US Hereditary Angioedema Association


IN THIS ISSUE
HAE IN-MOTION^® Quality of Life Survey HAEA Round Table Women With HAE Health Team Update Physician Spotlight	Community Blog HAE Speaks Podcast #BeyondHAE Youth Podcast Clinical Trial Updates Education Webinar Virtual Meet & Greets

Get ready to raise HAE awareness with our 2022 HAE IN-MOTION^® Virtual Challenge!

Grab your sneakers and rally your friends and family to raise awareness for HAE while being active through the HAE IN-MOTION^® Virtual Challenge. Registration opened on June 1st!

This fun HAEA event will take place from June 1st through July 31st, and there is no cost to participate! Challenge yourself and help us reach our goal of completing 50,000 miles by July 31st to spread HAE Awareness.

Walk, run or bike, invite your family and friends and compete for awards!

If you have any questions, please contact Mike Mallory at mikemallory@haea.org.

The US HAEA is Sponsoring a Research Survey that will help You and Your Physician Better Manage Your HAE

Dear HAEA Friend,

I am reaching out to personally invite you to participate in an important survey to gather the information needed to ensure that the HAEA quality of life (QoL) tool provides practical data that you and your physician can use to better manage your Hereditary Angioedema (HAE) over time.

This HAEA-initiated survey involves a three-step process, first by completing this first initial survey, and completing two shorter follow-up surveys in July and September. The HAEA will send you separate invitations to participate in the follow-up surveys once you complete the initial survey.

The success of this HAEA-initiated research effort depends on the involvement of all people with HAE!

The time commitment for this study involves completing an anonymous online survey with a total time commitment of approximately 35-40 minutes. We recognize that your time is valuable, so we will send you a check for $40 after you fully complete the survey.

To participate in this important research study, please reach out to the HAEA Research Team at research@haea.org with any questions or for more information.

We thank you in advance for your willingness to participate in this unique and timely research effort designed to benefit the HAEA community!

My warmest regards,

Tony Castaldo

Anthony Castaldo
US HAEA President & CEO

HAEA Round Table

Join us at the HAEA Round Table, where members of our community discuss how they have dealt with the unique challenges of HAE. The HAEA Round Table events are broadcast on Facebook Live, so make sure to tune in to the US HAEA official Facebook page to learn more about how others manage their HAE! The next event in the HAEA Round Table series will be on Tuesday, June 14th at 6:30 PM ET. This event will feature children ages 16-20 and will showcase their answers to questions related to their personal experiences in living with HAE.

HAEA Round Table Calendar:

• June 14th
For Teens and Young Adults with HAE Ages 16-20 - How Do You Talk to Others About Your HAE?
• August 9th
For Young Adults with HAE - Growing Into Adulthood with HAE
• October 11th
For BabyBoomers with HAE - How Does HAE Affect Aging?
• November 1st
For Caregivers and Parents - What is the Best Way to Support Someone with HAE?

If you missed the February and April HAEA Round Tables, you can access the recordings here!

Women with HAE - Are you considering starting a family?

Join HAE expert physician Sandra Christiansen for a special webinar on Family Planning and Pregnancy

Connect with Dr. Christiansen and HAE Advocate, Lisa Facciolla, on Tuesday, July 26 at 7:30 PM ET / 4:30 PM PT, for the second of the Women with HAE special series webinars: Family Planning & Pregnancy. The event will be a great resource for anyone who is interested in learning more about what to consider when planning for a family, and what women with HAE should expect and plan for during pregnancy.

We invite you to submit questions in advance to Lisa Facciolla at lisa@haea.org, and Dr. Christiansen will answer these live during the webinar.

Individuals who register for this event will be mailed a FREE printed copy of the recently released Women with Hereditary Angioedema guidebook that has been developed to help women and young girls with HAE navigate three specific life stages:

1) pediatrics through puberty
2) family planning and pregnancy
3) menopause and aging

Order your copy of the Women and HAE guidebook here.

For more information or questions on the Women and HAE guidebook or the pregnancy webinar, please contact Lisa Facciolla at lisa@haea.org.

Health Team Update

Are You Struggling with Insurance Reimbursement Claims?

A recent HAEA survey found that 50% of our community experienced medication reimbursement issues in the past two years.

Are you one of them?

To help the HAEA community navigate these insurance hurdles, we developed Insurance Reimbursement Guidebooks. There are two versions of the Guidebook available; one is tailored for people living with HAE and the other is designed for Healthcare Professionals.

You can download a Reimbursement Guidebook, or order a hard copy that will be mailed to you if you visit this page. From the same page, you can also download or order a version for Healthcare Professionals and share it with your HAE treating physician.

The HAEA Health Team is always here for you! Please reach out to us at 866-798-5598 for assistance with 1.) insurance issues, 2.) finding an HAE expert physician, 3.) updating your member information, 4.) joining a virtual support group, or 5.) an HAE-related question.

