Dilated Cardiomyopathy (DCM)Phase 1/2UPBEATNCT07426419
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
A Phase 1/2 study testing AFTX-201 in people with Dilated Cardiomyopathy (DCM) and BAG3 Mutation Associated Dilated Cardiomyopathy, run by Affinia Therapeutics at 5 sites in 2 countries.
Trial summary
This is a Phase 1/2, open-label, dose-exploration and dose-expansion, clinical trial evaluating the safety, tolerability, pharmacodynamics, and preliminary efficacy of a single intravenous infusion of AFTX-201 in adults with dilated cardiomyopathy caused by a BAG3 gene mutation Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Dilated Cardiomyopathy (DCM), BAG3 Mutation Associated Dilated Cardiomyopathy
- What is being tested?
- AFTX-201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 5 sitesin 2 countries
- Study dates
- Oct 2026to Dec 2032
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female, 18 to 70 years of age
- Truncating mutation in BAG3
- Dilated cardiomyopathy with left ventricular ejection fraction (LVEF) < 50% by echocardiogram or cardiac MRI
- NYHA Class II or III heart failure symptoms
- Body weight ≤ 120 kg
- Willing and able to sign informed consent and comply with study procedures
Who cannot join
- Prior myocardial infarction when, in the investigator's judgment, the totality of clinical and imaging evidence supports that left ventricular dysfunction is due to coronary artery disease rather than BAG3-associated dilated cardiomyopathy
- Prior heart transplant
- Presence or requirement of a left ventricular assist device (LVAD)
- IV therapy with positive inotropes, vasodilators, or diuretics for heart failure within 30 days prior to enrollment
- Positive neutralizing antibodies to ATC-0187
- Any condition that, in the investigator's opinion, would place the subject at undue risk or interfere with study participation or interpretation of results
Trial locations and contacts
5 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 2 countries
United States4 sites, 4 recruiting
- Recruiting
Houston Methodist Hospital
Houston, Texas, 77030
Jennifer (Garrett) Medley Sr. Clinical Trials Manager, 346-238-4516, jmgarrett@houstonmethodist.org
Cindy Martin, MD
- Recruiting
St. Lukes Hospital
Kansas City, Missouri, 64111
Amanda Huffman, RN, BSN, 816-932-0391, amanda.huffman@bjc.org
Andrew Kao, MD
- Recruiting
University of Miami Hospital and Clinics
Miami, Florida, 33136
Lina Carceres, 954 249 3587, caceresl@miami.edu
Joshua Hare, MD
- Recruiting
NYU Langone
New York, New York, 10016
Loriya Thao, 646-877-6343, loriya.thao@nyulangone.org
Tajinderpal Saraon, MD
Canada1 site, 1 recruiting
- Recruiting
Institut de Cardiologie de Montréal / Montreal Heart Institute
Montreal, Quebec, QC H1T 1C8
Jérémie Coordonnateur de recherche EPS / EP research coordinator, BScN, 514-376-3330 ext. 2293, Jeremie.Cortial@icm-mhi.org
Rafik Tadros, MD
About this study
Official title: 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, open-label, dose-exploration and dose-expansion, clinical trial evaluating the safety, tolerability, pharmacodynamics, and preliminary efficacy of a single intravenous infusion of AFTX-201 in adults with dilated cardiomyopathy caused by a BAG3 gene mutation
Read the detailed description
Participants will receive a single intravenous infusion of AFTX-201 and will be followed for safety and clinical assessments. The study includes short-term safety monitoring and long-term follow-up for up to 60 months
- Being tested
- AFTX-201
- Keywords
- AFTX-201, Gene Therapy, AAV, BAG3, Dilated Cardiomyopathy, DCM, ATC-0187
- Medical subject headings
- Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AFTX-201Genetic
AFTX-201 is a gene therapy product consisting of the ATC-0187 capsid containing the BCL2-associated Athanogene 3 (BAG3) transgene. It is administered as a single intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| AFTX-201 | Experimental | Participants will receive a single intravenous dose of AFTX-201 Receives: Genetic: AFTX-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Safety and tolerability of AFTX-201 over 12 months.
Measured: Baseline through 12 months
Secondary outcomes (8)
Change from baseline in pharmacodynamic biomarkers
Assessment of the extent of AFTX-201 transduction and myocardial BAG3 protein expression after a single IV dose
Measured: Baseline through 12 months
Change from baseline in measurement of cardiac function including left ventricular ejection fraction (LVEF)
Assessment of the impact of a single IV dose of AFTX-201 on measurement of cardiovascular function
Measured: Baseline through 12 months
Change from baseline in measurement of cardiac function including peak oxygen consumption (pVO2) by cardiopulmonary exercise test (CPET)
Assessment of the impact of a single IV dose of AFTX-201 on measurement of cardiovascular function
Measured: Baseline through 12 months
Change from baseline in New York Heart Association (NYHA) functional class
Assessment of the impact of a single IV dose of AFTX-201 on features of heart failure (HF) including functional classification by New York Heart Association (NYHA) class
Measured: Baseline through 12 months
Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)
Assessment of the impact of a single IV dose of AFTX-201 on quality of life
Measured: Baseline through 12 months
Change from baseline in NT-proBNP levels through 12 months
Assessment of the change from baseline in NT-proBNP levels following a single intravenous dose of AFTX-201
Measured: Baseline through 12 months
Change from baseline in NT-proBNP levels during long-term follow-up
Assessment of the change from baseline in NT-proBNP levels during long-term follow-up following a single intravenous dose of AFTX-201
Measured: Baseline through 60 months
Number of participants with adverse events during long-term follow-up
Safety and tolerability of AFTX-201 over 60 months
Measured: Baseline through 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2026
- Study startOct 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dilated Cardiomyopathy (DCM)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Affinia Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07426419
- Sponsor's protocol ID
- AFTX-201-001
- Acronym
- UPBEAT
- Status last verified
- Oct 2026
- First submitted
- 3 Feb 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No