ALS Observational Studies

iAdds context

“Your ALS/MND team or neurologist is a starting point for finding registries and studies, and may run studies you can join.”

Tells people to begin with their own neurologist and ask whether their ALS clinic is a study site, and says that if it is, taking part may be built into normal clinic visits. That reaches the enrolling half of the sentence; it does not say that teams know which registries are available locally.

Start by asking your neurologist if your ALS clinic is a study site.

Where can I find observational studies?

!Qualifies this

“Many registries ask mainly for information, sometimes updated over time, rather than for visits or treatments.”

Its description of registry studies is survey-based, which fits. But the same page says studies of this kind vary a lot in what they ask, and its list of what participation can involve includes travelling for in-person assessments and giving blood, saliva or urine. So the low-burden picture holds for many but not for all.

Some require in-person visits, some can be done completely from home, and others are hybrid.

Will I need to travel?

✓Supports this

“Information contributed to registries helps researchers understand how ALS/MND varies and how it progresses, and helps in finding and testing treatments.”

Treats registry studies as one kind of observational study and says what the whole group of them is for: learning about the causes, the different types and the progression of ALS. It goes on to say that this understanding helps identify biomarkers and improve clinical trials, which is the finding-and-testing half of the sentence.

Observational studies help researchers learn more about ALS, including potential causes, its different types, and how it progresses.

Why are observational studies so important?

✓Supports this

“Joining a registry involves an informed consent step: you are given information about it and asked to agree before you take part.”

Says a signed informed consent form is required for every study of this kind, and that the form is where you are told how your data will be used and what the team will do to protect it. It also says the potential risks of taking part are listed in that same form.

Every study requires you to sign an informed consent form that will contain information about how your data will be used and what the study team will do to protect it.

Are there different types of observational studies?

✓Supports this

“Information passed to researchers is normally handled so that they cannot directly identify you.”

States the same expectation, in terms of the outcome rather than the method: data shared with researchers should not reveal who you are. It does not use the words de-identified or coded, and it does not describe how this is done.

Data collected in observational studies should be shared with ALS researchers in a way that doesn't reveal your personal or medical information.

Are there different types of observational studies?

✓Supports this

“You should be able to ask questions about a registry, find out how your data is handled, and withdraw.”

Covers all three: it tells people to ask the study team how they handle personal data if they have questions or concerns, and it says taking part is voluntary and can be stopped at any time. It does not say what happens to data already collected, which is the question the Compass bullet goes on to advise asking.

Participation in observational studies is voluntary, so if needed, you can withdraw at any time.

Is participation right for me?

✓Supports this

“Registries differ in what they ask of you, from sharing information only, through giving samples, to more involved participation.”

Answers the question of what taking part involves by saying it depends on the study, then lists the range: online questionnaires, submitting medical records, travelling for in-person assessments such as physical exams or breathing tests, giving periodic blood, saliva or urine samples, and recording your voice.

This depends on the specific study.

What will participation involve?

✓Supports this

“You can usually take part in more than one registry or study at the same time.”

Says you should be able to take part in several observational studies at once, and that enrolling in more than one advances research further because different studies collect different information. It adds one caution: some large studies pool participant data in the same database, so it is worth asking the teams whether joining both would just duplicate data.

You should be able to participate in multiple observational studies at the same time.

Can I enroll in more than one observational study?

✓Supports this

“Taking part in research means being given information and asked to consent, and being able to withdraw at any time.”

Says every study requires you to sign an informed consent form setting out how your data will be used and how the team will protect it, and that taking part is voluntary and can be stopped. Written about observational studies rather than about every kind of research.

Participation in observational studies is voluntary, so if needed, you can withdraw at any time.

Is participation right for me?

✓Supports this

“Taking part in research helps move ALS/MND research forward.”

Sets out the chain this sentence is describing, for observational studies, which is the kind of study most biomarker research uses. It says taking part helps researchers learn what causes ALS and how it progresses, and that this in turn helps identify biomarkers and improve clinical trials. It does not cover taking part in a treatment trial.

Observational studies help researchers learn more about ALS, including potential causes, its different types, and how it progresses.

Why are observational studies so important?