Hypoplastic Left Heart Syndrome Clinical Trial
Official title:
Mesoblast Stem Cell Therapy for Patients With Single Ventricle and Borderline Left Ventricle
Verified date | April 2023 |
Source | Boston Children's Hospital |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 19 |
Est. completion date | January 2024 |
Est. primary completion date | December 19, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 5 Years |
Eligibility | Inclusion Criteria: - 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. Exclusion Criteria: - 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. |
Country | Name | City | State |
---|---|---|---|
United States | Boston Children's Hospital | Boston | Massachusetts |
Lead Sponsor | Collaborator |
---|---|
Boston Children's Hospital |
United States,
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. — View Citation
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. — View Citation
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. — View Citation
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. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | 24 months | |
Primary | 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, | 24 months | |
Secondary | Efficacy- rate of biventricular conversion | Rate of those who are successfully converted to biventricular conversion in the treatment group compared to the control group. | 12 months | |
Secondary | 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. | 12 months | |
Secondary | 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. | 12 months |
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