Non-Ischemic Heart FailurePhase 2NCT02467387
A Study to Assess the Effect of Intravenous Dose of (aMBMC) to Subjects With Non-ischemic Heart Failure
A Phase 2 study testing Allogeneic Mesenchymal Bone Marrow Cells (aMBMC) and Lactated Ringer's Solution in people with Non-Ischemic Heart Failure, run by CardioCell LLC at 5 sites in United States.
Trial summary
A phase IIa study to assess the safety and preliminary efficacy of intravenous dose of ischemia-tolerant Allogeneic Mesenchymal Bone Marrow Cells in subjects with non-ischemic heart failure. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non-Ischemic Heart Failure
- What is being tested?
- Allogeneic Mesenchymal Bone Marrow Cells (aMBMC) and Lactated Ringer's Solution
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 23 participants
- Where
- 5 sitesUnited States
- Study dates
- 1 Jun 2014to 11 May 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
Who may be able to join
- Males and females ≥18 years of age
- LVEF ≤35% on echocardiogram within 12 months of randomization to undergo MRI
- Screening cardiac MRI at baseline with:
- Ejection fraction ≤40% No significant hyper enhancement on MRI scan in the opinion of the central imaging lab reviewer
- Patients with non-ischemic heart failure etiology, as documented by absent or non-obstructive coronary artery disease on x-ray angiography or coronary computed tomography
- Patients with history of heart failure and treated for at least three months with GDMT
Show all 9Show fewer
- NYHA class II-III symptoms
- Ability to understand and provide signed informed consent
- Reasonable expectation that patient will receive standard post-treatment care and attend all scheduled safety follow-up visits
Who cannot join
- Pregnant or nursing women or those of childbearing age and not using an effective method of contraception
- History of stroke within 3 months
- Cardiac surgery within 3 months prior to randomization or the likelihood of a requirement for such procedures during the study period
- Current ICD or CRT or implantation planned within 6 months of infusion
- Presence of clinically significant, uncorrected valvular heart disease, hypertrophic or restrictive cardiomyopathy, active myocarditis, or uncontrolled hypertension
- History of cardiac arrest or life-threatening arrhythmias within 3 months
Show all 29Show fewer
- Treatment with parenteral inotropic agents within 1 month of randomization
- Anticipated cardiac transplantation within 1 year
- Illness other than heart failure with life expectancy less than 1 year
- Received an experimental drug or device within 30 days of randomization
- Left ventricular assist device or implantation planned in the next 6 months
- Patients with complex congenital heart disease
- Uncontrolled seizure disorder
- Presence of immune deficiency
- Clinically significant hematologic, hepatic, or renal impairment as determined by screening clinical laboratory tests:
- Liver disease = ALT or AST > 3x normal, alkaline phosphatase or bilirubin >2x normal)
- Renal disease = estimated glomerular filtration rate as assessed by the MDRD formula <30 ml/min
- Hematologic = Unexplained leukocytosis >10 or hemoglobin < 9gm/dl
- Presence of any other clinically-significant medical condition, psychiatric condition, or laboratory abnormality, that in the judgment of the investigator or sponsor for which participation in the study would pose a safety risk to the subject
- Inability to comply with the conditions of the protocol
- Malignancy within the previous five years, except adequately treated basal cell carcinoma, provided that it is neither infiltrating nor sclerosing, and carcinoma in situ of the cervix
- Active myocarditis or early postpartum cardiomyopathy (within six months).
- Systemic corticosteroids, cytostatics, immunosuppressive drug therapy (cyclophosphamide, methotrexate, cyclosporine, azathioprine, etc.), and DNA depleting or cytotoxic drugs taken within four weeks prior to study treatment
- Porphyria
- Allergy to sodium citrate or any "caine" type of local anesthetic
- Any contraindication for gadolinium use for MRI
- Patient scheduled for hospice care
- Clinically relevant abnormal findings in the clinical history, physical examination, ECG, or laboratory tests at the screening assessment that would interfere with the objectives of the study or would preclude safe completion of the study. Abnormal findings could include: known HIV infection or other immunodeficiency state, chronic active viral infection (such as hepatitis B or C), acute systemic infections (defined as patients undergoing treatment with antibiotics), gastrointestinal tract bleeding, or any severe or acute concomitant illness or injury
- Any other medical, social, or geographical factor that would make it unlikely that the patient could comply with study procedures (e.g., alcohol abuse, lack of permanent residence, severe depression, disorientation, distant location, or noncompliance)
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Emory University Hospital
Atlanta, Georgia, 30322
Northwestern University Centers for Heart Failure Therapy
Chicago, Illinois, 60611
Hospital of the University of Pennsylvania, Heart Failure and Transplant Program
Philadelphia, Pennsylvania, 19104
Stony Brook Heart Institute
Stony Brook, New York, 11794
MedStar Washington Hospital Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Phase IIa Randomized Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Ischemia-tolerant Allogeneic Mesenchymal Bone Marrow Cells to Subjects With Non-ischemic Heart Failure
A phase IIa study to assess the safety and preliminary efficacy of intravenous dose of ischemia-tolerant Allogeneic Mesenchymal Bone Marrow Cells in subjects with non-ischemic heart failure.
