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NCT ID: NCT04443907 Active, not recruiting - Sickle Cell Disease Clinical Trials

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

Start date: August 26, 2020
Phase: Phase 1
Study type: Interventional

This study is evaluating a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

NCT ID: NCT04443153 Active, not recruiting - Clinical trials for Diabetes Mellitus, Type 1

Adapting Diabetes Treatment Expert Systems to Patient in Type 1 Diabetes

DSS-2
Start date: September 4, 2020
Phase: N/A
Study type: Interventional

This study is evaluate the superior efficacy of a Continuous Glucose Monitor (CGM)-based advisory system in Type 1 Diabetes Mellitus (T1DM), as compared to Sensor Augmented Mode (SAM) therapy, and with characterizing the impact of psycho-behavioral factors on system performance, which will enable system individualization and lead to automated adaptation of advice delivery to optimize glycemic control and reduce the system's psychological impact.

NCT ID: NCT04442347 Active, not recruiting - Clinical trials for Ornithine Transcarbamylase Deficiency

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

Start date: November 3, 2020
Phase: Phase 1
Study type: Interventional

Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in clinically stable patients (stable on standard of care treatment, e.g. diet ± ammonia scavengers) with ornithine transcarbamylase deficiency (OTCD).

NCT ID: NCT04441905 Active, not recruiting - Clinical trials for Chikungunya Virus Infection

Phase 1 Study of SAR440894 vs Placebo

Start date: October 14, 2020
Phase: Phase 1
Study type: Interventional

A single, ascending-dose design with five dose-cohorts of 8 subjects. Forty healthy adults aged 18 to 45, inclusive, will be recruited and admitted at multiple sites. Each subject will be randomized to receive either SAR440894 or matching placebo via 60-minute intravenous infusion. In each cohort of 8 subjects, the randomization ratio will be 6 active to 2 placebo, and 2 sentinel subjects (one from each active and placebo group) will be dosed first. Dosing of the next dose-cohort will be dependent on acceptable meeting predefined safety criteria in the preceding cohort. Each subject's participation will take place over approximately 150 days, not including the screening visit. There are no hypotheses for this phase I study. The primary objective will be to determine the safety of single ascending intravenous (IV) infusions of SAR440894 when administered in healthy adults.

NCT ID: NCT04441749 Active, not recruiting - Clinical trials for Diagnosis of Peripheral Lung Nodules

nCLE For Diagnosis Of Peripheral Lung Nodules By Robotic Navigational Bronchoscopy

Start date: June 29, 2020
Phase:
Study type: Observational

Needle based confocal laser endomicroscopy (nCLE) employs a small fiber which can be passed through a biopsy needle to enable real time microscopic imaging of cells. With resolution of 3.5 microns it is possible to identify key features consistent with malignancy and pulmonary fibrosis. (Wijmans, et al., 2019). The effectiveness of nCLE with intravenous fluorescein has been demonstrated in central lung cancers using esophageal ultrasound guided needle placement (Wijmans, et al., 2019). In this study, the proposed nCLE lung cancer criteria had high accuracy for lung cancer detection and were consistently recognized by multiple raters. Probe based confocal microscopy (pCLE) has also been used in conjunction with a radial probe EBUS in evaluating solitary pulmonary nodules with a diagnostic accuracy of 79.2%. (Hassan, et al., 2017) Needle based confocal laser endomicroscopy (nCLE) has not previously been used in conjunction with robotic navigational bronchoscopy.

NCT ID: NCT04439968 Active, not recruiting - Cerebral Hypoxia Clinical Trials

Neonatal Brain Oxygenation Study

BOX
Start date: August 10, 2021
Phase: N/A
Study type: Interventional

Implementing target ranges for regional cerebral saturations in extremely preterm infants in the first week of life may improve neurodevelopmental outcomes at 22-26 months corrected age compared to those without targeted cerebral saturations (Csat) using near-infrared spectroscopy (NIRS). Infants will be randomized to a targeted cerebral saturation monitoring group with visible reading of Csat or to a control group with cerebral saturation monitoring, but with blinded Csat measures. Those in the targeted Csat group will follow a treatment guideline to maintain cerebral oxygenation in the target range. The primary outcome is neurodevelopmental outcome as determined by Bayley III cognitive scale score.

NCT ID: NCT04439357 Active, not recruiting - Clinical trials for Hematopoietic and Lymphoid Cell Neoplasm

Testing Trametinib as a Potential Targeted Treatment in Cancers With GNAQ or GNA11 Genetic Changes (MATCH-Subprotocol S2)

Start date: February 25, 2016
Phase: Phase 2
Study type: Interventional

This phase II MATCH treatment trial identifies the effects of trametinib in patients whose cancer has genetic changes called GNAQ or GNA11 mutations. Trametinib may block proteins called MEK1 and MEK2, which may be needed for cancer cell growth when GNAQ or GNA11 mutations are present. Researchers hope to learn if trametinib will shrink this type of cancer or stop its growth.

NCT ID: NCT04439344 Active, not recruiting - Clinical trials for Hematopoietic and Lymphoid Cell Neoplasm

Testing Binimetinib as a Potential Targeted Treatment in Cancers With NRAS Genetic Changes (MATCH-Subprotocol Z1A)

Start date: May 31, 2016
Phase: Phase 2
Study type: Interventional

This phase II MATCH treatment trial investigates the good and bad effects of binimetinib in patients whose cancer has a genetic change called NRAS mutation. Binimetinib blocks proteins called MEK1 and MEK2, which may be needed for cancer cell growth when an NRAS mutation is present. Researchers hope to learn if binimetinib will shrink this type of cancer or stop its growth.

NCT ID: NCT04439331 Active, not recruiting - Clinical trials for Hematopoietic and Lymphoid Cell Neoplasm

Testing VS-6063 (Defactinib) as a Potential Targeted Treatment in Cancers With NF2 Genetic Changes (MATCH-Subprotocol U)

Start date: August 12, 2015
Phase: Phase 2
Study type: Interventional

This phase II MATCH treatment trial identifies the effects of VS-6063 (defactinib) in patients whose cancer has a genetic change called NF2 mutation. Defactinib may block a protein called FAK, which may be needed for cancer cell growth when NF2 mutations are present. Researchers hope to learn if defactinib will shrink this type of cancer or stop its growth.

NCT ID: NCT04439318 Active, not recruiting - Clinical trials for Hematopoietic and Lymphoid Cell Neoplasm

Testing Trametinib as a Potential Targeted Treatment in Cancers With NF1 Genetic Changes (MATCH-Subprotocol S1)

Start date: February 25, 2016
Phase: Phase 2
Study type: Interventional

This phase II MATCH treatment trial identifies the effects of trametinib in patients whose cancer has a has a genetic change called NF1 mutation. Trametinib blocks proteins called MEK1 and MEK2, which may be needed for cancer cell growth when an NF1 mutation is present. Researchers hope to learn if trametinib will shrink this type of cancer or stop its growth.