Clinical trials: Newborn Infant
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012) in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy.
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases
- Trial name
- RADICHOL 1 (NCT00607373)
- Testing
- mipomersen
- Where
- 10 sites in United States, Brazil and 5 more
- Volunteers
- 51 people
- Sponsor
- Kastle Therapeutics, LLC
Retinopathy of Prematurity (ROP)
IGF-1/IGFBP3 Prevention of Retinopathy of Prematurity
To compare the severity of retinopathy of prematurity (ROP) among treated infants with an untreated control population, matched for gestational age at birth while confirming the dose of rhIGF-1/rhIGFBP-3 is safe and efficacious.
- Trial name
- NCT01096784
- Testing
- rhIGF-I/rhIGFBP-3
- Where
- 24 sites in United States, Italy and 4 more
- Volunteers
- 121 people
- Sponsor
- Shire
Cystic Fibrosis
A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
The primary purpose of study is to evaluate the treatment effect of tezacaftor in combination with ivacaftor (TEZ/IVA) on chest imaging endpoints using low-dose computed tomography (LDCT) at Week 72, and to evaluate the safety of…
- Trial name
- NCT02730208
- Testing
- Tezacaftor/Ivacaftor, Ivacaftor
- Where
- 9 sites in Australia
- Volunteers
- 41 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis
This is a first-in-human and proof-of-concept study of VX-445. The study includes 6 parts. Parts A, B, and C were conducted in healthy subjects.
- Trial name
- NCT03227471
- Testing
- IVA, TEZ/IVA, VX-445
- Where
- 38 sites in United States, Australia and 2 more
- Volunteers
- 225 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis, Pancreatic Insufficiency
Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)
Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic…
- Trial name
- NCT00880100
- Testing
- Ultrase® MT12
- Where
- 15 sites in United States
- Volunteers
- 49 people
- Sponsor
- Forest Laboratories
Lysosomal Acid Lipase Deficiency
Lysosomal Acid Lipase Deficiency in Risk Groups
A multicenter real-world observational study of the prevalence, diagnostic pathways, and clinical characteristics of lysosomal acid lipase deficiency in pediatric and adolescent risk groups in the Russian Federation (HELIOS)
- Trial name
- HELIOS (NCT07455864)
- Where
- 2 sites in Russia
- Volunteers
- 25 people
- Sponsor
- AstraZeneca
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.
Infantile Colic
The COLIC Study: Colonization of the Infant Gut With B. Infantis EVC001 to Reduce Symptoms of Colic
To investigate whether B. infantis EVC001 colonization in the infant gut can reduce symptoms of colic
- Trial name
- COLIC (NCT05129384)
- Where
- United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Evolve BioSystems, Inc.
Cystic Fibrosis, Mucociliary Clearance
A Trial to Assess the Safety, Tolerability, and Pharmacokinetics of GS-9411 in Healthy Male Volunteers
The purpose of this study is to evaluate the safety and tolerability of escalating doses of GS-9411 in healthy male volunteers. GS-9411 is a sodium channel inhibitor, that may restore airway hydration and mucociliary clearance in…
- Conditions
- Cystic Fibrosis, Mucociliary Clearance, Airway Hydration
- Trial name
- NCT00951522
- Testing
- GS-9411
- Where
- Australia
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Gilead Sciences
Cystic Fibrosis, Advanced Lung Disease
Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation
- Trial name
- NCT02390219
- Testing
- Lumacaftor, Ivacaftor
- Where
- 6 sites in United States
- Volunteers
- 46 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pseudomonas Aeruginosa, Cystic Fibrosis
Open Label Extension to Bridging Study CTBM100C2303
This was an open-label, single arm (uncontrolled) study in participants suffering from cystic fibrosis, who had completed their study participation in CTBM100C2303 (all visits) and who were proven infected with Pseudomonas…
- Trial name
- NCT00982930
- Testing
- Tobramycin inhalation powder
- Where
- 2 sites in Estonia, Russia
- Volunteers
- 55 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of single and multiple ascending doses of VX-272 in combination with deutivacaftor (D-IVA) with or without tezacaftor (TEZ) in healthy…
- Trial name
- NCT07437105
- Testing
- VX-272, Tezacaftor, Deutivacaftor
- Where
- United States
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Single Dose Escalation Study of TR02 (Sustained Lipid Inhalation Technology [SLIT™] Amikacin) in Participants With Cystic Fibrosis (CF) Having Chronic Infections of Pseudomonas Aeruginosa
The primary purpose of this study is to evaluate the safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.
- Trial name
- NCT06238856
- Testing
- SLIT™ Amikacin
- Volunteers
- 18 people
- Sponsor
- Insmed Incorporated
Lysosomal Acid Lipase Deficiency
Screening for Lysosomal Acid Lipase Deficiency
The primary outcome of this study is the development of a clinical profile of pediatric patients with LAL-D, which will enable the Sponsor to provide more focused guidance to the medical community as to which pediatric patients…
- Trial name
- NCT02926872
- Testing
- There is no intervention in the study
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Atopic Dermatitis, Skin Condition
Clinical Study of Partially Hydrolysed Protein Infant Formula on Trans-epidermal Water Loss (TEWL)
This is a single-centre, prospective, randomized, open-label, controlled trial of 200 infants 42±7 days of age. Subjects will be randomized to one of two open label feeding intervention group: Intact Cow's Milk Protein Formula…
- Trial name
- NCT04763512
- Testing
- Intact Cow's Milk Protein Formula, Partially Hydrolysed Whey Formula
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
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
Surgery
Calibration and Validation of Masimo's O3 Regional Oximetry Device in Neonates, Infants and Children
The study will collect cerebral oximetry data from pediatric and neonatal subjects using the Masimo O3 regional oximetry device, for the purposes of device calibration and validation.
- Trial name
- NCT03123354
- Testing
- O3 regional oximeter sensor
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Masimo Corporation
Cystic Fibrosis, Bronchiectasis
A Study to Test How Well BI 1291583 is Tolerated by People With Cystic Fibrosis Bronchiectasis (Clairafly™)
This study is open to adults aged 18 years and older with cystic fibrosis bronchiectasis. The purpose of this study is to find out whether a medicine called BI 1291583 is tolerated by people with cystic fibrosis bronchiectasis.
- Trial name
- NCT05865886
- Testing
- BI 1291583
- Where
- 24 sites in United States, Belgium and 5 more
- Volunteers
- 22 people
- Sponsor
- Boehringer Ingelheim
Magnetic Resonance Imaging
Gadobutrol Pharmacokinetic and Safety Study in Pediatric Subjects Aged <2 Years (Term Newborn Infants to Toddlers 23 Months of Age Inclusive)
The main purpose of this study is to collect data on the way gadobutrol is taken into, moves around, and is eliminated from, the body of children aged 0 to less than 2 years.
- Trial name
- NCT01544166
- Testing
- Gadobutrol (Gadavist, BAY86-4875)
- Where
- 9 sites in United States, Canada and 1 more
- Volunteers
- 44 people
- Sponsor
- Bayer
Jaundice, Neonatal
A mHealth Application as a Screening Tool for Neonatal Jaundice in Filipino Neonates
The purpose of the study is to test the reliability of the Picterus smartphone application in Filipino neonates. A descriptive cross-sectional study will be the method to understand the correlation of the modalities and to…
- Trial name
- NCT05616845
- Testing
- Picterus Jaundice Pro
- Where
- Philippines
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Picterus AS