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NCT ID: NCT04671433 Active, not recruiting - Clinical trials for X-Linked Retinitis Pigmentosa

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Start date: December 4, 2020
Phase: Phase 3
Study type: Interventional

A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

NCT ID: NCT04670679 Active, not recruiting - Clinical trials for Advanced or Metastatic Solid Tumors

A Dose Escalation/Expansion Study of ERAS-601 in Patients With Advanced or Metastatic Solid Tumors

FLAGSHP-1
Start date: December 15, 2020
Phase: Phase 1
Study type: Interventional

- To evaluate the safety and tolerability of escalating doses of ERAS-601 when administered as a monotherapy and in combination with other cancer therapies in study participants with advanced or metastatic solid tumors. - To determine the Maximum Tolerated Dose (MTD) and/or recommended dose (RD) of ERAS-601 when administered as a monotherapy and in combination with other cancer therapies. - To characterize the pharmacokinetic (PK) profile of ERAS-601 when administered as a monotherapy and in combination with other cancer therapies. - To evaluate the antitumor activity of ERAS-601 when administered as a monotherapy and in combination with other cancer therapies.

NCT ID: NCT04670445 Active, not recruiting - Clinical trials for Stage IV Breast Cancer

Improving Patient and Caregiver Understanding of Risks and Benefits of Immunotherapy for Advanced Cancer

UPLIFT
Start date: January 23, 2021
Phase: N/A
Study type: Interventional

The purpose of this study is to refine and pilot test educational material developed to educate and support patients receiving immunotherapy for advanced cancer. The intervention is an educational video and question prompt list (QPL) to promote communication between patients, caregivers, and the oncology team about the risks and benefits of immunotherapy.

NCT ID: NCT04669743 Active, not recruiting - Clinical trials for Pulmonary Disease, Chronic Obstructive

Innate Immunity in Ozone-induced Airway Inflammation in COPD

CO3PD
Start date: April 7, 2016
Phase: N/A
Study type: Interventional

Chronic obstructive pulmonary disease (COPD) is the third leading cause of death in the United States. Patients with COPD are routinely exposed to indoor and outdoor air pollution, which appears to cause escalation of their respiratory symptoms, a process called exacerbation, with resulting need to seek medical attention. This research plan proposes to evaluate the impact of lung immune cells in susceptibility to develop exacerbation through an experimental model of inhalational exposure using ambient levels of a component of air pollution (ozone) in COPD patients and longitudinal sampling of their lung immune cells.

NCT ID: NCT04669535 Active, not recruiting - Sandhoff Disease Clinical Trials

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

Start date: January 15, 2021
Phase: Phase 1
Study type: Interventional

The AXO-GM2-001 study is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of a bilateral thalamic and intracisternal/intrathecal infusion of AXO-AAV-GM2 in pediatric participants with GM2 Gangliosidosis (also known as Tay-Sachs or Sandhoff Diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the β-hexosaminidase A (HexA) enzyme. AXO-AAV-GM2 is an investigational gene therapy that aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via co-administration of two vectors utilizing the neurotropic adeno-associated virus recombinant human 8 serotype (AAVrh.8) capsid carrying the human HEXA or HEXB cDNA. The trial is expected to enroll pediatric participants with Tay-Sachs or Sandhoff Diseases, where infantile-onset participants will range from 6 months to 20 months old, and juvenile-onset participants will range from 2 years to 12 years old.

NCT ID: NCT04669444 Active, not recruiting - Critical Illness Clinical Trials

Biomarkers, Genomics, Physiology in Critically Ill and ECMO Patients

IGNITE
Start date: April 14, 2020
Phase: N/A
Study type: Interventional

Patients in end-stage cardiac failure and/or respiratory failure may be started on a rescue therapy known as Extracorporeal Membrane Oxygenation (ECMO). One of the major clinical questions is how to manage the ventilator when patients are on ECMO therapy. Ventilator Induced Lung Injury (VILI) can result from aggressive ventilation of the lung during critical illness. VILI and lung injury such as Acute Respiratory Distress Syndrome (ARDS) can further increase the total body inflammation and stress, this is known as biotrauma. Biotrauma is one of the mechanisms that causes multi-organ failure in critically ill patients. One advantage of ECMO is the ability to greatly reduce the use of the ventilator and thus VILI by taking control of the patient's oxygenation and acid-base status. By minimizing VILI during ECMO we can reduce biotrauma and thus multi-organ failure. Since the optimal ventilator settings for ECMO patients are not known, we plan to study the impact of different ventilator settings during ECMO on patient's physiology and biomarkers of inflammation and injury.

NCT ID: NCT04669197 Active, not recruiting - Clinical trials for Locally Advanced Pancreatic Adenocarcinoma

Study of Paclitaxel Protein Bound + Gemcitabine + Cisplatin + Hydrochloroquine as Treatment in Untreated Pancreas Cancer

Start date: December 1, 2020
Phase: Phase 2
Study type: Interventional

To evaluate the normalization rate of CA 19-9 of individuals with non-metastatic pancreas cancer following up to 6 months of neoadjuvant chemotherapy.

NCT ID: NCT04669158 Active, not recruiting - Clinical trials for Non Alcoholic Steatohepatitis

Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis

Start date: July 30, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

This is a randomized, placebo-controlled phase 1/2a study to investigate the safety and tolerability of Idebenone in patients 18 years of age or older with non-alcoholic steatohepatitis, with stage 1-3 fibrosis. As secondary end point target engagement and fibrosis improvement will be assessed.

NCT ID: NCT04669067 Active, not recruiting - Clinical trials for Acute Myeloid Leukemia

TL-895 and KRT-232 Study in Acute Myeloid Leukemia

Start date: March 31, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

This study evaluates TL-895, a potent, orally available and highly selective irreversible tyrosine kinase inhibitor combined with navtemadlin (KRT-232), a novel oral small molecule inhibitor of MDM2 for the treatment of adults with FLT3 mutated Acute Myeloid Leukemia. Participants must be relapsed/refractory (e.g., having failed prior therapy) to be eligible for this study.

NCT ID: NCT04668885 Active, not recruiting - Clinical trials for Myelodysplastic Syndromes

CPX-351 as a Novel Approach for the Treatment of Older Patients With AML and MDS

Start date: January 14, 2021
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate how effective lower doses of CPX-351 are in older participants with relapsed/refractory acute myeloid leukemia (AML) who are not eligible to receive intensive chemotherapy and in participants with myelodysplastic syndromes (MDS) after Hypomethylating Agents (HMA) failure.