Clinical trials: Neonatal Care
Metastatic Urothelial Carcinoma, Unresectable Urothelial Carcinoma
Pemigatinib + Pembrolizumab vs Pemigatinib Alone vs Standard of Care for Urothelial Carcinoma (FIGHT-205)
The purpose of this study is to evaluate the safety and efficacy of pemigatinib plus pembrolizumab or pemigatinib alone versus the standard of care for participants with metastatic or unresectable urothelial carcinoma who are not…
- Trial name
- NCT04003610
- Testing
- Pemigatinib, Pembrolizumab, Gemcitabine
- Where
- 79 sites in United States, Austria and 14 more
- Volunteers
- 7 people
- Sponsor
- Incyte Corporation
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care…
- Trial name
- NCT06192576
- Testing
- Olipudase alfa
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Sanofi
Growth, Neonates
Improving Artificial Intelligence-derived Algorithms for Estimating Length and Weight in NEonateS and infanTs up to 6 Months of Age (NEST)
The NEST study is a prospective, observational research study designed to collect clinical measurements and image data to develop and evaluate artificial intelligence (AI)-derived algorithms for estimating anthropometric…
- Conditions
- Growth, Neonates, AI (Artificial Intelligence)
- Trial name
- NEST (NCT07514182)
- Where
- Singapore
- Volunteers
- 60 people
- Sponsor
- Danone Asia Pacific Holdings Pte, Ltd.
Hemophilia B, Hemophilia A
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
- Trial name
- NCT03587116
- Testing
- Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 212 people
- Sponsor
- Pfizer
Neonatal Intensive Care
Estimation of Cardiac Output in Neonatal ICU Using an Ultrasound Dilution Technique
Presently there is no apparatus, clinically available, for routine determination of cardiac output (CO) in children. The goal of this research is to develop new technology that will help determine CO in neonates.
- Trial name
- CH (NCT01610622)
- Testing
- COstatus cardiac output measurement with isotonic saline
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Transonic Systems Inc.
Retinopathy of Prematurity (ROP)
Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)
The main purpose of this study is to evaluate the long-term efficacy and safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (NCT01096784).
- Trial name
- PEDAL (NCT02386839)
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 16 sites in United States, Italy and 4 more
- Volunteers
- 76 people
- Sponsor
- Shire
Dermatitis, Atopic
Curing Atopic Dermatits in Children With a Commerical Medical Device and Maintaining Healthy Skin by Using a New Cosmetic Product
1. Curing mild atopic dermatis in children with a commerical medical device (Bepanthen Itch Relief Cream). 2. Maintaining healthy skin by using a new cosmetic Bepanthen product or a commercially available cosmetic product
- Trial name
- NCT02615561
- Testing
- Bepanthen Itch Relief Cream: Phoenix 2 (Bepanthen Sensiderm, BAY207543), New cosmetic Bepanthen product: Pumpkin (BAY207543), Stelatopia (cosmetic comparator)
- Where
- 14 sites in Germany
- Volunteers
- 136 people
- Sponsor
- Bayer
Urea Cycle Disorder
UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder
UNLOCKED: A Phase 2 Trial to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
- Trial name
- UNLOCKED (NCT03933410)
- Testing
- KB195
- Where
- 19 sites in United States, Belgium and 5 more
- Volunteers
- 13 people
- Sponsor
- Kaleido Biosciences
Hemophilia A
Study Evaluating Refacto For Pharmacovigilance
The purpose of this study is to investigate the effectiveness and safety of treatment with ReFacto under conditions of routine therapy. Furthermore a continuous benefit/risk assessment will be done.
- Trial name
- NCT00195442
- Testing
- Moroctocog alfa
- Where
- 26 sites in Austria, Germany
- Volunteers
- 288 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Cystic Fibrosis
A 4-week Study to Test Different Doses of BI 1265162 in Adolescents and Adults With Cystic Fibrosis Using the Respimat® Inhaler - BALANCE - CF™1
The primary objective of this trial is to assess the efficacy, safety and pharmacokinetics of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler versus placebo in adolescents and adult patients with cystic…
- Trial name
- NCT04059094
- Testing
- BI 1265162
- Where
- 26 sites in United States, Belgium and 6 more
- Volunteers
- 52 people
- Sponsor
- Boehringer Ingelheim
Cystic Fibrosis
A Phase 2b Randomised, Placebo Controlled Study of OligoG in Patients with Cystic Fibrosis
A double-blind, randomised study of OligoG DPI compared to placebo DPI, both on top of standard-of-care, to assess safety, efficacy and tolerability. Adult patients with Cystic Fibrosis will be included in the study.
