2024 Amyloidosis Forum Annual Meeting

October 23, 2024 | FDA White Oak Campus | Zoom

Participants including clinicians, researchers, FDA and global health authorities, industry partners, patients, and the Amyloidosis Research Consortium (ARC) will detail and highlight the advancements cultivated by Amyloidosis Forum working groups and outline goals and touchstones for future programs and research.

Public Private Partnership with FDA

Participants

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Adam Castaño, MD, MS
Endpoints Working Group
BIO+
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Ahmad Masri, MD, MS
Co-Chair, Imaging Standardization Working Group
BIO+
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Angela Dispenzieri, MD
Chair, Endpoints Working Group
BIO+
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Ashutosh Wechalekar, MD, FRCP, FRCPath, DM
Co-Chair, Prognostic Factors Working Group
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Christie Nie, MD
Endpoints Working Group
BIO+
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Cynthia Welsh, MD
Regulatory Subcommittee
BIO+
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Frederick L. Ruberg, MD
Imaging Standardization Working Group
BIO+
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Isabelle Lousada
Host & Moderator
BIO+
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James Signorovich, PhD
Chair, Federated Analytics Working Group
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Jan Griffin, MD
Prognostic Factors Working Group
BIO+
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Jersey Chen, MD
Endpoints Working Group
BIO+
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Joban Vaishnav, MD
Prognostic Factors Working Group
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John Vest, MD
Endpoints Working Group
BIO+
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Julian Gillmore, MBBS, MD, PhD
Endpoints Working Group
BIO+
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Kevin Alexander, MD
Federated Analytics Working Group Working Group
BIO+
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Kristen Hsu
Host & Moderator
BIO+
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Mathew S. Maurer, MD
Chair, Prognostic Factors Working Group
BIO+
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Michelle Carty, PhD
Co-Chair, Endpoints Working Group
BIO+
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Norman Stockbridge, MD, PhD
Endpoints Working Group
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Phaedra Johnson, MS
Co-Chair, Federated Analytics Working Group
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Preston Dunnmon, MD, MBA
Federated Analytics Working Group
BIO+
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Rajshekhar Chakraborty, MD
Prognostic Factors Working Group
BIO+
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Raymond Comenzo, MD
Endpoints Working Group
BIO+
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Rosalyn Adigun, MD, PharmD, MSc (FDA)
Host & Moderator
BIO+
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Sharmila Dorbala, MD, MPH, MASNC
Chair, Imaging Standardization Working Group
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Spencer Guthrie
Imaging Standardization Working Group
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Vaishali Sanchorawala, MD
Endpoints Working Group
BIO+
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Xin Jiang, PhD
Prognostic Factors Working Group
BIO+

Agenda

(Draft Agenda; Subject to Change)

8:45 am
Welcome & Introduction
8:55 am
Evolution of the Amyloidosis Forum
Speaker: Kristen Hsu

9:05 am
Moderators Introduction
Speaker: Sharmila Dorbala
9:10 am
Echocardiography repeatability study proposal
Speaker: Frederick L. Ruberg
9:20 am
Discussion
Discussants: Frederick L. Ruberg, Monica Stanley, Alex Hoffling, Libero Marcello, Lori Baylor, Spencer Guthrie
10:05 am
10-Minute Break

10:15 am
Moderators Introduction
Speaker: Mathew S. Maurer
10:20 am
AL Prognostic Factors + Disease Progression
Presenter: Raj Chakraborty
10:30 am
ATTR Prognostic Factors + Disease Progression
Presenter: Joban Vaishnav
10:40 am
Discussion
Discussants: Raj Chakroberty, Jioban Vaishnav, Jan Griffin, Gamze Tombak, Ricardo Rocha, Charu Gandotra, Xin Jiang
10:50 am
CPET Project Proposal + Questions for Feedback
Presenter: Jan Griffin
11:05 am
Discussion
Discussants: Raj Chakroberty, Jioban Vaishnav, Jan Griffin, Gamze Tombak, Ricardo Rocha, Charu Gandotra, Xin Jiang
11:35 am
AmyLite Detection of dLCCD Biomarker in Biofluids as a Quantitative Prognostic Biomarker in lambda AL Patients
Presenter: Xin Jiang
11:45 am
Discussion
Discussants: Raj Chakroberty, Jioban Vaishnav, Jan Griffin, Gamze Tombak, Ricardo Rocha, Charu Gandotra, Xin Jiang
12:10 pm
Lunch

1:10 pm
Moderators Introduction
Speakers: Angela Dispenzieri
1:15 pm
Hematologic Endpoints/Background + Proposal
Presenter: Angela Dispenzieri
1:30 pm
Incorporating KCCQ in AL Amyloidosis Clinical Trial Endpoints
Presenter: Vaishali Sanchorawala
1:45 pm
Renal Response and Progression in AL Amyloidosis
Presenter: Eli Muchtar
2:00 pm
Discussion
Discussants: Michelle Carty, Vaishali Sanchorawala, Eli Muchtar, Julian Gillmore, Justin Grodin, Sami Khella, Ray Comenzo, Adam Castano, Charu Gandotra, Christie Nie, Cristina Quarta, Jersey Chen, John Vest, Norman Stockbridge, Aliza Thompson
3:15 pm
10-Minute Break

3:25 pm
Federated Analytics and Learning from Patient Data
Speaker: James Signorovitch
3:30 pm
Ongoing NT-proBNP study via Federated Analytics
Presenter: James Signorovitch
3:40 pm
When is Federated Analytics workable and what is required?
Presenter: James Signorovitch
3:45 pm
Decline in Renal Function in ATTR-CM
Presenter: Julian Gillmore
4:00 pm
Discussion
Discussants: James Signorovitch, Kevin Alexander, Julian Gillmore, Jessica Vermuelen, Preston Dunnmon, Adam Castano, John Vest, Lori Baylor, Aliza Thompson

4:15 pm
Closing Remarks
4:25 pm
Adjorn

Recordings


Recording of the Morning Sessions

Recording of the Afternoon Sessions

How We Work

The Amyloidosis Forum has established a collaborative framework with appropriate stakeholders, including government, academic, scientific, patient, and industry organizations, to foster scientific collaborations. The Forum convenes regular meetings and has defined both workstreams and working groups to focus on key areas. The Forum is committed to publishing the outcomes from these initiatives.

Thank You to our Supporters

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Bronze

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About the Fourm

In 2019, the Amyloidosis Research Consortium established a public private partnership with the FDA, named the Amyloidosis Forum with the goal of bringing together the entire amyloidosis community to identify and bridge scientific gaps that are acting as barriers to drug discovery and development for the treatment of amyloidosis. The Forum has held a number of meetings over the past few years and have several workstreams currently ongoing, including work to develop a multidomain composite endpoint, evaluating the performance of key biomarkers as surrogate endpoints, and establishing pathways for the development of imaging measures to serve as endpoints in clinical trials for AL and ATTR amyloidosis. The work through the Amyloidosis Forum is a collaborative effort led by clinicians, researchers, patients, regulators across the US, Europe, and UK, and industry.

Learn More

 

Anonymous Reporting

The Amyloidosis Research Consortium (ARC) is dedicated to providing a harassment-free conference. To read our Safety Plan detailing our commitment or submit a secure, anonymous report of an incident, please use the corresponding buttons below.

Safety Plan Reporting Form