Dilated Cardiomyopathy (DCM)Phase 1DYNAMICNCT02293603
Dilated cardiomYopathy iNtervention With Allogeneic MyocardIally-regenerative Cells (DYNAMIC)
A Phase 1 study testing Allogeneic Cardiosphere-Derived Cells (CDCs) in people with Dilated Cardiomyopathy (DCM) and Ischemic Cardiomyopathy, run by Capricor Inc. at 1 site in United States.
Trial summary
To determine the safety profile of CAP-1002 administered by multi-vessel intracoronary infusion in subjects with DCM. The study will further explore safety and exploratory efficacy endpoints of CAP-1002. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Dilated Cardiomyopathy (DCM), Ischemic Cardiomyopathy, Nonischemic Cardiomyopathy
- What is being tested?
- Allogeneic Cardiosphere-Derived Cells (CDCs)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 42 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2014to Apr 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Major Inclusion Criteria:
- DCM with left ventricular ejection fraction (LVEF) ≤ 35% as determined by a historical TTE within the previous 6 months
- New York Heart Association (NYHA) Class III or ambulatory Class IV heart failure
- Use of evidence based medical-therapy (beta-blockers, ACE-inhibitors/angiotensin receptor blockers, aldosterone antagonist) and with or without device-therapy (Implantable cardioverter-defibrillator or cardiac resynchronizing therapy), in accordance with the ACC/AHA guidelines for the management of heart failure, for at least three months prior to enrollment or documented contraindication or intolerance or patient preference
- Coronary anatomy suitable for Investigational Product (IP) infusion, as determined by the Eligibility Committee (a team of cardiology experts)
- Ability to provide informed consent and follow-up with protocol procedures
- Screening cardiac CT left ventriculogram ejection fraction <40% with left ventricular dilatation
- Age ≥ 18 years
Major Exclusion Criteria:
- Diagnosis of active myocarditis
- Immunologic incompatibility with all available Master Cell Banks (MCBs) by single-antigen bead (SAB) serum antibody profiling
- Left Ventricular Assist Devices (LVAD) or those actively in the process of acquiring one
- Recent placement of a cardiac pacemaker and/or resynchronization pacing therapy within the past three months or those actively in the process of acquiring one
- History of sustained ventricular tachycardia (VT) requiring cardiopulmonary resuscitation (with the exception of subjects who subsequently received an ICD)
- Non-cardiovascular disease with life expectancy of < 3 years
- Known hypersensitivity to contrast agents
- Estimated glomerular filtration rate (GFR) < 50 mL/min
- Active infection not responsive to treatment
- Active allergic reactions, connective tissue disease or autoimmune disorders
- History of cardiac tumor, or cardiac tumor demonstrated on screening
- History of previous stem cell therapy
- History of treatment with immunosuppressive agents, including chronic systemic corticosteroids, biologic agents targeting the immune system, anti-tumor and anti-neoplastic drugs or anti-vascular endothelial growth factor (VEGF) within 6 months prior to enrollment (not including drug eluting coronary stents)
- History of receipt of chemotherapeutic agents known to be implicated in cardiac dysfunction [Adriamycin, trastuzumab (Herceptin)]
- Known moderate-severe aortic stenosis/insufficiency or severe mitral stenosis/regurgitation
- Participation in an on-going protocol studying an experimental drug or device
- Current active alcohol or drug abuse or inability to comply with protocol-related procedures
- Pregnant/nursing women and women of child-bearing potential without use of active and highly reliable contraception
- Known history of Human Immunodeficiency Virus (HIV) infection
- Known history of chronic viral hepatitis
- Abnormal liver function (serum glutamic pyruvic transaminase (SGPT) > 10 times the upper reference range) and/or abnormal hematology (hematocrit < 25%, white blood cells (WBC) < 3000 µl, platelets < 100,000 µl) studies without a reversible, identifiable cause
- Evidence of tumor on screening of chest/abdominal/pelvic (body) CT scan
- Any prior organ transplant
- Being actively listed for, or under active consideration (i.e., work-up) for, a solid organ transplant of any kind
- Known hypersensitivity to bovine products
- Known hypersensitivity to dimethyl sulfoxide (DMSO)
- Any malignancy within past 2 years (except for in-situ non-melanoma skin cancer and in-situ cervical cancer)
- Any prior radiation therapy/treatment to the chest
- Uncontrolled diabetes (HbA1 >9.0)
- Any condition or other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for the study
Trial locations and contacts
1 site in 1 country.
