Baia Mare, Maramures, Romania
Study to Investigate the Efficacy, Safety, and Tolerability of FBL-MTX in Patients With Rheumatoid Arthritis
The Sponsor is developing folate-based liposomes encapsulating methotrexate (FBL-MTX) as a putative therapy for RA, by intravenous (IV) or subcutaneous (SC) administration. Considering the presented non-clinical and clinical data for FBL-MTX and that SC administration is the most adequate route for patient self-administration, the Sponsor intends to proceed the clinical development of FBL-MTX with the objective of providing a more patient-friendly product with at least the same efficacy. This proof-of-concept study intends to demonstrate the plausibility of FBL-MTX SC administration in patients through the exploratory evaluation of SC FBL-MTX efficacy in patients with moderate-to-severe active RA, and collection data on its safety and tolerability.
Phase
2Span
54 weeksSponsor
SOLFARCOS - Pharmaceutical and Cosmetic Solutions LtdAveiro
Recruiting
Evaluation of Two Isometric Exercises in the Reduction of the Blood Pressure in People With Resistant Hypertension
Phase
N/ASpan
12 weeksSponsor
Universidade do PortoAveiro
Recruiting
A Phase 3 Study of Barzolvolimab in Participants With Chronic Spontaneous Urticaria
This is a global, multicenter, randomized, double-blind, parallel group, placebo-controlled phase 3 study investigating the efficacy, safety and tolerability of barzolvolimab in adult participants with Chronic Spontaneous Urticaria (CSU) who are symptomatic despite treatment with non-sedating second generation H1-antihistamines at 1-4 times the locally approved dose. There is a screening period of up to 4 weeks, followed by a 24-week placebo-controlled treatment period, a 28-week active treatment period where all participants receive barzolvolimab followed by a 16-week treatment free period. Approximately 915 adult participants (610 in the active arms and 305 in the placebo arm) will be randomly assigned to the treatment arms.
Phase
3Span
147 weeksSponsor
Celldex TherapeuticsAveiro
Recruiting
TAKINGCARE - TAcKlING the Needs of Carers of People With Chronic respirAtoRy disEases
Chronic respiratory diseases (CRD) are the 3rd leading cause of death and disability worldwide, with high burden for individuals, but also for their families and society, including health services. They are systemic, with pulmonary and extrapulmonary manisfestations, and are characterized by a progressive decline on multiple activities of daily living making this people depend on others along diseases' trajectory. Daily, informal/family carers, who may be relatives, partners, friends or neighbours, with a significant personal relationship with an elderly person or an adult with a disabling chronic disease, are the main source of support of people with CRD. They have a multiplicity of roles in providing physical, financial, psychological/emotional, social, and spiritual support. It is assumed that informal/family carers have the appropriate level of health literacy, disease knowledge, financial security, household conditions, psychological readiness, and medical care abilities to support the person with CRD. Although they report positive experiences (e.g., personal growth, satisfaction), there has been an acknowledgement of negative impacts (e.g., burden, difficulty in realizing personal interests/projects) and specific needs related to their roles. Nevertheless, their needs are usually unmet and rarely assessed, and they are highly neglected by the social/health system, as well as in research. Assessment of this needs is the first step to provide meaningful support to this hidden workforce who provides most of the care to people with CRD and it has been emphasised by the European Union's recommendations to support and empower informal carers. However, only two instruments have been explicitly designed to assess their needs: the Carers' Alert Thermometer and the Carer Support Needs Assessment Tool (CSNAT), but their routine use is limited, their measurement properties have been scarcely explored and only for COPD, and studies have focused on end-of-life situations. Therefore, the primary aim of this study is to develop a new instrument to assess the needs of loved ones across CRD' trajectory. Secondary aims are to map available instruments that assess the needs of informal/family carers and their measurement properties, and characterize the needs of informal carers of people with CRD and their health literacy. This study will be carried out at the University of Aveiro, at the Laboratory of Research and Respiratory Rehabilitation of the School of Health Sciences of the University of Aveiro, at Unidade Local de Saúde de Gaia/Espinho, at Unidade Local de Saúde da Região de Aveiro, and at Unidade Local de Saúde de Entre o Douro e Vouga, in Portugal. It comprises three tasks: task 1 will consist of a systematic review, in which the instruments that assess the needs of informal/family carers and their measurement properties will be identified; task 2 will be a cross-sectional study where the needs of informal carers of people with CRD and their health literacy will be characterized, through a mixed-methods study, integrating people with CRD, their informal carers, and health professionals; task 3 will be the development of an instrument that assesses the needs of informal carers of people with CRD. The items will be generated based on tasks 1 and 2 and following existing guidelines. The instrument will be pilot-tested, and the measurement properties of the instrument will