Troina

Researchers are evaluating the effects of AEF0217 on adaptive behaviors and daily life activities in adults and older adolescents with Down Syndrome. This randomized, double-blind, placebo-controlled Phase 2B trial will also assess the safety and tolerability of AEF0217. The study aims to determine whether AEF0217 improves cognitive functions, quality of life, and sleep after 24 weeks of daily treatment compared to a placebo. Participants will be randomly assigned to receive one of three doses of AEF0217 or a matching placebo daily for 24 weeks. The trial includes visits at baseline, weeks 4, 12, and 24, with additional phone or video calls at weeks 1, 2, 8, and 18. After the treatment period, there is an 8-week follow-up visit at week 32. Participants completing 24 weeks may be invited to join a 12-month open-label extension study. During the study, participants and their caregivers will attend six clinic visits and receive five phone calls for monitoring. Assessments include adaptive behavior tests, cognitive evaluations, quality of life and sleep questionnaires, and safety labs including blood and urine tests. Researchers will measure changes in adaptive behavior scores from baseline to week 24 as the primary outcome. Safety will be closely monitored throughout the trial, with a total participation duration of up to 36 weeks.

Electrocardiogram (ECG) Q-T prolongation is a heart rhythm disorder linked to potentially dangerous arrhythmias. Certain psychotropic drugs can increase the risk of QT prolongation, so doctors usually perform a baseline ECG before prescribing these medications. However, there is no established guideline on when to repeat ECG tests during ongoing treatment or which clinical signs should prompt additional monitoring. This research studies how often QTc prolongation occurs as a side effect in children and adolescents taking psychotropic drugs long term. ECGs will be recorded at the start of treatment and then again at 3, 6, and 12 months to analyze changes and factors that could affect QT intervals. Participants will have ECGs recorded over the course of their treatment, with assessments at several points to track heart rhythm changes. The main measure is how the QT interval changes from baseline through 18 months after enrollment. This monitoring aims to better understand the timing and risk of QT prolongation with psychotropic medications in young patients.

Parkinson's disease is a neurological disorder that worsens over time and affects movement, causing symptoms like tremors, stiffness, and slowness. This study is evaluating how effective Foslevodopa/Foscarbidopa is in treating adults with advanced Parkinson's disease in Italy, also considering the quality of life of caregivers if present. Approximately 270 adult participants prescribed this treatment will be enrolled across about 19 sites in Italy. Participants will receive Foslevodopa/Foscarbidopa through a subcutaneous infusion as prescribed by their doctors during routine clinical care. They will be treated continuously and followed for up to 12 months. There are no additional burdens expected beyond their usual clinical visits. During the study, participants will attend regular hospital or clinic visits aligned with their normal care schedule. Researchers will assess changes from baseline in participants’ quality of life using the PD Questionnaire-39 (PDQ-39) over approximately six months. Caregiver quality of life will also be evaluated if a caregiver is involved. The study monitors participants throughout the 12-month period with no extra procedures beyond routine practice.

Researchers are investigating the unmet informational, clinical, and psychosocial needs of patients diagnosed with isolated REM Sleep Behavior Disorder (iRBD) and their significant others. iRBD is a sleep disorder marked by dream-enactment behaviors during REM sleep and is known as a strong early sign of neurodegenerative diseases like Parkinson's disease. The study aims to better understand the emotional and practical challenges faced by both patients and caregivers, as well as how the diagnosis affects their quality of life and future health concerns. This multicenter cross-sectional observational study collects information through structured online questionnaires completed by patients and their significant others or caregivers. Patients provide details about their experiences with iRBD, their informational needs, interactions with healthcare providers, emotional well-being, and quality of life. Caregivers complete a parallel questionnaire focusing on caregiving burden, support needs, and their own emotional challenges. The study gathers data at a single time point without interventions or treatments. Participants will provide responses about their disease knowledge, psychosocial impact, and support needs through the online survey platform. Researchers will analyze these responses to identify key unmet needs and challenges associated with iRBD. The study's findings aim to inform improved patient education, clinical care approaches, and support services tailored for individuals living with iRBD and their families.