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Hypoplastic Left Heart SyndromePhase 1/2NCT03079401

Mesoblast Stem Cell Therapy for Patients With Single Ventricle and Borderline Left Ventricle

A Phase 1/2 study testing MPC; rexlemestrocel-L in people with Hypoplastic Left Heart Syndrome and Atrioventricular Canal, run by Boston Children's Hospital at 1 site in United States.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

Patients under the age of 5, with a diagnosis of hypoplastic left heart syndrome (HLHS), unbalanced atrioventricular canal (uAVC), or borderline left heart who are undergoing staged LV recruitment following bidirectional Glenn (BDG) or undergoing BDG with plans for LV recruitment will be considered for enrollment in… Read more about this study

Who is it for?
Ages Up to 5 years
What is being tested?
MPC; rexlemestrocel-L
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
1 siteUnited States
Study dates
27 Nov 2017to Jan 2024
Phase
Phase 1/2
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 5 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Patients with a history of single ventricle palliation (Stage 1 palliation, PA band, or hybrid procedure) undergoing bidirectional Glenn (BDG) with simultaneous left ventricle (LV) recruitment procedures or those patients undergoing LV recruitment procedures will be considered for enrollment.

Who cannot join

  • Patients with current or history of myocardial tumors
  • Patients with aortic or mitral atresia
  • Patients with a history of high grade ventricular arrhythmias
  • Patients with a known allergy to dimethyl sulfoxide (DMSO)
  • Patient has known allergy to mouse and/or cow products.
  • Patient is prior recipient of stem cell therapy for cardiac repair.
  • Patient has received treatment and/or is within an incomplete follow-up treatment of any investigational cell based therapy within 6 months prior to randomization.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

Other trials in United States

About this study

Patients under the age of 5, with a diagnosis of hypoplastic left heart syndrome (HLHS), unbalanced atrioventricular canal (uAVC), or borderline left heart who are undergoing staged LV recruitment following bidirectional Glenn (BDG) or undergoing BDG with plans for LV recruitment will be considered for enrollment in this study. Those patients enrolled in the study will be randomized to either the experimental arm or control arm of the study. Those patients randomized to the experimental arm will receive mesenchymal precursor cells (MPCs) injected directly into the LV endocardium during their LV recruitment or BDG procedure. Those patients randomized to the control arm will receive normal standard of care during their procedure with no injection of MPCs.

It is believed that injection of MPCs will help improve the chances of those patients with single ventricle or borderline left ventricle being converted to biventricular circulation which could improve quality of life and longevity over palliation.

Read the detailed description

This is a prospective, single center, safety and feasibility, blinded, randomized trial to evaluate the use of MPCs in children with complex cardiac anatomy requiring surgical repair. Patients scheduled to undergo bidirectional Glenn (BDG) with future plans for LV recruitment, or patients with a history of BDG who are currently scheduled to undergo LV recruitment will be eligible.

Twenty-four subjects will be enrolled, 12 to the MPC treatment arm and 12 to the control arm following a 1:1 randomization schema. Randomization will be stratified according to surgeon to assure random distribution of subjects by surgeon. Families, the biostatistician, all clinical staff outside the operating room, and research staff completing data analysis will be blinded to randomization assignment. The PI, operating room staff, and research staff assisting with delivery will be unblinded to the randomization assignment. Families will be made aware of their randomization assignment once all subjects have completed their study Visit 4. Those randomized to the treatment arm will receive MPCs injected directly into the LV endocardium following clinical surgical maneuvers to recruit the LV (mitral valve repair, aortic valve repair, and/or resection of endocardial fibroelastosis) or BDG. Those subjects randomized to the control arm will receive standard LV recruitment or BDG with no injection.

All cardiac tissue acquired as part of a clinically indicated procedure will be collected on enrolled subjects. Most of the tissue sample will be analyzed at the time of collection (histology and H+E stain); the remainder will be banked for potential future testing.

Being tested
MPC; rexlemestrocel-L
Keywords
biventricular conversion
Medical subject headings
Hypoplastic Left Heart Syndrome, Endocardial Cushion Defects

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MPC; rexlemestrocel-LBiological

    Also called Allogeneic Mesenchymal Precursor Cell

    MPCs will be injected into the patient's myocardium during planned surgical procedures.