Throughout 2022, the US HAEA is spotlighting HAE treating physicians who have made an impact within the HAE community.

This month, we will be recognizing HAE treating physician, Katharine Woessner, MD!

"My one piece of advice to members of the HAE community is to continue to advocate and educate. There always remains an under-appreciation of the impact of these rare diseases on patients and their families. Despite increased awareness and multiple therapies, there are still patients who are not being diagnosed."

Thank you Dr. Woessner for your dedicated service to the HAE community!

"I embraced my diagnosis and I started educating everyone I knew and every doctor that I saw about HAE. Getting a diagnosis was empowering, and finally being able to treat my attacks with medication was incredibly freeing. I finally felt like myself again."

You can read the rest of Casey's HAE journey and her experience receiving a rare disease diagnosis in this month's featured HAEA Community Blog article!

Access the HAEA Community Blog today here.

Do you have a unique story or opinion to share about your HAE? The HAEA is excited to share your experiences in your own words with the HAEA community! You can share your story here.

Contact HAE Advocate, Hannah Carroll at hannah@haea.org for more information!

Podcasts

HAE Speaks Podcast

Download and listen to HAE Speaks, a podcast where people with HAE get the chance to broadcast their stories to the world! HAE Speaks gives you the opportunity to learn more about your own HAE through the shared experiences presented by our monthly hosts. You can download HAE Speaks on Apple Podcasts, Spotify, or wherever you listen to your podcasts!

Episode 26: The 2022 HAE IN-MOTION^® - Step it up for HAE virtual event registration is open. In this episode, hear tips, tricks, and advice from a past participant.

LISTEN NOW

Do you have a topic, question, or idea that you would like us to cover? Send your suggestion to Mike Mallory at mikemallory@haea.org.

#BeyondHAE Youth Podcast - Managing my HAE in Middle School

Youth Podcast series

Episode 39 welcomes youth host Paige, a young person with HAE who talks about how she copes with her attacks and her experience with attacks while in middle school. Paige shares how she has overcome embarrassment and interviews a couple of friends who talk about how they support Paige when she swells. Paige wraps up her episode with some advice to others who are not comfortable sharing their HAE diagnosis with others.

#BeyondHAE is the only podcast made exclusively by young people with HAE for young people with HAE. Listen to #BeyondHAE to hear different perspectives of how you can live a normal life with HAE. #BeyondHAE is available to download on Apple Podcasts, Spotify, or wherever you get your latest podcasts!

LISTEN NOW

Clinical Trial Updates

What is a Clinical Trial?

Clinical trials are research studies conducted with people affected by HAE to evaluate whether a drug is safe and effective in humans. They are one of the final stages of the medical research and development process and follow strict standards to protect the health and safety of participants. Clinical trials are a critical part of drug development and are required before a treatment can be approved by the Federal Drug Administration (FDA) and become available by prescription.

What are the benefits of participating in a Clinical Trial?

Clinical trials can offer access to HAE medicines at no cost to the participant for the duration of the trial, before it is available to everyone. Participation in a clinical trial also provides regular touchpoints with an HAE physician throughout the trial period, and allows you to have a more active role in your health care. Your participation in a trial is your decision, and you have the ability to withdraw if the trial is not working for you, without judgment or jeopardizing your health care.

Following is a list of clinical trials currently underway. If you are interested in receiving more information on clinical trials, or are interested in participating in any of the trials listed below, please reach out to the HAEA Research Team at research@haea.org.

CSL312 Study

The US HAEA is currently assisting CSL Behring in recruiting people with HAE for the CSL312_3002, a worldwide clinical research study to investigate the safety and effectiveness of Garadacimab, an investigational subcutaneous medication given once a month to prevent Hereditary Angioedema (HAE) attacks. In this study, doctors will be evaluating the long term use of garadacimab.

The trial is currently fully recruited for adult participants, however, a few more adolescent participants (ages 12 - 17) are needed to meet the required numbers.

CSL312_3002 is an open label study with no placebo phase required, which means that all participants will receive garadacimab for at least one year.

RAPIDe-1 & CHAPTER-1 Studies

The US HAEA is currently assisting Pharvaris in recruiting people with HAE for two clinical research studies to investigate the safety and effectiveness of an investigational oral drug for both acute and prophylactic use of Hereditary Angioedema (HAE) attacks.

In the RAPIDe-1 study, doctors will be evaluating how effective three different doses of the study medication (PHVS416) are in relieving symptoms associated with HAE attacks. The study drug is in the form of soft capsules taken by mouth. In the CHAPTER-1 study, doctors will be evaluating the effectiveness of PHVS416 as a potential oral treatment to prevent HAE attacks.

To qualify for the RAPIDe-1 study, participants must be diagnosed with HAE Type I or II and be between the ages of 18 and 75 years old, with at least 3 attacks in the last four months or 2 attacks in the last two months.

