Clinical trials: Newborn Infant
Cystic Fibrosis
Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)
The goal of the study is to evaluate the safety and tolerability of P-1037 and to determine whether the combination of P-1037 with hypertonic saline or P-1037 alone has a greater effect on lung function in patients with CF than…
- Trial name
- CLEAN-CF (NCT02343445)
- Testing
- P-1037, Hypertonic Saline, Saline
- Where
- 33 sites in United States
- Volunteers
- 142 people
- Sponsor
- Parion Sciences
Cystic Fibrosis
Study of VX-770 on Midazolam and Rosiglitazone and the Effect of Fluconazole on VX-770
The objectives of this study are to evaluate the effects of VX-770 on Midazolam and Rosiglitazone, and the effect of Fluconazole on VX-770.
- Trial name
- NCT01060566
- Testing
- VX-770
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics
Cystic Fibrosis
A Phase IIb Study of OligoG in Subjects With Cystic Fibrosis
The purpose of the study is assessment of efficacy and safety of OligoG as a dry powder formulation, in adult subjects with cystic fibrosis.
- Trial name
- SMR-2984 (NCT02157922)
- Testing
- alginate oligosaccharide
- Where
- 18 sites in Denmark, Germany and 3 more
- Volunteers
- 65 people
- Sponsor
- AlgiPharma AS
Atopic Dermatitis
Comparison of Pimecrolimus Cream 1% Twice-Daily to Once-Daily Dosing in the Management of Atopic Dermatitis in Pediatric Subjects
This study is not being conducted in the United States. To investigate the relative efficacy of pimecrolimus cream 1% applied twice daily (b.i.d.) versus once daily (o.d.) in preventing the progression to disease "relapse".
- Trial name
- NCT00139581
- Testing
- Pimecrolimus
- Where
- Switzerland
- Sponsor
- Novartis
Cystic Fibrosis
Prevalence of Antibodies to Selected Porcine Viruses in Patients With Cystic Fibrosis Receiving Porcine-derived Pancreatic Enzyme Replacement Therapy
This is a point prevalence study conducted entirely in the United States (US) to establish the prevalence of antibodies to hepatitis E virus (HEV) and other selected porcine viruses in cystic fibrosis (CF) patients receiving…
- Trial name
- NCT01858519
- Where
- 47 sites in United States
- Volunteers
- 1,310 people
- Sponsor
- AbbVie
Insomnia
Registry to Collect Information on Pregnancy, Neonatal, and Infant Outcomes in Pregnant Women Exposed to QUVIVIQ®
This study will investigate pregnancy, neonatal, and infant outcomes in women exposed to QUVIVIQ during pregnancy compared to women unexposed to QUVIVIQ during pregnancy.
- Trial name
- NCT06498128
- Testing
- Daridorexant, Non-orexin receptor antagonist medications for insomnia, No insomnia medication
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Cystic Fibrosis
A Single Arm 48-Week Follow-on Safety Study to a Core Study Comparing the Efficacy and Tolerability of Tobrineb®/Actitob®/Bramitob® Versus TOBI®
This is a 48-week extension study to CMA-0631-PR-0010 Core Study. Patients who have a positive culture for P. aeruginosa at visit 4 of the first 8-week core study period and/or if deemed appropriate by the Investigators will be…
- Trial name
- CT03Ext (NCT01111383)
- Testing
- tobramycin
- Where
- 20 sites in France, Poland and 1 more
- Volunteers
- 209 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Jaundice, Neonatal
Validation of an Innovative Neonatal Jaundice Detection System (Picterus) in Indonesia
The long-term goal of this project is to establish Picterus as a permanent tool to detect NNJ in the healthcare services of Indonesia.
