Clinical trials: Newborn Infant
Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
This was an extension study to Study LAL-CL01 (NCT01307098). The primary objective of the study was to evaluate the long-term safety and tolerability of sebelipase alfa in participants with liver dysfunction due to lysosomal acid…
- Conditions
- Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
- Trial name
- NCT01488097
- Testing
- sebelipase alfa
- Where
- 10 sites in United States, Canada and 3 more
- Volunteers
- 8 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Neonates
NICU-TECH RM9L-RS Probe ME Feasibility Study
Use of a new ultrasound device designed for babies to look at the catheters in veins and arteries as well as the blood vessel itself.
- Trial name
- NCT01181596
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- GE Healthcare
Cystic Fibrosis
A Phase 1 Study to Evaluate the Relative Bioavailability of a VX-828 Tablet Formulation
The purpose of this study is to evaluate the relative bioavailability of a VX-828 tablet formulation compared to a suspension and evaluate its safety and tolerability.
- Trial name
- NCT06861413
- Testing
- VX-828
- Where
- United States
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Study to Assess the Safety, Tolerability, and Pharmacokinetics of Brensocatib Tablets in Adults With Cystic Fibrosis
The main objective of the study is to evaluate the pharmacokinetics of brensocatib in participants with cystic fibrosis following once daily oral administration of study drug and to evaluate the safety of brensocatib compared to…
- Trial name
- NCT05090904
- Testing
- Brensocatib
- Where
- 14 sites in United States
- Volunteers
- 29 people
- Sponsor
- Insmed Incorporated
Cystic Fibrosis, CFTR Gene Mutation
Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor…
- Trial name
- LunairCF (NCT06747858)
- Testing
- ARCT-032
- Where
- 13 sites in United States
- Volunteers
- 33 people
- Sponsor
- Arcturus Therapeutics, Inc.
Premature Birth of Newborn, Intestinal Malabsorption
Study to Assess the Efficacy and Safety of NTRA-2112 on Intestinal Malabsorption in Preterm Infants
The study will evaluate the effect of NTRA-2112 on intestinal malabsorption in preterm infants.
- Trial name
- NCT02510560
- Testing
- NTRA-2112
- Where
- 50 sites in United States, Belgium and 8 more
- Volunteers
- 300 people
- Sponsor
- Elgan Pharma Ltd.
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
OPTION 2: A Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Cystic Fibrosis
The primary objectives of this study are to assess the safety and efficacy of MS1819 in enteric capsules vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to…
- Trial name
- NCT04375878
- Testing
- MS1819, Porcine PERT
- Where
- 15 sites in United States, Poland
- Volunteers
- 27 people
- Sponsor
- Entero Therapeutics
Non-Tuberculous Mycobacterial Pneumonia, Cystic Fibrosis
A Pilot Study to Assess the Effect of Intermittent iNO on the Treatment of NTM Lung Infection in CF and Non-CF Patients
The purpose of this open-label, multicenter, non-randomized, pilot study is to assess the safety of high dose intermittent iNO for treatment of NTM infection in CF and non-CF patients.
- Conditions
- Non-Tuberculous Mycobacterial Pneumonia, Cystic Fibrosis, Mycobacterial Pneumonia, Mycobacterium Abscessus Infection
- Trial name
- NCT04685720
- Testing
- LungFit
- Where
- Australia
- Volunteers
- 15 people
- Sponsor
- Beyond Air Inc.
Achondroplasia
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery
- Trial name
- NCT04554940
- Testing
- vosoritide
- Where
- 3 sites in Australia, United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Pulmonary Hypertension of Newborn
A Real-world Study of Nitric Oxide Generator and Delivery System
The study is aimed to observe the efficacy and safety of Nitric Oxide Generator and Delivery System in Pulmonary Hypertension of Newborn in real clinical settings.
- Trial name
- NCT05703191
- Testing
- Nitric Oxide Generator and Delivery System
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Novlead Inc.
Cerebrotendinous Xanthomatosis
The Prevalence of CTX Disorder in Juvenile Cataract Cases in Turkey
Epidemiologic observational study. Study will include two phases: 1. Retrospective cross-sectional phase: At this stage of the study, retrospective screenings are carried out at study sites and all juvenile cataract cases are…
- Trial name
- GEN-EYE-I (NCT03584893)
- Testing
- Blood sampling for Cholestanol Analysis
- Where
- 36 sites in Turkey (Türkiye)
- Volunteers
- 1,000 people
- Sponsor
- TRPHARM
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
Cystic Fibrosis, Pseudomonas Aeruginosa
Ph 1/2 Study Evaluating Safety and Tolerability of Inhaled AP-PA02 in Subjects With Chronic Pseudomonas Aeruginosa Lung Infections and Cystic Fibrosis
Phase 1b/2a, double-blind, randomized, placebo-controlled, single and multiple ascending dose study to evaluate the safety, tolerability and phage recovery profile of AP-PA02 multi-bacteriophage therapeutic candidate administered…
- Conditions
- Cystic Fibrosis, Pseudomonas Aeruginosa, Pseudomonas, Lung Infection
- Trial name
- SWARM-Pa (NCT04596319)
- Testing
- AP-PA02
- Where
- 20 sites in United States
- Volunteers
- 29 people
- Sponsor
- Armata Pharmaceuticals, Inc.
Cystic Fibrosis
A Cross-over Study of OligoG in Subjects With Cystic Fibrosis. Fibrosis
The purpose of this study is to determine the safety and local tolerability of multiple dose administration of inhaled OligoG in CF subjects.
- Trial name
- OligoG (NCT01465529)
- Testing
- OligoG CF-5/20, Saline
- Where
- 3 sites in Ireland, United Kingdom
- Volunteers
- 26 people
- Sponsor
- AlgiPharma AS
Cystic Fibrosis
A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation
This is a Phase 4, randomized, double-blind, placebo-controlled, parallel-group study in subjects aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation.
- Trial name
- NCT02875366
- Testing
- LUM/IVA
- Where
- 12 sites in Australia, United Kingdom
- Volunteers
- 70 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Respiratory Distress Syndrome, Bronchopulmonary Dysplasia
The Effect of Surfactant Dose on Outcomes in Preterm Infants With RDS
A prospective observational study using de-identified data from the Neonatal Network Research Database (NNRD) supplemented by additional information on dose, method of surfactant administration and dosing frequency to assess…
- Trial name
- OPTI-SURF (NCT03808402)
- Where
- 29 sites in United Kingdom
- Volunteers
- 2,973 people
- Sponsor
- Chiesi UK
Candidiasis
Open, Pharmacokinetic Study of Caspofungin in Neonates and Infants (0991-058)
The purpose of this study is to test the amount of caspofungin found in the blood of neonate and infants <3 months of age following treatment with caspofungin at 25 mg/m2.
- Trial name
- NCT00330395
- Testing
- caspofungin acetate
- Volunteers
- 16 people
- Sponsor
- Merck Sharp & Dohme LLC
Netherton Syndrome
A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome
Netherton Syndrome (NS) is a severe rare disease characterized by generalized scaling, erythema, and epidermal barrier defects. This study assessed the safety, pharmacokinetics (PK), and efficacy of DS-2325a in patients with NS.
- Trial name
- NCT05979831
- Testing
- DS-2325a
- Where
- France
- Volunteers
- 9 people
- Sponsor
- Daiichi Sankyo
Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.
- Trial name
- NCT00299000
- Testing
- Naglazyme
- Where
- 4 sites in United States, France and 1 more
- Volunteers
- 4 people
- Sponsor
- BioMarin Pharmaceutical
Growth, Colic
Safety and Tolerance Study of Alpha-Lactalbumin Enriched and Probiotic-Supplemented Infant Formula in Infants With Colic
The purpose of this study is to evaluate the nutritional adequacy, the digestive tolerance and the effect on colic of an alpha-lactalbumin-enriched and probiotic-supplemented infant formula.
- Trial name
- NCT00929292
- Testing
- Modilac Dahlia 1, Modilac 1
- Where
- 12 sites in France
- Volunteers
- 66 people
- Sponsor
- Sodilac