To qualify for the CHAPTER-1 study, participants must be diagnosed with HAE Type I or II and be between the ages of 18 and 75 years old, with at least 3 attacks in the last 3 months or 2 qualifying attacks during the screening period. The use of an approved on-demand medication to treat acute attacks is permitted.

The OASIS Study

The US HAEA is currently assisting IONIS in recruiting people with HAE for the OASIS-HAE study, a Phase 3 worldwide placebo-controlled trial, which aims to determine whether its study drug (called donidalorsen) is safe and effective at reducing or preventing HAE attacks.

Participants may be eligible to participate in an open-label extension study at the end of the OASIS-HAE study, where all participants receive donidalorsen.

To qualify for the OASIS-HAE study, participants must be 12 years of age or older with a documented diagnosis of HAE. Participants must experience at least two attacks during the screening period, and have access to, and the ability to use at least one or more acute medications to treat HAE attacks.

KOMPLETE & KONFIDENT Studies

The US HAEA is currently assisting Kalvista in recruiting people with HAE for the KOMPLETE and KONFIDENT studies to understand the effects of investigational oral medications called KVD824 and KVD900.

The KOMPLETE study is a placebo-controlled trial where doctors are studying three different doses of KVD 824-201 to see which one may be best as a safe prophylaxis treatment for HAE attacks. To qualify for the KOMPLETE study, participants must be 18 years of age or older; diagnosed with HAE Type I or II; have access to an on-demand medication to use for HAE attacks; and be willing to use a study-approved method of birth control during the study.

The KONFIDENT study is a placebo-controlled trial where doctors are studying two different doses of KVD 900 to see which may be best as a safe on-demand treatment for HAE attacks. To qualify for the KONFIDENT study, participants must be 12 years of age or older; diagnosed with HAE Type I or II; experienced two attacks in the last three months; have access to an on-demand medication to use for HAE attacks; and be willing to use a study-approved method of birth control during the study.

HAERMONY1 Study

The US HAEA is currently assisting BioMarin in recruiting people with HAE for the HAERMONY1 study, an investigational gene therapy called BMN 331 to improve HAE symptoms in adults 18 years of age and older diagnosed with HAE due to C1-INH deficiency.

To qualify for the HAERMONY1 study, participants must be 18 years of age or older; diagnosed with HAE due to C1-INH deficiency; have 6 months of a continuous prophylaxis or on-demand treatment regimen (prior to study enrollment); and a documented attack frequency of at least 1 attack per month on average. *Additional study requirements apply.

For those who qualify, participation lasts about 5 years after receiving the study drug. There are weekly and monthly visits during the first year. After the first year, the visits occur every three or every six months.

If you have HAE Type I, Type II, or HAE with Normal C1-Inhibitor and are interested in learning about new clinical studies to prevent attacks, please contact an HAE advocate at 866-798-5598 to see if there will be an active clinical trial site in your area.

HAEA Treatment Education Series Webinar

Re-treating? Let's Talk About It.

Date: Thursday, June 16th, 2022
Time: 7:00 PM ET / 4 PM PT
Speaker: John Anderson, MD
Sponsored by: Pharming
Webinar Link: https://haea.zoom.us/j/89988813893
Phone Number: +1 646 558 8656
(Webinar ID: 899 8881 3893)

HAEA Virtual Meet & Greet

Are You Interested In Meeting Others In The HAE Community?

Do you have questions for others living with HAE or would like to network with other HAEA members? The HAEA Virtual Meet & Greet Events are a perfect way to reach out and get to know others affected by HAE!

Each month, HAEA staff will be presenting on programs, resources, and relevant HAE topics, as well as providing an opportunity for the HAE community to meet each other. If you are interested in participating in an HAEA Virtual Meet & Greet, please check out the HAEA Events Calendar, or reach out to Mike Mallory at mikemallory@haea.org.

What would you tell someone who is considering attending an HAEA Meet & Greet?

"It's a wonderful experience. You will find a place of love and support."
~ Brandie M.

Upcoming HAEA Virtual Meet & Greet Events

Tuesday, June 21st, 6:30 PM CT

Tuesday, July 19th, 6:30 ET


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The HAEA is an advocacy and research organization committed to actively engaging our community in a wide variety of grassroots activities that promote HAE education and awareness. We provide personalized services to address the unique needs of people with HAE and their families, which includes helping them secure access to and reimbursement for modern HAE medicines. Our great success in supporting clinical research has resulted in a variety of FDA-approved therapeutic options. We work closely with expert physicians to continuously upgrade our community's quality of life through improving diagnosis and knowledge of the condition, and encourage an individualized approach to selecting an optimal treatment. The HAEA is product and company neutral, and continues to enthusiastically support drug discovery research aimed at the next generation of HAE therapies. Copyright © (2022) US Hereditary Angioedema Association All rights reserved. No part of this publication may be reproduced, stored in a retrieval system, or transmitted in any form by any means, mechanical, electronic, photocopying, recording, or otherwise without the prior written permission of the US Hereditary Angioedema Association. For information, send an email to info@haea.org.