Clinical trials: Congenital Diseases
Musculoskeletal Diseases, Genetic Disease
Turkish Version of the Affordance in the Home Environment for Motor Development-Toddler (AHEMD-T)
This study aimed to translate the Affordance in the Home Environment for Motor Development -Toddler (AHEMD-T) into Turkish and examine its psychometric properties.
- Conditions
- Musculoskeletal Diseases, Genetic Disease, Metabolic Disease, Delays Developmental
- Trial name
- NCT04556500
- Testing
- investigating the psychometric properties of Turkish Affordance in the Home Environment for Motor Development-Toddler (AHEMD-T)
- Where
- Turkey (Türkiye)
- Volunteers
- 139 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Cryopyrin Associated Periodic Syndrome
The Safety and Efficacy of Canakinumab in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada
This study will assess the safety, tolerability and efficacy of canakinumab treatment in Canadian patient diagnosed with cryopyrin-associated periodic syndrome over a period of 18 months.
- Trial name
- NCT01105507
- Testing
- canakinumab (company code: ACZ885D)
- Where
- 2 sites in Canada
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Mandibular Deficiency
Accuracy of Implant Placement for Patients With Mandibular Reconstruction Using Dynamic Navigation and Static Guides
To evaluate and compare the accuracy of implant placement using dynamic navigation technique versus static template technique in patients with bone tissue flap reconstruction of mandibular defects, thus further guiding the…
- Trial name
- NCT06250907
- Testing
- The guidance of dynamic navigation, The guidance of static template
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University
PICC Line Placement, Vessels; Anomaly
Image-Guided Central Catheter Insertion With Concurrent Assessment of Vascular Wall Integrity
The insertion of peripherally inserted central catheters (PICCs) represents a minimally invasive and safe method for establishing long-term central venous access in patients requiring prolonged intravenous therapy, including…
- Trial name
- NCT07466472
- Volunteers
- 80 people
- Sponsor
- University Medical Centre Ljubljana
Diabetes, Endocrine System Diseases
Evaluating Innovations in Transition From Pediatric to Adult Care - The Transition Navigator Trial
The Transition Navigator Trial (TNT) is a pragmatic randomized controlled trial evaluating the effectiveness of usual care plus a patient navigator service versus usual care plus newsletters and other educational materials, to…
- Conditions
- Diabetes, Endocrine System Diseases, Gastro-Intestinal Disorder, Neuro-Degenerative Disease
- Trial name
- TNT (NCT03342495)
- Testing
- Patient Navigator, Usual Care
- Where
- 3 sites in Canada
- Volunteers
- 337 people
- Sponsor
- University of Calgary
Systemic Lupus Erythematous
Pilot Study Related to the Effect of Clopidogrel on Plasmatic Soluble CD40 Ligand During Systemic Lupus Erythematous
CD40 Ligand (CD40L) has been identified as a key feature in systemic lupus erythematosus (SLE) pathogenesis, a systemic autoimmune disease characterized by a multiorgan involvement.
- Trial name
- CLOPUS (NCT02320357)
- Testing
- Treatment by clopidogrel
- Where
- 3 sites in France
- Volunteers
- 18 people
- Sponsor
- University Hospital, Bordeaux
Fetal Hypoxia
Scalp Sampling for Fetal Surveillance
The purpose of this study is to compare lactate and total acid-base status in fetal scalp blood with lactate and total acid-base status in maternal blood and amniotic fluids to provide information on how lactate and base deficit…
- Trial name
- FBS (NCT01533701)
- Testing
- Fetal scalp blood scalp, amniotic fluid, maternal blood and umbilical cord sampling
- Where
- Sweden
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Uppsala University Hospital
Hypofibrinogenemia, Congenital, Afibrinogenemia, Congenital
Human Fibrinogen Concentrate (FGTW) in Pediatric Patients With Congenital Fibrinogen Deficiency
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.
- Trial name
- NCT02094430
- Testing
- biological: human fibrinogen concentrate
- Where
- 4 sites in France, Lebanon and 2 more
- Volunteers
- 16 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Pregnancy Complications, Pregnancy, High Risk
Active Pregnancy Against COVID-19
Historically and traditionally, the recommendations related to physical exercise during pregnancy have been based more on moral or cultural issues than on scientific evidence.
- Conditions
- Pregnancy Complications, Pregnancy, High Risk, Pregnancy Induced Hypertension, Newborn Morbidity
- Trial name
- ACPREGCOV (NCT04563065)
- Testing
- Exercise program, Healthy lifestyle advise
- Where
- 2 sites in Spain
- Volunteers
- 280 people, healthy volunteers welcome
- Sponsor
- Universidad Politecnica de Madrid
Lennox-Gastaut Syndrome
Transcranial Direct Current Stimulation, Treatment of Childhood Drug-Resistant Lennox-Gastaut Syndrome, A Pilot Study
Background: Lennox-Gastaut syndrome (LGS) is a severe childhood epileptic syndrome with high pharmacoresistance. The treatment outcomes are still unsatisfied.