then be tested in a cross-sectional observational study. On task 2, the Carer Support Needs Assessment Tool v3.0 and the Preparedness for Caregiving Scale will also be translated to European Portuguese. Adults who are diagnosed with CRD, who are fluent in Portuguese and identify an informal/family carer will be included. If they show signs of cognitive impairment, they will not be eligible for the interviews. Informal carers will be included in the study if they are ≥18 years old and are fluent in Portuguese. They will be excluded if they show signs of cognitive impairment. Health professionals will be included if they contact with people with CRD in their clinical practice. Sociodemographic and general clinical data will be collected. Instruments assessing cognitive impairment, functional capacity, disease's impact, health related quality of life, health status, level of anxiety and depression, satisfaction with life, social network, functionality, burden, positive aspects of caregiving, preparedness for caregiving, health literacy and informal carers' needs, as well as questions regarding care, will be applied. Interviews will be conducted. Sample size will be determined by the saturation of qualitative data in task 2. For the assessment of health literacy of informal carers of people with CRD, it is not possible to determine the sample number. The maximum number of informal carers will be recruited and estimates a posteriori will be made. For task 3, following the Consensus-based Standards guidelines for the Selection of health status Measurement Instruments (COSMIN), a minimum of 100 participants will be required. Data analysis will be conducted using the Statistical Package for the Social Sciences (SPSS) software. For the organization and visualization/analysis of qualitative data, the NVivo program will be used. Descriptive statistics, comparison of distributions, and associations. Validity and reliability will be assessed. The analysis of qualitative data will be divided into content analysis and thematic analysis.
Phase
N/ASpan
214 weeksSponsor
Aveiro UniversityAveiro
Recruiting
Healthy Volunteers
LetS Get fUnctional! FuNctional Status in pEople With intersTitial Lung Disease
Functional status is a fundamental, patient-centred and multidimensional outcome for individuals with interstitial lung disease (ILD). Functional status decline is the most frequent impact reported in this population. It is often associated with an increased dependency on others, loss of productivity and premature death. It is also an important predictor of exacerbations, hospitalisations, readmissions, and mortality. Functional status decline is so overwhelming for individuals and society, that it has been recently recognised as an international top priority for patient-centred research in ILD. Yet, little is known how functional status is impaired and progresses in ILD, limiting the help health professionals are able to provide to optimise care. ILD are a highly disabling group of chronic respiratory diseases characterised by widespread inflammation and scarring (fibrosis) of the lung. In people with ILD, the destruction of the pulmonary capillary bed and vasoconstriction, results in impaired gas exchange and circulatory limitation, which can lead to reduced exercise capacity. Exercise capacity may be further limited due to peripheral muscle dysfunction caused by physical deconditioning and drug-induced myopathy from ILD treatment with corticosteroids. Taken together these factors contribute to the high symptom burden experienced. This population, therefore, presents pulmonary (e.g., dyspnoea, cough) and extra-pulmonary (e.g., decreased exercise tolerance, skeletal muscle dysfunction, fatigue) traits and experience a downward spiral of debilitating symptoms and functional status decline. Functional status is defined as the ability to perform normal daily activities required to meet basic needs, fulfil usual roles, and maintain health and well-being. It includes functional capacity, i.e., an individual's maximum capacity to perform daily life activities in a standardized environment; and functional performance, i.e., activities people actually do during the course of their daily life. Adequate assessment of functional status must, therefore, include measures of functional capacity and performance, to identify the presence or absence of decline and inform on preventive and/or rehabilitative strategies. Studies focussing on the comprehensive assessment of functional status in people with ILD using reliable and validated instruments are, however, lacking. Therefore, the primary aim of SUNSET study is to characterize the functional status and explore the determinants of functional status decline of people with ILD. Secondary aims are to: i) To determine the measurement properties of functional status instruments in people with ILD ii) To identify the impact of ILD and the participants' perspectives on functional status through interviews iii) Explore the progression of functional status progression in people with ILD and iv) Develop a multidimensional index, incorporating functional status parameters, to predict mortality in people with ILD. According to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guideline, a minimum sample size of 100 will be required for task 1. For task 2, number of participants needed for interviews will be determined by data saturation. For task 3, a sample size estimation was performed in GPower to detect a small effect size (eta^2=0.02), with 80% power, 5% significance