Study groups in NCT03079401
Study groupRoleWhat they receive
Treatment ArmExperimentalThose randomized to the treatment arm will receive MPCs injected directly into the LV endocardium following clinical surgical maneuvers to recruit the LV (mitral valve repair, aortic valve repair, and/or resection of endocardial fibroelastosis) or BDG. MPCs will be delivered directly into the LV endocardium via a 23-25 gauge needle following completion of all surgical procedures. A total dose of 20 million cells will be delivered, divided evenly into ~11 injections of 50 µL each. The total volume is not to exceed 2.0 mL.
Receives: Biological: MPC; rexlemestrocel-L
Control ArmNo interventionThose subjects randomized to the control arm will receive standard LV recruitment or BDG with no injection.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety- Incidence of severe adverse events

    Subjects will be SAE free for 24 months following injection of MPCs (in comparison of treatment arm to control arm.

    Measured: 24 months

  2. Safety- Absence of PRA status change or local inflammation

    Subjects will be free from Panel Reactive Antibody (PRA) status change for 24 months following injection of MPCs (in comparison of treatment arm to control arm). If there is a PRA of >5%, a donor specific antibody test will be completed,

    Measured: 24 months

Secondary outcomes (3)

  1. Efficacy- rate of biventricular conversion

    Rate of those who are successfully converted to biventricular conversion in the treatment group compared to the control group.

    Measured: 12 months

  2. Efficacy- improvement of LV end diastolic pressure

    Rate of those who have improvement in these measurements in the treatment group compared to the control group.

    Measured: 12 months

  3. Efficacy- improvement of LV mass/volume ratio

    Rate of those who have improvement in these measurements in the treatment group compared to the control group.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Mar 2017
  2. Study start27 Nov 2017
  3. Primary completion19 Dec 2022
  4. Study completionJan 2024 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Outcomes assessor
Enrolment
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03079401
Sponsor's protocol ID
IRB-P00020575
Status last verified
Apr 2023
First submitted
8 Mar 2017
Last updated
21 Apr 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Emani SM, del Nido PJ. Strategies to maintain biventricular circulation in patients with high-risk anatomy. Semin Thorac Cardiovasc Surg Pediatr Card Surg Annu. 2013;16(1):37-42. doi: 10.1053/j.pcsu.2013.01.003.

    PubMed 23561816

  • Emani SM, McElhinney DB, Tworetzky W, Myers PO, Schroeder B, Zurakowski D, Pigula FA, Marx GR, Lock JE, del Nido PJ. Staged left ventricular recruitment after single-ventricle palliation in patients with borderline left heart hypoplasia. J Am Coll Cardiol. 2012 Nov 6;60(19):1966-74. doi: 10.1016/j.jacc.2012.07.041. Epub 2012 Oct 10.

    PubMed 23062531

  • Ascheim DD, Gelijns AC, Goldstein D, Moye LA, Smedira N, Lee S, Klodell CT, Szady A, Parides MK, Jeffries NO, Skerrett D, Taylor DA, Rame JE, Milano C, Rogers JG, Lynch J, Dewey T, Eichhorn E, Sun B, Feldman D, Simari R, O'Gara PT, Taddei-Peters WC, Miller MA, Naka Y, Bagiella E, Rose EA, Woo YJ. Mesenchymal precursor cells as adjunctive therapy in recipients of contemporary left ventricular assist devices. Circulation. 2014 Jun 3;129(22):2287-96. doi: 10.1161/CIRCULATIONAHA.113.007412. Epub 2014 Mar 28.

    PubMed 24682346

  • Psaltis PJ, Carbone A, Nelson AJ, Lau DH, Jantzen T, Manavis J, Williams K, Itescu S, Sanders P, Gronthos S, Zannettino AC, Worthley SG. Reparative effects of allogeneic mesenchymal precursor cells delivered transendocardially in experimental nonischemic cardiomyopathy. JACC Cardiovasc Interv. 2010 Sep;3(9):974-83. doi: 10.1016/j.jcin.2010.05.016.

    PubMed 20850099

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing MPC; rexlemestrocel-L in people with Hypoplastic Left Heart Syndrome and Atrioventricular Canal, run by Boston Children's Hospital 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?

The sponsor lists 1 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 21 Apr 2023. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined 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.

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?

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?

Safety- Incidence of severe adverse events (time frame: 24 months). The study lists 2 primary outcomes in all. 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 NCT03079401, which also lists 4 linked publications.

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 Apr 2023.

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