Home / What are Umbrella Studies? A New Approach in Clinical Research
In the realm of clinical research, innovation is constant and crucial. One recent innovation is the “umbrella study,” a streamlined approach to gathering comprehensive data. During a recent discussion with Grace Tougher, Study Coordinator Team Lead, we delved into the mechanics and benefits of umbrella studies, using the Merino study as a prime example.
An umbrella study combines a healthy volunteer study and a patient study into a single protocol or study methodology. This integration not only speeds up the research process but also simplifies the ethics approval procedures, making it a more efficient approach for sponsors.
From a sponsor’s perspective, the primary benefit of an umbrella study is speed. By eliminating the need to submit a new protocol for the patient phase, sponsors can continue collecting data without interruption. This method accelerates the research timeline, providing quicker access to vital data that can drive decisions on the efficacy and safety of new treatments.
The Merino study is an exemplary case of an umbrella study. It focuses on four disease areas: Myasthenia Gravis (MG), and three dermatological conditions—Bullous pemphigoid (BP), Pemphigus vulgaris (PV)/Pemphigus foliaceus (PF), or
Cutaneous lupus erythematosus (CLE).
Initially, the study involved healthy volunteers and having completed this phase, it now transitions to patients.
The healthy volunteer portion of the Merino study has been completed, and preparations are underway for the patient portion.
The primary objective of the Merino study is to assess the safety and tolerability of the investigational drug, aimed at lowering immunoglobulin (Ig) levels to manage autoimmune diseases. The study measures the presence of the drug in the blood and its impact on Ig levels, using various assessments including blood tests, monitoring vital signs, ECGs, and disease-specific evaluations.
The patient portion involves weekly dosing for 12 weeks, followed by a period of monitoring. While the healthy volunteers received single or multiple doses, patients will receive the same dosage, and will all receive the study drug (no placebo).
Recruitment is often a significant challenge, especially when dealing with specific disease criteria. For the Merino study, patients must not be too mild or too severe in their condition. Additionally, some of the conditions targeted are more prevalent in older populations, which adds another layer of complexity to recruitment. Collaboration with dermatologists and neurologists for patient referrals is crucial to identify and screen potential participants.
From the healthy volunteer phase, common side effects included headaches and injection site reactions. These findings help inform what might be expected in the patient phase.
Merino study is the first time the specific medication is being tested, although similar drugs from the sponsor have undergone prior trials. This phase is crucial for gathering new data to potentially bring the medication closer to FDA approval.
Umbrella studies represent a significant advancement in clinical research, offering a faster, more efficient pathway to gather essential data. The Merino study illustrates how this approach can be effectively implemented, with the potential to expedite the development of new treatments for various conditions. As clinical research continues to evolve, umbrella studies will likely become a staple protocol in the quest for medical advancements.