- Trial name
- NCT02731300
- Testing
- tDCS
- Volunteers
- 22 people
- Sponsor
- Khon Kaen University
Microsatellite Stable (MSS) Colon Cancer, Locally Advanced Colon Cancer
Node-Sparing Short-Course Radiation With CAPOX and Sintilimab for MSS Locally Advanced Colon Cancer: A Randomized, Prospective, Multicenter Study
This study aims to evaluate the use of node-sparing short-course radiotherapy combined with chemotherapy and Sintilimab, or chemotherapy alone, as neoadjuvant therapy for MSS-type locally advanced colon cancer.
- Trial name
- mRCAT-C (NCT06594692)
- Testing
- Node-Sparing Short-Course Radiotherapy Combined with CAPOX and Sintilimab, CAPOX Chemotherapy
- Where
- 2 sites in China
- Volunteers
- 140 people
- Sponsor
- Sir Run Run Shaw Hospital
Hypophosphatasia (HPP)
Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)
This clinical trial studies the safety, tolerability, and pharmacology of asfotase alfa when given to adults with HPP.
- Trial name
- NCT00739505
- Testing
- Asfotase Alfa
- Where
- 3 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Preterm Birth Complication, Neonatal Morbidity and Mortality
Delayed Versus Immediate Cord Clamping in Preterm Birth
The goal of this study is to evaluate whether delayed cord clamping improves early neonatal outcomes compared with immediate clamping in preterm birth.
- Conditions
- Preterm Birth Complication, Neonatal Morbidity and Mortality, Cord Clamping, Delayed Cord Clamping
- Trial name
- PREM-CORD (NCT07699666)
- Testing
- Delayed Umbilical Cord Clamping, Immediate Umbilical Cord Clamping
- Where
- Tunisia
- Volunteers
- 200 people
- Sponsor
- Faculty of Medicine of Tunis
Hyperbilirubinemia, Neonatal
Effectiveness of Phototherapy With and Without Probiotics
Raised serum bilirubin levels can cause kernicterus and premature infants are at increased risk due to metabolic immaturity. The standard treatment for neonatal jaundice is phototherapy but probiotics along with it can reduce the…
- Trial name
- NCT06173856
- Testing
- LED phototherapy
- Where
- Pakistan
- Volunteers
- 76 people
- Sponsor
- Fatima Jinnah Medical University
Retained Fetal Lung Fluid
Accuracy of Amniotic Fluid Lamellar Body Counting for Evaluating of Fetal Lung Maturity
The purpose of this study is to determine the accuracy of Amniotic fluid Lamellar Body Counting for Evaluating of Fetal Lung Maturity.
- Trial name
- NCT01463722
- Testing
- Amniotic fluid Lamellar Body Counting
- Where
- Iran
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- Isfahan University of Medical Sciences
Surgery for Congenital Heart Disease, Pediatric Patients
The Predictability of Intraoperative Rotational Thromboelastometry on Postoperative Bleeding and Transfusion Requirements
Viscoelastic hemostatic assay has been reported to be superior to predict perioperative bleeding in cardiac surgical patients compared with conventional blood coagulation test.
- Trial name
- NCT02081222
- Testing
- surgery for congenital heart disease with cardiopulmonary bypass
- Where
- South Korea
- Volunteers
- 119 people
- Sponsor
- Samsung Medical Center
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Monoblastic Leukemia (M5a)
Decitabine Followed by Idarubicin and Cytarabine in Treating Patients With Relapsed or Refractory AML and MDS
The goals of this study are to learn about the effectiveness, the side-effects, if waiting to give the idarubicin and cytarabine may change the side effects or effectiveness, and to identify factors to predict for responses to…
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2)
- Trial name
- NCT01607645
- Testing
- decitabine, idarubicin, cytarabine
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Fred Hutchinson Cancer Center
Spinocerebellar Ataxia Type 2
Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Adult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose…
- Trial name
- NCT06672445
- Testing
- ARO-ATXN2 Injection
- Where
- 16 sites in Australia, Canada and 6 more
- Volunteers
- 39 people
- Sponsor
- Arrowhead Pharmaceuticals
Pulmonary Hypertension
Imatinib and Carvedilol for High Blood Pressure in the Lungs in Adults With Sickle Cell Disease
Background: About one-tenth of adults with sickle cell disease have pulmonary hypertension (high blood pressure in the lungs). This condition can cause shortness of breath, pain crisis, and congestive heart failure.
- Trial name
- NCT01568645
- Testing
- Carvedilol, Imatinib
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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