Hypoplastic Left Heart SyndromePhase 2ELPIS IINCT04925024
Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS): A Phase IIb Clinical Trial.
A Phase 2 study testing Lomecel-B medicinal signaling cells in people with Hypoplastic Left Heart Syndrome, run by Longeveron Inc. at 10 sites in United States.
Trial summary
The purpose of this study is to test whether Lomecel-B™ works in treating patients with hypoplastic left heart syndrome (HLHS) and to gather additional information about the safety of Lomecel-B. Lomecel-B contains human mesenchymal stem cells (MSCs) as the active ingredient. Read more about this study
- Who is it for?
- Ages Up to 12 months
- Conditions the study is for
- Hypoplastic Left Heart Syndrome
- What is being tested?
- Lomecel-B medicinal signaling cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 10 sitesUnited States
- Study dates
- 25 Jun 2021to 31 Aug 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- All participants must have HLHS (includes all types) requiring Stage II palliation (Glenn or Hemi-Fontan operation).
Who cannot join
- Requirement for ongoing mechanical circulatory support immediately prior to Stage II palliation within 5 days
- Need for concomitant surgery for aortic coarctation or tricuspid valve repair or Endocardial fibroelastosis (EFE) resection or left ventricle recruitment procedures
- Undergoing the Stage I (Norwood) procedure that does not have HLHS
- Serum positivity for: human immunodeficiency virus (HIV); hepatitis B virus surface antigen (HBV BsAg); and/or viremic hepatitis C virus (HCV). This criterion can be ascertained by one of three ways:
- Documented history of mother's testing conducted during pregnancy
- Documented history of participants testing.
Show all 11Show fewer
- If above documentation is not available blood will be obtained from participant at Screening/Baseline.
- Parent/guardian that is unwilling or unable to comply with necessary follow-up
- Unsuitability for the study based on the Investigator's clinical opinion
- Known hypersensitivity to dimethyl sulfoxide (DMSO)
- Presence of a pacemaker, or anticipated placement of a pacemaker, at the time of the Stage II palliation
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Advocate Children's Hospital
Chicago, Illinois, 60453
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Children's Nebraska
Omaha, Nebraska, 68114
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Primary Children's Hospital/University of Utah
Salt Lake City, Utah, 84113
About this study
Official title: Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS) : A Phase IIb Clinical Trial.
The purpose of this study is to test whether Lomecel-B™ works in treating patients with hypoplastic left heart syndrome (HLHS) and to gather additional information about the safety of Lomecel-B. Lomecel-B contains human mesenchymal stem cells (MSCs) as the active ingredient. MSCs are special cells in the body that are able to change into other types of cells, such as heart, blood, and muscle cells. MSCs are found in various tissues of the body, such as the bone marrow, which is the spongy tissue inside of your bones. Lomecel-B uses MSCs from bone marrow of unrelated young healthy donors. These are called "allogeneic", and do not require donor matching to the patient.
- Conditions
- Hypoplastic Left Heart Syndrome
- Being tested
- Lomecel-B medicinal signaling cells
- Keywords
- Hypoplastic Left Heart Syndrome, Pediatrics, Heart Defects, Congenital Cardiovascular Diseases, Stem Cells
- 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.
Lomecel-B medicinal signaling cellsBiological
A single administration of Lomecel-B will be performed via 6-10 intramyocardial injections into the right ventricle during the participant's standard of care stage II palliation. Dosing is based on body weight. Each patient will be given 2.5 x 10^5 cells per kg of body weight. The entire dose of the cells will be roughly 600 microliters.
| Study group | Role | What they receive |
|---|---|---|
| Lomecel B Group | Experimental | Participants randomized to receive Lomecel-B injections during their Stage II palliation. Receives: Biological: Lomecel-B medicinal signaling cells |
| No Study Intervention Control Group | No intervention | Participants randomized to receive no study intervention during their Stage II palliation. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in right ventricular ejection fraction (RVEF)
Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.
Measured: Baseline, 12 Months
Secondary outcomes (18)
Change in right ventricular ejection fraction (RVEF)
Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.
Measured: Baseline, 6 Months
Change in right ventricular mass index at diastole
Efficacy will be reported as change in right ventricular mass index at diastole assessed as g/m^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Measured: Baseline, 12 Months
Change in right ventricular end-diastolic volume index (RVEDVI)
Efficacy will be reported as change in right ventricular end diastolic volume index (RVEDVI) assessed as ml/m^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Measured: Baseline, 12 Months
Change in right ventricular end-systolic volume index (RVESVI)
Efficacy will be reported as change in right ventricular end systolic volume index (RVESVI) assessed as ml/m^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Measured: Baseline, 12 Months
Change in right ventricular global longitudinal strain and strain rate
Efficacy will be reported as change in right ventricular global longitudinal strain and strain rate assessed as % and s^-1 respectively and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Measured: Baseline, 12 Months
Change in right ventricular global circumferential strain and strain rate
Efficacy will be reported as the change in right ventricular global circumferential strain and strain rate as % and s^-1 respectively, and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Measured: Baseline, 12 Months
Change in right atrial volume index
Efficacy will be reported as the change in right atrial volume index as ml/m^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Measured: Baseline, 12 Months
Change in tricuspid regurgitation severity
Efficacy will be reported as change in the categorical qualitative assessment of tricuspid valve regurgitation (trivial, mild, moderate, severe) and will be measured via transthoracic echocardiography.
