NINDS ALS Expanded Access Program (RFA-NS-26-001)

NINDS ALS Expanded Access Program (RFA-NS-26-001) is sponsored by National Institute of Neurological Disorders and Stroke (NINDS). This program supports research aimed at expanding access to promising investigational therapies for Amyotrophic Lateral Sclerosis (ALS). The goal is to provide access for patients who are not eligible for or cannot participate in ongoing clinical trials.

RFA-NS-26-001: Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required) Department of Health and Human Services Part 1.

Overview Information Participating Organization(s) National Institutes of Health ( NIH ) Components of Participating Organizations National Institute of Neurological Disorders and Office of The Director, National Institutes of Health ( OD ) Funding Opportunity Title Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required) U01 Research Project – Cooperative Agreements Check for any recent Notices of NIH Policy Changes that may impact application requirements.

Funding Opportunity Number (FON) Companion Funding Opportunity See Section III. Eligibility Information .

Assistance Listing Number(s) Funding Opportunity Purpose The purpose of this Notice of Funding Opportunity (NOFO) is to solicit applications for the conduct of scientific research utilizing data from expanded access (EA) for investigational drugs or biological products as described in section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) .

These applications will target intermediate size populations of patients living with amyotrophic lateral sclerosis (ALS) who are not eligible for ongoing clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of ALS.

Funding Opportunity Goal(s) To support extramural research funded by the National Institute of Neurological Disorders and Stroke (NINDS) including: basic research that explores the fundamental structure and function of the brain and the nervous system; research to understand the causes and origins of pathological conditions of the nervous system with the goal of preventing these disorders; research on the natural course of neurological disorders; improved methods of disease prevention; new methods of diagnosis and treatment; drug development; development of neural devices; clinical trials; and research training in basic, translational, and clinical neuroscience.

Open Date (Earliest Submission Date) Renewal / Resubmission / Revision (as allowed) AIDS - New/Renewal/Resubmission/Revision, as allowed All applications are due by 5:00 PM local time of applicant organization. Applicants are encouraged to apply early to allow adequate time to make any corrections to errors found in the application during the submission process by the due date.

No late applications will be accepted for this Notice of Funding Opportunity (NOFO). Required Application Instructions It is critical that applicants follow the instructions in the Research (R) Instructions in the How to Apply - Application Guide , except where instructed to do otherwise (in this NOFO or in a Notice from NIH Guide for Grants and Contracts ).

Conformance to all requirements (both in the Application Guide and the NOFO) is required and strictly enforced. Applicants must read and follow all application instructions in the Application Guide as well as any program-specific instructions noted in Section IV. When the program-specific instructions deviate from those in the Application Guide, follow the program-specific instructions.

Applications that do not comply with these instructions may be delayed or not accepted for review. There are several options available to submit your application through Grants. gov to NIH and Department of Health and Human Services partners.

You must use one of these submission options to access the application forms for this opportunity. Use the NIH ASSIST system to prepare, submit and track your application online. Use an institutional system-to-system (S2S) solution to prepare and submit your application to Grants.

gov and eRA Commons to track your application. Check with your institutional officials regarding availability. Use Grants.

gov Workspace to prepare and submit your application and eRA Commons to track your application. Part 1. Overview Information Part 2.

Full Text of Announcement Section I. Notice of Funding Opportunity Description Section II. Award Information Section III.

Eligibility Information Section IV. Application and Submission Information Section V. Application Review Information Section VI.

Award Administration Information Section VII. Agency Contacts Section VIII. Other Information Part 2.

Full Text of Announcement Section I. Notice of Funding Opportunity Description The purpose of this Notice of Funding Opportunity (NOFO) is to solicit applications for the conduct of scientific research utilizing data from expanded access (EA) for investigational drugs or biological products as described in section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) .

These applications will target intermediate size populations of patients living with amyotrophic lateral sclerosis (ALS) who are not eligible for ongoing clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of ALS ("intermediate EA protocol for ALS").

Provision of the investigational drug or biological product under an intermediate EA protocol for ALS must not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval or otherwise compromise the potential development of medical products for the prevention, diagnosis, mitigation, treatment, or cure of ALS.

ALS is a rapidly progressive, ultimately fatal, neurodegenerative disease causing weakness and wasting of skeletal muscles including the diaphragm. There are approximately 34,000 cases in the United States (U.S.) equivalent to a prevalence of 10. 1 per 100,000 U.S. population.

