Francavilla Fontana, Italy

  • A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

    The primary objective of this study is to assess the long-term safety of omaveloxolone as prescribed to participants with FA in the real-world setting, including characterization of all drug-induced liver injury (DILI) and congestive heart failure (CHF) AEs. The secondary objective of this study is to capture the reasons and timing of omaveloxolone treatment interruptions, discontinuations, and drug overdose.

    Phase

    N/A

    Span

    251 weeks

    Sponsor

    Biogen

    Conegliano

    Recruiting

  • Peripheral Extreme Revascularization in "No-option" Patiens With Chronic Limb Threatening Ischemia

    Critical lower Limb Threatening Ischemia is the most advanced stage of lower limb arterial disease and is associated with a high risk of mortality and major amputation.Literature data indicate that about 5-10% of patients with peripheral arterial disease have a risk of developing CLTI, therefore the most advanced stage of arterial disease of the lower limbs, within 5 years and have a mortality of 20% at 6 months after diagnosis of CLTI.Despite the continuous improvement of the techniques and materials used in the revascularization of these patients, the data of the Literature show that in about 10-15% of these patients revascularization is impossible or ineffective. This condition is defined as: "no-option" CLTI.Therefore, for patients with "no-option" CLTI an alternative treatment for limb rescue may be considered the use of arterialization of the venous plexus of the foot; that is, to use to bring the arterial flow to the foot a healthy conduit, such as the vein, instead of the diseased artery and no longer usable. The present study is designed as a multicentre, prospective, single-arm, observational study. All eligible subjects for undergoing PiPER procedure at sites participating in the study will be considered for enrolment and will be asked to give consent prior to participating. Subjects will be considered enrolled in the study at the time written informed consent is given to the use of their personal data. Once patients are enrolled, their demographics, medical history, disease-relevant conditions, treatment details and outcomes will be collected for up to 24 months from the procedure. The study will collect information about the medical care patients receive during their planned procedure. No additional testing or procedures will be done. The revascularization procedure will be performed as per the current clinical practice. After discharge all patients will attend clinic visits at 30 days (±7 days), 6 months (±14 days),12 months (±30 days), 24 months (±30 days).

    Phase

    N/A

    Span

    387 weeks

    Sponsor

    EndoCore Lab s.r.l.

    Conegliano

    Recruiting

  • DiamondTemp Global Registry

    The DiamondTemp™ Global Registry is a prospective, global, multi-center, observational post-market registry. The purpose of this clinical study is to describe clinical performance and safety data in a broad patient population treated with the commercially available DiamondTemp™ Family of Cardiac Ablation Catheters (hereafter referred to as the DiamondTemp™ Ablation System) and followed according to the standard of care at each respective institution. The follow up period is intended to align with standard practice and subjects will be followed for a minimum of 12 months post-procedure.

    Phase

    N/A

    Span

    519 weeks

    Sponsor

    Medtronic Cardiac Ablation Solutions

    Conegliano

    Recruiting

  • A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

    The primary objective of Part 1 randomized controlled trial (RCT) is to evaluate the efficacy of omaveloxolone at Week 52 and the secondary objectives are to evaluate safety of omaveloxolone through Week 52 and the concentration of omaveloxolone after single and multiple dose administration. The primary objective of Part 2 open-label extension (OLE) trial is to evaluate the safety and tolerability of long-term omaveloxolone use and the secondary objective is to evaluate the efficacy of omaveloxolone following long-term use.

    Phase

    3

    Span

    233 weeks

    Sponsor

    Biogen

    Conegliano, Veneto

    Recruiting

  • Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

    The UNIFAI Study: Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study is a global research study designed to provide a deep and evolving understanding of the natural history of this rare and debilitating genetic disorder as well as inform clinical trial design and implementation. Friedreich's Ataxia is a rare, inherited, multi-system condition characterized by progressive neurological and cardiac symptoms. It is caused by mutations in the FXN (frataxin) gene. The UNIFAI study is a multicenter, prospective, longitudinal, observational study, which means that all data about the natural disease course will be captured at study visits conducted annually according to a single protocol for all sites. This study will recruit participants with FA worldwide, to be assessed annually for up to 25 years. Study participation involves yearly study visits with data collected from medical records and history along with clinical outcome assessments including, neurological exams, timed walking tests, upper limb function measures, and patient-reported quality of life assessments, with the option for sites to collect additional ancillary measures related to speech, vision, fatigue, balance and cognition. This global study is a harmonization of two well-established, long-standing natural history studies in FA that have provided a framework for further investigation of clinical measures that can quantitatively assess FA: FACOMS (US, Canada, Australia, New Zealand, and India) and EFACTS (European countries). These studies have been conducted in parallel with many similarities in study conduct. Uniting these two existing studies and groups of researchers gives greater power to data previously collected as well as future data. It also continues efforts to expand the network of clinical research centers specializing in FA called the FA-Global Clinical Consortium. Due to advances in understanding the genetic and cellular dysfunction of FA that lead to symptoms observed in affected individuals, there has been significant growth in the discovery and development of therapeutic approaches, many currently being evaluated in clinical trials and a first approved treatment in the United States in 2023. The UNIFAI study aims to build upon this momentum by focusing on several key objectives. UNIFAI will assess the natural history of FA by collecting data from diagnosed individuals of all ages and stages of disease progression. The data collected includes demographics, medical history, medications, neurological and functional assessments, cardiac examinations, laboratory studies, and health questionnaires. The study will assess and evaluate clinical outcomes in individuals with FA, such as disease progression, symptom severity, and overall quality of life, by various factors that might influence such outcomes such as genetic mutation, demographics (age, geography), co-existing conditions, medications, or treatments. By tracking outcomes over time, researchers aim to discern patterns, trends, and potential variations in subgroups or in the effectiveness of therapies or interventions across a diverse group of participants. The UNIFAI study aims to play a pivotal role in identifying clinical milestones and changes in natural history over time as new treatments emerge. The study will monitor how these interventions alter the trajectory of the disease, potentially leading to the identification of crucial tipping points, disease landmarks, or stages where interventions can be most impactful. This study has the potential to significantly improve our understanding of FA and lead to more effective treatments and improved outcomes for those living with FA. The study aims to inform clinical trial design and the development and validation of novel clinical outcome assessments and biomarkers that can be used in clinical trials. The UNIFAI study aims to capture data from a wide and diverse cohort of individuals with FA so that this dataset can be used to inform the selection of inclusion and exclusion criteria and power calculations for trial designs with specific clinical outcome measures.

    Phase

    N/A

    Span

    1283 weeks

    Sponsor

    Friedreich's Ataxia Research Alliance

    Conegliano

    Recruiting

    Healthy Volunteers

  • A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

    The study will have Part 1-dose confirmations and Part 2 with dose levels to be decided based on the cumulative PK, electroencephalography (EEG), and safety data emerging from Part 1. The dose levels for the first cohort of Part 2 will be decided based on the cumulative PK, EEG, and safety data emerging from Part 1. Part 2 will explore the change in EEG beta-band power relative to baseline at Week 2, Week 4 (i.e., approximately 2 weeks after the start of the Dose B), and at the end of the 12-week treatment period after daily administration of alogabat.

    Phase

    2

    Span

    119 weeks

    Sponsor

    Hoffmann-La Roche

    Conegliano, Veneto

    Recruiting

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