Clinical trials: Congenital Malformation
Myelomeningocele
Cellular Therapy for In Utero Repair of Myelomeningocele - The CuRe Trial
Spina bifida, or myelomeningocele (MMC), is a birth defect that results in paralysis, excess fluid on the brain (hydrocephalus), and impaired ability to urinate and have bowel movements normally.
- Trial name
- CuRe (NCT04652908)
- Testing
- Placental Mesenchymal Stem Cells seeded on a commercially available dural graft extracellular matrix, Untreated contemporaneous cohort
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- University of California, Davis
Haemophilia B
A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic…
- Trial name
- B-SURE (NCT03655340)
- Testing
- Alprolix
- Where
- 18 sites in France
- Volunteers
- 91 people
- Sponsor
- Swedish Orphan Biovitrum
Atopic Dermatitis
Oregano Ointment vs. Standard Treatment for Pediatric Atopic Dermatitis
This study is designed to evaluate and compare the efficacy of 3% oregano extract ointment prepared in aqueous solution versus 1% hydrocortisone ointment, a standard treatment, in decreasing the inflammation associated with mild…
- Trial name
- NCT02289989
- Testing
- Oregano extract cream, Hydrocortisone
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Rutgers, The State University of New Jersey
Retinitis Pigmentosa, Maculopathy, Age Related
High Resolution, High-speed Multimodal Ophthalmic Imaging
Knowledge of the pathogenesis of ocular conditions, a leading cause of blindness, has benefited greatly from recent advances in ophthalmic imaging.
- Conditions
- Retinitis Pigmentosa, Maculopathy, Age Related, Macular Dystrophy, Macular Edema
- Trial name
- IMA-MODE (NCT04129021)
- Testing
- High-resolution retinal imaging through adaptive optics, High-resolution retinal imaging through holographic systems
- Where
- France
- Volunteers
- 1,200 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Leukemia, Lymphoma
Alemtuzumab Plus Fludarabine and Melphalan With or Without Cyclosporine, Mycophenolate Mofetil, and Low-Dose Total-Body Irradiation Therapy Followed by Donor Peripheral Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, monoclonal antibodies, and radiation therapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00085449
- Testing
- alemtuzumab, cyclosporine, fludarabine phosphate
- Where
- 24 sites in United States
- Sponsor
- Alliance for Clinical Trials in Oncology
MPS IVA, MPS VI
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI.
- Trial name
- NCT05845749
- Testing
- Vosoritide Injection [Voxzogo]
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease
A Pilot Study of Fecal Microbiome and Neutrophil Cellular Adhesion Molecules in Patients With Sickle Cell Disease (SCD)
Patients with sickle cell disease often develop painful crisis without any obvious reasons. Some patients are more likely to develop this complication than others.
- Trial name
- NCT03007849
- Volunteers
- 50 people
- Sponsor
- Rhode Island Hospital
Dermatitis, Atopic
Tacrolimus Ointment Long Term Safety in Young Children With Atopic Dermatitis
The objective of this study is to assess the safety and efficacy of 0.03% tacrolimus ointment as long-term treatment in paediatric patients with atopic dermatitis.
- Trial name
- NCT00560326
- Testing
- Tacrolimus Ointment 0.03%
- Where
- 8 sites in Canada, Finland and 3 more
- Volunteers
- 50 people
- Sponsor
- Astellas Pharma Inc
Age-Related Osteoporosis
Genetic Regulators of Bone Health That Are Unique to Vertebral Bone
Osteoporosis is an age related disease in which a person's bone slowly becomes weaker with time. The bones may become so weak that they break easily such as a fall from standing height.
- Trial name
- NCT05946278
- Testing
- Sample collection for gene expression
- Where
- United States
- Volunteers
- 550 people
- Sponsor
- University of Colorado, Denver
Alpha-Mannosidosis
Lamazym Aftercare Study
The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial.
- Trial name
- rhLAMAN-09 (NCT01908725)
- Testing
- Lamazym
- Where
- Denmark
- Volunteers
- 8 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Atrial Fibrillation, Tachycardia, Ventricular
Ajmaline Utilization in the Diagnosis and Treatment of Cardiac Arrhythmias
The study evaluates 3 different populations: It is an open, randomized, parallel-group study comparing the effectiveness of intravenous (iv) ajmaline with currently used antiarrhythmic drugs in the acute treatment of : 1.
