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1/2 Allogeneic Human Mesenchymal Stem Cell (MSC) Injection in Patients with Hypoplastic Left Heart Syndrome: A Phase IIb Clinical Trial

Award Number: UH3HL148318
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: COOPERATIVE AGREEMENT
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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This application for the "Allogeneic Human Mesenchymal Stem Cell (MSC) Injection in Patients with Hypoplastic Left Heart Syndrome: A Phase IIb Clinical Trial (ELPIS)" Clinical Coordinating Center (CCC) is collaborative with the ELPIS Data Coordinating Center (DCC) application. The CCC will have ultimate responsibility for managing the ELPIS clinical consortium of six clinical sites, meeting, planning enrollment milestones, distributing CCC resources, and overseeing all aspects of clinical design of the studies. Hypoplastic Left Heart Syndrome (HLHS) is one of the most complex forms of congenital heart disease (CHD), with a reported incidence of 0.2 per 1000 live births or 3% of children born with CHD. Once a universally fatal diagnosis, dramatic improvements in three staged palliative operations now allow the single right ventricle (RV) to functionally support the circulation. Despite these strides in medical care, the mortality rate of these infants remains as high as 25 to 35 percent during the first year of life and continues with a high mortality rate for the rest of their life. Those surviving childhood are likely to progress towards cardiac transplantation, usually due to failure of the systemic RV. To address this RV dysfunction, our stem cell trial is based on a decade of basic research and the ELPIS Phase I study using allogeneic MSCs in HLHS patients at the Stage II operation. Our completed enrolled ELPIS Phase I study supports both the feasibility for this investigative strategy and the safety profile of the allogeneic MSCs. Secondary endpoints demonstrated that the initial MSC treated patients showed by cardiac MRI (CMR) no difference in RV ejection fraction, or end diastolic volumes, however a significant decrease in RV mass was present, a surrogate for decreased hypertrophy. Our hypothesis for this trial is that intramyocardial delivery of allogeneic MSCs will improve the performance of the single systemic RV in HLHS patients at the Stage II operation. A total of 36 HLHS patients who are undergoing the Stage II operation will be single blinded and randomized to either receive MSC treatment or standard of care. Patients will be followed for one year. The primary outcome measurement will be decrease in RV mass as determined by CMR studies at baseline, 6 and 12 months following MSC injection. Secondary outcomes will include improvements in clinical and physiologic endpoints, improvements seen by other global RV cardiac function parameters measured by serial CMR studies, exosome biomarker analysis and safety endpoints. This single blinded randomized clinical trial is designed to address the following specific aims: (1) to analyze MSC delivery for RV function improvement at the Stage II operation; (2) to determine dynamic changes of the amount and composition of plasma biomarkers derived from the transplanted MSCs; and (3) to analyze safety of MSC treatment. Although not planned as a survival study, we anticipate that the proposed study will provide valuable data on the safety and benefit of MSC treatment in HLHS patients. The sufficiently definitive information will inform the decision on whether to proceed with a Phase III clinical trial of therapeutic MSC delivery to reduce mortality in HLHS patients and to direct its study design.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2025 ( Subtotal = $0 )
2025	2024	ANN & ROBERT H LURIE CHILDRENS HOSPITAL OF CHICAGO	225 E CHICAGO AVE	CHICAGO	IL	60611	COOK	USA	Cardiovascular Diseases Research	000	6	12/12/2024	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2024 ( Subtotal = $408,351 )
2024	2024	ANN & ROBERT H LURIE CHILDRENS HOSPITAL OF CHICAGO	225 E CHICAGO AVE	CHICAGO	IL	60611	COOK	USA	Cardiovascular Diseases Research	001	6	8/6/2024	NON-COMPETING CONTINUATION	$408,351
2024	2023	ANN & ROBERT H LURIE CHILDRENS HOSPITAL OF CHICAGO	225 E CHICAGO AVE	CHICAGO	IL	60611	COOK	USA	Cardiovascular Diseases Research	000	5	11/14/2023	NON-COMPETING CONTINUATION	$0
														Subtotal = $408,351

Issue Date FY: 2023 ( Subtotal = $898,221 )
2023	2023	ANN & ROBERT H LURIE CHILDRENS HOSPITAL OF CHICAGO	225 E CHICAGO AVE	CHICAGO	IL	60611	COOK	USA	Cardiovascular Diseases Research	001	5	7/18/2023	NON-COMPETING CONTINUATION	$898,221
2023	2022	ANN & ROBERT H LURIE CHILDRENS HOSPITAL OF CHICAGO	225 E CHICAGO AVE	CHICAGO	IL	60611	COOK	USA	Cardiovascular Diseases Research	000	4	6/8/2023	NON-COMPETING CONTINUATION	$0
														Subtotal = $898,221

Issue Date FY: 2022 ( Subtotal = $1,278,411 )
2022	2022	ANN & ROBERT H LURIE CHILDRENS HOSPITAL OF CHICAGO	225 E CHICAGO AVE	CHICAGO	IL	60611	COOK	USA	Cardiovascular Diseases Research	001	4	8/17/2022	NON-COMPETING CONTINUATION	$1,036,319
2022	2021	ANN & ROBERT H LURIE CHILDRENS HOSPITAL OF CHICAGO	225 E CHICAGO AVE	CHICAGO	IL	60611	COOK	USA	Cardiovascular Diseases Research	000	3	5/24/2022	EXTENSION WITH OR WITHOUT FUNDS	$242,092
														Subtotal = $1,278,411

Issue Date FY: 2021 ( Subtotal = $1,178,321 )
2021	2021	ANN & ROBERT H. LURIE CHILDREN'S HOSPITAL OF CHICAGO	225 E CHICAGO AVE	CHICAGO	IL	60611	COOK	USA	Cardiovascular Diseases Research	000	3	9/9/2021	EXTENSION WITH OR WITHOUT FUNDS	$1,178,321
														Subtotal = $1,178,321

Grand Total All Awards = $3,763,304

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1/2 Allogeneic Human Mesenchymal Stem Cell (MSC) Injection in Patients with Hypoplastic Left Heart Syndrome: A Phase IIb Clinical Trial

Award Number: UH3HL148318

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: COOPERATIVE AGREEMENT

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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