Clinical trials: Congenital Diseases
Craniosynostoses, Hypovolemia
Hemodynamic Monitoring During Craniosynostosis Surgery: Comparing Traditional and Newer Technology Monitors (CRASY-PRAM)
Hemodynamic evaluation during pediatric anesthesia is essential to care management. Intraoperative cardiovascular instability is frequent in major surgeries, and appropriate monitoring is necessary to ensure safe anesthetic…
- Conditions
- Craniosynostoses, Hypovolemia, Hemorrhage, Hemodynamic Instability
- Trial name
- CRASY-PRAM (NCT06263075)
- Testing
- Intraoperative monitoring
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Hereditary Angioedema
A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II
This study is a randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adult subjects.
- Trial name
- NCT04208412
- Testing
- KVD900
- Where
- 25 sites in United States, Austria and 8 more
- Volunteers
- 84 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Huntington Disease
Move to Improve: Telehealth Exercise to Music for HD
The purpose of this study is to determine if a movement to music exercise program delivered via telehealth is feasible and safe for individuals with neurodegenerative disease and their caregivers (Aim 1).
- Trial name
- MtI (NCT05270681)
- Testing
- Movement to Music
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Ohio State University
Hemophilia A, Hemophilia B
Pre-clinical Models for Mesenchymal Stem Cell Therapy in Hemophilic Arthropathy
Hemophilia is a constitutional coagulation disorder responsible for a hemorrhagic phenotype in patients from an early age. Hemarthrosis is one of the most frequent complications in hemophiliacs and leads to the development of…
- Conditions
- Hemophilia A, Hemophilia B, Arthropathy
- Trial name
- NCT05187936
- Where
- France
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Metastatic Cancer
A Study of Reduced-dose Radiation in People With Metastatic Tumors With a Genetic Change
This study will test whether reduced-dose radiotherapy is an effective treatment for metastatic tumors with an ATM mutation. The researchers want to find the lowest dose of radiation that would still be effective to treat these…
- Trial name
- NCT05010031
- Testing
- Palliative radiotherapy
- Where
- 7 sites in United States
- Volunteers
- 45 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Infantile Spinal Muscular Atrophy
Test of Aquatic Mobility of SMA Infants
Spinal muscular atrophy (SMA) is a genetic neurodegenerative disease impacting spinal cord motor neurons, leading to motor and respiratory issues and, ultimately, death.
- Trial name
- BAINSMA (NCT06152302)
- Testing
- Mobility study in dry conditions, Mobility study in water conditions
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Bronchopulmonary Dysplasia
Pressure-Sensing Mattresses and Mechanical Ventilation Weaning in Neonatal
This study aims to explore the correlation between using ballistocardiography for monitoring respiration and heart rate in neonates under invasive and non-invasive respiratory support.
- Trial name
- NCT06579157
- Testing
- Pressure-Sensing Mattresses
- Where
- Taiwan
- Volunteers
- 60 people
- Sponsor
- Fu Jen Catholic University
Hypercholesterolemia, Familial Hypercholesterolemia
A Clinical Trial to Assess the Long Term Safety and Tolerability of MK-0653H in Japanese Participants With Hypercholesterolemia (MK-0653H-833)
The study will assess the safety and tolerability of Ezetimibe 10 mg+ Rosuvastatin 2.5 mg and Ezetimibe 10 mg+ Rosuvastatin 5.0 mg for up to 52 weeks in Japanese participants with hypercholesterolemia uncontrolled with…
- Trial name
- NCT02748057
- Testing
- Ezetimibe, Rosuvastatin
- Volunteers
- 135 people
- Sponsor
- Organon and Co
Rare Diseases, Genetic Disease
The VetSeq Study: a Pilot Study of Genome Sequencing in Veteran Care
The VetSeq Study is a pilot intervention study exploring the feasibility of integrating genome sequencing into clinical care at the VA Boston Healthcare System.
