Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexMale
Age18+
SponsorSardocor Corp.
About this trial
This research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. The goal of SRD-001 gene therapy is to provide the heart muscle cells with extra copies of the SERCA2a gene so that they can produce more SERCA2a protein to help the heart muscle cells squeeze/contract better. Researchers will compare SRD-001 treated participants with no-treatment participants; all participants will continue to take their current heart medications. All participants will be followed very closely for 2 years and undergo cardiac magnetic resonance imaging of their heart at baseline, year 1 and year 2 along with assessment of upper limb function and lung function. After the 2 years of close follow-up, all participants will roll over into long-term follow-up where they will be called biannually for information on their current medical status.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Diagnosis of DMD with confirmatory genetic testing
Cardiomyopathy with left ventricular scar in at least 3 of 16 segments
Left ventricular ejection fraction < 40%
Individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months prior to enrollment
Disqualifiers
Abnormal blood pressure
Non-DMD-related liver function test elevations
Cystatin C ≥ 1.2 mg/L
Thrombocytopenia
Trial design
Design model
Sequential
Treatments tested in this trial
SRD-001
GeneticSRD-001 is an adeno-associated virus serotype 1 (AAV1) based gene therapy designed to deliver a copy of the gene encoding the human sarcoplasmic/endoplasmic reticulum Ca(2+) ATPase 2a (SERCA2a). It is administered as a one-time intracoronary infusion.
Treatment groups
12 Participants
are divided into 3 treatment groupsGroup A: Low DoseExperimental treatment 1 intervention
Group B: High DoseExperimental treatment 1 intervention
Group C: ControlNo intervention 0 interventions
Trial outcomes
Primary outcomes
1
Rate of all-cause mortality
Death
Time frame
From Day 1 to Week 52 and Week 1042
Rate and severity of related treatment-emergent adverse events
Adverse events related to the investigational product or the administration procedure
Time frame
From Day 1 to Week 52 and Week 1043
Rate and severity of all treatment-emergent adverse events
Adverse events
Time frame
From Day 1 to Week 52 and Week 1044
Rate of cell-mediated immune reaction
Cell-mediated immune reaction as assessed by enzyme-linked immunosorbent spot (ELISpot)
Time frame
From Day 1 to Week 52Secondary outcomes
1
Change, including normal/abnormal shifts, in 12-lead electrocardiogram (ECG)
Change in the heart's electrical activity
Time frame
From Day 1 to Week 52 and Week 1042
Change, including normal/abnormal shifts, in laboratory evaluations
Hematology, serum chemistries, urinalysis, cardiac enzymes and anti-AAV1 antibodies
Time frame
From Day 1 to Week 52 and Week 104Other outcomes
1
Change in myocardium and left ventricular structure and function
Assessed by cardiac magnetic resonance imaging with late gadolinium enhancement
Time frame
From baseline to Week 52 and Week 1042
Change in skeletal muscle function
Assessed by PUL 2.0, grip strength, key and tip-to-tip pinch strength and elbow flexion strength
Time frame
From baseline to Week 52 and Week 1043
Change in pulmonary function
Assessed by slow vital capacity, forced expiratory volume in 1 second, forced vital capacity, peak expiratory flow, maximum inspiratory pressure, maximum expiratory pressure, peak cough flow and inspiratory flow reserve
Time frame
From baseline to Week 52 and Week 1044
Change in quality of life
Assessed by the Duchenne muscular dystrophy quality of life questionnaire
Time frame
From baseline to Week 52 and Week 104Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov