Clinical trials: Newborn, Infant, Disease
Cystic Fibrosis
A Phase 3 Study of VX-659 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-659 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
- NCT03447249
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 101 sites in United States, Australia and 9 more
- Volunteers
- 385 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Invasive Group B Streptococcus Disease
Antibody Levels Associated With Reduced Risk of Invasive Group B Streptococcus Disease in Infants Aged Less Than 90 Days
Compare anticapsular antibody levels against Group B Streptococcus at delivery in mothers and their infants who develop disease versus those who do not.
- Trial name
- NCT02099149
- Where
- South Africa
- Volunteers
- healthy volunteers welcome
- Sponsor
- Novartis
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
Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects and Adults With Cystic Fibrosis.
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-032 in healthy adult subjects (Phase 1) and of two doses in Adults with Cystic Fibrosis (Phase 1b).
- Trial name
- NCT05712538
- Testing
- ARCT-032
- Where
- New Zealand
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Arcturus Therapeutics, Inc.
Pediatric Glaucoma, Elevated IOP in Pediatric Patients
Study of Travoprost Ophthalmic Solution, 0.004% Compared to Timolol (0.5% or 0.25%) in Pediatric Glaucoma Patients
The purpose of this study is to evaluate the intraocular pressure (IOP)-lowering efficacy of Travoprost 0.004% POLYQUAD (PQ) ophthalmic solution compared to Timolol ophthalmic solution (0.5% or 0.25%) in pediatric glaucoma…
- Conditions
- Pediatric Glaucoma, Elevated IOP in Pediatric Patients, Ocular Hypertension in Pediatric Patients
- Trial name
- NCT01652664
- Testing
- Travoprost 0.004% PQ ophthalmic solution, Timolol, 0.5% or 0.25% ophthalmic solution, Travoprost Vehicle
- Volunteers
- 184 people
- Sponsor
- Alcon Research
Cystic Fibrosis, Mucociliary Clearance
A Phase 1 Trial to Assess the Safety, Tolerability, and Pharmacokinetics of GS 9411 in Subjects With Cystic Fibrosis (CF)
The purpose of this study is to evaluate the safety and tolerability of GS-9411 in patients with Cystic Fibrosis. GS-9411 is a sodium channel inhibitor, that may restore airway hydration and mucociliary clearance in the lung.
- Trial name
- NCT01025713
- Testing
- GS-9411
- Where
- Australia
- Sponsor
- Gilead Sciences
Ichthyosis, Pruritus
Study Determining Safety and Efficacy of Avena Sativa (Oat) Skincare Products for Treating Skin Dryness and Itching in Cancer Patients
This study will test the safety and efficacy of three topical agents containing oat kernel flour to determine how well they relieve skin dryness and itching related to cancer therapies.
- Trial name
- NCT03796052
- Testing
- Avena Sativa Skincare Regimen
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Johnson & Johnson Consumer Inc. (J&JCI)
Pulmonary Hypertension, Familial Persistent, of the Newborn
A Study To Evaluate Safety And Efficacy Of IV Sildenafil In The Treatment Of Neonates With Persistent Pulmonary Hypertension Of The Newborn
This study will evaluate whether IV sildenafil can reduce the time on inhaled nitric oxide treatment and reduce the failure rate of available treatments for persistent pulmonary hypertension of the newborn.
- Trial name
- NCT01720524
- Testing
- iv sildenafil
- Where
- 42 sites in United States, Belgium and 10 more
- Volunteers
- 59 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
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
Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
The purpose of this study is to determine the efficacy and safety of chronic treatment with inhaled dry powder mannitol in subjects with cystic fibrosis.
- Trial name
- NCT00446680
- Testing
- Mannitol
- Where
- 29 sites in Australia, Ireland and 1 more
- Volunteers
- 340 people
- Sponsor
- Syntara
Cystic Fibrosis
Safety, Tolerability and Pharmacokinetics of BIIL 284 BS in Adult and Pediatric Cystic Fibrosis (CF) Patients
Safety, tolerability and pharmacokinetics following repeated doses (15-day dosing)
- Trial name
- NCT02269189
- Testing
- BIIL 284 BS, high dose, BIIL 284 BS, low dose
- Volunteers
- 36 people
- Sponsor
- Boehringer Ingelheim
HIV Infections
A Study of Delavirdine Mesylate in Combination With Other Anti-HIV Drugs in HIV-Infected Children and Babies
The purpose of this study is to see if it is safe and effective to give delavirdine mesylate (Rescriptor) plus two nucleoside reverse transcriptase inhibitors (NRTIs) to HIV-infected children and babies.
- Trial name
- NCT00002210
- Testing
- Delavirdine mesylate
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Pharmacia and Upjohn
Cystic Fibrosis
Study to Evaluate the Safety and Efficacy of Ciprofloxacin (Inhaled) in Patients With Cystic Fibrosis
To evaluate the change in forced expiratory volume (FEV1) from baseline to Day 28-30 between Cipro Inhale-treated and placebo-treated subjects after a 4-week treatment period.
- Trial name
- NCT00645788
- Testing
- Ciprofloxacin (Cipro Inhale, BAYQ3939)
- Where
- 86 sites in United States, Australia and 7 more
- Volunteers
- 288 people
- Sponsor
- Bayer
Metastatic Breast Cancer, Benign Breast Disease
China CellSearch Study
The study is designed to confirm the current indication (below) of the CellSearch® Circulating Tumor Cell Kit in metastatic breast cancer (MBC) patients for use of the kit in China.
- Trial name
- NCT01116869
- Where
- China
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Johnson & Johnson Medical, China
Cystic Fibrosis
A Phase 1 Study to Investigate the Food Effect of Lumacaftor in Combination With Ivacaftor
This study is designed to investigate the effect of food on the relative bioavailability of 2 different strengths of fixed-dose combinations of lumacaftor and ivacaftor tablet formulations.
- Trial name
- NCT01899105
- Testing
- lumacaftor, ivacaftor
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Farber Disease, Farber's Disease
Farber Disease Natural History Study
The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber…
- Conditions
- Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency
- Trial name
- NCT03233841
- Where
- 15 sites in United States, Argentina and 7 more
- Volunteers
- 45 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Neonatal Abstinence Syndrome
Study to Collect Data for Neonatal Abstinence Syndrome (NAS) and Evaluate the Automated Data Collection Process
Substance abuse during pregnancy is on the rise through both prescribed and illicit use of controlled substances, which has increased neonatal abstinence syndrome (NAS).
- Trial name
- NCT06303986
- Testing
- NeoMonki
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Rekovar Inc.
Cystic Fibrosis
Study of GLPG1837 in Subjects With Cystic Fibrosis (G551D Mutation)
32 cystic fibrosis patients with the G551D mutation will be treated for 4 weeks, consisting of three consecutive treatment periods: two 1-week periods followed by one 2-week period, evaluating one dose of GLPG1837 each.
- Trial name
- SAPHIRA1 (NCT02707562)
- Testing
- GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3
- Where
- 16 sites in Australia, Czechia and 3 more
- Volunteers
- 26 people
- Sponsor
- Lakefront Biotherapeutics NV
Cystic Fibrosis
GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis
This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d.
- Trial name
- PELICAN (NCT03474042)
- Testing
- GLPG2737
- Where
- 9 sites in Germany
- Volunteers
- 22 people
- Sponsor
- Lakefront Biotherapeutics NV
Cystic Fibrosis
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
- Trial name
- NCT04183790
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 21 sites in United States, Australia and 3 more
- Volunteers
- 64 people
- Sponsor
- Vertex Pharmaceuticals Incorporated