- Trial name
- NCT03822455
- Testing
- OligoG DPI
- Where
- Australia
- Volunteers
- 15 people
- Sponsor
- AlgiPharma AS
Hypoxemia
Oxygen Saturation Alarms in the Neonatal Intensive Care Unit
Infants will be observed in the neonatal intensive care unit for 4 hours. The observer will note the timing of oxygen saturation alarms, staff response, and interrupted staff activities.
- Trial name
- NCT01085539
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- Medtronic - MITG
Homozygous Familial Hypercholesterolemia
Effects of Lomitapide on Carotid and Aortic Atherosclerosis
Study to assess changes in carotid and aortic atherosclerosis in patients being treated with lomitapide.
- Trial name
- NCT02399852
- Testing
- Lomitapide
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Autosomal Dominant Hypocalcemia (ADH)
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1
The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
- Trial name
- CALIBRATE (NCT05680818)
- Testing
- Encaleret, Standard of Care
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 67 people
- Sponsor
- Calcilytix Therapeutics, Inc., a BridgeBio company
Cryopyrin-Associated Periodic Syndromes
Kineret CAPS Post Authorisation Study
A non-interventional, post authorization safety study to evaluate the safety of Kineret in the treatment of Cryopyrin Associated Periodic Syndromes (CAPS) in routine clinical care with regard to serious infections, malignancies…
- Trial name
- NCT02326376
- Testing
- anakinra (Kineret)
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Swedish Orphan Biovitrum
Pregnancy, Newborn Health
Study to Identify Factors Influencing Access of Pregnant Women and Their Infants to Local Healthcare Systems
This study collected information regarding the factors that might influence access of pregnant women to the local healthcare system. The main visits of the study screened \& enrollment, delivery and 90 day infant follow-up.
- Trial name
- NCT01734434
- Where
- 10 sites in Dominican Republic, Mozambique and 2 more
- Volunteers
- 3,243 people
- Sponsor
- Novartis Vaccines
Atopic Dermatitis (Eczema)
The Effects of a Botanical Moisturizer and an Unscented Soap on Eczema and Itch
The purpose of this study is to examine the effects of a botanical moisturizer and an unscented soap on eczema severity, itch, mood, and various skin measures (such as skin hydration) in those with mild to moderate eczema.
- Trial name
- NCT06804070
- Testing
- Botanical Moisturizer + Unscented Soap
- Where
- Poland
- Volunteers
- 34 people
- Sponsor
- Codex Labs Corporation
Rod Cone Dystrophy
Natural History Study of Advanced RCD
ECSTASI is a Natural History Study, which will be monitoring how standard of care (SoC) affect advanced rod-cone dystrophy (RCD), a type of eye disease also known as retinitis pigmentosa.
- Trial name
- ECSTASI (NCT07854899)
- Testing
- Eye examinations, Questionnaire
- Where
- 3 sites in Belgium, France and 1 more
- Volunteers
- 100 people
- Sponsor
- SparingVision
Intussusception, LRTI-related Post Neonatal Deaths
Implementing Hospital-based Active Surveillance Procedures for Vaccine Safety Monitoring at IMSS Hospitals in Mexico
This is an epidemiology study designed to implement hospital-based active surveillance for intussusception (IS) in children <1 year of age and lower respiratory tract infection (LRTI)-related deaths among children between 29 days…
- Trial name
- NCT00436358
- Testing
- Not applicable: none
- Where
- Mexico
- Volunteers
- 134 people
- Sponsor
- GlaxoSmithKline
Von Willebrand Disease (VWD)
A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers
The main aim of this study is to describe the experience and unmet needs of persons living with VWD and their caregivers in Canada.
- Trial name
- NCT05695560
- Testing
- No Intervention
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- Takeda