While considerable variability in presentation and progression exists, mean survival from symptom onset is typically only 3–5 years, and treatment options for ALS remain severely limited. Existing disease-modifying drugs for ALS have only modest effects on slowing disease progression in most ALS cases, and no known treatment prevents, halts or reverses ALS progression.

Thus, development of new, effective treatments to prevent disease onset, make ALS a livable disease, or cure ALS is a pressing need. To address this challenge, individuals with ALS may enroll in ongoing phase 3/efficacy clinical trials of investigational drugs and biological products for ALS.

However, trial sponsors typically set inclusion criteria that restrict participation to only a subset of people with ALS to increase the likelihood of detecting efficacy with feasible sample size and trial duration. Thus, a potentially large segment of people with ALS may be ineligible to participate in clinical trials because they do not meet these inclusion criteria.

EA, sometimes referred to as “compassionate use”, was established to provide treatment access to investigational medical products (i.e., drugs, biological products, medical devices) for people with serious, life-threatening diseases like ALS when no comparable or satisfactory therapy is available to diagnose, monitor, or treat the disease or condition. This pathway is defined and regulated by the FDA (see https://www. fda.

gov/news-events/public-health-focus/expanded-access ). Further, the FDA has not yet determined if these products are safe and effective for their intended use.

One requirement of the EA pathway is that providing the investigational medical product under EA must not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of a medical product for the EA use, or otherwise compromise its development.

An intermediate-size patient population EA protocol is generally used when more than one patient will be treated with an investigational drug/biological product under the EA ( 21 CFR 312. 315 ), and can involve hundreds or even thousands of patients if appropriate. Section 2 of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS; P.

L. 117-79) established a grant program for scientific research utilizing data from EA to investigational drugs for ALS. Since fiscal year 2022, NIH has been implementing this grant program through issuing NOFOs, including this one, and funding new EA research grants.

The objective of this NOFO is to support the conduct of scientific research utilizing data from intermediate-size patient population EA protocols for ALS ("intermediate EA protocol for ALS", see https://www. fda. gov/news-events/expanded-access/expanded-access-categories-drugs-including-biologics ).

To be eligible, the proposed intermediate EA protocol for ALS will provide access to an investigational drug or biological product for people with ALS and also generate data to support research on the prevention, diagnosis, mitigation, treatment, or cure of ALS.

The scientific research goal(s) informed by these data could include the investigational drug’s or biological product’s effect on biomarkers related to the pathophysiology of ALS or target engagement, the collection of safety or outcome information, such as survival or other significant medical events (e.g., hospitalization or need for ventilatory support), or patient experience data that are not specifically tied to assessing efficacy.

Additionally, this NOFO allows for competitive renewal of currently funded EA protocols through the NINDS. (See instructions under Section IV under Research Strategy.) Applicants are encouraged to discuss the submission of a renewal application with NINDS and seek approval to submit as necessary prior to submitting the application.

Applicants should take note of the following special requirements and considerations: EA New Drug Application (IND): An intermediate-size patient population EA IND application must be submitted to the FDA by the day of submission of the grant application to the NINDS. Approval notification from the FDA, such as a "may proceed" email or letter, must be submitted to the NINDS prior to funding.

Applicants should describe how real world data (RWD) from the EA cohort will complement existing trials. The investigational drug or biological product must not be approved under a New Drug Application (NDA) or licensed under a Biologics License Application (BLA).

Institutional Review Board (IRB) approval of the intermediate EA protocol for ALS and the informed consent document are not required at the time of application submission but must be documented prior to funding and initiation of treatment of participants. As such, the NINDS encourages investigators to begin these processes as early as possible.

Funding for the intermediate EA protocol for ALS will continue until one of the following events: Marketing authorization of the drug or biological product by the FDA Withdrawal or termination of the IND for the investigational drug or biological product by the sponsor Decision by the FDA to put the EA protocol on hold, e.g., should information emerge that alters the acceptability of the EA use Scenarios such as: (a) failure to implement the study protocol, (b) a substantial shortfall in study participant recruitment, follow-up, data reporting and dissemination, quality control, or other major breach of the protocol, (c) substantive changes in the agreed-upon protocol with which the NINDS does not concur, (d) reaching a major study objective substantially ahead of schedule with persuasive statistical evidence, (e) study participant safety or ethical issues that may dictate a premature termination, or (f) a change in the state of science that has a significant impact on the relevance of the question The project period has ended.

