Clinical trials: Newborn Diseases
Hemophilia
LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures
The purpose of this study, PerSept 3, is to evaluate LR769 for the prevention of excessive bleeding and achievement of hemostasis in congenital hemophilia A or B patients who have inhibitors to Factor VIII or Factor IX , are aged…
- Trial name
- PerSept3 (NCT02548143)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 9 sites in United States, Mexico and 4 more
- Volunteers
- 12 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Flatfoot
Dynamic Plantar Pressures Changes After Dry Needling in Flexor Digitorum Brevis Versus Non Emission Laser
Thirty-six subjects randomly distributed between a dry needling intervention group and a control group whose intervention is a non-emission laser.
- Trial name
- NCT05875415
- Testing
- Bilateral dry needling in latent trigger points of the Flexor digitorum Brevis Muscle, Bilateral non emission Laser in latent trigger points of the Flexor digitorum Brevis Muscle
- Where
- Spain
- Volunteers
- 36 people
- Sponsor
- Mayuben Private Clinic
Syringomyelia, Arnold Chiari Deformity
A Prospective Natural History Study of Patients With Syringomyelia
Background: Syringomyelia is a disorder in which a cyst (syrinx) forms within the spinal cord and causes spinal cord injury, with symptoms worsening over many years, including paralysis, loss of sensation, and chronic pain.
- Trial name
- NCT01150708
- Where
- United States
- Volunteers
- 180 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Hiv
Pharmacokinetics and Safety of DolutegravIr in Neonate
A Phase I/II, open-label, single arm, two-stage trial to evaluate the single and multi-dose PK and safety of DTG in HIV-exposed neonates on ARV prophylaxis.
- Trial name
- PETITE-DTG (NCT05590325)
- Testing
- Dolutegravir
- Where
- South Africa
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Desmond Tutu TB Centre
Colorectal Cancer
Survivorship in Lynch Syndrome Families
Objectives: Specific Aims: 1. Researchers will evaluate and compare predictors of health-related quality of life (QOL) among colorectal cancer (CRC) survivors who have Lynch syndrome (LS) with those patients who have sporadic CRC…
- Trial name
- NCT01126840
- Testing
- Telephone Interview, Questionnaire
- Where
- United States
- Volunteers
- 303 people, healthy volunteers welcome
- Sponsor
- M.D. Anderson Cancer Center
Diabetes Mellitus, Hyperglycemia
Durban Diabetes Study:A Study of the Epidemiology of Diabetes Mellitus in Urban South Africans of African Descent (DDS)
Type 2 diabetes (T2D) is an emerging epidemic in sub-Saharan Africa, with an estimated prevalence of 6%. With around seven million cases of T2D in 2000, it is anticipated that over 18 million Africans will have the disease by…
- Conditions
- Diabetes Mellitus, Hyperglycemia, Hereditary Diseases, Glucose Intolerance
- Trial name
- DDS (NCT02416440)
- Testing
- blood sample
- Where
- South Africa
- Volunteers
- 1,200 people, healthy volunteers welcome
- Sponsor
- University of KwaZulu
Hemophilia A, Musculoskeletal Assessment
Effects of Emicizumab Treatment in Patients With Hemophilia A
The aim of our study is to investigate the effects of emicizumab treatment on the musculoskeletal system in children with hemophilia A.
- Conditions
- Hemophilia A, Musculoskeletal Assessment, Musculoskeletal Abnormalities
- Trial name
- NCT07190118
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Hasan Kalyoncu University
Multiple Endocrine Neoplasia Type 1, Pancreatic Neuroendocrine Carcinoma
Ziv-Aflibercept in Treating and Computed Tomography Perfusion Imaging in Predicting Response in Patients With Pancreatic Neuroendocrine Tumors That Are Metastatic or Cannot Be Removed by Surgery
This phase II trial studies ziv-aflibercept in treating and perfusion computed tomography perfusion imaging in predicting response in patients with pancreatic neuroendocrine tumors that have spread to other parts of the body or…
- Trial name
- NCT02101918
- Testing
- Computed Tomography Perfusion Imaging, Laboratory Biomarker Analysis, Ziv-Aflibercept
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- National Cancer Institute (NCI)
Premature Birth, Obstetric Labor, Premature
Family Nurture Intervention in the NICU at The Valley Hospital
The purpose of this study is to compare neurodevelopment and activity in infants born preterm (25 to 34 1/7 weeks gestational age (GA)) receiving Standard Care (SC) or Family Nurture Intervention (FNI) in the neonatal intensive…
- Trial name
- NCT03267043
- Testing
- Family Nurture Intervention (FNI), Standard Care
- Where
- 2 sites in United States
- Volunteers
- 150 people
- Sponsor
- Columbia University
Intracranial Arteriovenous Malformations (AVM)
Protocol for Staged Stereotactic Radiosurgery for Large Arteriovenous Malformations
Stereotactic radiosurgery is a well established treatment option for arteriovenous malformations (AVMs). The potential complications related to radiosurgery are well documented and are predominately related to radiation effects…
- Trial name
- NCT02576535
- Testing
- Fractionated stereotactic radiosurgery, Leskell gamma unit
- Volunteers
- 18 people
- Sponsor
- NYU Langone Health
Rare Diseases, Marfan Syndrome
Marfan Syndrome (MFS) and Facial Dysmorphism: Non-invasive 3D Assessment
The goal of this study observational prospective study is to define the facial morphological features associated with Marfan syndrome (MFS). The main qustion it aims to answer are: 1.
