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
- Dec 2026to Dec 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 startDec 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
- Nov 2026
- First submitted
- 3 Feb 2026
- Last updated
- 3 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07426419
- Acronym / sponsor ID
- UPBEAT / AFTX-201-001
- Keywords
- AFTX-201, Gene Therapy, AAV, BAG3, Dilated Cardiomyopathy, DCM, ATC-0187
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 22 participants (estimated)
- Lead sponsor
- Affinia TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Dec 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2032 (estimated)
- First posted
- 23 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AFTX-201 | Experimental | Genetic: AFTX-201Participants will receive a single intravenous dose of AFTX-201 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AFTX-201AFTX-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 | Genetic | AFTX-201 |
Primary outcome
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame: Baseline through 12 months
Safety and tolerability of AFTX-201 over 12 months.
8 secondary outcomes
Change from baseline in pharmacodynamic biomarkers
Time frame: Baseline through 12 months
Change from baseline in measurement of cardiac function including left ventricular ejection fraction (LVEF)
Time frame: Baseline through 12 months
Change from baseline in measurement of cardiac function including peak oxygen consumption (pVO2) by cardiopulmonary exercise test (CPET)
Time frame: Baseline through 12 months
Change from baseline in New York Heart Association (NYHA) functional class
Time frame: Baseline through 12 months
Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)
Time frame: Baseline through 12 months
Change from baseline in NT-proBNP levels through 12 months
Time frame: Baseline through 12 months
Change from baseline in NT-proBNP levels during long-term follow-up
Time frame: Baseline through 60 months
Number of participants with adverse events during long-term follow-up
Time frame: Baseline through 60 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trials Information ((617) 430-7817 ext. 2, clinicaltrials@affiniatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) (time frame: Baseline through 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2026.