Found 4304 clinical trials
Safety Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in healthy adult subjects.
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- 19 Feb, 2024
AAV Gene Therapy Study for Subjects With PKU
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
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- 19 Feb, 2024
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency. The study consists of a 6 month randomized, double blind, placebo-controlled period, followed by 1 year of …
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- 19 Feb, 2024
Gene Therapy for X Linked Severe Combined Immunodeficiency
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCIDsevere combined immune deficiency .The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number …
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- 19 Feb, 2024
A Multinational Randomized Double-blind Placebo-controlled Study to Assess the Efficacy Pharmacodynamics Pharmacokinetics and Safety of Venglustat in Late-onset GM2
Primary Objectives: Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period Secondary population (participants with juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile/adult galactosialidosis): To assess …
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- 19 Feb, 2024
- 2 locations
Determinants of Neonatal Anemia in Women Carrying Multiples
Recent data indicate that multiple births now comprise 3-4.5% of all births in the United States. Pregnant women are at risk for iron (Fe) deficiency anemia yet there are virtually no data on Fe status in women carrying multiples and current recommendations do not necessitate Fe screening among this high …
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- 10 Jan, 2025
- 2 locations
Tissue-engineered Skin Graft Repair of Autologous Scar Dermal Scaffolds
It affects the appearance and some scar contracture often leads to joint dysfunction.Patients have low quality of life, long treatment cycle, heavy social burden and high medical costs.Skin grafting is currently the gold standard for scar repair.However, there are often insufficient skin sources, easy to scar recurrence, lack of skin …
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- 19 Feb, 2024
Do We Need to Taper Down Steroid Therapy for Bell's Palsy
Tapering must be done carefully to avoid both recurrent activity of the underlying disease and possible cortisol deficiency resulting from hypothalamic-pituitary-adrenal axis (HPA) suppression. However, according to a review by Furst et al (2019), a patient who has received any dose of glucocorticoid for less than 3 weeks or patients …
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- 19 Feb, 2024
Effect of Preoperative Intervention With Folic Acid and Vitamin B12 on Postoperative Neurobehavioral Changes in Children
Comparison of preoperative folic acid and VitB12 intervention on postoperative delirium and long-term neurobehavioral changes in children under general anesthesia
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- 19 Feb, 2024
A Trial of Traffic Light Labeling With Behavioral Nudges and a Healthy Recipe Database to Increase Selection of Healthier Foods in Client-choice Food Pantries
This study is a pilot evaluation of the Healthy Pantry Program, a new behavioral economics-based training that allows pantry staff to learn how to implement nudges integrating traffic-light nutrition labeling and a healthy recipe database in the pantry environment. The hypothesis is that participation the Healthy Pantry Program will lead …
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- 19 Feb, 2024