Clinical trials: Neonatal Disease
Preterm Birth, Hypothermia, Newborn
Kangaroo Mother Care With Plastic Bag (Trials 1A & 1B)
The purpose of this study is to determine whether the combination of Kangaroo Mother Care (KMC) as continuously as possible together with the use of a plastic bag in combination with routine World Health Organization (WHO)…
- Trial name
- NCT03141736
- Testing
- KMC & WHO protocol (0-1 hour), KMC, WHO protocol & bag (0-1 hour), KMC & WHO protocol (1-24 hours)
- Sponsor
- University of Alabama at Birmingham
Kidney Cancer
Studying Genes Using Cord Blood and Placenta Samples From Relatively Healthy Newborns and Samples From Younger Patients With Wilms Tumor
RATIONALE: Studying samples of blood and tissue from newborns and from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.
- Trial name
- NCT01576211
- Testing
- DNA methylation analysis, RNA analysis, allele-specific oligonucleotide real-time quantitative polymerase chain reaction
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Children's Oncology Group
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
High-Dose Busulfan and High-Dose Cyclophosphamide Followed By Donor Bone Marrow Transplant in Treating Patients With Leukemia, Myelodysplastic Syndrome, Multiple Myeloma, or Recurrent Hodgkin or Non-Hodgkin Lymphoma
RATIONALE: Giving high doses of chemotherapy drugs, such as busulfan and cyclophosphamide, before a donor bone marrow transplant helps stop the growth of cancer cells.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT01177371
- Testing
- busulfan, cyclophosphamide, allogeneic bone marrow transplantation
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Case Comprehensive Cancer Center
Fetal Growth Retardation
Antenatal Allopurinol in Intrauterine Growth Restriction
Growth retardation in utero may be caused by uteroplacental vascular insufficiency. When Doppler ultrasound studies of the umbilical artery are abnormal pathological intrauterine growth restriction (IUGR) can be diagnosed.
- Trial name
- NCT00346463
- Testing
- Allopurinol
- Where
- Netherlands
- Volunteers
- 50 people
- Sponsor
- UMC Utrecht
Fanconi's Anemia
Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia
OBJECTIVES: I. Determine the probability of engraftment with total body irradiation, cyclophosphamide, fludarabine, and anti-thymocyte globulin followed by HLA nongenotypically identical donor, T-cell depleted hematopoietic cell…
- Trial name
- NCT00005898
- Testing
- anti-thymocyte globulin, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Minnesota
Cleft Palate
Validation of a Production Method of Stem Cell Isolated From the Nasal Cavity for an Innovative Cell Therapy of Cleft Palate
Cell therapy appears to be an alternative to treat bone defects. Scientific advances have shown in animal models and in humans that mesenchymal stem cells were good candidates to support cellular bone regeneration after…
- Trial name
- NCT02900014
- Testing
- sampling mesenchymal stem cells
- Where
- France
- Volunteers
- 2 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Hemolytic Disease of the Newborn, Fetal Anemia
Neonatal Outcomes on Direct Fetal Intravenous Immunoglobulin During Intrauterine Transfusion for Rhesus Isoimmunization
The goal of this clinical trial is to learn if giving intravenous immunoglobulin directly to the fetus during intrauterine transfusion can improve newborn outcomes in pregnancies affected by rhesus isoimmunization.
- Conditions
- Hemolytic Disease of the Newborn, Fetal Anemia, Rhesus Isoimmunization, Erythroblastosis Fetalis Due to RH Antibodies
- Trial name
- NCT07885592
- Testing
- Intrauterine Transfusion, Intravenous immunoglobulin (IVIG)
- Where
- India
- Volunteers
- 30 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
Developmental and Epileptic Encephalopathies, Epileptic Spasms
Ketogenic Diet for Prevention of Epileptic Spasms in Infantile Onset Genetic Epilepsies
Epileptic spasms (ES) are a predominantly infantile seizure type observed frequently in certain genetic disorders. Ketogenic diet (high ratio of fat to carbohydrate/protein) is an established non-medication treatment for…
- Conditions
- Developmental and Epileptic Encephalopathies, Epileptic Spasms, Genetic Epilepsy, Neonatal and Infant Epilepsy
- Trial name
- NCT06700811
- Testing
- Ketogenic diet
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Heather Olson
Heart Defects, Congenital
Effects of Perioperative Nesiritide or Milrinone Infusion on Recovery From Fontan Surgery
The staged surgical pathway to treat children with single ventricle heart defects culminates with the Fontan operation. In this procedure, systemic venous return is rerouted directly to the pulmonary arteries, which serves to…
- Trial name
- NCT00543309
- Testing
- nesiritide, milrinone
- Where
- United States
- Volunteers
- 106 people
- Sponsor
- John M Costello
Fractures, Bone, Deformity; Bone
Virtual Reality Distraction During Outpatient Pediatric Orthopedic Procedures
Virtual Reality(VR) has had promising applications in science and medicine, including intervention delivery. The use of VR interventions has been studied in a wide range of medical conditions, including anxiety, phobias, obesity…
- Trial name
- NCT05510232
- Testing
- A treatment group that will watch a non-VR video via an iPad, A treatment group that will have headset on and will watch a non-VR video, A treatment group that will be immersed in the VR game (Bear Blast) via the headset.
