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20 Hereditary Angioedema Trials

Power is an online platform that helps thousands of Hereditary Angioedema patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:16+
Sex:All
60 Participants Needed
The goal of this trial is to enable the collection of information about long-term safety and clinical activity of STAR-0215 in participants with hereditary angioedema (HAE). Participants will receive repeat doses of STAR-0215 for up to 5 years.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
56 Participants Needed
This trial is testing donidalorsen, a medication for people with Hereditary Angioedema (HAE) Types I and II. The goal is to see if it can reduce the number of swelling attacks and improve quality of life. Donidalorsen works by targeting pathways in the body that cause these attacks. Donidalorsen treatment resulted in a significantly lower rate of angioedema attacks.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12+
Sex:All
144 Participants Needed
This trial tests BMN 331, a gene therapy given through an IV, for people with hereditary angioedema (HAE) who lack a specific protein. The therapy uses a virus to deliver a healthy gene to liver cells, enabling them to produce the missing protein. The study aims to see if this treatment is safe and effective over several years. BMN 331 is a gene therapy that uses a virus to deliver a healthy gene to liver cells, a method that has shown promise in treating various genetic disorders.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
44 Participants Needed
The goal of this clinical trial is to test the drug STAR-0215 in participants with hereditary angioedema (HAE). One group of participants will get 1 dose of STAR-0215, and two other groups will get 2 doses of STAR-0215. Researchers will study the effects of STAR-0215 in participants with HAE as this is the first time that the drug has been given to participants with HAE.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
28 Participants Needed
This trial is testing a medication called KVD900 in teenagers with a rare condition that causes sudden swelling. The goal is to see how the medication behaves in their bodies and if it can help manage their symptoms.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12 - 17
Sex:All
12 Participants Needed
This trial tests the long-term safety of KVD900 in individuals with HAE. KVD900 aims to reduce symptoms by blocking proteins that cause swelling. Another treatment, Berotralstat, is used to prevent HAE attacks.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:All
145 Participants Needed
This phase 3b study will evaluate long-term safety and efficacy of CSL312 (also known as garadacimab) when administered subcutaneously (SC)
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:All
171 Participants Needed
The purpose of this study is to investigate the safety, PK / PD, and efficacy of SC CSL312 for prophylactic treatment of pediatric subjects with HAE.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2 - 11
Sex:All
20 Participants Needed
This trial uses donidalorsen, given as an injection under the skin, to help people with hereditary angioedema. The goal is to reduce the frequency and severity of their swelling episodes.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
20 Participants Needed
KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old with HAE Type I or II.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:2 - 11
Sex:All
24 Participants Needed
Prospective, multicenter, randomized, double-blind, parallel group, placebo- controlled, efficacy and safety phase 3 study of an intravenous human plasma- derived C1 esterase inhibitor (C1-INH) concentrate in participants with congenital C1-INH deficiency for the treatment and pre-procedure prevention of acute hereditary angioedema attacks
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:2+
Sex:All
124 Participants Needed
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2-period, 2-treatment cross-over study to evaluate the efficacy and safety of orally administered deucrictibant compared to placebo for the on-demand treatment of HAE attacks, including non-severe laryngeal attacks, in participants ≥12 to ≤75 years of age with HAE type 1 or type 2 (HAE-1/2), a proportion of whom are using long-term prophylactic medication for HAE.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12 - 75
Sex:All
120 Participants Needed
This study evaluates the safety and efficacy of PHA-022121 administered orally for prophylaxis against angioedema attacks in patients with hereditary angioedema (HAE). The study consists of 2 parts, with patients completing participation in Part 1 prior to initiation of treatment in Part 2. Part 1 of the study has 3 parallel arms and approximately 30 patients will be equally randomized to one of two dose regimens of PHA-022121 or matching placebo. Patients will continue to the single open-label arm in Part 2 of the study after completion of Part 1. The screening period is up to 8 weeks and the treatment periods are 12 weeks (Part 1) and 30 months (Part 2) in duration.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18 - 75
Sex:All
34 Participants Needed
This study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks, in participants with HAE type 1 or type 2 (HAE-1/2). The study will enroll patients from Study PHA022121-C201 (NCT04618211) and Study PHA022121-C306 (NCT06343779) who elect to participate in this extension study and meet the eligibility requirements.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2, 3
Age:12+
Sex:All
140 Participants Needed
Image of trial facility.

BW-20805 for Angioedema

Hershey, Pennsylvania
A Phase 2 in Adult Subjects with Hereditary Angioedema
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 2
Age:18 - 70
Sex:All
24 Participants Needed
Image of trial facility.

Deucrictibant for Angioedema

Saint Louis, Missouri
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of once-daily orally administered deucrictibant extended-release tablet compared to placebo for prophylaxis to prevent angioedema attacks in participants aged ≥ 12 years with hereditary angioedema.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12+
Sex:All
81 Participants Needed
The purpose of this study is to evaluate the pharmacokinetics (PK) and safety of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to \< 12 years old for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2 - 11
Sex:All
29 Participants Needed
Image of trial facility.

Navenibart for Angioedema

Little Rock, Arkansas
This is a Phase 3 multicenter, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of subcutaneous administration of navenibart in adult and adolescent participants with type 1 or type 2 hereditary angioedema (HAE). The goal of this clinical trial is to evaluate the efficacy and safety of navenibart compared to placebo in preventing HAE attacks in participants with HAE.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12+
Sex:All
145 Participants Needed
This study is designed to evaluate the safety after switching to garadacimab from another prophylactic hereditary angioedema (HAE) treatment (marketed kallikrein \[KK\] inhibitor or plasma-derived C1-esterase inhibitor \[pdC1INH\]prophylactic) when administered once monthly for approximately 3 months in participants aged greater than or equal to (\>=) 12 years with HAE.
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 4
Age:12+
Sex:All
30 Participants Needed
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Frequently Asked Questions

How much do Hereditary Angioedema clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Hereditary Angioedema clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Hereditary Angioedema trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Hereditary Angioedema is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Hereditary Angioedema medical study ?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Hereditary Angioedema clinical trials ?

Most recently, we added Garadacimab for Hereditary Angioedema, Navenibart for Angioedema and BW-20805 for Angioedema to the Power online platform.