Clinical trials: Newborn Diseases
Craniosynostosis, Bleeding
Tranexamic Acid for Craniofacial Surgery
This is a randomized, blinded, prospective study that will investigate the potential benefit of tranexamic acid to reduce the intraoperative bleeding and blood transfusions in pediatric patients undergoing craniofacial surgeries.
- Trial name
- NCT00722436
- Testing
- Tranexamic acid, saline
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Pittsburgh
HIV Infections, Neonatal Infection
Immune Response to BCG Vaccination in Neonates Born to HIV and LTBI Infected and Non-infected Mothers
Maternal infections affect the basal immune status of neonates. One of the possible mechanism is the fetomaternal microchimerism, in which some cells and active substances are exchanged bi-directionally between maternal and fetal…
- Conditions
- HIV Infections, Neonatal Infection, LTBI - Latent Tuberculosis Infection, Immune Tolerance
- Trial name
- IMMUNEO (NCT03383211)
- Testing
- RNAseq
- Where
- 2 sites in Moldova
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- George Washington University
Fabry Disease
Data Collection in Women With Fabry Disease
This protocol will collect information needed to design a clinical study for the symptoms and problems of women with Fabry disease, an inherited metabolic disorder.
- Trial name
- NCT00030134
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cleidocranial Dysostosis
Cleidocranial Dysplasia (CCD): From Genotype to Phenotype and Considerations for Care
Cleidocranial Dysplasia (CCD) is a rare, autosomal dominant disorder characterized by dysplasia of bones and teeth. Given the rarity of this condition (prevalence of 1 in 1,000,000), the variable phenotype and lack of correlation…
- Trial name
- NCT05368064
- Testing
- observational
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Tetralogy of Fallot, Congenital Heart Disease
Tetralogy of Fallot for Life
The aim is to conduct a prospective multi-centre international inception cohort study with an enrollment goal of 3,000 TOF patients and 2 year follow-up post-repair.
- Conditions
- Tetralogy of Fallot, Congenital Heart Disease, Congenital Heart Defect
- Trial name
- TOF-LIFE (NCT02968264)
- Where
- 20 sites in United States, Australia and 10 more
- Volunteers
- 1,108 people
- Sponsor
- Population Health Research Institute
Diabetes Mellitus, Type 1, Monogenic Diabetes
Monogenic Diabetes Misdiagnosed as Type 1
The study has two aims: 1. To (1a) determine the frequency of monogenic diabetes misdiagnosed as type 1 diabetes (T1D) and (2) to define an algorithm for case selection. 2.
- Conditions
- Diabetes Mellitus, Type 1, Monogenic Diabetes, Neonatal Diabetes, Maturity-onset Diabetes in the Young (MODY)
- Trial name
- ADDAM (NCT03988764)
- Testing
- None AHT
- Where
- Canada
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Hemophilia A
Joint Health, Balance and Quality of Life in Adults With Hemophilia A
Hemophilia is a hereditary bleeding disorder characterized by recurrent bleeding episodes, particularly into joints and muscles, leading to chronic musculoskeletal complications.
- Trial name
- NCT07523399
- Where
- Turkey (Türkiye)
- Volunteers
- 36 people
- Sponsor
- Yuzuncu Yil University
Osteoarthritis, Post-traumatic Arthritis
Postmarketing Study to Determine Performance of the SIGMA HP® PARTIAL KNEE SYSTEM
This is a postmarketing study to assess the overall performance and survivorship of the SIGMA HP® PARTIAL KNEE SYSTEM.
- Conditions
- Osteoarthritis, Post-traumatic Arthritis, Gout, Pseudo-gout
- Trial name
- NCT01529099
- Testing
- SIGMA HP PARTIAL KNEE
- Where
- 3 sites in Italy, Switzerland
- Volunteers
- 64 people
- Sponsor
- DePuy International
Leukemia
Idarubicin and High-Dose Cytarabine in Treating Patients With Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as idarubicin and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00528398
- Testing
- cytarabine, idarubicin
- Where
- 2 sites in United States
- Volunteers
- 111 people
- Sponsor
- City of Hope Medical Center
Cystic Fibrosis With Gastrointestinal Manifestation
Cohort Study Evaluating the Clinical Effectiveness, Safety and Immunogenicity to the Pandemic Influenza Vaccination
Multicenter prospective cohort of approximately 1000 cystic fibrosis patients followed in the Ile de France during the 2009-2010 influenza season with the main objective to assess the effectiveness of antiviral vaccination…
- Trial name
- MUCOFLU (NCT01499914)
- Testing
- influenza vaccination
- Where
- France
- Volunteers
- 439 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cardiovascular Disease, Hyperhomocysteinemia
Prevalence Of Hyperhomocysteinemia In Thai Chronic Kidney Disease (CKD) Patients
It is well recognized that patients with chronic kidney disease (CKD) have an increased risk for cardiovascular disease (CVD) and hyperhomocysteinemia appears to be a predictor of future CVD events.
