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NCT ID: NCT04093349 Active, not recruiting - Pompe Disease Clinical Trials

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Start date: October 1, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

NCT ID: NCT04091386 Active, not recruiting - Hemophilia A Clinical Trials

Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study)

LIFE-ACTIVE
Start date: June 10, 2021
Phase:
Study type: Observational

In this study researcher want to learn more about the change of physical activity of hemophilia A patients treated with damoctocog alfa pegol. Patients enrolled in this study will be wearing a small device measuring daily active time. Researcher will compare these physical activity data with patient's clinical data including bleeding events to gain information on how bleeding levels are related to the activity level of the patients. The study aims to enroll about 80 patients who are at the same time also participating in the HEM-POWR study (NCT03932201) evaluating the effectiveness and safety of damoctocog alfa pegol.

NCT ID: NCT04091048 Active, not recruiting - Clinical trials for Symptomatic Aortic Stenosis

Optimize PRO Study

Start date: September 16, 2019
Phase:
Study type: Observational

The purpose of this study is to collect clinical evidence on valve performance and procedural outcomes associated with an "optimized" TAVR care pathway and post-TAVR conduction disturbance pathway while using the Evolutâ„¢ PRO and Evolutâ„¢ PRO+ devices. The purpose of the addendum is to collect post-market clinical evidence on valve performance and procedural outcomes associated with the Evolut FX Device.

NCT ID: NCT04088409 Active, not recruiting - Uveitis Clinical Trials

A Study of Baricitinib (LY3009104) in Participants From 2 Years to Less Than 18 Years Old With Active JIA-Associated Uveitis or Chronic Anterior Antinuclear Antibody-Positive Uveitis

Start date: October 16, 2019
Phase: Phase 3
Study type: Interventional

The reason for this study is to see if the study drug baricitinib given orally is safe and effective in participants with active juvenile idiopathic arthritis (JIA)-associated uveitis or chronic anterior antinuclear antibody-positive uveitis from 2 years to less than 18 years old.

NCT ID: NCT04086264 Active, not recruiting - Clinical trials for Acute Myeloid Leukemia

IMGN632 as Monotherapy or With Venetoclax and/or Azacitidine for Participants With CD123-Positive Acute Myeloid Leukemia

Start date: November 6, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

This is an open-label, multicenter, Phase 1b/2 study to determine the safety and tolerability of IMGN632 and assess the antileukemia activity of IMGN632 when administered in combination with azacitidine and/or venetoclax in participants with relapsed and frontline CD123-positive AML.

NCT ID: NCT04083976 Active, not recruiting - Clinical trials for Advanced Solid Tumor

A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations

RAGNAR
Start date: November 20, 2019
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) in adult and pediatric participants with advanced solid tumors with fibroblast growth factor receptor (FGFR) alterations (mutations or gene fusions). It will also evaluate ORR in pediatric participants with advanced solid tumors and FGFR alterations.

NCT ID: NCT04083781 Active, not recruiting - Clinical trials for Haemophilia A With Inhibitors

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors

explorer7
Start date: October 21, 2019
Phase: Phase 3
Study type: Interventional

This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B with inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group, participants will get study medicine from the start of the study. In the other group, participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will get 1 injection with the study medicine every day under the skin. This participants will have to do themselves and can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for about six years. The length of time the participants will be in the study depends on when they agreed to take part or when the medicine is available for purchase in their country (12 November 2025 at the latest). Participants will have to come to the clinic for up to 41 times. The time between visits will be approximately 4 weeks for the first 6 to 12 months, depending on the group participants are in and approximately 8 weeks for the rest of the study. Participants will be asked to record information into an electronic diary during the study and may also be asked to wear an activity tracker.

NCT ID: NCT04083235 Active, not recruiting - Clinical trials for Metastatic Adenocarcinoma of the Pancreas

A Study to Assess the Effectiveness and Safety of Irinotecan Liposome Injection, 5-fluorouracil/Leucovorin Plus Oxaliplatin in Patients Not Previously Treated for Metastatic Pancreatic Cancer, Compared to Nab-paclitaxel+Gemcitabine Treatment

NAPOLI 3
Start date: February 11, 2020
Phase: Phase 3
Study type: Interventional

The purpose of this study is to look at the efficacy and safety of Irinotecan liposome injection in combination with other approved drugs used for cancer therapy, namely 5 fluorouracil/leucovorin (5FU/LV) plus oxaliplatin compared to nab-paclitaxel + gemcitabine treatment in improving the overall survival of patients not previously treated for metastatic pancreatic cancer.

NCT ID: NCT04082936 Active, not recruiting - Clinical trials for Mantle Cell Lymphoma

A Study of Imvotamab Monotherapy and in Combination in Subjects With Relapsed/Refractory Non-Hodgkin Lymphoma

Start date: September 30, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

This is a Phase 1/2 study of imvotamab in adult subjects with relapsed or refractory B-cell Non-Hodgkin Lymphoma. This study will consist of a dose-escalation stage, a combination stage, and a randomized dose-expansion stage where subjects will be enrolled into indication-specific expansion cohorts. imvotamab will be administered intravenously (IV). Additional CD20-positive NHL histologies (e.g. MZL and MCL), may be allowed with Medical Monitor approval during the Dose-Escalation Phase of the study.

NCT ID: NCT04082897 Active, not recruiting - CLL Transformation Clinical Trials

ObinutuzuMab AtezOlizumab and VenetocLax in RichTer transfOrmation

MOLTO
Start date: October 4, 2019
Phase: Phase 2
Study type: Interventional

This study is a multicenter, open-label, uncontrolled, phase II trial aimed to establish the safety and tolerability of venetoclax, atezolizumab and obinutuzumab combination in Richter Transformation of CLL.