NIH/NIAID – Expanding Extramural Research Opportunities at the NIH Clinical Center (U01 Clinical Trial Optional)

August 1, 2018 by School of Medicine Webmaster

The NIH Clinical Center is the largest biomedical research hospital in the world, and it is a unique local, regional, and national research resource. Although this Clinical Center represents a key resource for NIH intramural scientists to conduct clinical research and clinical trials, extramural scientists funded by NIH grants and contracts also may access this distinctive facility. To ensure the Clinical Center maximizes its potential to support the best possible extramural science, the NIAID wants to leverage these valuable inpatient and outpatient clinical research resources with the needs of the extramural community.

The NIAID mission is to conduct and fund research to understand, treat, and prevent infectious, immunologic and allergic diseases. Scientific priorities are described in the NIAID Strategic Plan, 2017pdf. Extramural scientists proposing research within NIAID’s priorities have an opportunity to utilize the unique resources of the Clinical Center (for a description of resources, see Support for clinical research and clinical trials conducted by NIAID and NIAID-funded scientists is also available through the NIAID Division of Clinical Research (DCR). DCR provides multi-disciplinary trans-NIAID services to facilitate clinical research, such as assisting in protocol development, study design, statistics and regulatory compliance (see

In particular:

Research Objectives and Scope

The goal of this FOA is to support extramural investigator-initiated clinical research in partnership with the NIH Clinical Center in Bethesda, MD. This new FOA will leverage the resources (inpatient and outpatient) and assets of the NIH Clinical Center (e.g., scientific and clinical expertise, nursing, beds, critical care services, ambulatory care services, laboratories, imaging, biostatistics, protocol development, regulatory guidance, clinical trials management and safety oversight) in accelerating the discovery and translation from laboratory to clinic of therapies for infectious (including primary immunodeficiency diseases), immunologic, and allergic diseases. Specifically, it will support hypothesis-driven mechanistic studies alone or within clinical projects employing Phase 0, 1, and/or 2a clinical trial designs. In the context of the FOA, a clinical project is a clinical trial together with integrated mechanistic or genetic studies.

Types of clinical trials and clinical research that may be proposed include but are not limited to the following:

  • Phase 0 clinical trials in healthy subjects (e.g., assess safety and explore pharmacokinetic (PK) characteristics of promising interventional agents (e.g., drugs, cells, and molecules) or new formulations of FDA approved drugs (e.g., calcineurin inhibitor immunosuppression)
  • Observational clinical research with associated mechanistic studies and/or genetic studies
  • Chronic allograft injury and destruction after organ transplantation
  • Treatment of post-transplant cancers
  • Treatment of post-transplant lymphoproliferative disease (PTLD)
  • Identification of genetic determinants of outcome or response to therapy in organ transplant recipients
  • Infectious disease challenge studies in healthy volunteers
  • Phase 1 (initial safety trial) and 2a pilot clinical trials to evaluate efficacy (and safety) in patients with the immune-mediated disease or condition to be treated, diagnosed, or prevented
  • Repurposing drugs approved for other indications for diseases within the NIAID mission based on the known mechanism of the drug
  • Cross-sectional and epidemiologic studies in specific disease cohorts looking at intermediate and long-term follow-up
  • Evaluation of immune response to licensed vaccines in special populations
  • Infectious diseases studies in special populations, including primary immunodeficiency diseases, concurrent diseases states, cystic fibrosis
  • New therapeutic approaches applying cutting edge advances in fundamental immunity and biology. Examples include: microbiome, gene therapy, regulating gene transcription, RNA metabolism, cellular therapy, cytokines, chemokines, and proteins mediating signal transduction
  • Develop diagnostic, predictive, and critical biomarkers that will facilitate routine surveillance, early diagnosis and ongoing monitoring of processes that contribute to morbidity and mortality for immune mediated diseases (e.g., organ transplantation, autoimmune disease, primary immunodeficiency diseases, allergy and asthma)

Specific areas of interest include:

  • Clinical research focused on the high priority areas in HIV/AIDS within the NIAID mission ( )
  • Allergy
  • Food allergy
  • eosinophilic and/or mast cell disorders
  • anaphylaxis
  • drug allergy including interest in immunologic basis of severe adverse drug reactions, diagnosis of drug allergy, role of IgE in drug allergy
  • Atopic dermatitis (eczema) and other allergies (e.g., rhinitis, rhinosinusitis)
  • Primary immunodeficiency diseases
  • Rare diseases in the areas of infectious, primary immunodeficiency, autoimmune, solid organ transplantation and allergic diseases
  • Acute and chronic inflammatory disease (e.g., inflammatory bowel disease)
  • Human immunology in context of biodefense
  • Transplantation immunology (organs, tissues, cells, and molecules)
  • New interventional immunotherapeutics
  • Host defense
  • Infectious diseases
  • Novel therapeutic vaccination approaches

NIAID reserves the right to specify: 1) whether an Investigational New Drug (IND)/ Investigational Device Exemption (IDE) application should be submitted to an appropriate regulatory agency and determine the entity (NIAID, primary awardee, etc.) who will hold the IND/IDE. In most cases, it is expected NIAID will not hold the IND/IDE and the primary extramural awardee has engaged with appropriate regulatory agencies with the IND/IDE in hand (as necessary or required) at the time of grant application. Potential applicants are encouraged to discuss their research proposal and potential need for formal regulatory consultation with the NIAID Program Officer in consultation with CC personnel. It is expected that the IND/IDE would be finalized within 6 months of award.


Milestones are a key component of the clinical trials awarded under this announcement and are only required if proposing a clinical trial. A milestone is defined as a scheduled event in the project timeline, signifying completion of a major project stage or activity. In the case of clinical trials, milestones commensurate with opening enrollment, quantitative yearly milestones, and completing clinical trial(s) within the period of award will be incorporated into the terms of award. Completion of research reports, preparation of peer-reviewed manuscripts, and data sharing are required milestones when individual study end points are met.

Planning Activities

It is expected that all necessary activities associated with the proposed research, especially in the case of clinical trials, will not be complete at the time of submission. Up to one year may be used to support planning, design, and preparation of documentation necessary for implementation of the proposed research with the clinical center.

Planning activities that may be proposed include but are not limited to the following:

  • Final protocol development
  • IRB approval
  • Review from regulatory agency (if applicable)
  • Development of data safety monitoring plan
  • Recruitment plan

It is anticipated that the clinical research activities would largely be conducted at the Clinical Center, with some additional NIAID support for work to be performed in the extramural investigator’s laboratory. While collaborations with intramural investigators are allowed, it is not required. Moreover, projects that take only minimal advantage of Clinical Center resources would not be considered for NIAID support. For guidance on whether a project sufficiently utilizes CC resources, contact the CC Research contact in Section VII.

For more information regarding this FOA, please refer to the NIAID FAQ webpage at:

The following will not be supported by this FOA:

  • Select agent research
  • Multisite clinical trials
  • Phase 2b and above clinical trials
  • Research focused only on clinical samples or data
  • Research using animal models and/or “humanized” animals
  • On-going clinical trials
  • Research Projects without integrated mechanistic studies
  • Xenotransplantation studies
  • Studies of islet transplantation for treatment of Type 1 diabetes
  • Studies of hematopoietic stem cell transplantation (HSCT) unless HCST is a component of a study/therapy for primary immunodeficiency disease or organ transplantation
  • Studies requiring organ transplantation at the NIH Clinical Center
  • Research projects without significant Clinical Center utilization
  • Projects only utilizing banked samples or data
  • Studies that could easily be performed within existing academic medical centers or outpatient clinical facilities around the country

Specific Areas of Research Interest

Prior to submitting to this FOA, applicants are strongly encouraged to consult with the Scientific/Research contacts for the area of science for which they are planning to develop an application. Early contact (at least 12 weeks prior to submission or longer if IDE/IND is needed) is encouraged, especially with the contact for the NIH Clinical Center ( This period of time provides an opportunity for NIAID staff to discuss the scope and goals, and to provide information and guidance to the applicants.

Deadlines:  December 3, 2018, December 3, 2019, December 3, 2020 (letters of intent due 30 days prior to the deadline)


Filed Under: Funding Opportunities