Measured: Baseline, 12 Months
Change in weight
Efficacy measured as change in participant's weight in kg and will be measured serially to assess change in somatic growth.
Measured: Baseline, 12 Months
Change in length (height)
Efficacy measured as change in participant's length (height) in cm and will be measured serially to assess change in somatic growth.
Measured: Baseline, 12 Months
Change in head circumference
Efficacy measured as change in participant's head circumference in cm and will be measured serially to assess change in somatic growth.
Measured: Baseline, 12 Months
Change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)
Efficacy measured as change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) in pg/ml and will be measured serially by blood draw.
Measured: Baseline, 12 Months
Change in modified Ross Heart Failure Classification score
The Ross Heart Failure Classification provides a global assessment of heart failure severity in infants. Efficacy measured as change in classification and will be measured serially via physician's assessment using the modified Ross Heart Failure Classification; classifications defined as Class 1 (no limitations of physical activity); Class 2 (may experience symptoms during moderate exercise but not during rest); Class 3 (symptoms with minimal exertion that interfere with normal daily activity); Class 4 (unable to carry out physical activity/has symptoms at rest that worsen with exertion).
Measured: Baseline, 12 Months
Change in PedsQL™ Infant Scales
The PedsQL™ Infant Scales is a generic health related quality of life instrument specifically for healthy and ill infants ages 1-24 months. Efficacy measured as change in PedsQL total improvement score and will be measured serially by parental proxy-report. Higher scores are indicative of better quality of life.
Measured: Baseline, 12 Months
Freedom from unplanned catheter intervention needed to address the pulmonary arteries or aorta.
Efficacy measured as the number and percentage of participants who do not undergo unplanned catheter interventions to address pulmonary arteries or the aorta.
Measured: Baseline, 12 Months
Change in biomarkers/cytokines
Efficacy measured as change in biomarkers/cytokines in pg/ml and/or ng/ml and will be measured serially by blood draw.
Measured: Baseline, 12 Months
Participants experiencing treatment emergent serious adverse events (TE-SAEs)
Safety will be reported as the number of participants experiencing treatment emergent serious adverse events (TE-SAEs) assessed by treating physician within the 30 days following study procedure. These include: all-cause mortality; greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention; cardiogenic shock; unplanned cardiovascular operation for bleeding due to right ventricular intramyocardial injection site bleeding in the first five days after stage II palliation; sepsis; need for new permanent pacemaker; stroke or embolic event to the brain
Measured: 30 days post Stage II palliation
Participants experiencing major adverse cardiac events (MACE)
Safety will be reported as the number of participants with adjudicated events including worsening heart failure; listed for heart transplant; heart failure hospitalization; cardiovascular mortality
Measured: Baseline, 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2021
- Study start25 Jun 2021
- Primary completion30 Jun 2026 (estimated)
- Study completion31 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Longeveron Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Ann & Robert H Lurie Children's Hospital of Chicago, The University of Texas Health Science Center, Houston
- Responsible party
- Sponsor
- Investigators
- Stu Berger, MD, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04925024
- Sponsor's protocol ID
- 2021-4531
- Other IDs
- 7UG3HL148318-02, 1U24HL148316-01A1
- Acronym
- ELPIS II
- Status last verified
- Jun 2025
- First submitted
- 8 Jun 2021
- Last updated
- 25 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kaushal S, Hare JM, Hoffman JR, Boyd RM, Ramdas KN, Pietris N, Kutty S, Tweddell JS, Husain SA, Menon SC, Lambert LM, Danford DA, Kligerman SJ, Hibino N, Korutla L, Vallabhajosyula P, Campbell MJ, Khan A, Naioti E, Yousefi K, Mehranfard D, McClain-Moss L, Oliva AA, Davis ME. Intramyocardial cell-based therapy with Lomecel-B during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: the ELPIS phase I trial. Eur Heart J Open. 2023 Jan 11;3(2):oead002. doi: 10.1093/ehjopen/oead002. eCollection 2023 Mar.
PubMed 36950450
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS) : A Phase IIb Clinical Trial.
- ClinicalTrials.gov ID
- NCT04925024
- Acronym / sponsor ID
- ELPIS II / 2021-4531
- Conditions
- Hypoplastic Left Heart Syndrome
- Keywords
- Hypoplastic Left Heart Syndrome, Pediatrics, Heart Defects, Congenital Cardiovascular Diseases, Stem Cells
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Longeveron Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Ann & Robert H Lurie Children's Hospital of Chicago, The University of Texas Health Science Center, Houston
- Study start
- 25 Jun 2021 (actual)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 31 Aug 2026 (estimated)
- First posted
- 14 Jun 2021 (actual)
- 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.
The purpose of this study is to test whether Lomecel-B™ works in treating patients with hypoplastic left heart syndrome (HLHS) and to gather additional information about the safety of Lomecel-B. Lomecel-B contains human mesenchymal stem cells (MSCs) as the active ingredient. MSCs are special cells in the body that are able to change into other types of cells, such as heart, blood, and muscle cells. MSCs are found in various tissues of the body, such as the bone marrow, which is the spongy tissue inside of your bones. Lomecel-B uses MSCs from bone marrow of unrelated young healthy donors. These are called "allogeneic", and do not require donor matching to the patient.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Lomecel B Group | Experimental | Biological: Lomecel-B medicinal signaling cellsParticipants randomized to receive Lomecel-B injections during their Stage II palliation. |
| No Study Intervention Control Group | No intervention | –Participants randomized to receive no study intervention during their Stage II palliation. |
Who may qualify
- Ages
- Up to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- All participants must have HLHS (includes all types) requiring Stage II palliation (Glenn or Hemi-Fontan operation).
Key exclusion criteria (11 listed)
- Requirement for ongoing mechanical circulatory support immediately prior to Stage II palliation within 5 days
- Need for concomitant surgery for aortic coarctation or tricuspid valve repair or Endocardial fibroelastosis (EFE) resection or left ventricle recruitment procedures
- Undergoing the Stage I (Norwood) procedure that does not have HLHS
- Serum positivity for: human immunodeficiency virus (HIV); hepatitis B virus surface antigen (HBV BsAg); and/or viremic hepatitis C virus (HCV). This criterion can be ascertained by one of three ways:
- Parent/guardian that is unwilling or unable to comply with necessary follow-up
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 |
|---|---|---|
| Lomecel-B medicinal signaling cellsA single administration of Lomecel-B will be performed via 6-10 intramyocardial injections into the right ventricle during the participant's standard of care stage II palliation. Dosing is based on body weight. Each patient will be given 2.5 x 10^5 cells per kg of body weight. The entire dose of the cells will be roughly 600 microliters. | Biological | Lomecel B Group |
Primary outcome
Change in right ventricular ejection fraction (RVEF)
Time frame: Baseline, 12 Months
Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.
18 secondary outcomes
Change in right ventricular ejection fraction (RVEF)
Time frame: Baseline, 6 Months
Change in right ventricular mass index at diastole
Time frame: Baseline, 12 Months
Change in right ventricular end-diastolic volume index (RVEDVI)
Time frame: Baseline, 12 Months
Change in right ventricular end-systolic volume index (RVESVI)
Time frame: Baseline, 12 Months
Change in right ventricular global longitudinal strain and strain rate
Time frame: Baseline, 12 Months
Change in right ventricular global circumferential strain and strain rate
Time frame: Baseline, 12 Months
Change in right atrial volume index
Time frame: Baseline, 12 Months
Change in tricuspid regurgitation severity
Time frame: Baseline, 12 Months
Change in weight
Time frame: Baseline, 12 Months
Change in length (height)
Time frame: Baseline, 12 Months
Change in head circumference
Time frame: Baseline, 12 Months
Change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)
Time frame: Baseline, 12 Months
Change in modified Ross Heart Failure Classification score
Time frame: Baseline, 12 Months
Change in PedsQL™ Infant Scales
Time frame: Baseline, 12 Months
Freedom from unplanned catheter intervention needed to address the pulmonary arteries or aorta.
Time frame: Baseline, 12 Months
Change in biomarkers/cytokines
Time frame: Baseline, 12 Months
Participants experiencing treatment emergent serious adverse events (TE-SAEs)
Time frame: 30 days post Stage II palliation
Participants experiencing major adverse cardiac events (MACE)
Time frame: Baseline, 12 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2025.
- Background: PubMed 36950450Kaushal S, Hare JM, Hoffman JR, Boyd RM, Ramdas KN, Pietris N, Kutty S, Tweddell JS, Husain SA, Menon SC, Lambert LM, Danford DA, Kligerman SJ, Hibino N, Korutla L, Vallabhajosyula P, Campbell MJ, Khan A, Naioti E, Yousefi K, Mehranfard D, McClain-Moss L, Oliva AA, Davis ME. Intramyocardial cell-based therapy with Lomecel-B during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: the ELPIS phase I trial. Eur Heart J Open. 2023 Jan 11;3(2):oead002. doi: 10.1093/ehjopen/oead002. eCollection 2023 Mar.
- ELPIS II study websiteLink provided by the sponsor.
- Earlier phase I study (ELPIS)Link provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Stu Berger, MD principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
10 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 Lomecel-B medicinal signaling cells in people with Hypoplastic Left Heart Syndrome, run by Longeveron Inc. at 10 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 11 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: under way, not looking for participants, as of the sponsor's last update on 25 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 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. 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?
This study is still under way. Results are not available in the source record yet.
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?
Change in right ventricular ejection fraction (RVEF) (time frame: Baseline, 12 Months). 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 NCT04925024, 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 25 Jun 2025.