Efficacy, Safety and Cost-effectiveness of a Biomarker-based Predictive Model for Persistent Remission in Rheumatoid Arthritis Patients Undergoing Biological Therapy Optimization
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 184
- 试验地点
- 9
- 主要终点
- Percentage of patients maintaining sustained remission.
研究概览
简要总结
This study aims to evaluate a new tool designed to help doctors decide whether it is safe to reduce medication in patients with rheumatoid arthritis (RA) who are in remission.
Rheumatoid arthritis is a chronic inflammatory disease that affects the joints, causing pain, stiffness, and reduced mobility. Many patients receive long-term treatment with biological drugs to control the disease. When the disease is well controlled (remission), doctors may gradually reduce the medication dose. However, deciding when and in whom to reduce treatment is currently based on experience and trial-and-error.
The study evaluates a predictive tool (called OPTIBIO) that uses information from blood samples, genetic data, and clinical characteristics to estimate the risk that the disease will flare up if treatment is reduced.
Participants in the study will be randomly assigned to one of two groups:
- In one group, the decision to reduce medication will be made by their usual doctor.
- In the other group, the decision will be guided by the predictive tool.
The study lasts 12 months and includes several hospital visits. During these visits, participants will:
- Answer questionnaires about their health and quality of life
- Have physical examinations
- Provide blood samples for routine tests and additional research purposes
- Possibly undergo joint ultrasound (if they consent) Some additional blood samples may be stored in authorized biobanks for future research related to rheumatoid arthritis, but only if participants explicitly agree. These samples will be coded to protect personal identity and will only be used in ethically approved research projects.
Participation in the study is entirely voluntary. Participants can choose which procedures they agree to and may withdraw at any time without affecting their medical care.
The study may not provide direct benefit to participants, but it could help improve future treatment decisions and the overall management of rheumatoid arthritis.
详细描述
Background Rheumatoid arthritis (RA) is a chronic, immune-mediated inflammatory disease characterized by persistent synovitis, progressive joint damage, and reduced quality of life. The introduction of biological therapies, particularly tumor necrosis factor inhibitors (TNFi), has substantially improved disease outcomes, allowing many patients to achieve sustained remission.
In patients who reach remission, clinical guidelines recommend considering treatment optimization strategies, including dose tapering or discontinuation. However, in routine clinical practice, such decisions remain largely empirical and are primarily based on physician judgment. This approach introduces clinical uncertainty, as treatment reduction may lead to disease reactivation in a subset of patients, while continued treatment may expose patients to unnecessary risks and increase healthcare costs.
Rationale There is a clear unmet need for tools that support personalized treatment decisions in patients with RA in remission. A reliable method to predict the risk of disease flare could enable clinicians to better identify patients in whom treatment reduction can be safely implemented.
The OPTIBIO model has been developed as a predictive tool to address this need. It integrates clinical variables with biomarker data derived from peripheral blood, including protein expression, cellular components, and genetic information. By combining these data sources, the model aims to provide individualized risk predictions of disease reactivation following treatment optimization.
Study Purpose The purpose of this study is to evaluate the clinical utility of the OPTIBIO predictive model when incorporated into routine clinical decision-making, compared with standard practice.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Health Services Research
- 盲法
- Double (Participant, Investigator)
盲法说明
Three levels of blinding are implemented:
- Participants and blinded consulting investigators are aware of whether treatment has been optimized, but are not informed about the decision-making process that led to treatment optimization.
- Blinded outcome assessors are not aware of either the treatment optimization status or the decision-making process.
- Non-blinded investigators have full knowledge of both treatment optimization and the decision-making process.
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults aged ≥18 years.
- •Diagnosis of rheumatoid arthritis according to either the 1987 American College of Rheumatology (ACR) criteria or the 2010 ACR/EULAR classification criteria.
- •Clinical remission for at least 6 months prior to the baseline visit, defined as DAS28-CRP < 2.
- •Receiving biological anti-TNF therapy (infliximab, adalimumab, etanercept, golimumab, or certolizumab).
- •Ability and willingness to provide written informed consent to participate in the study.
排除标准
- •Patients in whom biological therapy was prescribed due to systemic manifestations of rheumatoid arthritis.
- •Patients with rheumatoid arthritis and any known associated condition that may interfere with the assessment of study outcomes (e.g., fibromyalgia or concomitant chronic inflammatory diseases).
- •Patients receiving chronic anti-TNF biological therapy who are already undergoing treatment tapering or are on reduced or extended dosing regimens prior to study inclusion.
结局指标
主要结局
Percentage of patients maintaining sustained remission.
时间窗: Up to 12 months
Proportion of patients who remain in sustained remission throughout the entire follow-up period, defined as DAS28-CRP \< 2.6 based on tender joint count (28 joints), swollen joint count (28 joints), C-reactive protein levels, and patient global assessment
Incidence of adverse events
时间窗: Baseline to 70 days after last dose
Incidence and characteristics of adverse events, including serious infections requiring systemic antibiotics or hospitalization, serious treatment-related adverse events, and specific adverse reactions (e.g., infusion or injection reactions), including severity.
次要结局
- Proportion of patients achieving sustained acceptable therapeutic target(Up to 12 months)
- Proportion of patients experiencing disease flare (0-6 months).(Up to 6 months)
- Proportion of patients experiencing disease flare (6-12 months)(6 to 12 months)
- Number of disease flares (0-6 months)(Up to 6 months)
- Number of disease flares (6-12 months)(6 to 12 months)
- Proportion of patients achieving acceptable therapeutic target at final visit(At 12 months)
- Proportion of patients in remission at final visit(At 12 months)
- Time to disease flare(Up to 12 months)
- Change in clinical disease activity parameters (joint count)(Baseline to 12 months)
- Change in clinical disease activity parameters: patient and physician global assessment(Baseline to 12 months)
- Change in clinical disease activity parameters: CRP(Baseline to 12 months)
- Change in health-related quality of life (EQ-5D-5L)(Baseline to 12 months)
- Change in functional disability (HAQ)(Baseline to 12 months)
- Direct and indirect healthcare costs and cost-effectiveness(Up to 12 months)
- Use of concomitant medication(Up to 12 months)
- Radiographic structural damage progression(Baseline and 12 months)
- Change in clinical disease activity parameters (ESR)(Baseline to 12 months)
研究者
Francisco J. Blanco
Principal Investigator
Hospital San Carlos, Madrid