level and moderate correlation among repeated measures (r=0.5) in a repeated measures analysis of variance. The minimum sample size estimation was 81 participants Considering a dropout rate of 50%, we will aim to recruit 122 participants. This study is a multicentric observational longitudinal research project comprising four tasks. It involves collaboration between institutions, namely the University of Aveiro, along with three healthcare facilities: Centro Hospitalar de Vila Nova de Gaia/Espinho (CHVNG/E), Centro Hospitalar do Baixo Vouga (CHBV), and Centro Hospitalar de Entre o Douro e Vouga (CHEDV). Task 1 aims to validate the measures of functional status. For this, two assessment moments will be performed. The first assessment will be conducted at baseline and will be in line with the routine appointment. The second assessment will be conducted 7 days after baseline. Task 2 will use the information collected in Task 1 and conduct semi-structured individual interviews to characterize the functional status from the perspective of people with ILD. Task 3 will then conduct an observational longitudinal study using the most valid and reliable measures of functional status identified in task 1 and mortality rates will be explored during task 3. For this, patients will be contacted to participate in four timepoints of assessment (at 6, 12, 18, and 24 months) which will be in line with the routine appointment. Table 1 details the measurements performed in each timepoint. If an outcome measure is found to not be valid and reliable during task 1, that outcome measure will not be collected in task 3 nor replaced by another outcome measure. In Task 4, the data collected in Task 3 will be used to develop a multidimensional index incorporating functional status parameters to predict mortality in people with ILD. The study assessments will take approximately 90 minutes and will take place at Centro Hospitalar de Vila Nova de Gaia/Espinho (CHVNG/E), Centro Hospitalar do Baixo Vouga (CHBV), and Centro Hospitalar de Entre o Douro e Vouga (CHEDV). Patients with ILD will be recruited via the pulmonology services at hospitals. Pulmonologists will explain the study briefly and inform the researchers about interested participants. The researchers will provide further information about the study, clarify any doubts and collect the informed consent. Participants will be eligible if they are: i) ≥18 years, ii) diagnosed with any type of ILD, iii) fluent in Portuguese and iv) able to provide informed consent. Participants will be excluded if they have: i) other respiratory diseases ii) a history of acute cardiac/respiratory condition in the previous month; iii) present signs of cognitive impairment; iv) significant cardiovascular, neurological and/or musculoskeletal disease that may limit their participation; v) current neoplasia, vi) other autoimmune diseases (aside from ILD). Once task 1 is finished, included participants will be contacted again to verify if they still match the inclusion/exclusion criteria and are still willing to participate in task 3. Baseline data will be collected: Sociodemographic, clinical characteristics (i.e., smoking habits, vital signs and symptoms), anthropometric (i.e., height and weight to compute body mass index) and general clinical data (i.e., medication, oxygen therapy, non-invasive ventilation, acute exacerbations, hospitalizations and number of hospital admissions in the last month and year, length of stay), as well as prior and follow-up lung function and arterial blood gas will be collected from clinical records for patients' characterization. Mortality and rehospitalizations will be explored during the study period. Peripheral muscle strength, functional status, daily physical activity, self-reported symptoms, functional status, impact of the disease and health-related quality of life. Qualitative data from interviews. Data analysis will be conducted using the Statistical Package for the Social Sciences (SPSS) software, incorporating both descriptive and inferential statistics. To examine changes in outcome measures, data from baseline and subsequent assessments will be compared. Additionally, the interviews will be transcribed and analysed using thematic analysis.
Phase
N/ASpan
226 weeksSponsor
Aveiro UniversityAveiro
Recruiting
Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM
Phase
3Span
161 weeksSponsor
CytokineticsAveiro
Recruiting
Exercise, PNE and Cognitive Training in Individuals With Chronic Low Back Pain
This study aims of to investigate whether combining cognitive training to exercise and Pain Neuroscience Education (PNE) will contribute to reduction of pain and associated symptoms (i.e., catastrophizing, fear of movement, disability) in individuals with low back pain (LBP). It will have three arms of which will receive exercise, PNE and/or cognitive training for 8 weeks (Group I - exercise and PNE; Group II - exercise, PNE and cognitive training; Group III - cognitive training). Participants will be assessed for pain characteristics, psychological, and cognitive aspects at baseline, post-intervention and at 3 months follow-up.
Phase
N/ASpan
47 weeksSponsor
Aveiro UniversityAveiro
Recruiting
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
All participants will continue their pre-study background therapy, defined as the participant's current treatment regimen. Options include, but are not limited to, granulocyte-colony stimulating factor (G-CSF), immunoglobulin replacement therapy, prophylactic antibiotics, or "watchful waiting".
