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NCT ID: NCT05604170 Enrolling by invitation - Clinical trials for Tuberous Sclerosis Complex

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

Start date: May 16, 2022
Phase: Phase 3
Study type: Interventional

This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001

NCT ID: NCT05517564 Enrolling by invitation - Clinical trials for Non-alcoholic Steatohepatitis

First-in-Human Study of GM-60106 in Healthy Adults and Otherwise Healthy Adults With an Increased Body Mass Index and Markers of Non-Alcoholic Fatty Liver Disease

Start date: August 1, 2022
Phase: Phase 1
Study type: Interventional

This is a Phase 1a/1b, randomised, double-blind, placebo-controlled single- and multiple-ascending dose study to evaluate the safety, tolerability, PK, and PD of GM-60106 in healthy adult male and female participants and otherwise healthy adults who have an increased BMI and markers of NAFLD.

NCT ID: NCT05515536 Enrolling by invitation - Friedreich Ataxia Clinical Trials

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Start date: December 8, 2022
Phase: Phase 3
Study type: Interventional

The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.

NCT ID: NCT05509855 Enrolling by invitation - Clinical trials for T-cell Acute Lymphoblastic Leukemia

A Long-Term Safety Follow-Up Study for Patients Treat With WU-CART-007

Start date: August 22, 2022
Phase:
Study type: Observational

This study will provide long-term follow-up for patients who have received treatment with WU-CART-007 in a previous clinical trial. In this study, patients will be followed for up to 15 years after their last dose of WU-CART-007 for evaluation of delayed adverse events, presence of persisting WU-CART-007 vector sequences, and overall survival and progression-free survival.

NCT ID: NCT05485961 Enrolling by invitation - Clinical trials for Atherosclerotic Cardiovascular Disease

Combined Dose-Finding and CV Outcomes Study With CSL300 (Clazakizumab) in Adult Subjects With ESKD Undergoing Dialysis

Start date: October 21, 2022
Phase: Phase 2/Phase 3
Study type: Interventional

This is a 2-part (phase 2b/3) prospective, interventional, multicenter, randomized, double blind, placebo controlled study. Part 1 (phase 2b) is a dose-finding study for CSL300 vs placebo. Part 2 (phase 3) aims to assess the efficacy of CSL300 on CV outcomes and safety in subjects with ASCVD or diabetes mellitus and evidence of systemic inflammation who are undergoing maintenance dialysis.

NCT ID: NCT05481827 Enrolling by invitation - Clinical trials for Age Related Macular Degeneration (AMD)

ORACLE: A Long-term Follow-up Study to Evaluate the Safety of GT005 in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration Treated in a Gyroscope-sponsored Antecedent Study

ORACLE
Start date: July 12, 2022
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the long-term safety of GT005 in participants with Geographic Atrophy (GA) secondary to AMD who have been treated in an antecedent study.

NCT ID: NCT05426733 Enrolling by invitation - Biliary Atresia Clinical Trials

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia

BOLD-EXT
Start date: July 5, 2022
Phase: Phase 3
Study type: Interventional

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia

NCT ID: NCT05374291 Enrolling by invitation - Heart Failure Clinical Trials

The RENAL LIFECYCLE Trial: A RCT to Assess the Effect of Dapagliflozin on Renal and Cardiovascular Outcomes in Patients With Severe CKD

Start date: November 8, 2022
Phase: Phase 3
Study type: Interventional

Rationale: Sodium glucose co transporter 2 (SGLT2) inhibitors are a relatively new class of agents, originally developed as oral antihyperglycemic drugs. SGLT2 inhibitors are clinically available since 2012 for the treatment of patients with diabetes mellitus type 2. Later, SGLT2 inhibitors appeared to have also specific reno- and cardioprotective effects. Remarkably, the trials that have been performed thus far excluded patients with an eGFR below 25 mL/min/1.73m2 at inclusion, prevalent dialysis patients, and kidney transplant recipients. This is unfortunate, because especially these patients are at high risk of reaching kidney failure requiring dialysis, cardiovascular complications and mortality, whereas there are only few proven effective therapies. There is emerging evidence from experimental studies and post hoc-analyses of randomized clinical trials that SGLT2 inhibitors may also be effective in preventing cardiovascular and mortality outcomes in these patients with severe CKD, including patients receiving dialysis or living with a kidney transplant. For instance, subgroup analysis of the DAPA-CKD trial comparing 624 patients with an eGFR<30 to the remainder of the trial population with better kidney function, demonstrated that the efficacy of the SGLT2 inhibitor dapagliflozin in reducing cardiovascular, heart failure and renal outcomes persisted in the population with impaired kidney function. Furthermore, in the DAPA-CKD trial patients continued to use dapagliflozin or placebo when dialysis was initiated. In the subgroup of patients who initiated dialysis, dapagliflozin was associated with a relative risk reduction for mortality of 21%. Finally, in kidney transplant recipients, SGLT2 inhibitors have been shown to be effective in lowering HbA1c, body weight, blood pressure and stabilize kidney function, and these agents were well tolerated and safe. Taken these findings together there is a sound rationale to study the long-term reno- and cardioprotective efficacy and safety of SGLT2 inhibitors in patients with severe CKD. There are two cardiac sub-studies: the cardiac MRI substudy and the echocardiography sub-study. The echocardiography sub-study is referred to as the "SGLT-2-inhibitors to Target Heart Failure in Peritoneal Dialysis" (STOP HF in PD) study. In STOP HF in PD the effect of dapagliflozin on cardiac function will be assessed in a subset of 100 patients treated with peritoneal dialysis.

NCT ID: NCT05303532 Enrolling by invitation - Cancer Clinical Trials

Roll Over StudY for Patients Who Have Completed a Previous Oncology Study With Durvalumab

ROSY-D
Start date: April 19, 2022
Phase: Phase 3
Study type: Interventional

The rationale of the ROSY-D study is to continue to provide study treatment for patients who have participated in a parent study with Durvalumab and who are continuing to derive clinical benefit from treatment at the end of such studies, as judged by the Investigator.

NCT ID: NCT05298982 Enrolling by invitation - Clinical trials for Mechanical Ventilation

The TEAM Long-Term Cohort Study (A Sub-study of TEAM(III))

Start date: February 11, 2020
Phase:
Study type: Observational

This study is a prospective cohort study to evaluate the long-term effects of early activity and mobilisation compared to standard care on disability, function and health status for patients at 1, 2 and 5 years after recruitment of patients randomised into the TEAM Phase III RCT (ClinicalTrials.gov NCT03133377). The primary outcome of the study will be the level of disability as measured by the World Health Organisation's Disability Schedule 2.0, 12 level (WHODAS) at 2 years after recruitment.