Read the detailed description
A phase IIa, single-blind, placebo-controlled, crossover, multi-center, randomized study to assess the safety, tolerability, and preliminary efficacy of a single intravenous dose of ischemia-tolerant allogeneic mesenchymal bone marrow cells to subjects with heart failure of non-ischemic etiology.
- Conditions
- Non-Ischemic Heart Failure
- Being tested
- Allogeneic Mesenchymal Bone Marrow Cells (aMBMC), Lactated Ringer's Solution
- Keywords
- Non-ischemic Heart Failure, Stem Cells
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Mesenchymal Bone Marrow Cells (aMBMC)Drug
Also called Mesenchymal Stem Cells, (MSC) Marrow Stromal Cells.
One time infusion Allogeneic Mesenchymal Bone Marrow Cells (aMBMC) 1.5 million cells/kg.
Lactated Ringer's SolutionDrug
Also called (LRS)
One time infusion 1.5mL/kg
| Study group | Role | What they receive |
|---|---|---|
| Experimental: Human (aMBMC) | Experimental | Intervention: One time intravenous infusion of 1.5 million (aMBMC) per kg administered at approximately 2mL/min. Maximum dose as for 100kg subject or 150 million cells for any subject 100kg or more. Receives: Drug: Allogeneic Mesenchymal Bone Marrow Cells (aMBMC) |
| Placebo:Lactated Ringer's Solution (LRS) | Placebo comparator | Intervention: One time intravenous infusion of 1.5mL/kg Lactated Ringer's Solution (LRS) administered at a constant rate of approximately 2mL/min. Receives: Drug: Lactated Ringer's Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety Will be Evaluated by Number of AE
As identified in the SAP, the safety analysis was the primary objective and was evaluated by the number of AEs
Measured: Total AEs and SAEs within 450 days post-infusion
Secondary outcomes (1)
Change in LVEF From Baseline to Day 90 Post-initial Infusion.
The secondary efficacy endpoint was the change in LVEF from baseline to Day 90 post-initial infusion. Participants with data available at each time point.
Measured: Baseline to Day 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2015 (estimated)
- Study start1 Jun 2014
- Primary completion11 May 2017
- Study completion11 May 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Non-Ischemic Heart FailureStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- CardioCell LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Stemedica Cell Technologies, Inc.
- Responsible party
- Sponsor
- Investigators
- Kristrun Stardal, RN, BSN, Study director, Clinical Operations Manager
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02467387
- Sponsor's protocol ID
- STEM-104-M-CHF
- Status last verified
- Dec 2019
- First submitted
- 2 Jun 2015
- Last updated
- 7 Jan 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Butler J, Epstein SE, Greene SJ, Quyyumi AA, Sikora S, Kim RJ, Anderson AS, Wilcox JE, Tankovich NI, Lipinski MJ, Ko YA, Margulies KB, Cole RT, Skopicki HA, Gheorghiade M. Intravenous Allogeneic Mesenchymal Stem Cells for Nonischemic Cardiomyopathy: Safety and Efficacy Results of a Phase II-A Randomized Trial. Circ Res. 2017 Jan 20;120(2):332-340. doi: 10.1161/CIRCRESAHA.116.309717. Epub 2016 Nov 16.
PubMed 27856497
Study at a glance
From the registry record, last updated by the sponsor on 7 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIa Randomized Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Ischemia-tolerant Allogeneic Mesenchymal Bone Marrow Cells to Subjects With Non-ischemic Heart Failure
- ClinicalTrials.gov ID
- NCT02467387
- Acronym / sponsor ID
- STEM-104-M-CHF
- Conditions
- Non-Ischemic Heart Failure
- Keywords
- Non-ischemic Heart Failure, Stem Cells
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- CardioCell LLCIndustry (pharma, biotech, devices)
- Collaborators
- Stemedica Cell Technologies, Inc.
- Study start
- 1 Jun 2014 (actual)
- Primary completion
- 11 May 2017 (actual)
- Study completion
- 11 May 2017 (actual)
- First posted
- 10 Jun 2015 (estimated)
- Results
- Posted 7 Jan 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A phase IIa study to assess the safety and preliminary efficacy of intravenous dose of ischemia-tolerant Allogeneic Mesenchymal Bone Marrow Cells in subjects with non-ischemic heart failure.
