibodies at the later stage when…
- Trial name
- NCT00621673
- Testing
- Kogenate (BAY 14-2222)
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Bayer
PPHN, Persistent Pulmonary Hypertension of the Newborn
Study To Investigate Safety And Efficacy Of Sildenafil In The Newborns With Persistent Pulmonary Hypertension (PPHN)
Sildenafil is efficacious in newborns with persistent pulmonary hypertension and its use will reduce the need for inhaled nitric oxide.
- Conditions
- PPHN, Persistent Pulmonary Hypertension of the Newborn, Hypoxic Respiratory Failure
- Trial name
- NCT01069861
- Testing
- sildanefil
- Where
- United Kingdom
- Volunteers
- 4 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Phenylketonuria, Inborn Errors of Metabolism
Evaluation of PKU Start
To evaluate the acceptability, tolerance and effect on metabolic control of PKU Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.
- Trial name
- PKU Start (NCT03058848)
- Testing
- PKU Start
- Where
- 3 sites in United Kingdom
- Volunteers
- 10 people
- Sponsor
- Vitaflo International, Ltd
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
Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
This study will assess the safety and tolerability of pancrelipase delayed release capsules in subjects up to 6 years of age with Pancreatic Exocrine Insufficiency (PEI) due to Cystic Fibrosis.
- Trial name
- NCT00775528
- Testing
- Pancrelipase Delayed Release
- Where
- 12 sites in United States
- Volunteers
- 19 people
- Sponsor
- Solvay Pharmaceuticals
Neonatal Jaundice
Evaluation of a Smartphone-based Screening Tool (Picterus JP) for Neonatal Jaundice (Chicago)
A cross-sectional conductive study at the University of Chicago Medicine facilities (hospital and outreach clinics) to enable high qualitative estimation of bilirubin levels in the blood of newborns, independent of skin color…
- Trial name
- NCT05521607
- Testing
- Picterus Jaundice Pro (JP)
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Picterus AS
Intracranial Aneurysm
Diversion-p64 Post Market Clinical Follow-Up Study to Assess Safety and Effectiveness of the p64 Flow Modulation Device
The purpose of this study is to assess safety and effectiveness of the p64 Flow Modulation Device.
- Trial name
- NCT02600364
- Where
- 26 sites in Argentina, Belgium and 8 more
- Volunteers
- 450 people
- Sponsor
- Phenox GmbH
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Cystic Fibrosis
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03525444
- Testing
- VX-445/TEZ/IVA, IVA
- Where
- 115 sites in United States, Australia and 11 more
- Volunteers
- 405 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Safety and Tolerability Study OF PUR118 In Subjects With Cystic Fibrosis
The purpose of this study is to determine whether PUR118 is safe and tolerable in a population of subjects with Cystic Fibrosis.
- Trial name
- NCT01543191
- Testing
- PUR118
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Pulmatrix Inc.