The consent process should clearly explain to the study participants that grant support for the EA protocol is limited to the project period.

This NOFO will only support applications from clinical trial sites that participate in a phase 2/3 or phase 3 efficacy clinical trial supported by a U.S.-based small business concern that is the FDA-designated sponsor of a drug or biological product which is the subject of an IND under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) to prevent, diagnose, mitigate, treat, or cure ALS.

This NOFO will only support applications that include a description of how the proposed intermediate EA protocol for ALS will not interfere with participant enrollment in ongoing clinical trials intended to support a regulatory decision for investigational therapies for the prevention, diagnosis, mitigation, treatment, or cure of ALS.

To maximize comparisons across datasets or studies and facilitate data integration and collaboration, this NOFO strongly encourages the use of standards and resources listed in Section IV. Application and Submission Information where applicable. This NOFO strongly encourages applications that include a plan for stakeholder engagement.

Stakeholder engagement in clinical studies typically involves people with lived experiences (PWLE), health professionals, and the clinical research team working in active partnership at various levels across the continuum of clinical research.

Leveraging the resources and support from advocacy groups, private research foundations, academic institutions, other government agencies, and the NIH Intramural Program is also strongly encouraged. Investigators proposing NIH-defined clinical trials may refer to the Research Methods Resources website for information about developing statistical methods and study designs. See Section VIII.

Other Information for award authorities and regulations. Section II. Award Information Cooperative Agreement: A financial assistance mechanism used when there will be substantial Federal scientific or programmatic involvement.

Substantial involvement means that, after award, NIH scientific or program staff will assist, guide, coordinate, or participate in project activities. See Section VI. 2 for additional information about the substantial involvement for this NOFO.

Application Types Allowed The OER Glossary and the How to Apply Application Guide provide details on these application types. Only those application types listed here are allowed for this NOFO. Required: Only accepting applications that propose clinical trial(s).

Need help determining whether you are doing a clinical trial? Funds Available and Anticipated Number of Awards NINDS intends to commit up to $40,000,000 per fiscal year to fund 4 new awards per year for the duration of this RFA. This estimate is contingent upon NIH appropriations and a sufficient number of meritorious applications.

Application budgets need to reflect the actual needs of the proposed project. The scope of the proposed project should determine the project period. The maximum project period is 4 years.

NIH grants policies as described in the NIH Grants Policy Statement will apply to the applications submitted and awards made from this NOFO. Section III.

Eligibility Information Higher Education Institutions - Includes all types Public/State Controlled Institutions of Higher Education Private Institutions of Higher Education Nonprofits Other Than Institutions of Higher Education Nonprofits with 501(c)(3) IRS Status (Other than Institutions of Higher Education) Nonprofits without 501(c)(3) IRS Status (Other than Institutions of Higher Education) For-Profit Organizations (Other than Small Businesses) City or Township Governments Special District Governments Indian/Native American Tribal Governments (Federally Recognized) Indian/Native American Tribal Governments (Other than Federally Recognized).

Eligible Agencies of the Federal Government U.S. Territory or Possession Independent School Districts Public Housing Authorities/Indian Housing Authorities Native American Tribal Organizations (other than Federally recognized tribal governments) Faith-based or Community-based Organizations Eligible applicants must be clinical trial sites that participate in a phase 2/3 or phase 3 efficacy clinical trial supported by a U.S.-based small business concern that is the FDA-designated sponsor of a drug or biological product which is the subject of an IND under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.

355(i)) to prevent, diagnose, mitigate, treat, or cure ALS. The definition of a small business concern can be found in section 3(a) of the Small Business Act ( 15 U.S.C. 632(a) ).

All activities proposed in your application and budget narrative must align with applicable law, including but not limited to statutes, executive orders, federal regulations and applicable judicial holdings.

Accordingly, discretionary awards shall not be used to fund, promote, encourage, subsidize, or facilitate; racial preferences or other forms of racial discrimination by the recipient, including activities where race or intentional proxies for race will be used as a selection criterion for employment or program participation; denial by the recipient of the sex binary in humans, or the belief that sex is a chosen or mutable characteristic; illegal immigration; or any other initiatives that compromise public safety.

If an application does not align, the application will not receive funding to the extent permitted by law and applicable court orders. Foreign Organizations/International Collaborations Non-domestic (non-U.S.) Entities (Foreign Organizations) are not eligible to apply. Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply.

Foreign components, as defined in the NIH Grants Policy Statement , are not allowed. Applicant organizations must complete and maintain the following registrations as described in the How to Apply- Application Guide to be eligible to apply for or receive an award. All registrations must be completed prior to the application being submitted.

Registration can take 6 weeks or more, so applicants should begin the registration process as soon as possible. Failure to complete registrations in advance of a due date is not a valid reason for a late submission, please reference the NIH Grants Policy Statement Section 2. 3.

9. 2 Electronically Submitted Applications for additional information. System for Award Management (SAM) – Applicants must complete and maintain an active registration, which requires renewal at least annually .

The renewal process may require as much time as the initial registration. SAM registration includes the assignment of a Commercial and Government Entity (CAGE) Code for domestic organizations which have not already been assigned a CAGE Code. Foreign organizations must obtain a NATO Commercial and Government Entity (NCAGE) Code (in lieu of a CAGE code) in order to register in SAM.

Unique Entity Identifier (UEI)- A UEI is issued as part of the SAM. gov registration process. The same UEI must be used for all registrations, as well as on the grant application.

eRA Commons - Once the unique organization identifier is established, organizations can register with eRA Commons in tandem with completing their Grants. gov registrations; all registrations must be in place by time of submission. eRA Commons requires organizations to identify at least one Signing Official (SO) and at least one Program Director/Principal Investigator (PD/PI) account in order to submit an application.

Grants. gov – Applicants must have an active SAM registration in order to complete the Grants. gov registration.

Program Directors/Principal Investigators (PD(s)/PI(s)) All PD(s)/PI(s) must have an eRA Commons account. PD(s)/PI(s) should work with their organizational officials to either create a new account or to affiliate their existing account with the applicant organization in eRA Commons. If the PD/PI is also the organizational Signing Official, they must have two distinct eRA Commons accounts, one for each role.

Obtaining an eRA Commons account can take up to 2 weeks. All PD(s)/PI(s) must be registered with ORCID . The personal profile associated with the PD(s)/PI(s) eRA Commons account must be linked to a valid ORCID ID.

For more information on linking an ORCID ID to an eRA Commons personal profile see the ORCID topic in our eRA Commons online help .

Eligible Individuals (Program Director/Principal Investigator) Any individual(s) with the skills, knowledge, and resources necessary to carry out the proposed research as the Program Director(s)/Principal Investigator(s) (PD(s)/PI(s)) is invited to work with their organization to develop an application for support.

For institutions/organizations proposing multiple PDs/PIs, visit the Multiple Program Director/Principal Investigator Policy and submission details in the Senior/Key Person Profile (Expanded) Component of the How to Apply-Application Guide. This NOFO does not require cost sharing as defined in the NIH Grants Policy Statement Section 1. 2 Definition of Terms .

3. Additional Information on Eligibility Applicant organizations may submit more than one application, provided that each application is scientifically distinct. The NIH will not accept duplicate or highly overlapping applications under review at the same time, per NIH Grants Policy Statement Section 2.

3. 7. 4 Submission of Resubmission Application .

This means that the NIH will not accept: A new (A0) application that is submitted before issuance of the summary statement from the review of an overlapping new (A0) or resubmission (A1) application. A resubmission (A1) application that is submitted before issuance of the summary statement from the review of the previous new (A0) application.

An application that has substantial overlap with another application pending appeal of initial peer review (see NIH Grants Policy Statement 2. 3. 9.

4 Similar, Essentially Identical, or Identical Applications ). Section IV. Application and Submission Information 1.

Requesting an Application Package The application forms package specific to this opportunity must be accessed through ASSIST, Grants. gov Workspace or an institutional system-to-system solution. Links to apply using ASSIST or Grants.

gov Workspace are available in Part 1 of this NOFO. See your administrative office for instructions if you plan to use an institutional system-to-system solution. 2.

Content and Form of Application Submission It is critical that applicants follow the instructions in the Research (R) Instructions in the How to Apply - Application Guide except where instructed in this notice of funding opportunity to do otherwise (in this NOFO, in a policy notice , or other notice from NIH Guide for Grants and Contracts ). Conformance to the requirements in the Application Guide is required and strictly enforced.

Applications that are out of compliance with these instructions may be delayed or not accepted for review. All page limitations described in the How to Apply- Application Guide and the Table of Page Limits must be followed. Instructions for Application Submission The following section supplements the instructions found in the How to Apply- Application Guide and should be used for preparing an application to this NOFO.

