This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
- Investigator
- Cesar A Trillo-Alvarez
- Ages
- 18 Years - N/A
- Sexes
- All
Clinical trials
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4D-710-C001 -
DBS Severe ET Closed Loop This is a feasibility study based on physician-initiated Investigational Device Exemption (IDE) including intraoperative experiments and chronic testing of implanted dual thalamic DBS lead systems. This study will inform protocols for optimal use of…
- Ages
- 21 Years - 99 Years
- Sexes
- All
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Alpha/beta T cell and CD19+ B cell depletion in Allogeneic Stem Cell Transplantation This study will assess the safety, efficacy, and feasibility of ⍺/β CD3+ T-cell and CD19+ B-cell depletion in allogeneic stem cell transplantation in patients with acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), juvenile…
- Investigator
- Jordan Milner
- Ages
- 6 Months - 39 Years
- Sexes
- All
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Weekend steroids and exercise as therapy for DMD The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular dystrophy (DMD).…
- Ages
- 5 Years - 9 Years
- Sexes
- Male
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DIFI This study evaluates the efficacy and safety of a single injection of iltamiocel (300 x 10^6 cells) compared to a placebo in the reduction of fecal incontinence episode frequency in adult female participants with chronic fecal incontinence and a…
- Investigator
- Johan Nordenstam
- Ages
- 18 Years - N/A
- Sexes
- Female
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Walking and mobility decline in older adults (Mind in Motion) The objective of this study is to determine the central neural control of mobility in older adults by acquiring data with multiple modalities of neuroimaging (EEG, fNIRS, MRI) and associating these data with a comprehensive set of diverse mobility…
- Ages
- 20 Years - 110 Years
- Sexes
- All
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DREAM The overriding objective of DREAM is to conduct a prospective longitudinal (36 months) observational clinical study to investigate the incidence, etiology, and pathophysiology of diabetes mellitus (DM) following acute pancreatitis (AP).
- Investigator
- Chris Forsmark
- Ages
- 18 Years - 75 Years
- Sexes
- All
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A Study to Compare Treatment With the Drug Selumetinib Alone Versus Selumetinib and Vinblastine in P This phase III trial investigates the best dose of vinblastine in combination with selumetinib and the benefit of adding vinblastine to selumetinib compared to selumetinib alone in treating children and young adults with low-grade glioma (a common…
- Investigator
- William Slayton
- Ages
- 2 Years - 25 Years
- Sexes
- All
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D1AMOND This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults in the treatment of Tourette's Disorder (TD). The study includes an open-label period followed by…
- Investigator
- Irene A Malaty
- Ages
- 6 Years - N/A
- Sexes
- All
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Dysfunctional Myelopoiesis and Myeloid-Derived Suppressor Cells in Sepsis Pathobiology - Investigator
- Philip A Efron