- Where
- United States
- Volunteers
- 226 people
- Sponsor
- Hospital for Special Surgery, New York
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
A Study of Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome
The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of seizures in children and adults with Dravet Syndrome (DS) or Lennox-Gastaut Syndrome (LGS).
- Trial name
- NCT05163314
- Testing
- Soticlestat
- Where
- 116 sites in United States, Australia and 18 more
- Volunteers
- 352 people
- Sponsor
- Takeda
Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa Dystrophica, Recessive
A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)
This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB).
- Conditions
- Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa Dystrophica, Recessive, Epidermolysis Bullosa Dystrophica Dominans
- Trial name
- NCT06563414
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Krystal Biotech, Inc.
Williams Syndrome
Fat Distribution and Glucose Metabolism in Williams Syndrome
Williams Syndrome (WS) is a genetic syndrome with features that may include vascular stenoses, neuro-developmental changes, and a variety of endocrine and metabolic abnormalities, including impaired glucose metabolism and…
- Trial name
- NCT01864304
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Respiratory Distress Syndrome, Newborn, Premature Birth
Prophylactic Minimally Invasive Surfactant Evaluation
The purpose of this study is to explore the question: Does prophylactic administration of exogenous surfactant in the delivery room, using a minimally-invasive technique, improve outcome in preterm infants 22-29 weeks' gestation…
- Trial name
- PreProMISe (NCT06007547)
- Testing
- Poractant Alfa
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Endeavor Health
Respiratory Distress Syndrome, Newborn, Chronic Lung Disease of Newborn (Diagnosis)
Diaphragmatic Electrical Activity in Preterm Infants on Non-Invasive Ventilation
Preterm babies have immature lungs and frequent pauses in their breathing which often necessitates breathing support. Nasal Continuous Positive Airway Pressure (CPAP) is one of the most commonly used tools but does not always…
- Trial name
- NCT03422549
- Testing
- Drager VN500 Ventilator
- Where
- Canada
- Volunteers
- 20 people
- Sponsor
- University of British Columbia
Duchenne Muscular Dystrophy
Evaluation of Home Based Assessments on Participants With DMD
This study is designed to evaluate the feasibility, wearability and participant satisfaction of novel outcome assessment tools in DMD patients which are performed in the home environment.
- Trial name
- NCT05657938
- Testing
- Wearable Device, Duchenne Video Assessment (DVA)
- Where
- United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Solid Biosciences Inc.
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma
Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors
This phase II trial studies how well cabozantinib-s-malate works in treating younger patients with sarcomas, Wilms tumor, or other rare tumors that have come back, do not respond to therapy, or are newly diagnosed.
- Conditions
- Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma, Central Nervous System Neoplasm, Childhood Clear Cell Sarcoma of Soft Tissue
- Trial name
- NCT02867592
- Testing
- Cabozantinib, Cabozantinib S-malate, Pharmacological Study
- Where
- 142 sites in United States
- Volunteers
- 109 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease
A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke
The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke.
- Trial name
- HIBISCUS 3 (NCT05953584)
- Testing
- Etavopivat, Hydroxyurea
- Where
- 9 sites in India, Nigeria and 1 more
- Volunteers
- 27 people
- Sponsor
- Forma Therapeutics, Inc.
MPS IVA, MPS VI
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI.
- Trial name
- NCT05845749
- Testing
- Vosoritide Injection [Voxzogo]
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, San Francisco
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