Phase
3Span
117 weeksSponsor
X4 PharmaceuticalsAveiro
Recruiting
Integrating Palliative Care Education in Pulmonary Rehabilitation
Chronic obstructive pulmonary disease (COPD) and interstitial lung diseases (ILD) have an overwhelming impact on individuals, society and health systems worldwide. Informal caregivers play an essential role in the lives of people with COPD or ILD, however little is known about how to support them. Palliative care addresses multiple unmet needs of people with COPD or ILD and informal caregivers, but it remains highly inaccessible. Pulmonary rehabilitation (PR) is a fundamental evidence-based intervention for the management of COPD and ILD, and may be an opportunity to introduce palliative care. However, the effects of this integrated care model on patients' and informal caregivers' outcomes are still unclear. Therefore, this study proposes to develop, implement and evaluate an innovative PR model with palliative care education. The primary aim is to explore the effects of palliative care education as part of PR on people with COPD or ILD and informal caregivers' knowledge about palliative care. The secondary aims are: a) to understand the perspectives of people with COPD or ILD and informal caregivers about integrating palliative care education in PR; and b) to explore the short and medium-term effects of palliative care education as part of PR on people with COPD or ILD and informal caregivers' attitude towards palliative care referral, symptoms, disease impact, health-related quality of life (HRQoL), needs, knowledge about the disease and burden. Recruitment will occur at the outpatient PR unit at Centro Hospitalar Baixo Vouga (CHBV). The pulmonologist of the PR program will identify eligible participants (i.e., people with COPD or ILD and informal caregivers) and explain the study. An appointment for the baseline assessment will be scheduled with those willing to participate. Participants will be randomised to experimental group (EG) and control group (CG). Both groups will follow a multidisciplinary team-based PR program, which will include two weekly supervised exercise sessions and weekly education and psychosocial support sessions in a group setting over a 12-week period. Informal caregivers will be invited to take part in all education and psychosocial support sessions. The key educational topics explored in the EG group will be: 1) information on chronic respiratory diseases; 2) medication, inhaler techniques, oxygen therapy and non-invasive ventilation; 3) symptom management and exacerbations; 4) palliative care; 5) exercise and physical activity; 6) action plan; 7) anxiety and depression management; and 8) nutrition. Additionally, there will be two sessions: "Peer-to-peer session" and "Get-apart session". In both the focus will be to discuss participants' own issues with the multidisciplinary team. Moreover, every two weeks, participants will have the opportunity to discuss any health-related issue through online sessions. Individual cases will also be referred for evaluation by a specialist palliative care team or by any other health and social care professional (e.g., psychologist or social worker) according to the specific unmet needs identified. The EG will receive the PR program as described above and the CG will receive the traditional PR program i.e., without the education session on palliative care, the "Peer-to-peer session", the "Get-apart session" and the online sessions. Quantitative data will be collected from all participants at baseline, at 12 weeks (i.e., end of PR) and at 6 months post-PR. Qualitative data will be collected only from the EG before and after PR. The following quantitative data will be collected from people with COPD or ILD: sociodemographic information; health status (e.g., exacerbation(s) within the last 12 months); health literacy; anthropometry; respiratory function; symptoms (pain, dyspnoea, fatigue, cough, and anxiety and depression) and disease impact; HRQoL; needs; functional performance and capacity; peripheral muscle strength; balance; knowledge about palliative care and the disease; and attitude towards palliative care referral. Informal caregivers will also provide information on: health status (e.g., limitation(s) in activities of daily living); health literacy; role of caring (e.g., relationship with the care receiver); symptoms (pain, fatigue, and anxiety and depression); needs; burden; knowledge about palliative care and the disease; and attitude towards palliative care referral. Qualitative data will be collected through focus groups. Sample size was estimated with the program G*Power 3.1.9.4, with an effect size specification "as in GPower 3.0", for the within-between interaction of mixed ANOVA with two groups (control and experimental) and two timepoints (pre and post PR). The investigators considered an α of 0.05, a power of 0.80, a correlation among repeated measures of 0.5, a nonsphericity correction of 1 and an expected effect size f of 0.25. The calculated sample size was 34 and considering a possible 40% dropout and missing data rate, the final sample size was determined to be 58.
Phase
N/ASpan
148 weeksSponsor
Aveiro UniversityAveiro
Recruiting
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
This is a multi-center, open-label extension (OLE) study of approximately 800 participants with SHTG who would be rolled over from studies ISIS 678354-CS5 (NCT05079919) or ISIS 678354-CS6 (NCT05552326). Day 1 of this study may be same as the Week 53 visit of either ISIS 678354-CS5 or ISIS 678354-CS6, as applicable. Participants will receive olezarsen during the 157-week treatment period. The study will include a 31-day qualification Period, a 157-week treatment period, and a 13-week post-treatment period.
Phase
3Span
225 weeksSponsor
Ionis Pharmaceuticals, Inc.Aveiro
Recruiting