Several gene therapy trials for Duchenne muscular dystrophy initiated in 2018. Trial decision making is complicated by non-curative, time-limited benefits; the progressive, fatal course; and high unmet needs. Here, caregivers and patients prioritize factors influencing decision making regarding participation in early-phase gene therapy trials.
Objects used to construct choice sets for the best-worst choice experiment
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Object
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Description
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Chance of improved muscle function
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Data are positive about the chance of maintaining, and maybe improving,
muscle function.
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Chance of improved heart function
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Data are positive about the chance of maintaining, and maybe improving,
heart function.
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Chance of improved lung function
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Data are positive about the chance of maintaining, and maybe improving,
lung function.
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Benefit
lasts about 10 years
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Data suggest that gene therapy will last for 10 years. It may be shorter
or longer, but no one knows. It is currently not possible to give a second
dose of gene therapy. It may be possible in the future, but no one knows.
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Chance of being in placebo group
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The trial uses a placebo group, where some participants are randomly
assigned to a group that gets an inactive (fake) treatment. People who get
placebo during the trial would be eligible for gene
therapy in the future.
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Lowest dose may be too low for benefit
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One of the trial’s goals is to test the right dose of gene therapy. If
participants get a dose that is too low to work, they will not get
another chance to use gene therapy.
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Two muscle
biopsies required
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Being in the trial requires 2 muscle biopsies (one from the arm and one
from the leg) to test for dystrophin production.
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Not eligible for future trials
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People who get gene therapy will most likely not be eligible for other
clinical trials for the rest of their lives. It may someday be possible, but no one knows.
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Limits later use of gene therapies or CRISPR
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People who get gene therapy may not be able to use some newer types of
gene therapy or gene editing (like CRISPR) for the rest of their lives. It may someday be possible, but no
one knows.
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Chance of
long hospitalization
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Data suggest a low risk of needing a long hospitalization of 4 weeks or
more to recover from serious side effects.
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Chance of death (low risk)
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Data suggest a very low risk of death soon after using gene therapy. That
risk should be even lower than we showed you in the first survey task.
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Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients
