A Phase 1/2, Open-Label, Dose Finding Study to Investigate the Safety, Tolerability, and Efficacy of ALXN2350 Gene Therapy in Adult Participants with Symptomatic BAG3 Mutation-Associated Dilated Cardiomyopathy

Conditions

BAG3 Mutation Associated Dilated Cardiomyopathy

Brief summary

Part A : Incidence and severity of TEAEs and SAEs, and clinically significant safety findings (physical examinations, vital sign measurements, clinical laboratory assessments, and ECG results).

Detailed description

Part B : Incidence and severity of TEAEs and SAEs, and clinically significant safety findings (physical examinations, vital sign measurements, clinical laboratory assessments, and ECG results)., Part A and B : CFB in cardiac serum biomarker NT‑proBNP through Week 52 and 78., Part A and B : Incidence of cardiac events including heart failure hospitalization, sustained ventricular arrhythmias, appropriate implantable cardioverter defibrillator shock through Week 52 and 78., Part A and B : Time to the first event of death, heart transplant, mechanical circulating support, or aborted sudden cardiac death through Week 52 and 78., Part A and B : CFB of cardiac serum biomarker hs-TnT Week 52 and 78., Part A and B : Amount of virus secreted in blood, feces, saliva, semen, and urine through Week 52 and 78., Part A and B : Incidence of anti-AAV9 and anti BAG3 antibodies, response categories, and titer, in participants with BAG3-associated DCM treated with ALXN2350 through Week 52 and 78.

Related papers

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Interventions

DRUGPREDNISONE VIATRIS 5 mg

DRUGcomprimé sécable

DRUGPrednisone Olikla 2.5 mg tablety

DRUGRapamune 0.5 mg coated tablets

DRUGRapamune 1 mg/mL oral solution

DRUGPrednisone Olikla 10 mg tablety

DRUGRapamune 1 mg coated tablets

DRUGPrednisone Olikla 50 mg tablety

DRUGPREDNISONE VIATRIS 1 mg

DRUGcomprimé

DRUGCo-Trimoxazole Forte 160/800mg Tablets

DRUGPREDNISONE VIATRIS 20 mg

DRUGadeno-associated virus

DRUGserotype 9 (AAV9)

DRUGRapamune 2 mg coated tablets

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Part A : Incidence and severity of TEAEs and SAEs, and clinically significant safety findings (physical examinations, vital sign measurements, clinical laboratory assessments, and ECG results).	—

Secondary

Measure	Time frame
Part B : Incidence and severity of TEAEs and SAEs, and clinically significant safety findings (physical examinations, vital sign measurements, clinical laboratory assessments, and ECG results)., Part A and B : CFB in cardiac serum biomarker NT‑proBNP through Week 52 and 78., Part A and B : Incidence of cardiac events including heart failure hospitalization, sustained ventricular arrhythmias, appropriate implantable cardioverter defibrillator shock through Week 52 and 78., Part A and B : Time to the first event of death, heart transplant, mechanical circulating support, or aborted sudden cardiac death through Week 52 and 78., Part A and B : CFB of cardiac serum biomarker hs-TnT Week 52 and 78., Part A and B : Amount of virus secreted in blood, feces, saliva, semen, and urine through Week 52 and 78., Part A and B : Incidence of anti-AAV9 and anti BAG3 antibodies, response categories, and titer, in participants with BAG3-associated DCM treated with ALXN2350 through Week 52 and 78.	—

Measure

Time frame

Part B : Incidence and severity of TEAEs and SAEs, and clinically significant safety findings (physical examinations, vital sign measurements, clinical laboratory assessments, and ECG results)., Part A and B : CFB in cardiac serum biomarker NT‑proBNP through Week 52 and 78., Part A and B : Incidence of cardiac events including heart failure hospitalization, sustained ventricular arrhythmias, appropriate implantable cardioverter defibrillator shock through Week 52 and 78., Part A and B : Time to the first event of death, heart transplant, mechanical circulating support, or aborted sudden cardiac death through Week 52 and 78., Part A and B : CFB of cardiac serum biomarker hs-TnT Week 52 and 78., Part A and B : Amount of virus secreted in blood, feces, saliva, semen, and urine through Week 52 and 78., Part A and B : Incidence of anti-AAV9 and anti BAG3 antibodies, response categories, and titer, in participants with BAG3-associated DCM treated with ALXN2350 through Week 52 and 78.

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Countries

Germany, Italy, Spain

Outcome results

None listed