Detailed description
A phase IIa, single-blind, placebo-controlled, crossover, multi-center, randomized study to assess the safety, tolerability, and preliminary efficacy of a single intravenous dose of ischemia-tolerant allogeneic mesenchymal bone marrow cells to subjects with heart failure of non-ischemic etiology.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Experimental: Human (aMBMC) | Experimental | Drug: Allogeneic Mesenchymal Bone Marrow Cells (aMBMC)Intervention: One time intravenous infusion of 1.5 million (aMBMC) per kg administered at approximately 2mL/min. Maximum dose as for 100kg subject or 150 million cells for any subject 100kg or more. |
| Placebo:Lactated Ringer's Solution (LRS) | Placebo comparator | Drug: Lactated Ringer's SolutionIntervention: One time intravenous infusion of 1.5mL/kg Lactated Ringer's Solution (LRS) administered at a constant rate of approximately 2mL/min. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Males and females ≥18 years of age
- LVEF ≤35% on echocardiogram within 12 months of randomization to undergo MRI
- Screening cardiac MRI at baseline with:
- Ejection fraction ≤40% No significant hyper enhancement on MRI scan in the opinion of the central imaging lab reviewer
- Patients with non-ischemic heart failure etiology, as documented by absent or non-obstructive coronary artery disease on x-ray angiography or coronary computed tomography
Key exclusion criteria (29 listed)
- Pregnant or nursing women or those of childbearing age and not using an effective method of contraception
- History of stroke within 3 months
- Cardiac surgery within 3 months prior to randomization or the likelihood of a requirement for such procedures during the study period
- Current ICD or CRT or implantation planned within 6 months of infusion
- Presence of clinically significant, uncorrected valvular heart disease, hypertrophic or restrictive cardiomyopathy, active myocarditis, or uncontrolled hypertension
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 Mesenchymal Bone Marrow Cells (aMBMC)Also: Mesenchymal Stem Cells, (MSC) Marrow Stromal Cells.One time infusion Allogeneic Mesenchymal Bone Marrow Cells (aMBMC) 1.5 million cells/kg. | Drug | Experimental: Human (aMBMC) |
| Lactated Ringer's SolutionAlso: (LRS)One time infusion 1.5mL/kg | Drug | Placebo:Lactated Ringer's Solution (LRS) |
Primary outcome
Safety Will be Evaluated by Number of AE
Time frame: Total AEs and SAEs within 450 days post-infusion
As identified in the SAP, the safety analysis was the primary objective and was evaluated by the number of AEs
1 secondary outcome
Change in LVEF From Baseline to Day 90 Post-initial Infusion.
Time frame: Baseline to Day 90
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jan 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 27856497Butler J, Epstein SE, Greene SJ, Quyyumi AA, Sikora S, Kim RJ, Anderson AS, Wilcox JE, Tankovich NI, Lipinski MJ, Ko YA, Margulies KB, Cole RT, Skopicki HA, Gheorghiade M. Intravenous Allogeneic Mesenchymal Stem Cells for Nonischemic Cardiomyopathy: Safety and Efficacy Results of a Phase II-A Randomized Trial. Circ Res. 2017 Jan 20;120(2):332-340. doi: 10.1161/CIRCRESAHA.116.309717. Epub 2016 Nov 16.
Related trials for Non-Ischemic Heart Failure
Active, not recruiting
Assessment of Combined CCM and ICD Device in HFrEF
Heart Failure, Heart Failure With Reduced Ejection Fraction
RecruitingObservational study
CCM OPEN HF Registry
Heart Failure
RecruitingObservational study
CytOSorb TreatMent Of Critically Ill PatientS Registry
Septic Shock, Acute Respiratory Distress Syndrome
RecruitingPhase 1
A Phase I Study of Allogeneic Myocardium-Derived Mesenchymal Stem Cells (HB1011) in Patients With Ischemic Cardiomyopathy
Ischemic Cardiomyopathy
RecruitingPhase 4
Withdrawal of Heart Failure Treatment Patients With Non-ischemic Cardiomyopathy Who Have Been Super-responders to Cardiac Resynchronization Therapy.
Non-ischemic Cardiomyopathy, Cardiac Resynchronisation Therapy (CRT)
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.
Kristrun Stardal, RN, BSN study director
Clinical Operations Manager
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Allogeneic Mesenchymal Bone Marrow Cells (aMBMC) and Lactated Ringer's Solution in people with Non-Ischemic Heart Failure, run by CardioCell LLC at 5 sites 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?
The sponsor lists 9 inclusion and 29 exclusion criteria. 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: finished, as of the sponsor's last update on 7 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Jan 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Safety Will be Evaluated by Number of AE (time frame: Total AEs and SAEs within 450 days post-infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02467387, which also lists 1 linked publication.
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 7 Jan 2020.