Clinical trials: Birth Defect
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Biliary Atresia, Kasai Portoenterostomy Status
Clinical Study About the Role of COX-2 Inhibitor in Liver Cirrhosis With Biliary Atresia
In this clinical study, meloxicam will be used to the patients who are older than 2 years and underwent Kasai portoenterostomy to treat their biliary atresia before.
- Trial name
- NCT02298218
- Testing
- Meloxicam
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Yonsei University
Hemophilia A
Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A
This study is a multi-center, open-label, single-arm trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (≥12 years old) with severe hemophilia A who…
- Trial name
- NCT03815318
- Testing
- Recombinant Human Coagulation FVIII
- Where
- China
- Volunteers
- 73 people
- Sponsor
- Sinocelltech Ltd.
Congenital Heart Disease
Inhaled NO as an Anti-inflammatory and Anti-reperfusion Agent in Infants and Children Undergoing Cardiopulmonary Bypass
Each year, there are over 400,000 cardiac surgical operations performed in the United States; of which 10,000 are performed on children.
- Trial name
- NCT00585013
- Testing
- Nitric Oxide
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Washington University School of Medicine
Hallux Rigidus, Unspecified Foot
Radiological Stage and Efficacy of an Intra-articular Injection of Hyaluronic Acid for the Treatment of Hallux Rigidus
The hallux rigidus is the most frequent type of arthrosis at the age of 50 and beyond. For the moderate forms of the disease, the majority of the studies recommend the recourse to the viscosupplement because of its efficiency in…
- Trial name
- REPAR (NCT03622723)
- Where
- France
- Volunteers
- 65 people
- Sponsor
- Labrha
Hemochromatosis, Cerebrovascular Accident
Stroke With Transfusions Changing to Hydroxyurea
The purpose of this study is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iron overload in children with…
- Conditions
- Hemochromatosis, Cerebrovascular Accident, Anemia, Sickle Cell, Hematologic Diseases
- Trial name
- NCT00122980
- Testing
- Red Cell Transfusions, Iron Chelation, Hydroxyurea
- Where
- 28 sites in United States
- Volunteers
- 134 people
- Sponsor
- St. Jude Children's Research Hospital
Acute Respiratory Tract Infections, Diarrhea
Home Based Child Care to Reduce Mortality and Malnutrition in Tribal Children of Melghat, India: CRCT
Melghat is poorly developed tribal area in India with very high child mortality \& malnutrition prevalence (grossly inadequate medical facilities). Important health problems.
- Conditions
- Acute Respiratory Tract Infections, Diarrhea, Malaria, Neonatal Sepsis
- Trial name
- HBCC (NCT02473796)
- Testing
- Home based child care
- Where
- India
- Volunteers
- 7,594 people
- Sponsor
- MAHAN Trust
Sickle Cell Disease, Thalassemia
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who…
- Conditions
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy
- Trial name
- NCT03367546
- Testing
- Blood and Marrow Transplant
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Anophthalmia, Microphthalmia
National Cohort on Congenital Defects of the Eye
Congenital malformations of the eye comprise various developmental defects including microphthalmia, anophthalmia, aniridia, and anterior segment anomalies (such as Peters and Axenfeld-Rieger anomalies).
- Conditions
- Anophthalmia, Microphthalmia, Aniridia, Anterior Segment Dysgenesis 6, Peters Anomaly Subtype
- Trial name
- RaDiCoACOEIL (NCT05954403)
- Where
- France
- Volunteers
- 800 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Periventricular Leukomalacia, White Matter Disease
Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia
The goal of this study is to explore a new treatment that may help repair brain damage in individuals with periventricular leukomalacia (PVL), a condition that affects white matter in the brain.
- Conditions
- Periventricular Leukomalacia, White Matter Disease, Hypoxic-Ischemic Encephalopathy, Demyelinating Diseases
- Trial name
- NCT06985303
- Testing
- Investigational Combination Product
- Sponsor
- MGAM LLC
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Deformities Around the Knee in Pedietrics
Correction of Multiplanar Deformities Around the Knee With Monolateral External Fixator
Angular deformities around the knee are one of the common cause of presentation at pediatric orthopedic clinic. Although these conditions are commonly benign and self-limiting.
- Trial name
- NCT05331677
- Testing
- correction of deformities around the knee with external fixator
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Sohag University
Single-ventricle
Fibrosis and the Fontan
The purpose of this study is to non-invasively characterize the fibrotic consequences of single ventricle physiology, its possible solution and effect on lymphatics.
- Trial name
- NCT04901975
- Testing
- Spironolactone
- Where
- United States
- Volunteers
- 98 people
- Sponsor
- Children's Hospital of Philadelphia
Prader-Willi Syndrome
Probiotic Treatment for Prader-Willi Syndrome
A whole new research area studying the function of intestinal microorganisms, also known as gut microbiota, has emerged during the last decade.
- Trial name
- NCT04685057
- Testing
- Probiotic, Follow-up probiotic
- Where
- Spain
- Volunteers
- 41 people
- Sponsor
- Fundació Sant Joan de Déu
Eye Abnormalities
Modeling Ocular Developmental Diseases From 3D Cultures of Optic Vesicle Organoids Derived From hiPSCs of Patients With Ocular Malformations
Ocular morphogenesis is a complex process starting as early as the 4th week of embryonic life, involving interactions between varioustissues of different origin and conserved genes.
- Trial name
- OrganoEye (NCT06408701)
- Testing
- Blood sampling
- Where
- 2 sites in France
- Volunteers
- 20 people
- Sponsor
- University Hospital, Toulouse
DCM - Dilated Cardiomyopathy
Observational Study for Patients With Dilated Cardiomyopathy
Observational study on patients with dilated cardiomyopathy aims to investigate the correlation between cardiac fibrosis, as indicated by cardiac magnetic resonance, and the prognosis of patients with dilated cardiomyopathy, and…
- Trial name
- NCT04837612
- Testing
- No intervention nor exposure
- Where
- China
- Volunteers
- 500 people
- Sponsor
- Guangdong Provincial People's Hospital
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
MELAS Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of…
- Trial name
- NCT06644534
- Testing
- TTI-0102
- Where
- 2 sites in France, Netherlands
- Volunteers
- 9 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Club Foot
Low Dye Taping Technique Versus Robert Debre Method On Foot Posture And Range Of Motion In Children With Club Foot.
To Check the effects of the low dye taping technique versus the Robert Debre method on foot posture and range of motion in children with club feet.
- Trial name
- NCT05794334
- Testing
- Low Dye Taping
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- Riphah International University
Laryngomalacia
Laryngomalacia No Longer An Ambiguity
The purpose of this study is to expose some of the factors that may be associated or predisposing to development of laryngomalacia.
- Trial name
- NCT00972764
- Where
- Kuwait
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- Boushahri Clinic Medical Center
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