Clinical trials: Newborn Infant
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
Cystic Fibrosis
Lipid Formulation to Increase the Bioavailability of Fatty Acids in Cystic Fibrosis (CF) Patients
The purpose of this trial is to evaluate if the use of a newly developed lipid formulation versus fish oil would better enable the absorption of essential fatty acids after 12 weeks of supplementation.
- Trial name
- NCT02646995
- Testing
- modified lipid formulation, Fish oil
- Where
- Switzerland
- Volunteers
- 16 people
- Sponsor
- Société des Produits Nestlé (SPN)
Breastfeeding, Patient Simulation
Evaluation of Health Professional Learners' Competence in Newborn Positioning and Attachment Following a Standardized Patient-led Lactation Skills Workshop
Evaluating newborn positioning and attachment is a key skill for maternal-child healthcare providers. Trainees have limited opportunities to practice this skill in their clinical rotations due to a lack of access to lactating…
- Trial name
- NCT04717128
- Testing
- Breastfeeding Skills Workshop
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- LiquidGoldConcept
Bronchopulmonary Dysplasia
Safety and Efficacy of PNEUMOSTEM® in Premature Infants at High Risk for Bronchopulmonary Dysplasia (BPD) - a US Study
PNEUMOSTEM® consists of ex vivo cultured allogeneic, unrelated, human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs) and it is intended for use as a cellular therapy product for prevention of Bronchopulmonary…
- Trial name
- NCT02381366
- Testing
- Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Medipost, Inc.
Lung Diseases, Pulmonary Disease
A Study of the Safety and Tolerability of Inhaled SNSP113 in Healthy Subjects and Subjects With Stable Cystic Fibrosis
Although Cystic Fibrosis is a complex genetic disease affecting many organs, lung disease is the primary cause of mortality. The objective of this study is to determine the safety and tolerability of SNSP113 in healthy subjects…
- Conditions
- Lung Diseases, Pulmonary Disease, Cystic Fibrosis, Cystic Fibrosis Lung
- Trial name
- NCT03309358
- Testing
- Inhaled SNSP113
- Where
- 2 sites in United Kingdom
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Synspira, Inc.
Cystic Fibrosis
A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
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 homozygous for the F508del mutation (F/F).
- Trial name
- NCT03460990
- Testing
- VX-659/TEZ/IVA, TEZ/IVA, IVA
- Where
- 46 sites in United States, Australia and 4 more
- Volunteers
- 116 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis (CF)
Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 Combination Therapies in Adult Participants With Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40.
- Trial name
- NCT04853368
- Testing
- ABBV-576, Galicaftor, Navocaftor
- Where
- 41 sites in United States, Australia and 5 more
- Volunteers
- 48 people
- Sponsor
- AbbVie
Mucopolysaccharidosis II
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients
- Trial name
- NCT01301898
- Testing
- GC1111_0.5mg/kg, GC1111_1.0mg/kg, Elaprase_0.5mg/kg
- Where
- South Korea
- Volunteers
- 31 people
- Sponsor
- Green Cross Corporation
Retinopathy of Prematurity
Regulatory PMS Study for Lucentis® in Patients With Retinopathy of Prematurity
This study was an open-labeled, multicenter, single arm, observational post-marketing surveillance study under routine clinical practice with no mandated treatments, visits or assessments.
- Trial name
- NCT05304949
- Testing
- Lucentis
- Where
- 6 sites in South Korea
- Volunteers
- 69 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
A Study of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) in Healthy Adult Panelists
The purpose of this study is to evaluate the flavor (basic tastes, aroma, texture, mouthfeel) of VNZ/TEZ/D-IVA fixed dose combination (FDC) granules.
- Trial name
- NCT06299696
- Testing
- VNZ/TEZ/D-IVA
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation
The purpose of this study is to investigate the safety, tolerability and pharmacokinetics of N6022, and to obtain descriptive information on the effect of N6022 on biomarkers of CFTR function and inflammation in adult cystic…
- Trial name
- SNO-1 (NCT01746784)
- Testing
- N6022, Normal saline
- Where
- 17 sites in United States
- Volunteers
- 66 people
- Sponsor
- Nivalis Therapeutics, Inc.
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Liprotamase Efficacy Trial in Patients With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency
This is a clinical trial which will evaluate the efficacy of Liprotamase treatment in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (PI).
- Trial name
- DIGEST (NCT00449878)
- Testing
- Liprotamase
- Where
- 29 sites in United States
- Volunteers
- 163 people
- Sponsor
- Anthera Pharmaceuticals
Respiratory Syncytial Virus Infections
Prospective Study for the Use of Palivizumab (Synagis®) in High-risk Children in Germany
The SYNAGIS Registry was carried out in order to gather comprehensive real-world data on the use of palivizumab in children at high risk for serious respiratory syncytial virus (RSV) disease.
- Trial name
- NCT01155193
- Volunteers
- 30,804 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Cystic Fibrosis
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
- Trial name
- NCT06237335
- Testing
- RCT2100, Ivacaftor
- Where
- 23 sites in United States, France and 3 more
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- ReCode Therapeutics
Pulmonary Disease, Chronic Obstructive
Repeat Dose Study in Male Healthy Volunteer Smokers
This is a 4 to 6 week study to look at the safety and PK profile of SB656933 in healthy male smokers
- Trial name
- NCT00615576
- Testing
- SB-656933
- Where
- United Kingdom
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Cystic Fibrosis, Steatorrhea
An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption
The purpose of this study is to evaluate the effectiveness and safety of PANCREASE MT (pancrelipase microtablets) to improve steatorrhea (excessive excretion of fat in feces) in infants and toddlers with cystic fibrosis who have…
- Trial name
- NCT00217204
- Testing
- Pancrelipase microtablets
- Volunteers
- 18 people
- Sponsor
- McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
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)
Any Single Gene Disorder (Cystic Fibrosis, Tay-Sachs)
Concurrent Single Gene and 24 Chromosome Aneuploidy Preimplantation Genetic Diagnosis (PGD)
Gene Security Network has developed a novel technology called Parental SupportTM (PS) which is used for Preimplantation Genetic Screening/Diagnosis (PGS/D) during in vitro fertilization (IVF).
- Trial name
- IVF008 (NCT01023048)
- Testing
- preimplantation diagnosis
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Retinopathy of Prematurity
Study to Assess the Efficacy, Safety, and Tolerability of Intravitreal Aflibercept Compared to Laser Photocoagulation in Patients With Retinopathy of Prematurity
The primary objective of the study is to assess the efficacy of aflibercept compared to laser in patients diagnosed with retinopathy of prematurity (ROP).
- Trial name
- BUTTERFLEYE (NCT04101721)
- Testing
- aflibercept, laser photocoagulation
- Where
- 52 sites in United States, Bulgaria and 11 more
- Volunteers
- 127 people
- Sponsor
- Regeneron Pharmaceuticals
Respiratory Insufficiency Syndrome of Newborn
A Randomized, Multicenter Study of the Safety and Performance of Fabian PRICO for Saturation Targeting With Non-invasive Respiratory Support
This study is planned as a part of the post market clinical follow-up (PMCF) on a CE marked product. The purpose of this study is to assess, in a routine clinical environment at two centers the use of fabian-PRICO with…
- Trial name
- NCT05823909
- Testing
- fabian Therapy evolution ventilators with PRICO (Predictive Intelligent Control of Oxygenation), fabian Therapy evolution ventilators with manual FiO2 control
- Where
- 2 sites in Italy
- Volunteers
- 47 people
- Sponsor
- Vyaire Medical