Clinical trials are the most reliable – and ultimately the fastest – way to translate promising laboratory science into new and better ways to treat and care for people living with ALS/MND. Researchers use clinical trials to find out whether new treatments are safe and beneficial for people living with ALS/MND. Clinical trials also help find better ways to prevent, detect and diagnose ALS/MND; and improve quality of life through things like assistive technologies or social support.
Participation of people living with ALS/MND is essential for clinical research. Individuals who enroll in a clinical trial are contributing to improved care for everyone living with the disease. Participation often comes with a commitment and burden to the person living with ALS/MND, and caregivers and families. Participating in trials may require individuals living with ALS/MND to travel long distances to trial sites, make more frequent trips than their care typically involves, adjust their standard of care medications, and undertake considerable time and financial commitments. Additionally, people living with ALS/MND are bearing the physical risks and psychological burden of being on an experimental therapy or placebo.
Trial sponsors have the responsibility towards people living with ALS/MND to do the right thing given the sacrifice and toll it takes to participate in often multi-year trials.
The International Alliance’s Scientific Advisory Council offers the following five recommendations that a trial sponsor should do after the clinical trial is complete to ensure the expectations of participants are met.
1. Disseminate the results of the trial quickly and responsibly: Trial sponsors should promptly convey the results of the trial to the community, regardless of whether the results are positive or negative.
Most sponsors provide top-line results through a press release. However, we recommend that additional approaches, such as directly contacting trial participants, publishing in peer-reviewed publications, presenting at scientific meetings, and holding patient webinars, should be undertaken to inform trial participants of the results of the study.
Communicating trial results in ALS/MND requires a balance of transparency and sensitivity. Below are some suggestions on how to structure the communication:
- Trial sponsors should present findings clearly and factually, avoiding overly optimistic language that can unintentionally foster false hope.
- When sharing biomarker results, sponsors should clearly communicate the biomarker’s context of use, its potential relevance to disease progression or treatment response, and whether it is a novel discovery, ensuring expectations are grounded in the current stage of validation and practical applicability.
- Sponsors should emphasize what the data definitively show, clearly acknowledge limitations and where post hoc analysis is used, and frame early-phase results as part of an ongoing scientific process rather than a breakthrough.
- Including patient advocates or clinicians in communication planning can guide messaging that resonates with the ALS/MND community while fostering realistic expectations. While social media has a place in creating awareness of clinical research and its outcomes, the use of influencers by sponsors to promote investigational therapies is strongly discouraged.
2. Share data and samples with the research community: Many people living with ALS/MND participate in trials with the expectation that their samples and data be widely used for research. Data and sample sharing is not just good practice, but in the health research context, it allows us to replicate, validate or develop new hypotheses and leads to more research engagement and improvement of patient care.
Sponsors should ensure that appropriate consent and deidentification processes are in place, so that data and samples can be shared, and that data ownership and data security are safeguarded. Sponsors should also promptly share data to open-source platforms and share biosamples through biorepositories whenever possible and authorized by the participants.
3. Clear guidance on access to treatment outside of the blinded trial: When clinical trials end, many people living with ALS/MND continue to get access to experimental treatment under an open label extension (OLE) or compassionate use (also known as Expanded Access, or EA). EA may also be available, unattached to a specific trial, in any phase of drug development.
Sponsors need to be clear upfront if they will be providing long-term access to experimental therapy through an OLE or EA pathway and for how long. They also need to be clear as to how participants will be selected, regardless of whether they were participants in the blinded trial or not. Clinics and telehealth organizations offering EA should inform prospective participants in advance if EA spots are limited, helping them avoid unnecessary time and expenses to become eligible through that site or organization.
We concede that for many sponsors, OLE and EA programs may be cost prohibitive. We recommend that sponsors take these costs into consideration when they are fundraising or building capital for the blinded trial so that such programs can be offered in a transparent, equitable, and objective manner. Sponsors should also inform participants prior to starting the blinded trial if OLE and EA programs will not be offered.
4. Share short-term and long-term development plans: ALS/MND is a devastating disease with few options for disease-modifying treatments. As such, people living with ALS/MND are always hopeful that new experimental treatments continue to move forward in the clinical trial pipeline toward approval. It is important for sponsors to describe the regulatory next steps that the company will undertake, regardless of whether the trial hit its endpoints, didn’t meet them, or there are subgroups that may have benefited from the therapy.
We recommend sponsors to do patient and lay-friendly and transparent presentations (see point#1) on what the results were and how it impacts their regulatory strategy. Sponsors should delineate if they are going to pursue regulatory approval and in which countries and which pathways are available to them to pursue future approval.
5. Unblind the participants: Blinding is essential for clinical trials to remove bias that can be caused if participants or the research team is aware of who is receiving an active or placebo treatment. Blinding is also important if a trial sponsor continues to collect data through an OLE period to assess the longer-term safety and therapeutic potential of their treatment.
However, once the trial and OLE period is officially closed, or the sponsor has an approval decision from regulatory agencies, participants should be promptly unblinded and informed as to their original treatment (e.g., drug or placebo) in the blinded portion of the trial. With the addition of numerous platform and adaptive trial designs in ALS/MND, there may be challenges in unblinding participants promptly, and in these cases participants should be informed in advance of unblinding timepoints and expectations.
International Alliance of ALS/MND Associations
April 2025
The original language of communication is English and any translation cannot be guaranteed for accuracy of messaging.