Cure HHT
Data sharing for patient reported outcome tools
We hope to have a qualified patient reported outcome tool for clinical trials.
Posted February 25, 2025
Background & Context
Our patient advocacy organization is running a phase II/III clinical trial. In 2018, a patient reported outcome (PRO) tool was published as a validated metric for measuring daily bleeding in the patient population we support. This PRO is currently being used in the clinical trial. We have several pharma/industry groups interested in getting the data from the publication of the PRO for their upcoming studies. It is our intent to bring everyone to the table with some kind of data sharing agreement to use the PRO exactly as written/intended. Groups usually change/tweak/optimize tools like this, but that gets us further from having a qualified metric to use broadly in clinical trials that is accepted by the FDA. We want to ensure this PRO can keep moving forward as an accepted tool for the entire community to use.
Work & Deliverables
We would like to develop a data sharing agreement for industry and academia around the use of a patient-reported outcome tool.
Preparation Phase
- Meeting with clinical trial team (Dennis Sprecher and Cassi Friday) to review background.
Collaboration Phase
Wrap Up
Cure HHT
Cure HHT is an international advocacy organization supporting and providing educational services patients and families with the rare blood vessel disease, Hereditary Hemorrhagic Telangiectasis (HHT). We were formed in 1991 and have evolved in supporting and funding research, collaborations, and have 32 multi-diciplinary Centers of Excellence in North America and 21 globally. HHT is the 2nd most common inherited bleeding disorder with no approved FDA therapies. We have increased capacity through the Rare As One Grant and opened a therapeutic arm of our organization. We participated in 3 clinical trials-1 in which we have taken forward ourselves and received $6.2M in funding for this Phase II III clinical trial from the Department of Defense and the FDA. We will be launching our patient reported registry in April 2023 to obtain important deidentified patient information on their disease. We received a 3 year grant from the Health Resources and Services administration to support 15 Centers of Excellence in the U.S. With focus on increasing access to coordinated care and development of a continuing medical education program, we are creating more awareness leading to increased diagnosis and treatment. A third element of the program is a research registry recruiting patients seen at HHt Centers of Excellence. We have received IRB approvals and are launching a biorepository in which we will make tissue samples available to researchers .
Cure HHT
Cure HHT is an international advocacy organization supporting and providing educational services patients and families with the rare blood vessel disease, Hereditary Hemorrhagic Telangiectasis (HHT). We were formed in 1991 and have evolved in supporting and funding research, collaborations, and have 32 multi-diciplinary Centers of Excellence in North America and 21 globally. HHT is the 2nd most common inherited bleeding disorder with no approved FDA therapies. We have increased capacity through the Rare As One Grant and opened a therapeutic arm of our organization. We participated in 3 clinical trials-1 in which we have taken forward ourselves and received $6.2M in funding for this Phase II III clinical trial from the Department of Defense and the FDA. We will be launching our patient reported registry in April 2023 to obtain important deidentified patient information on their disease. We received a 3 year grant from the Health Resources and Services administration to support 15 Centers of Excellence in the U.S. With focus on increasing access to coordinated care and development of a continuing medical education program, we are creating more awareness leading to increased diagnosis and treatment. A third element of the program is a research registry recruiting patients seen at HHt Centers of Excellence. We have received IRB approvals and are launching a biorepository in which we will make tissue samples available to researchers .