Clinical trials: Newborn Diseases
Huntington Disease, Tardive Dyskinesia
A Dose-escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetic of LPM3770164 in Healthy Subjects
This is a single-center, randomized, double-blinded, placebo-controlled, dose escalation trial to evaluate the safety, tolerability and pharmacokinetic of LPM3770164 sustained-release tablets orally administered in healthy…
- Trial name
- NCT05238701
- Testing
- LPM3770164 sustained release tablet, LPM3770164 sustained release tablet simulant
- Where
- China
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- Luye Pharma Group Ltd.
Hereditary Anemias
Utility of Fibroscan in Estimating Hepatic Iron Concentration
In patients with hereditary anemias (e.g. thalassemias), defective red blood cells are produced due to an error in the genes, or DNA, that provide the instructions for their synthesis.
- Trial name
- NCT02067130
- Testing
- Fibroscan
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- University of British Columbia
Sickle Cell Disease, Sickle Cell Crisis
Optimal Timing of Ketamine Initiation for SCD Pain
The goal of this clinical trial is to learn if the use of early ketamine decreases the chance of admission to the hospital in patients with sickle cell disease presenting with pain.
- Trial name
- NCT07450430
- Testing
- Ketamine hydrochloride injection
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Boston Children's Hospital
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Methadone, Morphine, or Oxycodone in Treating Pain in Patients With Cancer
RATIONALE: Methadone, morphine, or oxycodone may help relieve pain caused by cancer. It is not yet known whether methadone is more effective than morphine or oxycodone in treating pain in patients with cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00726830
- Testing
- methadone hydrochloride, morphine sulfate, oxycodone hydrochloride
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- M.D. Anderson Cancer Center
Port Wine Stain
A Randomized Trial to Study Combined Pulsed Dye Laser and Rapamycin Treatment of Port Wine Stain Birthmarks.
The researchers want to collect data on safety and efficacy of combined pulsed dye laser and rapamycin to improve fading/blanching of port wine stain birthmarks as compared to pulsed dye laser alone, which is the current standard…
- Trial name
- NCT00830466
- Testing
- Laser and rapamycin versus laser alone
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of California, Irvine
Developmental Dysplasia of the Hip, Acetabular Bone Loss
3D-Printed Pelvic Models for Planning Complex Total Hip Arthroplasty
Total hip arthroplasty (THA) in patients with complex acetabular anatomy can be challenging because of abnormal bone structure, deformity, or bone loss.
- Conditions
- Developmental Dysplasia of the Hip, Acetabular Bone Loss, Acetabular Fracture
- Trial name
- NCT07904637
- Testing
- Conventional Imaging-Based Preoperative Planning, CT-Based 3D-Printed Pelvic Model-Assisted Planning
- Where
- Egypt
- Volunteers
- 15 people
- Sponsor
- Ain Shams University
Retinopathy of Prematurity, Retinal Detachment
Efficient Study of ACULAR in Inhibiting Proliferative Retinopathy in Prematurity
The purpose of this study is to test whether ACULAR, a nonsteroidal anti-inflammatory eye drop medication, can prevent the development of retinopathy of prematurity (ROP) and/ or decrease its severity.In this study ACULAR will be…
- Conditions
- Retinopathy of Prematurity, Retinal Detachment, Blindness
- Trial name
- ROP (NCT00634972)
- Testing
- ACULAR, REFRESH TEARS
- Where
- United States
- Volunteers
- 83 people, healthy volunteers welcome
- Sponsor
- University of South Alabama
Inborn Errors of Metabolism, Pregnancy
Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol
Background: People with inborn errors of metabolism can t turn food into energy the right way. This can affect a person s growth and health. Researchers want to know how this condition affects a pregnant woman and her baby.
- Conditions
- Inborn Errors of Metabolism, Pregnancy, Acidemias
- Trial name
- NCT02322177
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Adenomatous Polyposis Coli
Trial In Pediatric Patients With Familial Adenomatous Polyposis (FAP)
To test whether celecoxib can be used to prevent colon polyp formation in children with familial adenomatous polyposis (FAP).
- Trial name
- CHIP (NCT00585312)
- Testing
- Celecoxib
- Where
- 42 sites in United States, Belgium and 12 more
- Volunteers
- 106 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Bicuspid Aortic Valve, Thoracic Aortic Aneurysm
The Effect of Losartan in Bicuspid Aortic Valve Patients
The specific aims of this study are to: Establish baseline levels of circulating MMP-2 and -9 , TIMP-1and- 2 and TGFB levels in individuals with bicuspid aortic valve and ascending aortic or aortic sinus measurements >40mm.
- Trial name
- NCT01390181
- Testing
- Cozaar
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Michigan
Congenital Heart Disease
Assessing Neurodevelopment in Congenital Heart Disease.
Congenital heart disease (CHD) is the most prevalent congenital malformation affecting 1 in 100 newborns per year. Children with CHD are a known risk population for brain injury, with neurodevelopmental alterations shown over…
- Trial name
- NEUROHEART (NCT02996630)
- Testing
- Sonography, Magnetic Resonance Imaging, Bailey Test
- Where
- Spain
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Ewing Sarcoma, Rhabdomyosarcoma
Vorinostat in Combination With Chemotherapy in Relapsed/Refractory Solid Tumors and CNS Malignancies
Investigators are testing new experimental drug combinations such as the combination of vorinostat, vincristine, irinotecan, and temozolomide in the hopes of finding a drug that may be effective against tumors that have come back…
- Conditions
- Ewing Sarcoma, Rhabdomyosarcoma, Wilms Tumor, Neuroblastoma
- Trial name
- NYMC195 (NCT04308330)
- Testing
- Vorinostat
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- New York Medical College
Wrinkles, Scars
Pilot Evaluation of the Cynosure Potenza™ System for Treatment of Cosmetic Dermatologic Skin Conditions
The primary objective of this pilot study is exploratory investigation evaluating the Potenza microneedle fractional radiofrequency (RF) device and may be used in combination with the Icon intense pulsed light (IPL) device.
- Conditions
- Wrinkles, Scars, Stretch Marks, Pigmentation
- Trial name
- NCT05847530
- Testing
- Potenza, Icon
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Cynosure, Inc.
Pulmonary Hypertension
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients With Pulmonary Arterial Hypertension (PAH)
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in sickle cell disease (SCD) patients diagnosed with pulmonary arterial hypertension.
- Trial name
- NCT00310830
- Testing
- Bosentan
- Volunteers
- 14 people
- Sponsor
- Actelion
Atopic Dermatitis, Eczema
Effects of an Emollient Containing Postbiotic Saccharomyces and Lactobacillus on Pediatric Atopic Dermatitis
The goal of this study is to investigate whether the use of emollients containing postbiotic Saccharomyces and Lactobacillus can significantly reduce the risk of atopic dermatitis (AD) recurrence in pediatric patients aged 0-6…
- Conditions
- Atopic Dermatitis, Eczema, Emollient
- Trial name
- NCT06474728
- Testing
- Treatment with an Emollient Containing Postbiotic Saccharomyces and Lactobacillus
- Where
- China
- Volunteers
- 98 people
- Sponsor
- Children's Hospital of Fudan University
Hemophilia A
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
Factor VIII (FVIII) is a large plasma glycoprotein that participates in blood coagulation. Loss of circulating FVIII activity due to mutations within the F8 gene results in the X-linked, recessive bleeding disorder hemophilia A.
- Trial name
- NCT05265767
- Testing
- Auto CD34+PBSC transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene
- Where
- India
- Volunteers
- 6 people
- Sponsor
- Christian Medical College, Vellore, India
Necrotizing Enterocolitis
Pentoxifylline in the Treatment of NEC in Premature Neonates
Pentoxifylline improves microcirculation and decreases TNF alpha levels associated with sepsis, rendering it of potential therapeutic value in necrotizing enterocolitis in premature neonates.
- Trial name
- NCT00271336
- Testing
- Pentoxifylline
- Where
- Israel
- Volunteers
- 50 people
- Sponsor
- Shaare Zedek Medical Center
Psoriasis Vulgaris, Atopic Dermatitis
The Role Of Neutrophil Proteases As Global Regulators Of Il-1 Family Cytokine Activity In Skin Disorders
Pro-inflammatory cytokines are critically important drivers of inflammatory and autoimmune diseases and cytokine-targeted biologics have been transformative in the treatment of several inflammatory and autoimmune diseases.
- Conditions
- Psoriasis Vulgaris, Atopic Dermatitis, Ichthyosis Vulgaris
- Trial name
- NEUTROPRO (NCT04750161)
- Testing
- No intervention
- Where
- Ireland
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- St. James's Hospital, Ireland
Neurofibromatosis Type 1
PASS of Paediatric Patients Initiating Selumetinib
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1.
- Trial name
- NCT05388370
- Where
- 46 sites in Austria, France and 8 more
- Volunteers
- 124 people
- Sponsor
- AstraZeneca
Persistent Ductus Arteriosus
Clinical Trial to Evaluate Two Guidelines for the Administration of Ibuprofen in the Treatment of Persistent Ductus Arteriosus Eco-guided: Impact in the Intestinal Prognosis
Persistent ductus arteriosus (DA) is a common entity in the premature newborn and is associated with high morbidity and mortality. There is still controversy about which is the best treatment for its closure.
- Trial name
- IBU24h-EchoG (NCT04282941)
- Testing
- Ibuprofen in continuous (24 hours) iv infusion and EchoG, IV bolus Ibuprofen slow (15 minutes) and EchoG
- Where
- 3 sites in Spain
- Volunteers
- 180 people
- Sponsor
- Instituto de Investigación Hospital Universitario La Paz
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