Clinical trials: Abnormalities
Type II Gaucher Disease
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
- Trial name
- NCT06272149
- Testing
- VGN-R08b
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Primary Ciliary Dyskinesia, Kartagener Syndrome
Genetics of Primary Ciliary Dyskinesia
This study is designed to study DNA sequencings for mutations in a research genetic test panel of genes (which contains all 32 known and/or published genes associated with PCD).
- Trial name
- NCT02389049
- Where
- 8 sites in United States, Canada
- Volunteers
- 320 people
- Sponsor
- University of North Carolina, Chapel Hill
Post-COVID-19 Condition, Ulcerative Colitis
Emerging Indications for Hyperbaric Oxygen Treatment
This study will evaluate the effectiveness of hyperbaric oxygen therapy (HBOT) on treating emerging indications (i.e., conditions that have shown to potentially benefit from HBOT) using the Multicenter Registry for Hyperbaric…
- Conditions
- Post-COVID-19 Condition, Ulcerative Colitis, Crohn Disease, Calciphylaxis
- Trial name
- NCT07240649
- Testing
- Hyperbaric oxygen treatment (HBOT), 100% Oxygen
- Volunteers
- 100 people
- Sponsor
- Jay C. Buckey Jr.
Fetal Alcohol Syndrome, Teen Pregnancy
Evaluating the Efficacy of a Video Based Intervention to Educate Teen Moms About Fetal Alcohol Syndrome (FAS)
Evaluating the efficacy of a video about FAS to determine whether it has an impact on knowledge about FAS and their likelihood to participate in risk taking behavior.
- Trial name
- NCT00747630
- Testing
- An educational video about Fetal Alcohol Syndrome
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- Texas Tech University Health Sciences Center
Congenital Bleeding Disorder, Haemophilia A
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who…
- Trial name
- pathfinder™5 (NCT01731600)
- Testing
- turoctocog alfa pegol
- Where
- 51 sites in United States, Brazil and 14 more
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S
Leukemia, Myelodysplastic Syndromes
Combination Chemotherapy With or Without Filgrastim and/or Tretinoin in Treating Patients With Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Colony-stimulating factors such as filgrastim may increase the number of immune cells found in bone marrow…
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00005863
- Testing
- filgrastim, cytarabine, daunorubicin hydrochloride
- Where
- United Kingdom
- Sponsor
- Medical Research Council
Muscular Dystrophy, Duchenne
Spironolactone Versus Prednisolone in DMD
This is a randomized, open-label, pilot clinical trial of spironolactone suspension versus oral prednisolone for use in Duchenne muscular dystrophy.
- Trial name
- NCT03777319
- Testing
- Spironolactone, Prednisolone
- Where
- 4 sites in United States
- Volunteers
- 2 people
- Sponsor
- Kevin Flanigan
Down Syndrome
Evaluation of a Simplified Method for Diagnosis of Sleep Apnea in Children and Adults With Down Syndrome
People with Down syndrome are at increased risk of sleep apnea, not only from obstruction of the upper airway, but also of central origin.
- Trial name
- SAOS/T21 (NCT00951730)
- Testing
- Polysomnography
- Where
- 3 sites in France
- Volunteers
- 97 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Sickle Cell Disease
Sickle-cell Disease Registry of the GPOH
Sickle cell disease is one of the most common hereditary diseases. Most severe complications can be avoided if the disease is detected early and treated appropriately.
- Trial name
- SichReg (NCT03327428)
- Where
- Germany
- Volunteers
- 1,000 people
- Sponsor
- University Hospital Heidelberg
Atopic Dermatitis
CorEvitas International Adolescent Atopic Dermatitis (AD) Drug Safety and Effectiveness Registry
Prospective observational registry for an adolescent cohort with AD under the care of a dermatology provider. Approximately 1500 subjects and 75 clinical sites in North America and select European countries will be recruited to…
- Trial name
- NCT06238765
- Where
- United States
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- CorEvitas
Congenital Heart Disease, Telerehabilitation
The Effect of Telerehabilitation Exercises in Children With Congenital Heart Disease
This study aims to investigate the effects of functional inspiratory muscle training on physical performance and quality of life in pediatric patients with congenital heart disease.
- Conditions
- Congenital Heart Disease, Telerehabilitation, Functional Inspiratory Muscle Training
- Trial name
- CHD (NCT07854171)
- Testing
- Inspiratory Muscle Training, Functional Inspiratory Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Izmir Democracy University
Cystic Fibrosis
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03525444
- Testing
- VX-445/TEZ/IVA, IVA
- Where
- 115 sites in United States, Australia and 11 more
- Volunteers
- 405 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cleft Lip and Palate
Liposomal Bupivacaine Use in Alveolar Bone Graft Patients
The goal of this clinical trial is to learn if a pain medication called liposomal bupivacaine (brand name EXPAREL®) with epinephrine will provide better pain control, increased activity, and reduced use of opioids compared with…
- Trial name
- NCT06284434
- Testing
- Liposomal Bupivacaine, Bupivacain, Epinephrine
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Kerry O'Rourke
Venous Malformation, Lymphatic Malformation
Weekly Sirolimus Therapy
In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months.
- Trial name
- NCT04861064
- Testing
- Sirolimus
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Medical University of South Carolina
GM1 Gangliosidosis
Interviews and Video Capture in Patients With GM1 Gangliosidosis
GM1 gangliosidosis is a rare disease for which there is a limited understanding of disease progression and meaningful outcome measures.
- Trial name
- NCT04310163
- Testing
- Natural history
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- LYSOGENE
Spina Bifida
The Study Aims to Improve the Accuracy of Detecting Spina Bifida During Early Ultrasound Scans. to Achieve This, an AI Model Has Been Developed to Provide Feedback About the Presence of Spina Bifida. a RCT Has Been Designed to Compare the Effectiveness of AI Feedback with No AI Feedback.
The study aims to improve the accuracy of detecting spina bifida during early ultrasound scans. To achieve this, an AI model has been developed to provide feedback about the presence of spina bifida.
- Trial name
- NCT06566014
- Testing
- Evaluation of XAI-assisted spina bifida diagnosis
- Where
- Denmark
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Copenhagen Academy for Medical Education and Simulation
Cystic Fibrosis
Coping Compliance and Adjustment in Adolescents With Cystic Fibrosis
In this research, we will use established surveys to look at the relationship between various styles of coping with a disease, religious coping styles, treatment compliance, locus of control, broad measures of mental health and…
- Trial name
- NCT00577252
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Akron Children's Hospital
Beta-Thalassemia
Thrombophilia In Beta Thalassemia
β-thalassemia disease is one of the most common congenital hemolytic anemia commonly found in the malarial belt areas including the Mediterranean, the Middle East, Africa, Southeast Asian countries, and China.
- Trial name
- NCT04219449
- Testing
- PT, Protein C, Platelet aggregation by ADP and arachidonic acid
- Volunteers
- 100 people
- Sponsor
- Assiut University
Neonatal Abstinence Syndrome, Substance Withdrawal, Neonatal
The Effect of Music Therapy on Newborns
The clinical study is evaluating the impact of music therapy on neonates, specifically infants with neonatal opioid withdrawal syndrome (NOWS).
- Trial name
- NCT03987165
- Testing
- Music Therapy
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Tulane University
Neutropenia, Sickle Cell Anemia
Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders
OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders. II.
- Conditions
- Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
- Trial name
- NCT00005893
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center