Found 7803 clinical trials
Gene Therapy for Fanconi Anemia Complementation Group A
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the …
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- 19 Feb, 2024
- 1 location
Efficacy of Near-Infrared Vein Imaging for Difficult IV Placement
The objective of this project is to define the effectiveness and therefore the role of NIR vein finders in adult patients with difficult peripheral venous access. The specific objective of the proposed randomized controlled trial is to test the clinical success rate of placing peripheral venous catheters in 'difficult' access …
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- 19 Feb, 2024
- 1 location
Lap-assisted vs. US-Guided Visualization of TAP Blocks
Peripheral nerve blocks have been effective in decreasing post-operative pain as well as the use of narcotics for numerous years. Typically, these blocks are placed by anesthesiologist via ultrasound. In more recent years, surgeons have been placing nerve blocks laparoscopically. Since there are few studies that looks compare the two …
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- 19 Feb, 2024
- 1 location
Topotecan Episcleral Plaque for Treatment of Retinoblastoma
This single site, single-arm, non-randomized, dose escalation phase I toxicity clinical trial will assess primarily the safety and secondarily the efficacy of episcleral topotecan in patients with active residual or recurrent intraocular retinoblastoma in at least one eye following completion of first-line therapy.
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- 19 Feb, 2024
- 1 location
BrainWeighve App Feasibility Study- Fahima
Childhood obesity within the United States has been a growing concern over the past number of years, and if not addressed, leads to detrimental health outcomes for youth as they move into adulthood. The literature suggests that over-eating, especially when framed in terms of food addiction, plays a key role …
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- 24 Apr, 2026
- 1 location
Rapid Analysis and Response Evaluation of Combination Anti-Neoplastic Agents in Rare Tumors (RARE CANCER) Trial: RARE 1 Nilotinib and Paclitaxel
Background People with rare cancers often have limited treatment options. The biology of rare cancers is not well understood. Researchers want to find better treatments for these cancers. They want to test 2 drugs that, taken separately, have helped people with non-rare cancers. They want to see if these drugs …
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- 19 Feb, 2024
- 1 location
Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome
This is a prospective, open-label, randomized, two-arm clinical trial conducted to evaluate the safety and efficacy of romiplostim in comparison with eltrombopag in the treatment of thrombocytopenia in patients with Wiskott-Aldrich syndrome
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- 19 Feb, 2024
- 1 location
Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3
Cohort 3 Evaluate the efficacy of venglustat in combination with Cerezyme in adult and pediatric GD3 patients by assessing: CSF Lyso-GL1 levels Modified Friedreichs Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Brain resting-state functional Magnetic Resonance Imaging (rs-fMRI) reflecting connectivity between parieto-occipital areas Bone disease manifestations Evaluate safety …
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- 19 Feb, 2024
- 7 locations
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
Primary Objective: To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objective: To characterize the safety and tolerability To characterize the pharmacokinetics (PK)
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- 19 Feb, 2024
- 11 locations
Study of a Pneumococcal Conjugate Vaccine When Administered Concomitantly With Routine Pediatric Vaccines in Healthy Toddlers and Infants
To assess the immune response (serotype specific IgG concentration) of the SP0202 formulations and Prevnar 13 1 month after the administration of one dose in toddlers (Groups 1-4) To assess the immune response (serotype specific IgG concentration) of the SP0202 formulations and Prevnar 13 1 month after the administration of …
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- 19 Feb, 2024
- 32 locations