United States1 site
Cedars-Sinai Medical Center
Los Angeles, California, 90048
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Phase I Study of the Safety of Multi-vessel Intra-coronary Delivery of Allogeneic Human Cardiosphere-Derived Stem Cells in Patients With Dilated Cardiomyopathy (DCM)
To determine the safety profile of CAP-1002 administered by multi-vessel intracoronary infusion in subjects with DCM. The study will further explore safety and exploratory efficacy endpoints of CAP-1002.
Read the detailed description
Eligible subjects will undergo sequential intracoronary infusion of CAP-1002 or placebo in up to three coronary arteries supplying three major cardiac territories to the heart (anterior, lateral, inferior/posterior). After completion of the screening procedures, Phase Ia subjects will receive CAP-1002 administered via intracoronary infusion in a dose escalation, stepwise manner. Phase Ia subjects will be followed at Week 2 and at Months 1, 2, 3, 6 and 12 after CAP-1002 infusion. The first fourteen (14) subjects will receive intracoronary infusion of CAP-1002 in an open-label fashion (Phase Ia). Once all 14 subjects in the Phase Ia have reached the primary safety endpoint (1 month visit), the DSMB will conduct a review of the Phase Ia data and recommend whether to proceed with enrollment of the next 28 subjects in the Phase Ib.
- Conditions
- Dilated Cardiomyopathy (DCM), Ischemic Cardiomyopathy, Nonischemic Cardiomyopathy, Heart Failure
- Being tested
- Allogeneic Cardiosphere-Derived Cells (CDCs)
- Keywords
- cardiomyopathy, cardiosphere-derived stem cells, intracoronary infusion, adult stem cells, heart disease, ventricular dysfunction, pathological processes, cardiovascular diseases, heart failure
- Medical subject headings
- Cardiomyopathy, Dilated, Heart Failure, Cardiomyopathies, Heart Diseases, Ventricular Dysfunction, Pathologic Processes, Cardiovascular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Cardiosphere-Derived Cells (CDCs)Biological
Also called CAP-1002
Intracoronary delivery of CAP-1002 or placebo
| Study group | Role | What they receive |
|---|---|---|
| Allogeneic Cardiosphere-Derived Cells | Experimental | The Phase I study consists of a Phase Ia portion and a Phase Ib portion. The Phase Ia portion (N=14 subjects) consists of an open-label, single-arm, study design. The potentially conducted Phase Ib portion of the study (N=28 subjects) consists of a double-blind, randomized, placebo-controlled study design.
The Phase Ia portion is an open-label, dose escalation of Allogeneic Cardiosphere-Derived Cells (CDCs). Receives: Biological: Allogeneic Cardiosphere-Derived Cells (CDCs) |
| Placebo | Placebo comparator | The placebo study arm only applies to the Phase Ib portion of the study design. The Phase Ia portion (N=14 subjects) consists of an open-label, single-arm, study design. The potentially conducted Phase Ib portion of the study (N=28 subjects) consists of a double-blind, randomized, placebo-controlled study design. Receives: Biological: Allogeneic Cardiosphere-Derived Cells (CDCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects that experience new TIMI flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2.
Measured: Intraprocedural
Proportion of subjects that experience acute myocarditis, possibly attributable to CAP-1002. In order to be considered related to CAP-1002, humoral or cellular or immune reaction specific to CAP-1002 must also be documented.
Measured: Within one month of intracoronary infusion
Proportion of subjects that experience ventricular tachycardia or ventricular fibrillation resulting in death, appropriate discharge of an ICD or requiring medical intervention.
Measured: During or within 72 hours of intracoronary infusion
Proportion of subjects that experience sudden unexpected death occurring within one hour of symptom onset, or un-witnessed death in a person previously observed to be well within the preceding 24 hours without an identified cause.
Measured: During or within 72 hours of intracoronary infusion
Proportion of subjects that experience major adverse cardiac events (MACE), including death, non-fatal myocardial infarction and re-hospitalization for cardiovascular event (including heart failure hospitalizations).
Measured: During or within 72 hours of intracoronary infusion
Secondary outcomes (10)
Acute myocarditis possibly attributable to CAP-1002. In order to be considered related to CAP-1002, humoral or cellular immune reaction specific to CAP-1002 must also be documented.
