Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)

Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required) is sponsored by National Institutes of Health (NIH) / National Institute of Neurological Disorders and Stroke (NINDS). This opportunity supports mission-aligned projects and measurable outcomes.

Expired RFA-NS-25-024: Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required) This notice has expired. Check the NIH Guide for active opportunities and notices. 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 October 10, 2024 - Notice of Information: Technical Assistance Webinars for RFA-NS-25-024 Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required).

See Notice NOT-NS-25-003 . April 4, 2024 - Overview of Grant Application and Review Changes for Due Dates on or after January 25, 2025. See Notice NOT-OD-24-084 .

August 31, 2022 - Implementation Changes for Genomic Data Sharing Plans Included with Applications Due on or after January 25, 2023. See Notice NOT-OD-22-198 . August 5, 2022 - Implementation Details for the NIH Data Management and Sharing Policy.

See Notice NOT-OD-22-189 . Funding Opportunity Number (FON) Companion Funding Opportunity See Section III. 3.

Additional Information on Eligibility . Assistance Listing Number(s) Funding Opportunity Purpose The purpose of this Notice of Funding Opportunity (NOFO) is to encourage grant applications for the conduct of scientific research utilizing data from expanded access (EA) for investigational drugs or biological products.

These applications will target EA for intermediate size populations of people living with amyotrophic lateral sclerosis (ALS) who are not eligible for ongoing clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of ALS. Open Date (Earliest Submission Date) Letter of Intent Due Date(s) 30 days prior to application due 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 How to Apply - Application Guide and the NOFO) is required and strictly enforced.

Applicants must read and follow all application instructions in the How to Apply - Application Guide as well as any program-specific instructions noted in Section IV. When the program-specific instructions deviate from those in the How to Apply - Application Guide , follow the program-specific instructions. Applications that do not comply with these instructions may be delayed or not accepted for review.

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 encourage grant 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 w ith over 32,000 estimated cases in the United States (U.S.) or a prevalence rate of 9. 9 per 100,000 U.S. population. The disease affects both upper and lower motor neurons, leading to weakness and wasting of most skeletal muscles including the diaphragm.

There is considerable variability in the phenotypic presentation and progression of ALS although the mean survival of people with diagnosis of ALS from symptom onset is 3-5 years. To date, treatment options for ALS remain severely limited.

Currently, there are three disease-modifying drugs for ALS on the market in the U.S. Another drug was approved by the FDA in 2022, but was removed from the market by the manufacturer based on topline results from a phase III clinical trial that ended in 2023. Two of these drugs are approved for all forms of ALS, and one drug is approved for SOD1-linked, familial ALS.

However, so far, these drugs only have modest effects on slowing progression of the disease, and there is no known treatment that prevents, halts or reverses the progression of ALS. Thus, the 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, some people with ALS may be eligible to 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 potential 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. The objective of this NOFO is to support the conduct of scientific research utilizing data from intermediate-size patient population EA protocols for ALS (see https://www.

fda. gov/news-events/expanded-access/expanded-access-categories-drugs-including-biologics ). 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 FDA, such as a "may proceed" email or letter, must be submitted to NINDS prior to funding 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 protocol 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, NINDS encourages investigators to begin these processes as early as possible. Applications must follow NINDS guidelines for monitoring clinical trials.

The EA protocol will provide access to the investigational drug or biological product for people with ALS. The protocol must also generate data that will support research or development related to the prevention, diagnosis, mitigation, treatment, or cure of ALS.

A description of the scientific research goal(s) informed by these data could include the investigational drug’s/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.

Applications are required to include a description of how the proposed EA program will not interfere with participant enrollment in ongoing clinical trials for investigational therapies for the prevention, diagnosis, mitigation, treatment, or cure of ALS.

Funding for the EA protocol will continue until one of the following events: Marketing authorization of the drug/biological product by the FDA Withdrawal or termination of the IND for the investigational agent 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 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 NINDS does not concur, (d) study participant safety or ethical issues that may dictate a premature termination, or (e) 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 strongly encourages the use of common data elements (CDEs). CDEs are data standards that have been identified and defined for use in multiple data sets across different studies.

This NOFO strongly encourages applications that include a plan for stakeholder engagement. Stakeholder engagement in clinical studies 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.

Applicants are strongly encouraged to establish relationships with PWLE(s) and/or advocacy groups and 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.

Applicants are encouraged to facilitate continued respectful, equitable and bidirectional knowledge transfer during stakeholder engagement to build trust in communities that may be fearful of participating in clinical research because of stigmas and medical mistrust. Furthermore, engaging stakeholders in these ways may also improve participant retention and adherence to the study protocol.

NINDS is committed to reducing the disproportionate burden of neurological disease borne by underserved groups of society, including racial and ethnic minority, rural, and socioeconomically disadvantaged populations. People from racial and ethnic minority groups and people living in rural areas are disproportionately affected by many medical conditions including ALS.

Additionally, socioeconomically disadvantaged populations are more likely to be at risk for poorer health outcomes. Therefore, ensuring appropriate inclusion of populations that experience health disparities is strongly encouraged. See Section VIII.

Other Information for award authorities and regulations. Investigators proposing NIH-defined clinical trials may refer to the Research Methods Resources website for information about developing statistical methods and study designs. 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 provides 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 $35,000,000 in FY 2025 to fund 2-5 new awards 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 All organizations administering an eligible parent award may apply for a supplement under this NOFO.

Higher Education Institutions Public/State Controlled Institutions of Higher Education Private Institutions of Higher Education The following types of Higher Education Institutions are always encouraged to apply for NIH support as Public or Private Institutions of Higher Education: Hispanic-serving Institutions Historically Black Colleges and Universities (HBCUs) Tribally Controlled Colleges and Universities (TCCUs) Alaska Native and Native Hawaiian Serving Institutions Asian American Native American Pacific Islander Serving Institutions (AANAPISIs) 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 (designed to provide pivotal data to support a marketing application) supported by a 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) ).

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 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. NATO Commercial and Government Entity (NCAGE) Code – Foreign organizations must obtain an 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 registration; 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.

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.

Individuals from diverse backgrounds, including underrepresented racial and ethnic groups, individuals with disabilities, and women are always encouraged to apply for NIH support. See, Reminder: Notice of NIH's Encouragement of Applications Supporting Individuals from Underrepresented Ethnic and Racial Groups as well as Individuals with Disabilities, NOT-OD-22-019 .

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. Conformance to the requirements in the How to Apply - Application Guide is required and strictly enforced.

Applications that are out of compliance with these instructions may be delayed or not accepted for review. Although a letter of intent is not required, is not binding, and does not enter into the review of a subsequent application, the information that it contains allows IC staff to estimate the potential review workload and plan the review. By the date listed in Part 1.

Overview Information , prospective applicants are asked to submit a letter of intent that includes the following information: Descriptive title of proposed activity Name(s), address(es), and telephone number(s) of the PD(s)/PI(s) Names of other key personnel Participating institution(s) Number and title of this funding opportunity The letter of intent should be sent to: 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 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 repository Costs to cover expenses incurred by participating clinical trial sites in conducting research with respect to such drugs/biological products including any analysis Personnel effort, support for study participant travel/meals, and other budget items within the overall budget cap to ensure that this goal of appropriate inclusion is met 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: Introduction to Application: For Resubmission applications, an Introduction to Application is required in the Overall component.

Significance: Describe how data generated through the 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 intermediate EA protocol for ALS, including preliminary data, clinical and/or preclinical studies, or data on biological mechanisms.

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 marketing 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 the NIH/NINDS Common Data Elements and, as applicable, other NIH or external resources to standardize the collection of clinical data (see additional details below).

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. The application should describe what (if any) non-traditional approaches to engage potential study participants for intervention delivery and data collection will be used to minimize burden (see section 2 below).

Use of CDEs in NIH-funded Research: Leading assessments and decision-making processes regarding adoption and implementation of data collection standards, a core set of CDEs and tools, and harmonization procedures including mapping to common data models to maximize comparability across the program and measurement modes.

Data availability and data collection processes will differ across different types of studies, including, for example, studies with data extracted from electronic health records, studies with data collected in research clinic settings, and studies with data collected in community settings via digital technology (e.g., mobile app, wearables, etc.). NINDS strongly encourages the use of CDEs available at www. commondataelements.

ninds. nih. gov ( ALS CDE Project ) .

Investigators should describe how standardization of data collection and data collection instruments will be used, including the use of existing NIH/NINDS CDEs. Investigators are expected to describe how they will use the NINDS CDEs and other data standards, creation of Case Report Forms (CRFs) and data dictionaries.

Whenever applicable, investigators should use the standardized CRFs and other instruments identified by the NINDS CDE Project. If other CDEs are used/reused, describe how they will be used and mapped. The CDE Project has developed uniform formats by which clinical data can be systematically collected, analyzed, and shared across the research community.

To maximize comparisons across datasets or studies and facilitate data integration and collaboration, researchers funded through this RFA are strongly encouraged to use the following standards and resources (where applicable): 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 Stakeholder Engagement: this NOFO strongly encourages applications that include a plan for stakeholder engagement.

Stakeholder engagement in clinical studies involves patients, families, their representatives, 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 are also strongly encouraged.

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. 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. If CTSA resources will be utilized, include letter of support from each site CTSA program officer concurring with the specific plan for using 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.

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 grantees utilizing this resource (see NOT-NS-15-046 ).

Applicants planning projects in which biospecimens will be collected are strongly advised to 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 (https://biosend. org/request_a_quote.

html). Please request a quote from BioSEND by filling out a quote form at https://biosend. org/request_a_quote.

html . 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 grantees 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 (https://nindsgenetics. org/contact-information/).

All instructions in the How to Apply - Application Guide must be followed, with the following additional instructions: All applicants planning research (funded or conducted in whole or in part by NIH) that results in the generation of scientific data are required to comply with the instructions for the Data Management and Sharing Plan.

All applications, regardless of the amount of direct costs requested for any one year, must address a Data Management and Sharing Plan. To increase the value of its funded research, an ALS Knowledge Platform managed by the NINDS will serve as a data 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 timeframe. Applicants are encouraged to make use of the NINDS CDEs for ALS, see https://www. commondataelements.

ninds. nih. gov/amyotrophic%20lateral%20sclerosis.

Only limited Appendix materials are allowed. Follow all instructions for the Appendix as described in the How to Apply - Application Guide . Applicants should include with appendix materials a statement regarding the qualification of the drug/biological product sponsor as a small business concern as defined in section 3(a) of the Small Business Act ( 15 U.S.C.

632(a) ). 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