All instructions in the How to Apply - Application Guide must be followed. SF424(R&R) Project/Performance Site Locations All instructions in the How to Apply- Application Guide must be followed. SF424(R&R) Other Project Information All instructions in the How to Apply- Application Guide must be followed.

SF424(R&R) Senior/Key Person Profile All instructions in the How to Apply- Application Guide must be followed. All instructions in the How to Apply- Application Guide must be followed. Funds may be requested for: Payment to the manufacturer or sponsor for the costs related to the investigational drug or biological product of the intermediate EA protocol for ALS, as authorized under section 312.

8(d) of title 21, Code of Federal Regulations (or successor regulations) (indirect costs not allowed) Costs incurred in providing such investigational drug/biological product consistent with the research objectives of the grant (indirect cost not allowed) Costs associated with biospecimen collection and banking through BioSEND or other repositories Costs to cover expenses incurred by participating clinical trial sites in conducting research with respect to such investigational drugs/biological products including any analysis of the clinical data or biospecimens collected Personnel effort, support for study participant travel/meals, and other budget items within the overall budget cap to ensure inclusion of all populations at risk for ALS All instructions in the How to Apply - Application Guide must be followed.

PHS 398 Cover Page Supplement All instructions in the How to Apply - Application Guide must be followed.

All instructions in the How to Apply - Application Guide must be followed, with the following additional instructions: Significance: Describe how data generated through the proposed intermediate EA protocol for the investigational drug/biological product will advance research or development to support prevention, diagnosis, mitigation, treatment, or cure of ALS.

Biological and/or Clinical Relevance : Applications should not only describe the research goal(s), but also describe how achieving the goal(s) will advance our understanding of ALS by addressing the investigational drug's/biological product's effect on biomarkers related to the pathophysiology of ALS or target engagement, collection of safety or outcome information (such as survival or other significant medical events [e.g., hospitalization or need for ventilatory support]), or patient experience data not specifically tied to assessing efficacy.

Preliminary Studies : Present the scientific rationale for the research goal(s) to be supported by data generated through the proposed intermediate EA protocol for ALS, including preliminary data from clinical and/or preclinical studies, or data from mechanistic studies.

For renewal applications, describe 1) details of progress made toward regulatory approval, especially progress in pivotal clinical trials of the investigational drug or biological product, with particular attention to progress made during the awarded grant period, and 2) the recruitment goals and associated timeline of the ongoing/planned pivotal trial, as applicable.

For renewal applications , describe 1) details of progress made toward regulatory approval, especially progress in pivotal clinical trials of the investigational drug or biological product, with particular attention to progress made during the awarded grant period, and 2) the body of existing data from phase 2/3 or phase 3 trials (if completed) that demonstrate the efficacy of the proposed therapeutic and 3) the recruitment goals and associated timeline of the ongoing/planned pivotal trial, as applicable.

Non-interference with Clinical Trials: Describe how the proposed intermediate EA protocol for ALS is designed to avoid 1) interfering with the initiation, conduct, or completion of clinical investigations that could support regulatory approval or 2) otherwise compromise the potential development of medical products for the prevention, diagnosis, mitigation, treatment, or cure of ALS.

Outline how data will be obtained, analyzed, and interpreted with sufficient rigor to quantitatively assess project outcomes.

Describe the use of common data elements (CDEs) , including those available at Amyotrophic Lateral Sclerosis | NINDS CDE , and, as applicable, other NIH or external resources to standardize the collection of clinical data such as: PhenX Toolkit Social Determinants of Health (SDOH) Collections Clinical and Translational Science Award (CTSA) Program HL7 FHIR® (Fast Healthcare Interoperability Resources) Standardized set of data classes, data elements, and associated vocabulary standards specified in the United States Core Data for Interoperability (USCDI) standards FDA Digital Health Center of Excellence As applicable, describe what non-traditional approaches to engage potential study participants for intervention delivery and data collection will be used to minimize participant burden.

In addition, describe the plans, if any, to use non-traditional data collection approaches (e.g., digital/mobile/sensor technologies and web-based systems) and why these are appropriate.

As appropriate, describe the plan for stakeholder engagement, such as establishing relationships with PWLE and/or advocacy groups to solicit their input (including during study design) on recruitment, the clinical meaningfulness of the question under study, the relevance of the proposed clinical outcomes, and approaches to minimizing the burden on study participants.

The summary of the proposed research should include a discussion of potential limitations and/or challenges in the protocol and strategies to address these concerns. Letters of Support : If there will be subcontracts or service agreements for personnel or facilities, include documentation of such commitments, co-signed by a business official and the investigator at the participating center.

All activities proposed in subcontracts must align with the current Executive Orders (EOs). If there are agreements with collaborating industry partners, include documentation of the agreements, co-signed by a business official and an appropriate official at the company.

If there are manufacturing agreements, include a letter of intent or letter of authorization from the manufacturer, co-signed by a business official and an appropriate official at the company. Should CTSA resources be used, include letter(s) of support from each site’s CTSA program officer(s) concurring with the specific plan for usage of these resources.

If some costs for the intermediate EA protocol for ALS are to be borne by sources other than NIH, include documentation of this support, signed by individuals who have the authority to make a commitment on behalf of the organization they represent.

Applicants are encouraged to include letters or other supporting documentation from relevant stakeholders (e.g., potential study participants, referring and treating physicians, patient advocacy groups) to show that they believe the study question to be important, consider the study design to be acceptable, and that patients were included as partners in the concept development and design of the EA program.

Resource Sharing Plan : Individuals are required to comply with the instructions for the Resource Sharing Plans as provided in the How to Apply - Application Guide . Applications proposing to collect biospecimens must use the BioSEND protocols and procedures, and all specimens collected and banked with BioSEND must come from individuals who have consented to banking and sharing broadly with academia and industry.

Note that costs for collection are NOT included as a component of the NINDS Biomarkers Repository award to offset some of the cost of biospecimen banking. Therefore, costs for the biospecimen collection (collection kits, shipping kits to sites, shipping samples to BioSEND) are borne by the recipients utilizing this resource (see NOT-NS-15-046 ).

Applicants planning projects in which biospecimens will be collected must consult the BioSEND website for more information about samples banked at the repository. In addition, applicants are advised to consult with BioSEND staff to obtain a quote for biospecimen banking costs . Please request a quote from BioSEND by filling out a quote form .

BioSEND staff will contact the PI(s). Applicants should include this information in the application. Whenever applicable, the NINDS Human Cell and Data Repository (NHCDR) must be used to bank and distribute cells collected from participants in these studies.

Note that costs for collection are NOT included as a component of the NHCDR award to offset some of the cost of biospecimen banking. Therefore, costs for the biospecimen collection (collection kits, shipping kits to sites, shipping samples to NHCDR) are borne by the recipients utilizing this resource.

Applicants planning projects in which biospecimens will be collected are strongly advised to consult the NHCDR website for more information about samples banked at the repository. In addition, applicants are advised to consult with NHCDR staff to obtain a quote for biospecimen banking costs.

All instructions in the How to Apply - Application Guide must be followed, with the following additional instructions: A Data Management and Sharing Plan (DMS Plan) is required for any NIH-funded or conducted research that will generate scientific data. Applicants must submit the DMS Plan at the time of application using the NIH DMS Plan Format Page .

The DMS Plan must address the elements in the structured format should not exceed two (2) pages. Where the DMS Plan Format Page requires a “Yes or No” response, no additional narrative is allowed. To increase the value of its funded research, the ALS Knowledge Portal managed by the NINDS serves as a repository for data sharing and analysis through a cloud-based infrastructure.

Applicants are expected to submit a complete de-identified dataset containing all variables collected in the intermediate EA protocol for ALS and a data dictionary within an agreed upon time frame. Applicants must should include a statement regarding the qualification of the investigational drug/biological product sponsor as a U.S. based small business concern as defined in section 3(a) of the Small Business Act ( 15 U.S.C. 632(a) ).

Appendix: Only limited Appendix materials are allowed. Follow all instructions for the Appendix as described in the How to Apply - Application Guide . No publications or other material, with the exception of blank questionnaires or blank surveys, may be included in the Appendix.

PHS Human Subjects and Clinical Trials Information When involving human subjects research, clinical research, and/or NIH-defined clinical trials (and when applicable, clinical trials research experience) follow all instructions for the PHS Human Subjects and Clinical Trials Information form in the How to Apply- Application Guide , with the following additional instructions: If you answered “Yes” to the question “Are Human Subjects Involved?

” on the R&R Other Project Information form, you must include at least one human subjects study record using the Study Record: PHS Human Subjects and Clinical Trials Information form or Delayed Onset Study record. Study Record: