Making clinical trials more comparable, consistent, and focused on the biological mechanisms underlying the onset and development of multiple sclerosis (MS) are all key to more quickly realizing new treatments for progressive MS, according to the International Progressive MS Alliance.
To attain these goals, the Alliance, a worldwide collaborative organization committed to the speedy development of new therapies for progressive forms of MS, published a series of recommendations on how early MS trials should be designed and conducted.
In addition to changes in trial design and protocol, the Alliance proposed a funding program to support Phase 2a MS trials into the safety and efficacy of treatment candidates. The Alliance will directly support this new funding scheme, and make its money available to studies following the organization’s recommendations.
Alliance recommendations and plans for its funding program are described in the paper, “Facing the urgency of therapies for progressive MS — a Progressive MS Alliance proposal,” published in the journal Nature Reviews Neurology.
“This paper is an important milestone in the Alliance’s goal of accelerating new treatments for people with progressive MS,” Alan Thompson, MD, chair of the group’s scientific steering committee, said in a press release from the National MS Society.
“We look forward to getting feedback from stakeholders so that our plans for a new funding program will do even more to speed this work,” Thompson added.
Phase 2 trials are smaller studies that normally seek to validate the safety and explore the preliminary efficacy of investigational therapies, before they move to be tested in larger Phase 3 studies that aim to generate convincing data supporting their approval.
The type of outcomes selected to assess whether a potential therapy is beneficial or not for a given disorder varies greatly. But this key choice has a direct impact on the design of Phase 2a trials, and influences the launch of the pivotal Phase 3 studies that depend on their success.
According to the Alliance, it is essential that early Phase 2 trials of potential of new MS treatments are comparable and consistent in the way they are conducted to best ensure success. These studies should also focus on expanding knowledge of the cellular and molecular mechanisms underlying MS to further treatment development.
An expert group, called the Alliance’s experimental medicine trial implementation planning team, released a series of recommendations on how these early trials should be designed and conducted.
According to the team, studies should be less restrictive on their eligibility criteria, and rely more on feedback from MS patients regarding the feasibility of their design.
Trials should also include a range of disease measures on top of a core set of clinical and exploratory outcomes — assessments of physical disability, cognition, mobility, and vision; and gathering data on gene activity — to evaluate the potential benefits of the therapy being tested.
Core outcome measures in MS trials should also include several markers of disease activity, and other biological response indicators of the therapy’s likely mechanism of action.
These studies should take advantage of advanced trial designs, testing different potential therapies at the same time, as well as in combination with approved MS medications.
In its paper, the Alliance also proposes establishing a funding program to offer grants to early Phase 2 trials of MS therapies that follow its recommendations regarding trial design.
The organization now welcomes feedback from the MS community on these recommendations before releasing applications.
“The objective of the International Progressive MS Alliance is to expedite the development of effective therapies for progressive MS through new initiatives that foster innovative thinking and concrete advancements,” the researchers wrote. “We are soliciting public and academic feedback, which will help shape the programme and future strategies of the Alliance.”
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