Purpose of the RAPIDe-3 Study

The RAPIDe-3 Study is a clinical research study for people living with hereditary angioedema (HAE) type 1 or 2. The study is evaluating the safety and efficacy of an investigational oral drug called deucrictibant as a potential on-demand treatment for HAE attacks as compared to a placebo (a substance that looks just like deucrictibant but contains no medicine).

Who may qualify

To be considered for participation in the RAPIDe-3 Study, interested individuals must:

  • Be between 12 and 75 years of age
  • Have a confirmed diagnosis of HAE type 1 or 2
  • Have had at least two HAE attacks within the past three months
  • Have had experience using standard-of-care treatment to manage on-demand treatment for HAE attacks

There are additional eligibility requirements that the study doctor can explain to interested individuals.

Study Participation

Participation in the RAPIDe-3 Study will vary in duration based on how long it takes for a participant to experience and treat two qualifying HAE attacks with the study drug (either deucrictibant or placebo). Participants will be remotely guided by the study doctor when treating two HAE attacks with the study drug in their home setting. They will have a maximum of four study center visits, as well as two telephone interviews of one hour each, within 10 days after an attack treated with the study drug.

Screening

Time Commitment

On-site: ~3 hours

At home: ~3 hours

Time Commitment

On-site: ~3 hours

At home: ~3 hours

Potential participants will undergo screening to confirm eligibility for the study.

Day 1 visit

Time Commitment for Adults: ~30 minutes

Time Commitment for Adolescents: ~9 hours

Time Commitment for Adults: ~30 minutes

Time Commitment for Adolescents: ~9 hours

If eligible, participants will be enrolled in the study to treat two qualifying HAE attacks (one with deucrictibant and one with placebo).

For adolescent participants: Adolescent participants will get one dose of deucrictibant at this visit while in a non-attack state, followed by multiple blood draws over the course of eight hours to check how their body is affected by the active study drug. Adolescent participants will then be enrolled in the study to treat two qualifying HAE attacks (one with deucrictibant and one with placebo).

Treatment phase: treatment of two qualifying attacks

Time Commitment for All Participants: 2 remote calls

Time point questionnaires: multiple within 2 days

Time Commitment for All Participants: 2 remote calls

Time point questionnaires: multiple within 2 days

For all participants: Upon experiencing symptoms of an HAE attack, participants will consult with their study doctor, who will determine if the participant should take the study drug. Participants will answer a series of questionnaires on their handheld device before taking the study drug and at various time points for up to two days after treatment. These will take about 2 to 3 minutes to complete each time.

If the study drug does not provide enough symptom relief during an HAE attack, the participant may contact their study doctor again to discuss how they feel and to find out if they should take a second dose of the study drug. However, if needed, the participant may use their own prescribed HAE medication or study-provided icatibant.

Follow-up visits after each treated HAE attack

Time Commitment for All Participants

Post-attack Visit 1: ~15–30 minutes

Post-attack Visit 2 (end-of-study): ~30 minutes to 1 hour

Time Commitment for All Participants

Post-attack Visit 1: ~15–30 minutes

Post-attack Visit 2 (end-of-study): ~30 minutes to 1 hour

For all participants: Following each HAE attack treated with the study drug, participants will have a study visit to check their health and a one-hour interview will be conducted by telephone or an online communication platform such as Webex. The second follow-up visit will also be the end-of-study visit.

After study participation

After completing the RAPIDe-3 Study, participants may be eligible to join an open-label extension (OLE) study, called the RAPIDe-2 Study, to continue receiving deucrictibant to treat their acute HAE attacks at no cost. This OLE study is planned to continue until availability of commercial supply or another means of continued treatment can be provided.

See if you may qualify