- Trial name
- NCT01391416
- Volunteers
- 98 people, healthy volunteers welcome
- Sponsor
- Bangkok Metropolitan Administration Medical College and Vajira Hospital
Adrenomyeloneuropathy, Adrenoleukodystrophy
Plasma Exchange With Albumin in AMN Patients
Adrenoleukodystrophy (X-ALD) is the most common genetic disorder of the brain white matter with an incidence of 1:14,700 births.
- Trial name
- NCT04303416
- Testing
- Albumin solution
- Where
- Spain
- Volunteers
- 5 people
- Sponsor
- Pujol, Aurora, M.D.
Hurler Syndrome
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an…
- Trial name
- NCT01173016
- Testing
- Laronidase
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Cystic Fibrosis, MRSA
Epidemiology and Treatment of Small-colony Variant Staphylococcus Aureus in Cystic Fibrosis
Methicillin-susceptible (MSSA) and Methicillin-resistant (MRSA) Staphylococcus aureus (SA) are two of the most important infectious pathogens in CF, with 69% of CF patients having lung infection with MSSA or MRSA in the last…
- Trial name
- NCT02547116
- Testing
- Rifampin
- Where
- United States
- Sponsor
- Johns Hopkins University
Bronchopulmonary Dysplasia (BPD)
Late Low-Dose Dexamethasone in Preterm Neonates Using DART Protocol
Premature babies, especially those who need breathing support for many days, are at risk of developing a long-term lung condition called bronchopulmonary dysplasia (BPD).
- Trial name
- NCT07904104
- Testing
- Dexamethasone
- Where
- Pakistan
- Volunteers
- 93 people
- Sponsor
- Shaheed Zulfiqar Ali Bhutto Medical University
Arteriovenous Malformations (Extracranial)
Cobimetinib In Extracranial Arteriovenous Malformations (COBI-AVM Study)
The purpose of this open-label study is to evaluate the safety and efficacy of cobimetinib in extracranial AVM.
- Trial name
- NCT05125471
- Testing
- Cobimetinib
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of Arkansas
Premature Birth
Assessment of Autonomic Regulation During the Listening of Mother Heart Beat in Newborns
Prematurity birth lead to an early breaking link with the in utero environment. A special attention is brought in the neonatal intensive care units to reduce noise and lighting surrounding aiming to protect the newborn.
- Trial name
- Cardio_Sound (NCT04821011)
- Testing
- electronic stethoscope, acoustic listening of mother's heartbeats, Recording of the newborn's heart rate
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Leukemia
Vaccine Therapy in Treating Patients With Acute Myeloid Leukemia
RATIONALE: Vaccines made from a person's white blood cells and cancer cells may make the body build an effective immune response to kill cancer cells.
- Trial name
- NCT00100971
- Testing
- autologous tumor cell vaccine, therapeutic autologous dendritic cells, tumor cell-derivative vaccine therapy
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Boston Medical Center
Wiskott-Aldrich Syndrome, Hematopoietic Stem Cell Transplantation
A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patient with…
- Conditions
- Wiskott-Aldrich Syndrome, Hematopoietic Stem Cell Transplantation, Graft Failure
- Trial name
- NCT03019809
- Testing
- G-CSF for Conditioning before HSCT., Plerixafor for Conditioning before HSCT.
- Where
- Russia
- Volunteers
- 30 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Hemophilia A or B
PF-06741086 Long-term Treatment in Severe Hemophilia
This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study.
- Trial name
- NCT03363321
- Testing
- PF-06741086
- Where
- 8 sites in United States, Brazil and 5 more
- Volunteers
- 20 people
- Sponsor
- Pfizer
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