- Trial name
- NCT05630495
- Testing
- Picterus Jaundice Pro
- Where
- 2 sites in Indonesia
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Picterus AS
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
A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients
The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of…
- Trial name
- NCT02722057
- Volunteers
- 368 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Dermatitis, Atopic
Atopic Dermatitis in Atopy Predisposed Infants
The primary objective of this study is to investigate the effectiveness of a standardized skin care regimen using a milk lotion once daily and a bathing addendum as needed during the first 52 weeks of life on the development of…
- Trial name
- ADAPI (NCT02906475)
- Testing
- standardized skin care regimen
- Where
- Germany
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- HiPP GmbH & Co. Vertrieb KG
Rare Diseases
NICUSeq: A Trial to Evaluate the Clinical Utility of Human Whole Genome Sequencing (WGS) Compared to Standard of Care in Acute Care Neonates and Infants
Prospective, multi-site, study to evaluate the clinical utility of cWGS in a proband. One group will receive cWGS and a clinical report approximately 15 days after blood samples are received, while the other group will continue…
- Trial name
- NICU-Seq (NCT03290469)
- Testing
- clinical whole genome sequencing (cWGS)
- Where
- 5 sites in United States
- Volunteers
- 355 people
- Sponsor
- Illumina, Inc.
Cystic Fibrosis
Pharmacokinetics of GLPG3067 in Male Subjects With Cystic Fibrosis.
This clinical study is a Phase I, open-label, single-center study designed to evaluate the pharmacokinetics profile of a single oral dose of GLPG3067 in adult male subjects with cystic fibrosis in fed state.
- Trial name
- NCT03589313
- Testing
- GLPG3067 single dose
- Where
- Belgium
- Volunteers
- 6 people
- Sponsor
- Lakefront Biotherapeutics NV
Bronchopulmonary Dysplasia
Mesenchymal Stem Cells for Prevention of Bronchopulmonary Dysplasia in Infants
The clinical study with UMC119-01 is designed to investigate the safety in patients with bronchopulmonary dysplasia ("BPD"). This will be a dose escalation, open-label, single-center study in infants at high risk for BPD.
- Trial name
- NCT03631420
- Testing
- Human Umbilical Cord Derived-Mesenchymal Stem Cells
- Where
- Taiwan
- Volunteers
- 9 people
- Sponsor
- Meridigen Biotech Co., Ltd.
Cystic Fibrosis
ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation
The purpose of this program is to provide elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) to CF patients in critical need who are 6 to 11 years of age with at least one F508del mutation in response to unsolicited physician…
- Trial name
- NCT04702360
- Testing
- ELX/TEZ/IVA, IVA
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation
The purpose of this study was to evaluate the safety and efficacy of ivacaftor in participants with cystic fibrosis (CF) who were aged 12 years or older and were homozygous for the F508del-CF transmembrane conductance regulator…
- Trial name
- DISCOVER (NCT00953706)
- Testing
- Ivacaftor
- Where
- 34 sites in United States
- Volunteers
- 140 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Chronic Obstructive Pulmonary, Idiopathic Pulmonary Fibrosis
EZ-2053 in the Prophylaxis of Acute Pulmonary Allograft Rejection
The purpose of this study is to assess the efficacy and safety of the study drug, known as "ATG Fresenius S," which is sometimes called "EZ-2053," to prevent a lung transplant patient's body from rejecting a transplanted lung or…
- Conditions
- Chronic Obstructive Pulmonary, Idiopathic Pulmonary Fibrosis, Cystic Fibrosis, Bronchiectasis
- Trial name
- NCT00105183
- Testing
- EZ-2053, EZ-2053 5mg/kg
- Where
- 19 sites in United States, Australia and 2 more
- Volunteers
- 223 people
- Sponsor
- Neovii Biotech
Cystic Fibrosis
A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR Mutation
The study will evaluate the impact of smart adherence technology for monitoring on lumacaftor/ivacaftor (LUM/IVA) adherence rates among subjects 16 years of age and older with Cystic Fibrosis (CF) who are homozygous for the…
- Trial name
- NCT02823470
- Testing
- LUM/IVA, activated smart device, de-activated smart device
- Where
- 11 sites in United States, Canada
- Volunteers
- 24 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures