Key Dates

NOT-HL-23-068: Notice of Intent to Publish a Funding Opportunity Announcement for The Blood and Marrow Transplant Clinical Trials Network - Core Clinical Centers (UG1)

National Heart, Lung, and Blood Institute (NHLBI)

The National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute (NCI) intend to publish a Request for Application (RFA) seeking applications from qualified institutions or consortia to participate in the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) as the Data Coordinating Center (DCC). The DCC will be expected to provide overall coordination, infrastructure, data management, and per-patient capitation to sites for participants enrolled in network studies evaluating hematopoietic cell transplantation (HCT) and adoptive cell therapies. The goal of these trials is to improve the health and quality of life for children and adults with life-threatening non-malignant and malignant blood diseases. Collaboration among clinical sites funded as Core Clinical Centers/or Consortia (CCC), the DCC, participating Affiliate clinical sites, NHLBI, NCI, and other stakeholders is expected. As in the past, BMT CTN will collaborate on trials led by the NCI Clinical Trials Network. BMT CTN will also engage Affiliate sites to allow a wider range of investigators from broader geographic areas to participate on trials. Additionally, NHLBI and NCI plan to issue a companion RFA for the CCCs.

This Notice is being provided to allow potential applicants sufficient time to develop meaningful collaborations and responsive applications.

The intended RFA is expected to be published in the summer of 2023, with an expected application due date in the Fall of 2023.

The RFA for the DCC will utilize the U24 Research Project Cooperative Agreement activity code.

Additional details of the planned RFA are provided below.

This Notice encourages investigators with the experience, knowledge, and skills needed from transplant programs with the commitment and capacity to further the BMT CTN mission to begin to consider applying for the new planned RFA.

The objective of the BMT CTN is to provide an infrastructure for collaborative multi-center clinical research to improve the safety and efficacy of HCT and novel adoptive cell therapies in children and adults with rare and life-threatening non-malignant and malignant blood diseases. To ensure that BMT CTN clinical trials include individuals from under-represented groups and broad geographic areas, the DCC will identify Affiliate centers to bring additional participants to network studies, as needed. Trial results are expected to be widely disseminated and to improve the scientific basis of care for affected individuals.

This Notice encourages organizations and investigators with the resources, infrastructure, and expertise to apply for the DCC. Successful applications will support the launch and simultaneously conduct of at least 4 clinical trials, in addition to conducting an observational study to build a shareable, prospective biospecimen collection from patients with non-malignant blood diseases. The successful applicant institution will demonstrate proficiency and plans for:

1. Comprehensive management of administrative, operational, and scientific activities related to study development, trial launch and coordination, quality assurance, medical oversight, manuscript preparation, and results dissemination.
2. Biostatistical support for study design, sample size calculations, safety monitoring, and data analysis.
3. Development and implementation of a plan to identify qualified Affiliate clinical sites that serve children, adults, individuals from under-represented racial and ethnic groups, and from geographically diverse areas.
4. Development and implementation of a plan for engagement of patients, caregivers, and patient advocates for BMT CTN trials.
5. Management of BMT CTN core regulatory and administration activities, including a single Institutional Review Board (sIRB); support for NHLBI-appointed committees that review protocols scientifically and oversee the safety of participants enrolled on trials.
6. Coordination of logistical and other support services, including convening an External Advisory Group, in collaboration with NHLBI and NCI, and all committees required for the governance of the network.
7. Management and distribution of funds to support the patient care costs associated with the protocols on a per-patient basis, subcontracts with central laboratories and pharmacies, and according to the approved protocol budgets.
8. Data management support for all studies, including studies conducted under an Investigational New Drug (IND) application, with high levels of regulatory reporting and trial master files, as well as patient reported outcomes.
9. Provision of expertise needed for interactions with the Food and Drug Administration and to serve as the Authorized Representative for trials conducted under INDs.
10. An infrastructure that supports investigator and site staff mentorship and training to ensure the conduct high-quality clinical trials.
11. A Leadership Succession Strategy, regardless of whether applications are single-PI or multi-PI; Plans for Enhancing Diverse Perspectives (PEDP); and a Data Management and Sharing Plan.
12. Collection and integration of long-term follow-up data from the Center for International Blood and Marrow Transplant Research (CIBMTR) into the BMT CTN trial datasets.
13. Securement of biorepository science expertise.

To help maintain a vibrant investigator community, the CCCs will provide mentorship for at least one junior faculty member (i.e., an early-career or mid-career researcher) to help equip them with the skills and experiences needed to conduct or lead a multi-center clinical trial successfully. The DCC will coordinate opportunities for the junior faculty to engage in Network activities more fully and will make funds for mentored projects or ancillary studies available. The BMT CTN Steering Committee will determine ways that these funds will be allocated shortly after the start of funding under this RFA.

This planned phase of the BMT CTN will include an observational protocol to build a shareable, prospective biospecimen collection linked to clinical data from patients with non-malignant blood diseases. A protocol team will determine the specimens and relevant, linked clinical data to be collected. The DCC will support the protocol and repository until transitioned to appropriate NHLBI resources programs for sharing with the extramural community for future research.

Applications are not being solicited at this time.

Please direct all inquiries to:
Nancy L. DiFronzo, PhD
NHLBI, Division of Blood Diseases and Resources
301-827-8267
[email protected]