Clinical trials: Congenital Diseases
Hemophilic Arthropathy
Rofecoxib Efficacy and Safety Evaluation Trial in Hemophilic Arthropathy
This is a multicenter, randomized, double-blind study to evaluate the efficacy and safety of TRM-201 (rofecoxib) versus Placebo in the treatment of patients with hemophilic arthropathy (HA) over a 12-week period (Part I) that is…
- Trial name
- RESET-HA (NCT04684511)
- Testing
- TRM-201 (Rofecoxib)
- Where
- 46 sites in United States, Australia and 6 more
- Volunteers
- 2 people
- Sponsor
- Tremeau Pharmceuticals, Inc.
Alpha-1 Antitrypsin Deficiency (AATD)
A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)
This study is the first study in the RestorAATion clinical program. The purpose of this first-in human (FIH), double-blind, randomized, placebo-controlled, single ascending dose (SAD) and multiple-dose Phase 1 study is to assess…
- Trial name
- RestorAATion-1 (NCT06186492)
- Testing
- WVE-006
- Where
- United Kingdom
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Wave Life Sciences USA, Inc.
Persistent Pulmonary Hypertension of the Newborn
Efficacy of Milrinone With Sildenafil in Persistent Pulmonary Hypertension in Children
Neonates with persistent pulmonary hypertension (PPH) should be administered inhaled nitric oxide (iNO) and extracorporeal membrane oxygenation (ECMO), but these are not available in most resource-constrained settings like ours.
- Trial name
- NCT06562036
- Testing
- Sildenafil, Milrinone
- Where
- Pakistan
- Volunteers
- 42 people
- Sponsor
- Muhammad Aamir Latif
Sickle Cell Disease
Genetic and Haematological Modifiers of SCD Severity in Kaduna State, Northern Nigeria
This study is aimed to assess the genetic and haematological modifiers of disease severity among patients with Sickle Cell Disease (SCD) in Kaduna State, northern Nigeria.
- Trial name
- SCA (NCT05837871)
- Volunteers
- 400 people
- Sponsor
- Ahmadu Bello University Teaching Hospital
Atrial Septal Defect (ASD), Dexmedetomidine
Effect of Dexmedetomidine Supplementation to General Anaesthesia in Paediatric Transcatheter Closure of Atrial Septal Defect
The aim of this randomized trial is to determine efficacy of dexmedetomidine in improving quality of recovery in pediatric patients undergoing interventional catheter closure of ASD while maintaining the hemodynamic parameters.
- Conditions
- Atrial Septal Defect (ASD), Dexmedetomidine, Delirium - Postoperative, Stress Response
- Trial name
- NCT06631534
- Testing
- Dexmedetomidine
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Alexandria University
Sickle Cell Disease
Assessing Physical Function in Sickle Cell Patients Taking Voxelotor
Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels.
- Trial name
- NCT06023199
- Testing
- Voxelotor
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Inova Health Care Services
Polycystic Ovary Syndrome, Hyperandrogenism
Metformine and CC Compared With Placebo and CC for Induction Ovulation in PCOS Patients With Insulin Resistant
Polycystic ovary syndrome (PCOS) is the most common cause of anovulatory infertility which affects 12-21% of the population.Several studies performed evaluate the possible benefit of metformin alone or in combination with…
- Trial name
- NCT02523898
- Testing
- metformine, clomiphene citrate
- Volunteers
- 388 people
- Sponsor
- Semnan University of Medical Sciences
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
The purpose of this study is: To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects treated previously with a single 7-day course of QLT091001 in Study RET IRD 01 To…
- Trial name
- NCT01521793
- Testing
- QLT091001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 27 people
- Sponsor
- QLT Inc.
Copy Number Abnormality, Prenatal Diagnosis
Technology Development for Noninvasive Prenatal Genetic Diagnosis Using Whole Fetal Cells From Maternal Peripheral Blood
Amniocentesis (amnio) and chorionic villus sampling (CVS) can reliably detect many smaller DNA/genetic abnormalities that cannot be reliably diagnosed by cell-free noninvasive prenatal testing (NIPT) that is in widespread use.
- Conditions
- Copy Number Abnormality, Prenatal Diagnosis, Genetic Disease
- Trial name
- NCT04285814
- Testing
- Whole Fetal Cell (WFC) Testing
- Where
- 2 sites in United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Columbia University
Cystic Fibrosis
Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults
The aim of this project is to evaluate the psychological reshuffle induced by ORKAMBI. The particular focus of this study is the consequence of its introduction on anxiety, depression, quality of life and adherence to all cystic…
- Trial name
- ORK-AJA (NCT03659214)
- Testing
- Questionnaires
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease
The purpose of this study is to estimate the prevalence, demographic, and clinical characteristics of PKD1/2 gene variant groups in the ADPKD population.
