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A Phase 1/2 study of ALXN2350 in adult participants with BAG3-associated dilated cardiomyopathy
EU CTIS ID: 2024-519674-40-00
What this study is testing
Part A: To determine the safety and tolerability after a single IV infusion of ALXN2350 in participants with BAG3-associated DCM. Part B : To evaluate clinical impact (efficacy) after a single IV infusion of ALXN2350 vs external control in participants with BAG3-associated DCM
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participant must be at least 18 years of age and no older than 70 years of age at the time of signing the informed consent.
- Documented heterozygous pathogenic or likely pathogenic mutation in BAG3, interpreted according to the ACMG Guidelines, as confirmed by central laboratory. Pathogenic and likely pathogenic mutations may include deletions, frameshifting indels, premature truncations, disruption to an essential splice site, and/or sequence-altering missense variation with evidence supporting loss of function of the affected allele.
- Have had medical history of diagnosis of DCM (as per national guidelines)
- Participants must be on a stable combination of HF SoC medications (including angiotensin receptor-neprilysin inhibitor [ARNi], angiotensin-converting enzyme [ACE] inhibitor, angiotensin receptor blocker [ARB], beta-blocker, mineral corticoid receptor antagonists, and sodium-glucose cotransporter 2 [SGLT2] inhibitor) for at least 12 weeks prior to Stage 2 consent and during the Screening Period. If the participant is currently taking diuretics, then diuretics must also be stable for at least 2 weeks prior to Stage 2 consent (minor dose adjustments [<50%] in diuretics are allowed based on the Investigator’s judgment).
- Participants must have adequate acoustic windows for echocardiography.
You likely can't join if
- Decompensated HF or any cardiopulmonary hospitalization within 4 weeks prior to Stage 2 consent, or during the screening period.
- Clinically significant pericardial disease in the opinion of the Investigator.
- Pathogenic or likely-pathogenic missense genotype specifically affecting BAG3 amino acid 209 (eg, NP_004272.2:p.Pro209Leu), or presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM
- Presence of antibodies to AAV9 based on TAb assay at an MRD of 1:20.
- Recipient of any major organ transplant (eg, heart, lung, liver, bone marrow, renal) or likelihood, in the Investigator’s opinion, of undergoing cardiac transplantation, LVAD, or cardiac surgery within 3 months of Stage 2 of Screening.
- Any of the following within 3 months prior to Stage 2 consent: Acute coronary syndrome, stroke, transient ischemic attack, and cardiac procedures (pacemaker/ICD/ CRT-D implantation/coronary artery bypass grafting or percutaneous coronary intervention, coronary revascularization, ablation of atrial fibrillation/flutter, valve repair/replacement, aortic aneurysm surgery, etc).
See the full eligibility criteria
Who can join
- Participant must be at least 18 years of age and no older than 70 years of age at the time of signing the informed consent.
- Documented heterozygous pathogenic or likely pathogenic mutation in BAG3, interpreted according to the ACMG Guidelines, as confirmed by central laboratory. Pathogenic and likely pathogenic mutations may include deletions, frameshifting indels, premature truncations, disruption to an essential splice site, and/or sequence-altering missense variation with evidence supporting loss of function of the affected allele.
- Have had medical history of diagnosis of DCM (as per national guidelines)
- Participants must be on a stable combination of HF SoC medications (including angiotensin receptor-neprilysin inhibitor [ARNi], angiotensin-converting enzyme [ACE] inhibitor, angiotensin receptor blocker [ARB], beta-blocker, mineral corticoid receptor antagonists, and sodium-glucose cotransporter 2 [SGLT2] inhibitor) for at least 12 weeks prior to Stage 2 consent and during the Screening Period. If the participant is currently taking diuretics, then diuretics must also be stable for at least 2 weeks prior to Stage 2 consent (minor dose adjustments [<50%] in diuretics are allowed based on the Investigator’s judgment).
- Participants must have adequate acoustic windows for echocardiography.
What rules you out
- Decompensated HF or any cardiopulmonary hospitalization within 4 weeks prior to Stage 2 consent, or during the screening period.
- Clinically significant pericardial disease in the opinion of the Investigator.
- Pathogenic or likely-pathogenic missense genotype specifically affecting BAG3 amino acid 209 (eg, NP_004272.2:p.Pro209Leu), or presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM
- Presence of antibodies to AAV9 based on TAb assay at an MRD of 1:20.
- Recipient of any major organ transplant (eg, heart, lung, liver, bone marrow, renal) or likelihood, in the Investigator’s opinion, of undergoing cardiac transplantation, LVAD, or cardiac surgery within 3 months of Stage 2 of Screening.
- Any of the following within 3 months prior to Stage 2 consent: Acute coronary syndrome, stroke, transient ischemic attack, and cardiac procedures (pacemaker/ICD/ CRT-D implantation/coronary artery bypass grafting or percutaneous coronary intervention, coronary revascularization, ablation of atrial fibrillation/flutter, valve repair/replacement, aortic aneurysm surgery, etc).
- Positive coronary artery ischemic evaluation or presence of obstructive coronary artery disease (defined by ≥70% obstruction in a major epicardial coronary artery or ≥50% left main disease). If a prior ischemic workup cannot be documented, a participant may undergo a coronary CCTA in Screening. A positive result would lead to exclusion. CCTA results that would exclude a participant are CAD RADS classifications 4, 5 or N.
- Congenital long QT syndrome or prolonged QTcF >500msec.
- Cardiac ventricular arrhythmia that requires treatment. However, participants with atrial fibrillation or flutter and controlled ventricular rate (eg resting HR < 110 bpm) are permitted. Participants with cardiac ventricular arrhythmia that are treated with antiarrhythmic agents (eg, amiodarone) and are stable are permitted.
- History of untreated clinically significant valve disease or a Screening confirmation of severe aortic stenosis, severe mitral stenosis, moderate or severe aortic insufficiency or severe mitral insufficiency.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.