Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD

Summary

This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in heart health over time in boys with DMD.

Currently, there is a lack of sufficiently well characterized cardiac MRI biomarkers that can serve as endpoints for detecting on-target and/or off-target cardiac effects during clinical drug trials for boys with DMD.

Consequently, the first objective is to identify and characterize several cardiac MRI biomarkers for boys with DMD.

Conditions

• Muscular Dystrophy, Duchenne

Interventions

OTHER

Cardiac MRI with contrast

Cardiac MRI

OTHER

Cardiac MRI without contrast

Cardiac MRI

OTHER

Blood Test

Hematocrit, Creatinine, Troponin, BNP

OTHER

Heart Rate

Holter Monitor

OTHER

Pulmonary Function Test

Pulmonary Function Test

OTHER

Genetic Testing

Genetic Testing

OTHER

Repeat MRI scan

Repeat MRI scan

Sponsors & Collaborators

• National Institutes of Health (NIH)

  collaborator NIH

• Children's Hospital of Orange County

  collaborator OTHER

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• University of California, Los Angeles

  collaborator OTHER

• Stanford University

  lead OTHER

Principal Investigators

• Daniel Ennis, PhD · Stanford University

Study Design

Allocation

NON_RANDOMIZED

Purpose

BASIC_SCIENCE

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

7 Years

Max Age

21 Years

Sex

MALE

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2017-02-01

Primary Completion

2022-03-30

Completion

2022-03-30

Countries

• United States

Study Locations