- Trial name
- NCT06747572
- Where
- 45 sites in United States, Belgium and 7 more
- Volunteers
- 401 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Combined Inspiratory Muscle and 'Whole Muscle' Training in Children With Cystic Fibrosis
To study the effects of an 8-week combined inspiratory muscle training and exercise (resistance+aerobic) program on of a lung volume, inspiratory muscle strength (maximal inspiratory pressure, PImax) and cardiorespiratory fitness…
- Trial name
- NCT01706445
- Testing
- Other
- Where
- Spain
- Volunteers
- 20 people
- Sponsor
- Universidad Europea de Madrid
Huntington's Disease, Parkinson's Disease
Treating Sleep/Wake Cycle Disturbances in Basal Ganglia Disorders With Ramelteon
The proposed study is a double-blind, placebo controlled pilot study of HD, PD, and DLB subjects with sleep disturbances. This study is designed to determine the effects of 4 weeks Ramelteon treatment on the sleep patterns of…
- Conditions
- Huntington's Disease, Parkinson's Disease, Dementia With Lewy Bodies, Sleep Disorders
- Trial name
- NCT00907595
- Testing
- Ramelteon
- Where
- United States
- Sponsor
- Massachusetts General Hospital
Leukemia, Lymphoma
Collecting Tissue Samples From Patients With Leukemia or Other Blood Disorders Planning to Enroll in an ECOG Leukemia Treatment Clinical Trial
RATIONALE: Collecting and storing samples of blood and bone marrow from patients with cancer to study in the laboratory may help doctors learn more about diagnosing cancer and determine a patient's eligibility for a treatment…
- Trial name
- NCT00897767
- Testing
- biologic sample preservation procedure
- Where
- 214 sites in United States
- Volunteers
- 1,873 people
- Sponsor
- Eastern Cooperative Oncology Group
Congenital Heart Disease, Single-ventricle
Extracellular Vesicle Micro RNA Profiling in Congenital Heart Disease: Fetal-Maternal Regulation in Neonatal Thrombosis
Newborns with congenital heart disease (CHD) are at increased risk of developing postpartum and postoperative blood clots after cardiac surgery.
- Conditions
- Congenital Heart Disease, Single-ventricle, Thrombosis
- Trial name
- EVmiRNA (NCT06434207)
- Testing
- Collecting discarded blood samples
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Boston Children's Hospital
Coronary Artery Disease, Myocardial Infarction
Comparative Effectiveness Research to Assess the Use of Primary ProphylacTic Implantable Cardioverter Defibrillators in Europe
The "EUropean Comparative Effectiveness Research to assess the use of primary prophylacTic Implantable Cardioverter Defibrillators (EU-CERT-ICD)" is a modular research project to study the effectiveness of prophylactic ICDs in a…
- Conditions
- Coronary Artery Disease, Myocardial Infarction, Dilated Cardiomyopathy, Implantable Defibrillator User
- Trial name
- EU-CERT-ICD (NCT02064192)
- Where
- 42 sites in Belgium, Bulgaria and 14 more
- Volunteers
- 2,285 people
- Sponsor
- University Medical Center Goettingen
Heart Defects, Congenital
Neuroprotective Effects of Remote Ischemic Preconditioning (RIPC) During Infant Cardiac Surgery
The purpose of this study is to look at whether the use of a simple technique before surgery, which involves inflating a blood pressure cuff on the infant's leg, can improve development of language, motor and thinking skills at…
- Trial name
- NCT01835392
- Testing
- Remote Ischemic Preconditioning, Control
- Where
- United States
- Volunteers
- 137 people
- Sponsor
- Nemours Children's Clinic
Single Ventricle
Perioperative Treprostinil in Pediatric Patients Undergoing the Fontan Operation
The purpose of this study is to test the efficacy of treprostinil (TRE) in the perioperative non-fenestrated, extracardiac Fontan patients in order to reduce duration of chest tube drainage (in days).
- Trial name
- NCT02498444
- Testing
- Treprostinil
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Stanford University
Rheumatoid Arthritis, Osteoarthritis
A Two Centre Study to Assess the Long-term Performance of the Pinnacle™ Cup With a Metal-on-Metal Bearing in Primary Total Hip Replacement
The purpose of this study is to monitor the performance and determine the metal ion release of the Pinnacle™ Cup with a metal-on-metal bearing combination in the treatment of patients with hip joint disease requiring a total hip…
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00208364
- Testing
- Pinnacle Acetabular Cup System
- Where
- 2 sites in Italy, United Kingdom
- Volunteers
- 163 people
- Sponsor
- DePuy International
Lymphatic Malformation, Lymphatic Abnormality
Institution of an Italian Registry and Biobank for Biological Sample Collection
Lymphatic malformations (ML), are benign non-neoplastic, rare, resulting from an embryologic abnormal development of the lymphatic system.
- Conditions
- Lymphatic Malformation, Lymphatic Abnormality, Lymphangioma, Lymphatic Abnormalities
- Trial name
- IReg-LM (NCT06892964)
- Testing
- Genetic profiling of all eligible patients
- Where
- Italy
- Volunteers
- 70 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.