Measured: During the six & twelve month follow-up period
Ventricular tachycardia or ventricular fibrillation resulting in death or requiring medical intervention or appropriate discharge of an ICD.
Measured: During the six & twelve month follow-up period
Sudden unexpected death defined as occurring within one hour of symptom onset, or unwitnessed death in a person previously observed to be well within the preceding 24 hours without an identified cause.
Measured: During the six & twelve month follow-up period
Major adverse cardiac events (MACE), including death, non-fatal myocardial infarction, hospitalization for cardiovascular event, emergency room treatment for heart failure, left ventricular assist device or heart transplant.
Measured: During the six & twelve month follow-up period
Any hospitalization due to a cardiovascular cause or related to CAP-1002 (or placebo in Phase Ib).
Measured: During the six & twelve month follow-up period
Any inter-current cardiovascular illness or one related to CAP-1002 (or placebo in Phase Ib) which prolongs hospitalization.
Measured: During the six & twelve month follow-up period
Development of, or an increase in the frequency of, VT with a duration of 30 seconds or longer ascertained by protocol-mandated ECG ambulatory monitoring.
Measured: During the six & twelve month follow-up period
Development of increased anti-Human Leukocyte Antigen (HLA) antibody levels with development of sensitization to HLA antigens specific to the CAP-1002 CDC donor at immunologically significant titers.
Measured: During the six & twelve month follow-up period
Total number of appropriate ICD firings.
Measured: During the six & twelve month follow-up period
Peak elevation in troponin and CKMB levels following CAP-1002 or placebo infusion.
Measured: Through Month 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2014 (estimated)
- Study startNov 2014
- Primary completionMay 2015
- Study completionApr 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dilated Cardiomyopathy (DCM)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Capricor Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cedars-Sinai Medical Center, National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Rajendra (Raj) Makkar, MD, Principal investigator, Cedars-Sinai Medical Center, Heart InstituteDeborah Ascheim, MD, Study director, Capricor Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02293603
- Sponsor's protocol ID
- CAP-1002 (DYNAMIC)
- Other IDs
- R44HL095203
- Acronym
- DYNAMIC
- Status last verified
- Jun 2016
- First submitted
- 5 Nov 2014
- Last updated
- 21 Jun 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 21 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Phase I Study of the Safety of Multi-vessel Intra-coronary Delivery of Allogeneic Human Cardiosphere-Derived Stem Cells in Patients With Dilated Cardiomyopathy (DCM)
- ClinicalTrials.gov ID
- NCT02293603
- Acronym / sponsor ID
- DYNAMIC / CAP-1002 (DYNAMIC)
- Conditions
- Dilated Cardiomyopathy (DCM), Ischemic Cardiomyopathy, Nonischemic Cardiomyopathy, Heart Failure
- Keywords
- cardiomyopathy, cardiosphere-derived stem cells, intracoronary infusion, adult stem cells, heart disease, ventricular dysfunction, pathological processes, cardiovascular diseases
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Capricor Inc.Industry (pharma, biotech, devices)
- Collaborators
- Cedars-Sinai Medical Center, National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Nov 2014
- Primary completion
- May 2015 (actual)
- Study completion
- Apr 2020 (estimated)
- First posted
- 18 Nov 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To determine the safety profile of CAP-1002 administered by multi-vessel intracoronary infusion in subjects with DCM. The study will further explore safety and exploratory efficacy endpoints of CAP-1002.
