Thoughts on the drugs involved in the StratosPHere trial

Respondents were asked to read short information sheets about the two drugs, phenylbutyrate and hydroxychloroquine, that will be used in the StratosPHere trial.

Phenylbutyrate 50%

said they would be prepared to take this drug as part of a clinical trial.

20% Yes, definitely 30% Yes, probably 37% I’m not sure 10% No, probably not 4% No, definitely not

Respondents were asked to list any concerns they may have about taking phenylbutyrate. Themes included:

• Side effects • Interactions with other drugs • Liquid form is inconvenient • Co-morbidities (other health conditions)

Hydroxychloroquine 54%

said they would be prepared to take this drug as part of a clinical trial

23% Yes, definitely 31% Yes, probably 23% I’m not sure 17% No, probably not 6% No, definitely not

Respondents were asked to list any concerns they may have about taking hydroxychloroquine.

Themes included: • Side effects • Interactions with other drugs • Severe allergy • Pre-existing eye disease

Some respondents were already taking the drug for another condition.

Most people said they would prefer to take a new drug for PH in tablet form. Preferences were as follows: 1. As a tablet 2. As a capsule 3. As liquid medicine 4. Using a nebuliser 5. Using intravenous infusion Respondents were asked how often they would be willing to take a new medication. Preferences were as follows: 1. Once a day 2. Twice a day 3. Three times a day 4. Four times a day infusion