Clinical trials: Congenital Diseases
Leukemia, Lymphoma
Total-Body Irradiation, Cyclophosphamide, and Stem Cell Transplantation in Treating Patients With Hematologic Cancer
RATIONALE: Adjusting the dose of drugs used in chemotherapy such as cyclophosphamide may decrease side effects while stopping cancer cells from dividing so they stop growing or die.
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Trial name
- NCT00062140
- Testing
- cyclophosphamide, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation
- Where
- United States
- Sponsor
- Fred Hutchinson Cancer Center
Pulmonary Artery Stenosis, Tetralogy of Fallot
Retrospective Review of Anesthetic Considerations for Pulmonary Artery Rehabilitation
Pulmonary artery rehabilitation procedure is done frequently in the catheterization suite for patients with pulmonary artery stenosis or small pulmonary arteries following surgical repair for patients with tetralogy of Fallot…
- Conditions
- Pulmonary Artery Stenosis, Tetralogy of Fallot, Pulmonary Atresia
- Trial name
- NCT02586740
- Testing
- Pulmonary artery rehabilitation
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Aymen N Naguib
Rare Diseases, Congenital Diaphragmatic Hernia
Intact Cord Resuscitation in CDH
Isolated CDH is a rare disease (1/3500) and displays a wide range of severity and outcome. Despite attempts to standardize the management of this disease at birth and during the first months of life, the mortality varies from 20…
- Trial name
- CHIC (NCT04429750)
- Testing
- Immediate umbilical cord clamping, intact cord resuscitation
- Where
- 3 sites in France
- Volunteers
- 180 people
- Sponsor
- University Hospital, Lille
Pulmonary Artery Pressure, Thalassemia
Effect of L-Glutamine on Pulmonary Artery Pressure in Patients With Non-Transfusion-Dependent Thalassemia
The goal of this clinical trial is to learn whether L-glutamine can help lower pulmonary artery pressure in adults with non-transfusion-dependent thalassemia (NTDT).
- Conditions
- Pulmonary Artery Pressure, Thalassemia, Non-transfusion Dependent Thalassemia
- Trial name
- NCT07210450
- Testing
- L-glutamine
- Where
- Iran
- Volunteers
- 8 people
- Sponsor
- Mazandaran University of Medical Sciences
Congenital Heart Disease
The Efficacy of Systematic Oral Feeding Education Program
After the intervention of Systematic Oral Feeding Education Program: 1. The feeding techniques scores of the main caregivers in the experimental group will be higher than those in the control group. 2.
- Trial name
- SOFEP (NCT06763497)
- Testing
- Systematic Oral Feeding Education Program
- Volunteers
- 60 people
- Sponsor
- National Taiwan University Hospital
Cystic Fibrosis
A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
- Trial name
- NCT03911713
- Testing
- VX-561, IVA
- Where
- 48 sites in United States, Australia and 5 more
- Volunteers
- 77 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Cystic Fibrosis
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis
The goal of this clinical trial is to learn if drug SPL84 is safe for adult patients with cystic fibrosis (CF). It will also learn if the drug works to treat works to treat CF with a specific mutation (3849 +10kb C-->T).
- Trial name
- NCT06429176
- Testing
- SPL84
- Where
- 3 sites in United States
- Volunteers
- 64 people
- Sponsor
- SpliSense Ltd.
Heart Failure Congenital, Single-ventricle
REHAB Fontan Failure: A Trial of Cardiac Rehabilitation
The goal of this clinical trial is to compare the impact of cardiac rehabilitation on Fontan failure patients' exertional tolerance, frailty, and quality of life. 1.
- Trial name
- NCT06150950
- Testing
- Cardiac rehabilitation, Usual care
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- Stanford University
Hypospadias
Effect of Caudal and Penile Block on Hypospadias Repair Complications
Hypospadias is one of the most common genitourinary (GU) malformations, seen in approximately 1 of 250 male live births. Common methods of local anesthesia administration for hypospadias surgery include caudal and dorsal penile…
- Trial name
- NCT02851290
- Testing
- Caudal block anesthesia, Dorsal penile nerve block anesthesia
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Baylor College of Medicine
Neck Disease, Trismus
Ideal Point of Transluminal Light in Tracheal Intubation With a Light Wand
If the patients's teeth are weak or the mouth dose not open well, the lightwand is a useful device when endotracheal intubation is necessary.
