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NCT ID: NCT04777201 Active, not recruiting - Clinical trials for Neovascular Age-related Macular Degeneration

A Study to Evaluate the Long-Term Safety and Tolerability of Faricimab in Participants With Neovascular Age-Related Macular Degeneration

AVONELLE-X
Start date: April 19, 2021
Phase: Phase 3
Study type: Interventional

This main long-term extension study is designed to evaluate the long-term safety and tolerability of faricimab 6 milligrams (mg) administered by intravitreal injection at a personalized treatment interval (PTI) to participants with neovascular age-related macular degeneration (nAMD) who enrolled in and completed one of the Phase III studies: GR40306 (NCT03823287) or GR40844 (NCT03823300), also referred to as the parent studies. Eligible patients who consent to participate in this main study will be enrolled upon completion of the end-of-study visit in the parent study. Additionally, there is a substudy that is being conducted. The aim of this substudy is to evaluate the impact of intravitreal faricimab on the health of the corneal endothelial cells in the study eyes of patients with nAMD to fulfill a U.S. Food and Drug Administration (FDA) post-marketing requirement. The fellow eyes of the same enrolled participants in the substudy will serve as the controls.

NCT ID: NCT04777045 Active, not recruiting - Clinical trials for Microvascular Angina

Efficacy of Diltiazem to Improve Coronary Microvascular Dysfunction: a Randomized Clinical Trial

EDIT-CMD
Start date: October 25, 2019
Phase: Phase 3
Study type: Interventional

Rationale: Up to 40% of patients undergoing a coronary angiogram for symptoms/signs of ischemia do not have obstructive coronary artery disease (CAD). In about half of them the mechanism underlying cardiac ischemia is coronary microvascular dysfunction (CMD). In CMD, myocardial ischemia is caused by impaired endothelial and/or non-endothelial coronary vasoreactivity resulting in the coronary microvasculature not dilating properly or becoming vasospastic. Recently published diagnostic criteria state that to confirm the diagnosis, CMD patients should either have an impaired coronary flow reserve (CFR), increased microvascular resistance (IMR) or have evidence of microvascular spasms. Hence, invasive coronary function testing (CFT) is considered the reference standard for a definitive diagnosis of CMD. Patients with microvascular angina often have continuing episodes of chest pain leading to frequent first aid visits and hospital re-admissions with associated high health care costs. Moreover, CMD is associated with a worsened cardiovascular prognosis. Therefore, adequate treatment is paramount. However, current treatment options are based on a limited number of small studies, most of which were not placebo-controlled. Based on prior studies and our clinical experience we believe diltiazem, a calcium channel blocker (CCB) could improve coronary microvascular function in patients with CMD. Objective: Our primary objective is to assess the effect of diltiazem on coronary microvascular function as assessed by CFT in symptomatic patients with CMD. Our secondary objective is to assess the effect of diltiazem on the individual coronary function parameters. Study design: This is a clinical multi-center randomized with 1:1 ratio, double-blind, placebo-controlled study. Patients with chronic angina in the absence of obstructive CAD will be screened for study enrollment. Eligible patients will be asked for informed consent after which the screening visit will take place. Within 8 weeks after screening they will undergo CFT with the assessment of the coronary flow reserve (CFR), index of microcirculatory resistance (IMR) and coronary spasm. - Intervention arm: if CFT shows either a CFR ≤ 2.0, an IMR ≥ 25 and/or coronary spasm, the patient will continue in the intervention arm of the trial and will be randomized to either diltiazem or placebo treatment for 6 weeks. After 6 weeks, a CFT will be repeated and the diltiazem/placebo treatment will be discontinued. Follow-up will be obtained after 6 weeks of treatment, and 1 year and 5 years after treatment discontinuation. - Registration arm: If the CFT at baseline shows no signs of vascular dysfunction, patients will enter in the registration arm of the study. These patients will not receive any study medication. Follow-up will be obtained after 1 year and 5 years. Study population: Adult patients with chronic angina in the absence of obstructive CAD will be screened for participation. They will be recruited from the outpatient clinic of the cardiology department of the participating sites. Patients with contra-indications for coronary function testing (with the use of adenosine and acetylcholine) and/or diltiazem treatment (i.e. severe AV conduction delay, hypersensitivity, reduced left ventricular function) will not be eligible. Intervention: After establishing an abnormal coronary vascular function, 6 weeks treatment with either diltiazem 120-360 mg or placebo will be initiated in a double-blind fashion. Every two weeks dose titration will be performed if possible, under the guidance of patient tolerance (dizziness, leg oedema, etc.), blood pressure and heart rate. Main study parameters/endpoints: The proportion of patients having a successful treatment with diltiazem, defined as normalization of at least one abnormal parameter and none of the normal parameters becoming abnormal.. A normal IMR is specified as IMR < 25, a normal CFR being a CFR > 2 and a normal acetylcholine test is specified as one without ECG abnormalities and without signs of spasm at the same acetylcholine dose used at baseline. Main secondary endpoints will be the change in the individual coronary function parameters. Nature and extent of the burden and risks associated with participation, benefit and group relatedness: The extensive experience with diltiazem and the favourable safety profile in combination with the short duration of treatment make the treatment risk low for participants. Related to the study procedure several reports show that CFT is a safe procedure with serious complication rates (death, myocardial infaction, etc.) ranging from 0 to 0.7%. The first CFT is clinically indicated by the treating physician. The second CFT will bring additive risk to the participants in the intervention arm. However, we believe it is essential to investigate the effect of diltiazem on coronary function to justify its use in CMD patients.