- Trial name
- NCT03380819
- Testing
- Genome sequencing
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- VA Boston Healthcare System
Cystic Fibrosis
Functional Analysis by Dynamic Imaging of the Respiratory Epithelium in Infants With Cystic Fibrosis
Cystic fibrosis (CF) is characterized by airway inflammation and infection leading to progressive destruction of lungs. One of the most important abnormalities in CF is an abnormal processing of the mutated CFTR protein through…
- Trial name
- NCT01605565
- Testing
- A nasal brushing
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
End Stage Renal Failure, Hemodialysis Access Failure
Outcomes for One-stage and Two-stage Brachial Basilic Arteriovenous Fistulas
An arteriovenous (AV) fistula is essential for patients with kidney failure on dialysis. There is no clear data on the best method to create a new brachial basilic AV fistula (BBAVF), so the choice between a single procedure and…
- Conditions
- End Stage Renal Failure, Hemodialysis Access Failure, Arterio-venous Fistula
- Trial name
- NCT04376567
- Testing
- One stage approach
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Tze-Woei Tan
Retinitis Pigmentosa, Retinal Dystrophy
Observational Study to Assess the Reliability and Validity of the MLYMT and MLSDT
This is a prospective Multi-Center Observational Study to assess the reliability and validity of the Multi-Luminance Y-Mobility Test (MLYMT) and Multi-Luminance Shape Discrimination Study (MLSDT) Main Outcome Measures: (i)…
- Trial name
- NCT05820100
- Testing
- Observational
- Where
- 8 sites in United States, Puerto Rico
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Nanoscope Therapeutics Inc.
Down Syndrome
Physiological Abnormalities Associated with Down Syndrome
The overall goal of this study is to evaluate biomarkers of oxidative stress, mitochondrial function, and DNA methylation (epigenetics) in order to determine the extent to which these biomarkers are related to cognitive…
- Trial name
- NCT03087500
- Testing
- There is no other intervention, only clinical treatment.
- Sponsor
- University of Arkansas
Sickle Cell Disease
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can…
- Trial name
- SUN (NCT03587272)
- Testing
- Alemtuzumab, low dose total body irradiation, Sirolimus
- Where
- 7 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Robert Nickel
Cystic Fibrosis
Fidgety Movements in Cystic Fibrosis
Very low birth weight infants who are at risk for chronic lung diseases may also be at risk for brain anomalies such as increased echogenicity, leukomalacia and intracranial hemorrhage.
- Trial name
- NCT03381157
- Testing
- Observational General Movements Assessments
- Where
- Turkey (Türkiye)
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Benign Breast Disease, Breast Cancer
Prolactin Receptor and Breast Diseases
Prolactin is known to play an important role in breast development and differentiation. Thus proliferative breast diseases are good models to unravel PRl / PRLR function in proliferative processes.
- Trial name
- Prolacsein (NCT00842465)
- Testing
- blood collection for hormonal status analysis, breast Biopsy or surgery, blood collection
- Where
- France
- Volunteers
- 735 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Meconium Aspiration
Phase III Randomized Study of Lucinactant in Full Term Newborn Infants With Meconium Aspiration Syndrome
OBJECTIVES: Evaluate the safety and efficacy of lucinactant administered by bronchoalveolar lavage (BAL) in the treatment of meconium aspiration syndrome (MAS) in newborn infants.
- Trial name
- NCT00004500
- Testing
- Lucinactant, Standard Care
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Windtree Therapeutics
Atopic Dermatitis
A Study Assessing Rocatinlimab (AMG 451) Monotherapy in Moderate-to-severe Atopic Dermatitis (AD) (ROCKET-Horizon)
The co-primary objectives of the study are to: Evaluate the efficacy of rocatinlimab compared with placebo at Week 24, assessed using Validated Investigator's Global Assessment for Atopic Dermatitis (vIGA-AD).
- Trial name
- ROCKET-Horizon (NCT05651711)
- Testing
- Rocatinlimab
- Where
- 197 sites in United States, Australia and 19 more
- Volunteers
- 726 people
- Sponsor
- Amgen
Congenital Heart Diseases
Ulinastatin Injection in in Pediatric Patients Undergoing Open Heart Surgery
1. Explore the efficacy of Ulinastatin use in improving post-operative pulmonary insufficiency and safety in pediatric patients undergoing scheduled CPB open heart surgery to treat Complex Congenital Heart Disease 2.
- Trial name
- NCT02527811
- Testing
- Ulinastatin
- Where
- 5 sites in China
- Volunteers
- 450 people
- Sponsor
- Techpool Bio-Pharma Co., Ltd.
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