- Conditions
- Neck Disease, Trismus, Tooth Abnormalities
- Trial name
- NCT03480035
- Testing
- lightwand intubation
- Where
- South Korea
- Volunteers
- 107 people
- Sponsor
- Keimyung University Dongsan Medical Center
Cytomegalovirus Infections
(Val)Ganciclovir TDM in Transplant Recipients
The aim of this study is to gain more insight into therapeutic drug monitoring and thus the pharmacodynamics and pharmacokinetics of ganciclovir, in the context of prophylaxis and treatment of CMV infections, in order to provide…
- Trial name
- NCT03698435
- Testing
- Ganciclovir, Valganciclovir
- Where
- Netherlands
- Volunteers
- 100 people
- Sponsor
- University Medical Center Groningen
Alpha-Mannosidosis
Expanded Access to Velmanase Alfa
Individual patient expanded access requests may be considered for patients who have no other treatment options.
- Trial name
- NCT04959240
- Testing
- Velmanase Alfa
- Sponsor
- Chiesi Farmaceutici S.p.A.
Retinitis Pigmentosa
The Perceptual Experience of Argus II Users
This Basic Experimental Studies Involving Humans (BESH) study investigates phosphene perception and performance factors in blind participants implanted with the Argus II retinal prosthesis system.
- Trial name
- NCT05285618
- Testing
- Argus II
- Where
- 3 sites in United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- University of California, Santa Barbara
Fabry Disease
Enzyme Replacement Therapy in Fabry Disease
Fabry disease is an X-linked rare metabolic disease, caused by a deficient activity of the hydrolase α-Galactosidase A, and characterized by a progressive and systematic deposition of glycosphingolipids in many organs.
- Trial name
- NCT00149318
- Testing
- Biochemical analyses.
- Where
- Italy
- Volunteers
- 2 people
- Sponsor
- Mario Negri Institute for Pharmacological Research
Down Syndrome
Effects of Pelvic Girdle Stabililty With Use of Therapeutic Taping in Down Syndrome
A Interventional study on 16 down syndrome pateints and check effects o therapeutic taping on pelvic girdle stability on 8 weeks sessions
- Trial name
- NCT06770218
- Testing
- pelvic girdle stability training and therapeutic tapping, pelvic girdle stability training only
- Where
- 2 sites in Pakistan
- Volunteers
- 16 people
- Sponsor
- Riphah International University
Primary Immune Deficiency
Multicenter Study of Long-Term Clinical Outcomes of Subcutaneous Immune Globulin IgPro20 in Subjects With Primary Immunodeficiency (Japan Study)
The purpose of this study is to assess the long-term efficacy, tolerability, and safety of IgPro20 in subjects with primary immunodeficiency (PID) as an extension to the preceding follow-up study ZLB07_001CR (NCT01458171).
- Trial name
- NCT01461018
- Testing
- Immune globulin subcutaneous (Human)
- Where
- 9 sites in Japan
- Volunteers
- 22 people
- Sponsor
- CSL Behring
Primary Antibody Deficiency Disorders
Exercise Intervention and Immunodeficiency
The purpose of this study is to assess if a 12-week exercise program is safe, practical, and acceptable for adults with primary antibody deficiencies.
- Trial name
- NCT07698197
- Testing
- App-based exercise intervention
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Yale University
Congenital Diaphragmatic Hernia
Safety and Efficacy of Fetoscopic Endoluminal Tracheal Occlusion in Congenital Diaphragmatic Hernia
The purpose of this study is to evaluate the safety and efficacy of Fetoscopic Endoluminal Tracheal Occlusion (FETO) in cases of severe Congenital Diaphragmatic Hernia (CDH).
- Trial name
- NCT06946576
- Testing
- Fetoscopic Endoluminal Tracheal Occlusion (FETO) Procedure
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Oregon Health and Science University
Congenital Bleeding Disorder, Congenital FXIII Deficiency
Safety and Pharmacokinetics of Recombinant Factor XIII in Patients With Congenital Factor Xlll Deficiency
This trial was conducted in the United States of America (USA). The aim of this trial was to investigate safety and pharmacokinetics of escalating single doses of catridecacog (recombinant factor XIII, rFXIII) in patients with…
- Trial name
- NCT00056589
- Testing
- catridecacog
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Novo Nordisk A/S
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