Are you currently living with BAG3 Cardiomyopathy?
If so, you may be interested in The UPBEAT Trial which is now enrolling adults with dilated cardiomyopathy caused by a BAG3 gene mutation.
SEE TRIAL LOCATIONS
What is the UPBEAT clinical trial?
This clinical research study is designed to evaluate the safety and tolerability of a single intravenous dose of an investigational gene therapy in adults with dilated cardiomyopathy caused by a BAG3 gene mutation.
The study will also collect additional information to better understand how the therapy behaves in the body and its effects on the heart.
All participants will receive a one-time intravenous infusion of the gene therapy and will be followed through scheduled study visits and long-term follow-up.
Participation in this study is voluntary.
All study related costs, including travel and lodging, will be provided by the study sponsor.
This study is being conducted by Affinia Therapeutics.
Who can join the UPBEAT clinical trial?
Difficulty in performing normal physical activity.
Participants must be 18-55 years of age inclusive
BAG3 mutation
What are the key exclusion criteria?
- Prior heart attack
- Prior heart transplant
- Presence or requirement of a left ventricular assist device (LVAD)
Note: This is not a complete list of inclusion and exclusion criteria. Eligibility will be determined by the study doctor based on the full inclusion and exclusion criteria.
What is involved in participating?
Clinical Trial – Currently Enrolling
Infusion
Participants will receive a one-time intravenous infusion of the investigational gene therapy.
Assessments
For 12 months after treatment, participants will attend scheduled study visits that include clinical assessments, laboratory testing, exercise testing, imaging tests of the heart, and other evaluations to monitor safety. The study visits are typically done on an outpatient basis, meaning hospitalization is not required..
Long-Term Follow-up
After the 12 month period, participants will be asked to return for annual visits over the next 4 years for a total of 5 years of follow up. This long-term follow-up is standard for gene therapy studies.
Clinical Trial Ends
Study Clinic Locations
If you are interested in participating in this trial, contact the principal investigator at the site location most convenient for you.
More trial locations will be opening soon. Please contact us at clinicaltrials@affiniatx.com to find out more.
More Information
Official name of trial – A Phase 1/2 Multicenter, Open-Label, Dose-Escalation and Dose-Expansion Trial to Assess the Safety, Tolerability, Pharmacodynamics, and Preliminary Efficacy of AFTX-201 Administered to Adult Participants with BCL2-Associated Athanogene 3 (BAG3) Mutation-Associated Dilated Cardiomyopathy: UPBEAT Trial.
This is a Phase 1/2 multicenter, open-label clinical research study in adults with dilated cardiomyopathy caused by a pathogenic BAG3 gene mutation. The primary end point is safety and tolerability over 12 months and the secondary end points include assessments of BAG3 gene and protein expression in the heart as well as potential for efficacy over 12 months and long term safety and efficacy over 60 months.
Adults (ages 18 to 55) with mutations in BAG3 and dilated cardiomyopathy will receive a onetime intravenous infusion of an AAV based gene therapy with the BAG3 gene and will be followed with scheduled clinical assessments and long-term follow-up to monitor safety.
A more detailed description of the trial can be found at:
Inquiry Form
Complete the form below to learn more about the Affinia Therapeutics- UPBEAT Trial. After you have submitted the form, a patient advocacy expert from our partners at the SADS Foundation will contact you to discuss the trial, answer your questions, and — if the trial is right for you — help connect you directly with a participating study site.