NCT ID: NCT04770779 Active, not recruiting - Clinical trials for Transfusion-dependent Beta-Thalassemia

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

ENERGIZE-T
Start date: November 30, 2021
Phase: Phase 3
Study type: Interventional

The primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT).

NCT ID: NCT04770753 Active, not recruiting - Clinical trials for Non-Transfusion-dependent Beta-Thalassemia

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

ENERGIZE
Start date: November 8, 2021
Phase: Phase 3
Study type: Interventional

The primary purpose of this study is to compare the effect of mitapivat versus placebo on anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).

NCT ID: NCT04770545 Active, not recruiting - Clinical trials for Geographic Atrophy Secondary to Age-related Macular Degeneration

An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD

GALE
Start date: March 4, 2021
Phase: Phase 3
Study type: Interventional

This is a phase 3, open-label, multicenter, extension study to evaluate the long-term safety and efficacy of pegcetacoplan (APL-2) in subjects with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) who participated in Study APL2-103 (NCT03777332) or completed the treatment at Month 24 of either Study APL2-303 (Derby, NCT03525613) or Study APL2-304 (Oaks, NCT03525600).

NCT ID: NCT04770220 Active, not recruiting - Clinical trials for Early Alzheimer's Disease

An Efficacy and Safety Study of ALZ-801 in APOE4/4 Early AD Subjects

APOLLOE4
Start date: May 19, 2021
Phase: Phase 3
Study type: Interventional

This study is being conducted to evaluate the safety and efficacy of ALZ-801 in Early Alzheimer's disease (AD) subjects with the APOE4/4 genotype. This is a double-blind, randomized trial with one dose of ALZ-801 compared to placebo.

NCT ID: NCT04768062 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)

Start date: April 13, 2021
Phase: Phase 3
Study type: Interventional

This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.

NCT ID: NCT04765345 Active, not recruiting - Clinical trials for Retinitis Pigmentosa

Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F

RUSH1F
Start date: June 8, 2021
Phase:
Study type: Observational

The overall goal of this project, co-funded by the Foundation Fighting Blindness and the USHER 1F Collaborative is to characterize the natural history of disease progression in patients with PCDH15 mutations in order to accelerate the development of outcome measures for clinical trials.

NCT ID: NCT04764448 Active, not recruiting - Clinical trials for Alpha 1-Antitrypsin Deficiency

A Study of Belcesiran in Patients With AATLD

ESTRELLA
Start date: February 12, 2021
Phase: Phase 2
Study type: Interventional

This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ AATD-associated liver disease (AATLD). The study will be conducted in 3 separate cohorts. A total of up to 16 participants may be enrolled in Cohort 1 and 2. A total number of 30 subjects will be enrolled in cohort 3. The 3 cohorts are differentiated by the duration of the treatment period, the number of doses administered, and the timing of the second liver biopsy.

NCT ID: NCT04757636 Active, not recruiting - Clinical trials for Neovascular Age-related Macular Degeneration

OPT-302 With Aflibercept in Neovascular Age-related Macular Degeneration (nAMD)

COAST
Start date: March 12, 2021
Phase: Phase 3
Study type: Interventional

A 2-year phase 3, multicentre, randomised, parallel-group, sham-controlled, double-masked study. Primary efficacy will be determined at Week 52.