Detailed description
Eligible subjects will undergo sequential intracoronary infusion of CAP-1002 or placebo in up to three coronary arteries supplying three major cardiac territories to the heart (anterior, lateral, inferior/posterior). After completion of the screening procedures, Phase Ia subjects will receive CAP-1002 administered via intracoronary infusion in a dose escalation, stepwise manner. Phase Ia subjects will be followed at Week 2 and at Months 1, 2, 3, 6 and 12 after CAP-1002 infusion. The first fourteen (14) subjects will receive intracoronary infusion of CAP-1002 in an open-label fashion (Phase Ia). Once all 14 subjects in the Phase Ia have reached the primary safety endpoint (1 month visit), the DSMB will conduct a review of the Phase Ia data and recommend whether to proceed with enrollment of the next 28 subjects in the Phase Ib.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Allogeneic Cardiosphere-Derived Cells | Experimental | Biological: Allogeneic Cardiosphere-Derived Cells (CDCs)The Phase I study consists of a Phase Ia portion and a Phase Ib portion. The Phase Ia portion (N=14 subjects) consists of an open-label, single-arm, study design. The potentially conducted Phase Ib portion of the study (N=28 subjects) consists of a double-blind, randomized, placebo-controlled study design. The Phase Ia portion is an open-label, dose escalation of Allogeneic Cardiosphere-Derived Cells (CDCs). |
| Placebo | Placebo comparator | Biological: Allogeneic Cardiosphere-Derived Cells (CDCs)The placebo study arm only applies to the Phase Ib portion of the study design. The Phase Ia portion (N=14 subjects) consists of an open-label, single-arm, study design. The potentially conducted Phase Ib portion of the study (N=28 subjects) consists of a double-blind, randomized, placebo-controlled study design. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CDCs)Also: CAP-1002Intracoronary delivery of CAP-1002 or placebo | Biological | Allogeneic Cardiosphere-Derived Cells, Placebo |
Primary outcome
Proportion of subjects that experience new TIMI flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2.
Time frame: Intraprocedural
Proportion of subjects that experience acute myocarditis, possibly attributable to CAP-1002. In order to be considered related to CAP-1002, humoral or cellular or immune reaction specific to CAP-1002 must also be documented.
Time frame: Within one month of intracoronary infusion
Proportion of subjects that experience ventricular tachycardia or ventricular fibrillation resulting in death, appropriate discharge of an ICD or requiring medical intervention.
Time frame: During or within 72 hours of intracoronary infusion
Proportion of subjects that experience sudden unexpected death occurring within one hour of symptom onset, or un-witnessed death in a person previously observed to be well within the preceding 24 hours without an identified cause.
Time frame: During or within 72 hours of intracoronary infusion
Proportion of subjects that experience major adverse cardiac events (MACE), including death, non-fatal myocardial infarction and re-hospitalization for cardiovascular event (including heart failure hospitalizations).
Time frame: During or within 72 hours of intracoronary infusion
10 secondary outcomes
Acute myocarditis possibly attributable to CAP-1002. In order to be considered related to CAP-1002, humoral or cellular immune reaction specific to CAP-1002 must also be documented.
Time frame: During the six & twelve month follow-up period
Ventricular tachycardia or ventricular fibrillation resulting in death or requiring medical intervention or appropriate discharge of an ICD.
Time frame: During the six & twelve month follow-up period
Sudden unexpected death defined as occurring within one hour of symptom onset, or unwitnessed death in a person previously observed to be well within the preceding 24 hours without an identified cause.
Time frame: During the six & twelve month follow-up period
Major adverse cardiac events (MACE), including death, non-fatal myocardial infarction, hospitalization for cardiovascular event, emergency room treatment for heart failure, left ventricular assist device or heart transplant.
Time frame: During the six & twelve month follow-up period
Any hospitalization due to a cardiovascular cause or related to CAP-1002 (or placebo in Phase Ib).
Time frame: During the six & twelve month follow-up period
Any inter-current cardiovascular illness or one related to CAP-1002 (or placebo in Phase Ib) which prolongs hospitalization.
Time frame: During the six & twelve month follow-up period
Development of, or an increase in the frequency of, VT with a duration of 30 seconds or longer ascertained by protocol-mandated ECG ambulatory monitoring.
Time frame: During the six & twelve month follow-up period
Development of increased anti-Human Leukocyte Antigen (HLA) antibody levels with development of sensitization to HLA antigens specific to the CAP-1002 CDC donor at immunologically significant titers.
Time frame: During the six & twelve month follow-up period
Total number of appropriate ICD firings.
Time frame: During the six & twelve month follow-up period
Peak elevation in troponin and CKMB levels following CAP-1002 or placebo infusion.
Time frame: Through Month 1
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jun 2016.
- Capricor's websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rajendra (Raj) Makkar, MD principal investigator
Cedars-Sinai Medical Center, Heart Institute
Deborah Ascheim, MD study director
Capricor Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Allogeneic Cardiosphere-Derived Cells (CDCs) in people with Dilated Cardiomyopathy (DCM) and Ischemic Cardiomyopathy, run by Capricor Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 21 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects that experience new TIMI flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2. (time frame: Intraprocedural). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jun 2016.