A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)

Study Identifier:
R0765C00001
ClinicalTrials.gov Identifier:
NCT05981092
EudraCT Identifier:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Recruitment Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
    Date
    Oct 2022 - Sep 2026
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: 18+ years
    Requirements Information
    Sex
    Female & Male
    Age
    18+ years

    Study Details

    Medical Condition
    • Unmapped
    Study Drug
      Date
      Oct 2022 - Sep 2026
      Phase 1
      Phase 2
      Phase 3
      Phase 4
      N/A
      Patient Requirements
      Sex: Female & Male
      Age: 18+ years years
      Requirements Information

      Protocol Summary

      The purpose of this study is to learn about the natural progression of DCM (dilated cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is weakened and the heart becomes enlarged. This makes it hard for the heart to pump enough blood for the body.

      The study is seeking up to about 35 participants who have:

      • BAG3 mutation (change in the gene) that causes or is likely to cause dilated cardiomyopathy
      • NYHA (New York Heart Association) Class I-IV at screening (Stage B-D)
      • Left Ventricular Ejection Fraction less than or equal to 50% (meaning reduced heart function)

      All participants in this study will receive their usual treatment. The investigators will observe the natural progression of people who have BAG3 DCM. This will help the investigators better understand the disease and aid in future research.

      Participants will take part in this study for one year. During this time, participants will visit the site at least 4 times (about every 3 months). Participants will undergo study procedures and give information about their health. These procedures will include a physical exam, cardiac magnetic resonance imaging, echocardiography, ECG monitoring, activity monitoring, cardiopulmonary exercise testing, and blood tests. Participants will answer questions about health and quality of life. The study team will also call participants about 1 time over the phone.

      Trial Locations

      Location
      Status
      Location
      Research Site
      Palo Alto, California, United States, 94305
      Status
      N/A
      Location
      Research Site
      Aurora, Colorado, United States, 80045
      Status
      N/A
      Location
      Research Site
      Boston, Massachusetts, United States, 02115
      Status
      N/A
      Location
      Research Site
      Rochester, Minnesota, United States, 55905
      Status
      N/A
      Location
      Research Site
      Bronx, New York, United States, 10467
      Status
      N/A
      Location
      Research Site
      Philadelphia, Pennsylvania, United States, 19104
      Status
      N/A