Clinical trials: Newborn Infant
Cystic Fibrosis, Motor Neuron Disease
MN4000 for Treatment of CF and MND Patients in the Home Setting
The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.
- Conditions
- Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
- Trial name
- NCT03797677
- Testing
- MN 4000
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Hill-Rom
Cystic Fibrosis
Cystic Fibrosis (CF) Flow Rates Study
We wish to measure the inspiratory flow and volumes generated by the subjects inhaling from a spirometer with a high resistance dry powder inhaler in series in subjects with cystic fibrosis.
- Trial name
- NCT00779077
- Volunteers
- 25 people
- Sponsor
- Syntara
Cystic Fibrosis
Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis
The study is a randomized, double-blind, placebo-controlled, study that will be conducted at multiple centers in subjects with Cystic Fibrosis (CF) who are either homozygous for the F508del mutation or heterozygous with at least…
- Trial name
- NCT03500263
- Testing
- PTI-808, PTI-801, PTI-428
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Proteostasis Therapeutics, Inc.
Hemophilia A
Clinical Trial of NXT007 for the Prophylactic Treatment of Hemophilia A in Infants and Children
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis in pediatric patients aged 0 to 11 years with severe or moderate congenital hemophilia A without factor…
- Trial name
- ZEBRHA 3 (NCT07838194)
- Testing
- NXT007
- Volunteers
- 50 people
- Sponsor
- Hoffmann-La Roche
Cystic Fibrosis
A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation
The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF) who are heterozygous for F508del-CFTR allele and…
- Trial name
- NCT02392234
- Testing
- VX-661/Ivacaftor, Ivacaftor
- Where
- 93 sites in United States, Australia and 9 more
- Volunteers
- 248 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Infantile Colic, Infantile Functional Gastrointestinal Disorders
A Phase I Study to Evaluate the Oral Absorption of Nepadutant in Infants
The purpose of the study is to evaluate the gastrointestinal absorption of nepadutant after single dose as oral solution (and the effect of age on its oral absorption) in infants.
- Trial name
- NCT00655083
- Testing
- Nepadutant
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- Menarini Group
Cystic Fibrosis
Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis
This is a Phase 2 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of cystic fibrosis in patients 12 years of age or older.
- Trial name
- NCT03451045
- Testing
- Lenabasum 20 mg, Lenabasum 5 mg
- Where
- 105 sites in United States, Austria and 19 more
- Volunteers
- 447 people
- Sponsor
- Corbus Pharmaceuticals Inc.
Ankyloglossia
Case Series of Infants With Anterior Ankyloglossia Being Treated by the Sublingual Neonatal Incision Platform Device
A study assessing the feasibility of a novel device designed specifically to treat infant tongue-tie.
- Trial name
- SNIP (NCT07655895)
- Testing
- Frenectomy of anterior ankylogossia using the novel device
- Where
- United Kingdom
- Volunteers
- 6 people
- Sponsor
- Europa Life Sciences Limited
Cystic Fibrosis
Safety and Efficacy of 12-wk Treatment With Two Doses of Tiotropium Respimat in Cystic Fibrosis
This study evaluates the effects of 12-week treatment with two doses of tiotropium bromide (2.5 mcg q.d. and 5 mcg q.d.) compared to placebo administered via the Respimat device on lung function in patients with Cystic Fibrosis.
- Trial name
- NCT00737100
- Testing
- Tiotropium bromide 5 mcg, tiotropium bromide-low dose-2.5mcg
- Where
- 107 sites in United States, Australia and 9 more
- Volunteers
- 510 people
- Sponsor
- Boehringer Ingelheim
Cystic Fibrosis
Continuous Cardiorespiratory Monitoring in Cystic Fibrosis
Cystic Fibrosis (CF) is a disease without a primary cure that requires lifelong care and is characterized by pulmonary exacerbations (PEx).
- Trial name
- NCT04489186
- Testing
- Wearable device
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Byteflies
Cystic Fibrosis
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
The study evaluates the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF participants 2 to less than (<) 24 months of age.
- Trial name
- NCT05882357
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 19 sites in Australia, Canada and 5 more
- Volunteers
- 67 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele
This is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele or phenotypically…
- Trial name
- NCT04126473
- Testing
- ELX-02, Ivacaftor
- Where
- 9 sites in Australia, Germany and 1 more
- Volunteers
- 17 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Bronchopulmonary Dysplasia
Safety and Efficacy of MSC-EVs in the Prevention of BPD in Extremely Preterm Infants
The phase 1/2 trial aims to evaluate the safety and efficacy of EXOB-001 consisting of extracellular vesicles derived from umbilical cord mesenchymal stromal cells in the prevention of bronchopulmonary dysplasia (BPD) in…
- Trial name
- EVENEW (NCT06279741)
- Testing
- Endotracheopulmonary Instillation, Suspension
- Where
- 8 sites in Belgium, Italy
- Volunteers
- 265 people
- Sponsor
- EXO Biologics S.A.
Pancreatic Exocrine Insufficiency
Evaluation of Safety and Tolerability of Creon Micro in Children Younger Than Four Years With Pancreatic Exocrine Insufficiency
The purpose of this study is to evaluate the safety and tolerability of Creon micro in children younger than four years with pancreatic exocrine insufficiency.
- Trial name
- NCT01747330
- Testing
- Pancreatin
- Where
- 9 sites in Russia
- Volunteers
- 40 people
- Sponsor
- Abbott Products
Respiratory Distress Syndrome, Newborn, Pulmonary Ventilation; Newborn, Abnormal
Continuous Monitoring of Lung Ventilation
Background: Patients that suffer from respiratory failure and need mechanical ventilation are at risk of further deterioration due to complications induced by progression of lung disease or the mechanical ventilation.
- Conditions
- Respiratory Distress Syndrome, Newborn, Pulmonary Ventilation; Newborn, Abnormal, Apnea, Respiration Disorders
- Trial name
- NCT00810186
- Where
- 3 sites in Israel
- Volunteers
- 77 people
- Sponsor
- Pneumedicare Ltd.
Pregnancy, Hyperlipidemias
Bempedoic Acid Pregnancy Surveillance Program
Bempedoic acid pregnancy surveillance program
- Trial name
- NCT05103254
- Testing
- Bempedoic Acid, Bempedoic Acid / Ezetimibe
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Esperion Therapeutics, Inc.
Cystic Fibrosis Lung
4D-710 in Adult Patients With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
- Trial name
- CF (NCT05248230)
- Testing
- 4D-710
- Where
- 20 sites in United States
- Volunteers
- 30 people
- Sponsor
- 4D Molecular Therapeutics
Prader-Willi Syndrome
Study to Assess the Efficacy and Safety of Eutropin in Prader-Willi Syndrome
Evaluate the efficacy and safety after treatment of Eutropin® inj. compared to Genotropin® in infants/toddlers with Prader-Willi syndrome
- Trial name
- NCT02204163
- Testing
- Eutropin, Genotropin
- Where
- 3 sites in South Korea
- Volunteers
- 34 people
- Sponsor
- LG Life Sciences
Cystic Fibrosis
A Phase 1 Study to Examine the Drug-Drug Interaction of Ciprofloxacin, Itraconazole, and Rifampin on the Combination of Lumacaftor With Ivacaftor in Healthy Adult Subjects
The purpose of this study is to examine the drug-drug interaction effects of ciprofloxacin, itraconazole, and rifampin on the pharmacokinetics of lumacaftor in combination with ivacaftor as well as to evaluate the potential…
- Trial name
- NCT01768663
- Testing
- Lumacaftor, Ivacaftor, Ciprofloxacin
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hemolytic Disease of the Fetus and Newborn
A Study of Nipocalimab in Pregnancies at Risk for Severe Hemolytic Disease of the Fetus and Newborn (HDFN)
The purpose of this study is to assess the effectiveness of nipocalimab when compared to placebo in decreasing the risk of fetal anemia (a condition in which a baby's red blood cell volume falls below normal levels while the baby…
- Trial name
- AZALEA (NCT05912517)
- Testing
- Nipocalimab
- Where
- 69 sites in United States, Argentina and 16 more
- Volunteers
- 120 people
- Sponsor
- Janssen Research & Development, LLC