NOT-NS-22-096: Notice of Intent to Publish a Funding Opportunity Announcement for Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)

Notice of Intent to Publish a Funding Opportunity Announcement for Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)

Notice Number:

NOT-NS-22-096

Key Dates

Release Date:

April 13, 2022

Estimated Publication Date of Funding Opportunity Announcement:

May 09, 2022

First Estimated Application Due Date:

June 09, 2022

Earliest Estimated Award Date:

September 13, 2022

Earliest Estimated Start Date:

October 04, 2022

Related Announcements

None

Issued by

National Institute of Neurological Disorders and Stroke (NINDS)

Purpose

The National Institute of Neurological Disorders and Stroke (NINDS) and the Office of the Director, National Institutes of Health (OD), intend to promote a new initiative by publishing a Funding Opportunity Announcement (FOA) to solicit applications for the conduct of scientific research utilizing data from expanded access (EA) to investigational drugs or biological products. These applications will target intermediate size populations of patients living with amyotrophic lateral sclerosis (ALS) who are not otherwise eligible for clinical trials for the diagnosis, mitigation, treatment, or cure of ALS ("intermediate EA protocol for ALS").

Providing 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 diagnosis, mitigation, treatment, or cure of ALS. Eligible applicants are clinical trial sites that participate in a phase 3 clinical trial supported by a United States (U.S.) small business concern (as defined in section 3(a) of the Small Business Act (15 U.S. C. 623(a)) that is also the U.S. Food and Drug Administration (FDA) designated sponsor of a drug or biological product for ALS that is the subject of an Investigational New Drug Application (IND).

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

The FOA is expected to be published in May 2022 with an expected application due date in June 2022.

This FOA will utilize the U01 activity code. Details of the planned FOA are provided below.

Research Initiative Details

Applicants should take note of the following special requirements and considerations:

EA IND application:
- A grant will not be awarded unless the applicant provides documentation such as a "may proceed" email or letter from the FDA prior to funding.
- The protocol should be submitted to the FDA under an intermediate size patient population EA IND prior to submission of the grant application to the NINDS.
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 are required prior to funding and the initiation of treatment of patients. As such, NINDS encourages investigators to begin these processes as early as possible.
The EA protocol will provide patient access to the investigational drug or biological product. The protocol must also collect data that will inform a scientific research objective that will further our understanding of ALS. This could include the drug's/biological product's effect on biomarkers tied to the pathophysiology of ALS, and the collection of safety information or outcome information such as survival or other significant medical events such as hospitalization or need for ventilatory support.

Funding Information

TBD

Estimated Total Funding

$25,000,000 over 4 years

Expected Number of Awards

TBD

Estimated Award Ceiling

$6,250,000; awards are for four years of support.

Primary Assistance Listing Number(s)

TBD

Anticipated Eligible Organizations

Public/State Controlled Institution of Higher Education
Private Institution of Higher Education
Nonprofit with 501(c)(3) IRS Status (Other than Institution of Higher Education)
State Government
Indian/Native American Tribal Government (Federally Recognized)
County governments
Independent school districts
Public housing authorities/Indian housing authorities
Indian/Native American Tribally Designated Organization (Native American tribal organizations (other than Federally recognized tribal governments)
U.S. Territory or Possession
Indian/Native American Tribal Government (Other than Federally Recognized)
Regional Organization
Eligible Agencies of the Federal Government

Applications are not being solicited at this time.

Inquiries

Please direct all inquiries to:

Amelie K. Gubitz, Ph.D.

National Institute of Neurological Disorders and Stroke (NINDS)/Division of Neuroscience

301-332-6453

gubitza@nih.gov

Emily Caporello, Ph.D.
National Institute of Neurological Disorders and Stroke (NINDS)/Division of Translational Research
Email: emily.caporello@nih.gov

Robin Conwit, M.D., FAAN
National Institute of Neurological Disorders and Stroke (NINDS)/Division of Clinical Research
Email: robin.conwit@nih.gov