Hypoplastic Left Heart SyndromePhase 1ELPISNCT02398604
Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome
A Phase 1 study testing Allo-hMSCs in people with Hypoplastic Left Heart Syndrome, run by Longeveron Inc. at 2 sites in United States.
Trial summary
This study is intended to evaluate the safety and feasibility of intramyocardial injection of allogeneic mesenchymal cells during the Bi-Directional Cavopulmonary Anastomosis (BDCPA) surgery for hypoplastic left heart syndrome (HLHS) pediatric patients. Read more about this study
- Who is it for?
- Ages Up to 30 days
- Conditions the study is for
- Hypoplastic Left Heart Syndrome
- What is being tested?
- Allo-hMSCs
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 2 sitesUnited States
- Study dates
- Apr 2015to Mar 2019
- 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 Up to 30 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Subjects with hypoplastic left heart syndrome (all types) requiring BDCPA surgery.
Who cannot join
- Have HLHS and restrictive or intact atrial septum.
- Be undergoing the Norwood procedure that do not have HLHS.
- Have significant coronary artery sinusoids.
- Require mechanical circulatory support prior to surgery.
- Have an underlying evidence of arrhythmia requiring anti-arrhythmia therapy.
- Parent or guardian unwilling or unable to comply with necessary follow-up(s)
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- Be serum positive for HIV, hepatitis BsAg (B Surface Antigen) or viremic hepatitis C.
- Be unsuitable for inclusion in the study, in the opinion of the investigator.
- Need for concomitant surgery for aortic coarctation or tricuspid valve repair.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Maryland Medical Center
Baltimore, Maryland, 21201
Johns Hopkins Hospital
Baltimore, Maryland, 21287
About this study
Official title: Allogeneic Human MEsenchymal Stem Cell (hMSC) Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
This study is intended to evaluate the safety and feasibility of intramyocardial injection of allogeneic mesenchymal cells during the Bi-Directional Cavopulmonary Anastomosis (BDCPA) surgery for hypoplastic left heart syndrome (HLHS) pediatric patients.
Read the detailed description
This is an open label study, intended as a safety and efficacy assessment prior to a full comparator study. In this Phase I study, cells administered will be delivered in 6-10 intramyocardial injections that will be tested in 20 patients and 10 patients will be controls with a total of 30 HLHS patients.
A total of 30 patients with HLHS will be enrolled in a staged enrollment process. In this open-labeled study, a maximum of 20 patients will eventually receive intramyocardial injection of the allogeneic mesenchymal stem cells and 10 control patients with no cell injection. The enrollment of the patients will occur in two stages groups: Group A and Group B. In Group A, 10 consecutive HLHS patients will be initially enrolled in the allogenic MSCs treatment arm to determine feasibility and safety. After 6 months of the last enrolled patient in Group A, all Group A patients will be assessed in order to determine whether it is feasible and safe, including the harvesting, processing, and administering of the allogeneic MSCs. Thereafter, Group B will start enrolling a total of 20 HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio, respectively, in order to have 10 allogeneic MSCs-treated patient and 10 control patients. At the completion of this Phase I clinical study, the total enrolled cohort will be 20 patients treated with allogeneic MSCs and 10 patients in the control arm.
- Conditions
- Hypoplastic Left Heart Syndrome
- Being tested
- Allo-hMSCs, Placebo
- Keywords
- Pediatrics, HLHS
- Medical subject headings
- Hypoplastic Left Heart Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allo-hMSCsDrug
Also called stem cells; Allogeneic Human Mesenchymal Stem Cells
Allogeneic Human Mesenchymal Stem Cells
PlaceboDrug
Also called Placebo comparator
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Group A - Allo-hMSCs | Experimental | Group A: Fifteen (20) patients will be treated with Allogenic human mesenchymal stem cells (Allo-hMSCs): A concentration of 5 million cells/ml delivered in a dose of 2.5 x 10^5 cells per kg of recipient (5 million/20kg) Allo-hMSCs. The entire dose of the cells will be divided and delivered in 6-10 open intramyocardial injections during the BDCPA operation. Receives: Drug: Allo-hMSCs |
| Group B | Placebo comparator | Group B: Fifteen (10) patients will be treated with a placebo comparator. The placebo will be divided and delivered in 6-10 open intramyocardial injections during the BDCPA operation. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Monitor major adverse cardiac events
Monitoring of events includes death, sustained/symptomatic ventricular tachycardia requiring intervention with inotropic support, aggravation of heart failure, new myocardial infarction, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards.
Measured: 1 month after injection
Secondary outcomes (4)
Composite Change from baseline in right ventricular function, right ventricular end-diastolic volume, right ventricular end-systolic volume, right ventricular end-systolic diameter and tricuspid regurgitation
as measured by serial echocardiograms and MRI scans.
Measured: baseline, 24 weeks and 1 year
Number of participants with incidence of mortality or need for transplantation after the BDCPA operation
Assessed through the number of participants with adverse events, hospitalizations, or transplantations.
Measured: One year after injections
Composite Changes in somatic growth velocity over time (weight, height, head circumference) from the BDCPA operation
Changes in weight, height, and head circumference will be measured
Measured: 12 months post operative
Assessment of Co-morbidity
Assessment of cardiovascular mortality, all-cause morbidity, Cardiovascular morbidity, re-hospitalizations, need for transplantation.
Measured: up to 12 months follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2015 (estimated)
- Study startApr 2015
- Primary completionMar 2019
- Study completionMar 2019
Why it stopped: Study was closed due to Sponsor switched to Longeveron. New Identifier is NTC03525418
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypoplastic Left Heart SyndromeStudies 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
- Parallel groups
- Model description
- 20 patients and 10 patients will be controls with a total of 30 HLHS patients.
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Longeveron Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anthony Oliva, MD, Principal investigator, Longeveron Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02398604
- Sponsor's protocol ID
- 20140718
- Acronym
- ELPIS
- Status last verified
- Jul 2021
- First submitted
- 24 Dec 2014
- Last updated
- 19 Jul 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Human MEsenchymal Stem Cell (hMSC) Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
- ClinicalTrials.gov ID
- NCT02398604
- Acronym / sponsor ID
- ELPIS / 20140718
- Conditions
- Hypoplastic Left Heart Syndrome
- Keywords
- Pediatrics, HLHS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was closed due to Sponsor switched to Longeveron. New Identifier is NTC03525418
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Longeveron Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2015
- Primary completion
- Mar 2019 (actual)
- Study completion
- Mar 2019 (actual)
- First posted
- 25 Mar 2015 (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.
This study is intended to evaluate the safety and feasibility of intramyocardial injection of allogeneic mesenchymal cells during the Bi-Directional Cavopulmonary Anastomosis (BDCPA) surgery for hypoplastic left heart syndrome (HLHS) pediatric patients.
Detailed description
This is an open label study, intended as a safety and efficacy assessment prior to a full comparator study. In this Phase I study, cells administered will be delivered in 6-10 intramyocardial injections that will be tested in 20 patients and 10 patients will be controls with a total of 30 HLHS patients.
A total of 30 patients with HLHS will be enrolled in a staged enrollment process. In this open-labeled study, a maximum of 20 patients will eventually receive intramyocardial injection of the allogeneic mesenchymal stem cells and 10 control patients with no cell injection. The enrollment of the patients will occur in two stages groups: Group A and Group B. In Group A, 10 consecutive HLHS patients will be initially enrolled in the allogenic MSCs treatment arm to determine feasibility and safety. After 6 months of the last enrolled patient in Group A, all Group A patients will be assessed in order to determine whether it is feasible and safe, including the harvesting, processing, and administering of the allogeneic MSCs. Thereafter, Group B will start enrolling a total of 20 HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio, respectively, in order to have 10 allogeneic MSCs-treated patient and 10 control patients. At the completion of this Phase I clinical study, the total enrolled cohort will be 20 patients treated with allogeneic MSCs and 10 patients in the control arm.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 20 patients and 10 patients will be controls with a total of 30 HLHS patients.
| Arm | Type | Receives |
|---|---|---|
| Group A - Allo-hMSCs | Experimental | Drug: Allo-hMSCsGroup A: Fifteen (20) patients will be treated with Allogenic human mesenchymal stem cells (Allo-hMSCs): A concentration of 5 million cells/ml delivered in a dose of 2.5 x 10^5 cells per kg of recipient (5 million/20kg) Allo-hMSCs. The entire dose of the cells will be divided and delivered in 6-10 open intramyocardial injections during the BDCPA operation. |
| Group B | Placebo comparator | Drug: PlaceboGroup B: Fifteen (10) patients will be treated with a placebo comparator. The placebo will be divided and delivered in 6-10 open intramyocardial injections during the BDCPA operation. |
Who may qualify
- Ages
- Up to 30 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Subjects with hypoplastic left heart syndrome (all types) requiring BDCPA surgery.
Key exclusion criteria (9 listed)
- Have HLHS and restrictive or intact atrial septum.
- Be undergoing the Norwood procedure that do not have HLHS.
- Have significant coronary artery sinusoids.
- Require mechanical circulatory support prior to surgery.
- Have an underlying evidence of arrhythmia requiring anti-arrhythmia therapy.
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 |
|---|---|---|
| Allo-hMSCsAlso: stem cells; Allogeneic Human Mesenchymal Stem CellsAllogeneic Human Mesenchymal Stem Cells | Drug | Group A - Allo-hMSCs |
| PlaceboAlso: Placebo comparatorPlacebo | Drug | Group B |
Primary outcome
Monitor major adverse cardiac events
Time frame: 1 month after injection
Monitoring of events includes death, sustained/symptomatic ventricular tachycardia requiring intervention with inotropic support, aggravation of heart failure, new myocardial infarction, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards.
4 secondary outcomes
Composite Change from baseline in right ventricular function, right ventricular end-diastolic volume, right ventricular end-systolic volume, right ventricular end-systolic diameter and tricuspid regurgitation
Time frame: baseline, 24 weeks and 1 year
Number of participants with incidence of mortality or need for transplantation after the BDCPA operation
Time frame: One year after injections
Composite Changes in somatic growth velocity over time (weight, height, head circumference) from the BDCPA operation
Time frame: 12 months post operative
Assessment of Co-morbidity
Time frame: up to 12 months follow-up
Results and safety
The study was stopped early (reason given: Study was closed due to Sponsor switched to Longeveron. New Identifier is NTC03525418). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2021.
- Interdisciplinary Stem Cell InstituteLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
Related trials for Hypoplastic Left Heart Syndrome
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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.
Anthony Oliva, MD principal investigator
Longeveron Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Allo-hMSCs in people with Hypoplastic Left Heart Syndrome, run by Longeveron Inc. at 2 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 1 inclusion and 9 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: stopped early, as of the sponsor's last update on 19 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 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?
The study was stopped early (reason given: Study was closed due to Sponsor switched to Longeveron. New Identifier is NTC03525418). 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?
Monitor major adverse cardiac events (time frame: 1 month after injection). 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 19 Jul 2021.