- Conditions
- Atrial Fibrillation, Tachycardia, Ventricular, Brugada Syndrome
- Trial name
- AJUAR (NCT00702117)
- Testing
- flecainide, ajmaline, procainamide
- Where
- 4 sites in Spain
- Volunteers
- 123 people
- Sponsor
- Hospital Clinic of Barcelona
Univentricular Heart
Aesculus Hippocastanum L. on Fontan Circulation
Patients after the Fontan operation at a single institution will be randomized to receive a standardized extract of Aesculus hippocastanum L. (horse chestnut) or placebo for 4 months in a crossover clinical trial.
- Trial name
- NCT04035317
- Testing
- Aesculus Hippocastanum / Horse Chestnut
- Where
- Brazil
- Sponsor
- Casa Espirita Terra de Ismael
Sickle Cell Disease
Collect of Cord Blood From Subjects at Risk for Sickle Cell Disease, for the Purpose of Laboratory Research
The study consists in collecting umbilical cord blood cells from newborns at risk of sickle cell disease, to perform laboratory experiments aiming to characterize the cells with HbS/HbS mutation, to develop methods to prepare, to…
- Trial name
- DREPACORD (NCT03876821)
- Where
- France
- Volunteers
- 44 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Venous Malformation, Pain
A Study of Enalapril in Treatment of Venous Malformations
The goal of this clinical trial is to explore if enalapril can be used to treat painful venous malformations. The main question it aims to answer are: Can enalapril reduce pain and volume of the malformation and increase quality…
- Conditions
- Venous Malformation, Pain, Quality of Life, Vascular Malformation
- Trial name
- EVA (NCT06788314)
- Testing
- Enalapril
- Where
- Norway
- Volunteers
- 12 people
- Sponsor
- Oslo University Hospital
Social Behaviour, Brain Neoplasms
Social Cognition in Children Treated for a Brain Tumour
There is ample evidence that children treated for a brain tumour (BT) often develop deficits in social and emotional functioning. The investigators wish to examine the cause of these deficits, i.e.
- Conditions
- Social Behaviour, Brain Neoplasms, Cystic Fibrosis
- Trial name
- NCT01599052
- Where
- 4 sites in Belgium, Netherlands
- Volunteers
- 152 people, healthy volunteers welcome
- Sponsor
- University Medical Center Groningen
Heart Defects, Congenital
DEX vs SEVO in Congenital Heart Surgery
Anesthesia-related neurotoxicity in the developing brain is still a concern although evidence in humans is debatable. Moreover, it is unclear whether repeated and/or prolonged exposures are harmless and whether their effects are…
- Trial name
- DEXLOSNeuro (NCT05369949)
- Testing
- DEX group, Control group
- Volunteers
- 150 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Duchenne Muscular Dystrophy, Cardiomyopathy
HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age.
- Trial name
- HOPE (NCT02485938)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Capricor Inc.
Vaso-occlusive Pain Episodes
Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial
Vaso-occlusion contributes significantly to morbidity in sickle cell disease (SCD). Vaso-occlusive painful episodes (VOE) are common and debilitating, causing the majority of emergency department visits.
- Trial name
- NCT01796678
- Testing
- Arginine
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Hemophilia A and B
Comparison of 30-Second Sit-to-Stand Test Performance Between Individuals With Hemophilia and Healthy Peers
Hemophilia is an inherited bleeding disorder characterized by recurrent musculoskeletal bleeding, particularly into joints, leading to impaired physical function and reduced functional performance.
- Trial name
- NCT07736976
- Where
- Turkey (Türkiye)
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Bahçeşehir University
Congenital Heart Disease
Effect of Feeding With Nutrient Dense Formula in Malnourished Infants and Children With Congenital Heart Defects
Infants with congenital heart disease (CHD) are usually born with a normal weight but develop malnutrition over time, and about 20-50% of these children are usually malnourished. Malnutrition in these patients is multifactorial.
- Trial name
- NCT04795076
- Testing
- Infant formula
- Where
- Iran
- Volunteers
- 65 people
- Sponsor
- Shahid Beheshti University
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