- Trial name
- FACE (NCT05702476)
- Where
- Italy
- Volunteers
- 140 people
- Sponsor
- IRCCS Policlinico S. Donato
Cleft Lip, Cleft Palate
Oral Cleft Prevention Program
The purpose of this study is to asses if taking 4 mg of folic acid per day at preconception and during the first three months of pregnancy decreases the risk of having a child with cleft lip and palate compared to 0.4 mg folic…
- Trial name
- NCT00397917
- Testing
- Folic acid: 4 mg versus 0.4 mg per day
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- University of Iowa
Bipolar Disorder, Psychiatric Morbidity
Bipolar Disorder in Pregnancy and the Postpartum Period: Predictors of Morbidity
The purpose of this study is to examine risk factors for relapse of bipolar disorder during pregnancy and the postpartum period.
- Conditions
- Bipolar Disorder, Psychiatric Morbidity, Neonatal Outcome
- Trial name
- NCT00580268
- Where
- 2 sites in United States
- Volunteers
- 118 people
- Sponsor
- Massachusetts General Hospital
Newborn Respiratory Distress Syndrome
HHHFNC (Heated Humidified High-Flow Nasal Cannula) Versus NCPAP for Respiratory Distress Syndrome of Prematurity
Preterm infants ranging from 29+0 to 36+6 weeks+days are randomly assigned to one of the following treatments as non invasive respiratory support if they develop mild to moderate Respiratory Distress Syndrome within 72 hrs from…
- Trial name
- NCT02570217
- Testing
- HHHFNC, NCPAP
- Where
- Italy
- Volunteers
- 316 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Hypophosphatasia (HPP)
A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1
This is a phase 1/2a randomised, placebo controlled, double-blind study investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of ALE1 on healthy adult subjects and adult patients with Hypophosphatasia…
- Trial name
- NCT07179640
- Testing
- ALE1
- Where
- 3 sites in Germany, New Zealand and 1 more
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Alesta Therapeutics
Autism Spectrum Disorder, Fragile X Syndrome
Speech-in-noise Perception in Autism and Fragile X
The goal of this study is to identify which brain regions are active during speech-in-noise perception, as well as how those regions interact.
- Trial name
- NCT06088589
- Testing
- Mismatch negativity
- Where
- United States
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Rheologic Disease, Sickle Cell Disease
Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease
The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of…
- Conditions
- Rheologic Disease, Sickle Cell Disease, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT01294891
- Testing
- Imaging
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University
Necrotising Enterocolitis, Late-Onset Neonatal Sepsis
Characterization of Intestinal Microbiota Stability in Preterm Born Neonates
Study around very-low birthweight preterm infants at high risk of developing necrotizing enterocolitis (NEC) or late-onset sepsis (LOS).
- Trial name
- NEC (NCT04792918)
- Where
- 2 sites in Switzerland
- Volunteers
- 81 people
- Sponsor
- Emma Marie Caroline Slack
Cerebrovascular Accident, Stroke, Acute
Fabry and Cardiomyopathy (FaCard)
Primary objective and endpoint is the analysis of the long-term course of lyso-Gb3 and its clinical correlation to the progression of the cardiomyopathy in N215S-Fabry patients.
- Conditions
- Cerebrovascular Accident, Stroke, Acute, Cerebral Stroke
- Trial name
- FaCard (NCT01429597)
- Where
- Germany
- Sponsor
- CENTOGENE GmbH Rostock
Morquio Disease
Establishment of Human Cellular Disease Models for Morquio Disease
Establishment of human cellular disease models for Morquio disease for an individualized therapy development having the capacity to address both hepatic and neurologic forms of the disease
- Trial name
- IPSMORQUIO (NCT03872713)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
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