R01FD007630

Clinic-Based Multicenter ALS Natural History Data Collection - Project Summary/Abstract

Amyotrophic Lateral Sclerosis (ALS) is a universally disabling and fatal neurological condition. Current treatments have only modest benefit. Development of effective therapeutics is hampered in part by inadequate knowledge of ALS natural history.

Though valuable, existing ALS registries have either large sample sizes but limited clinical information, focus on relatively small subsets of the ALS population, or provide information from a relatively brief period. However, as a part of routine clinical care, ALS Multidisciplinary Clinics (MDCs) develop a rich natural history dataset from all ALS patients, and most people living with ALS are seen at MDCs.

We have built a combined, de-identified natural history dataset from over 1,700 ALS patients from 9 MDCs, with racial representation that is reflective of the ALS population at large and broad geographic representation as well. We offer enrollment to all patients at our clinics.

We will build on our multicenter, longitudinal, prospective MDC dataset in collaboration with industry and the ALS patient community to provide information critical for clinical trial development and post-marketing evaluation. Specifically, we will do the following:

1. Record clinically relevant baseline characteristics and serum neurofilament light (NFL) measurements at enrollment as well as validated longitudinal measures of disease progression including ALSFRS-R and disease staging, respiratory vital capacity, speaking rate, and 10-meter walk test.

2. Record disease milestones based upon patient-reported outcomes, durable medical equipment orders in the electronic health record, and other events such as dates of gastrostomy, hospitalizations, and death.

3. Model time to events using baseline characteristics as well as retrospective and prospective longitudinal data.

We have demonstrated the ability to enroll the vast majority of patients seeking care in our clinics so as to best represent the ALS population. Baseline characteristics will include ALS phenotype and results of genetic testing. Datasets will be made available to academic and industry investigators to advance ALS care, drug development, and outcomes research.

We will expand existing engagement from industry and other stakeholders to include an advisory panel of people living with ALS and will encourage opportunities for data dissemination through these existing relationships and publications.

Regents Of The University Of Minnesota

TO ASSIST INSTITUTIONS AND ORGANIZATIONS, TO ESTABLISH, EXPAND, AND IMPROVE RESEARCH, DEMONSTRATION, EDUCATION AND INFORMATION DISSEMINATION ACTIVITIES, ACQUIRED IMMUNODEFICIENCY SYNDROME (AIDS), BIOLOGICS, BLOOD AND BLOOD PRODUCTS, THERAPEUTICS, VACCINES AND ALLERGENIC PROJECTS, DRUG HAZARDS, HUMAN AND VETERINARY DRUGS, CLINICAL TRIALS ON DRUGS AND DEVICES FOR ORPHAN PRODUCTS DEVELOPMENT, NUTRITION, SANITATION AND MICROBIOLOGICAL HAZARDS, MEDICAL DEVICES AND DIAGNOSTIC PRODUCTS, RADIATION EMITTING DEVICES AND MATERIALS, FOOD SAFETY AND FOOD ADDITIVES. THESE PROGRAMS ARE SUPPORTED DIRECTLY OR INDIRECTLY BY THE FOLLOWING CENTERS AND OFFICES: CENTER FOR BIOLOGICS EVALUATION AND RESEARCH (CBER), CENTER FOR DRUG EVALUATION AND RESEARCH (CDER), CENTER FOR DEVICES AND RADIOLOGICAL HEALTH (CDRH), CENTER FOR VETERINARY MEDICINE (CVM), CENTER FOR FOOD SAFETY AND APPLIED NUTRITION (CFSAN), NATIONAL CENTER FOR TOXICOLOGICAL RESEARCH (NCTR), THE OFFICE OF ORPHAN PRODUCTS DEVELOPMENT (OPD), THE CENTER FOR TOBACCO PRODUCTS (CTP), AND OFFICE OF REGULATORY AFFAIRS (ORA), AND THE OFFICE OF THE COMMISSIONER (OC). SMALL BUSINESS INNOVATION RESEARCH (SBIR) PROGRAMS: TO STIMULATE TECHNOLOGICAL INNOVATION, TO ENCOURAGE THE ROLE OF SMALL BUSINESS TO MEET FEDERAL RESEARCH AND DEVELOPMENT NEEDS, TO INCREASE PRIVATE SECTOR COMMERCIALIZATION OF INNOVATIONS DERIVED FROM FEDERAL RESEARCH AND DEVELOPMENT, AND TO FOSTER AND ENCOURAGE PARTICIPATION BY MINORITY AND DISADVANTAGED PERSONS IN TECHNOLOGICAL INNOVATION. FUNDING SUPPORT FOR SCIENTIFIC CONFERENCES THAT ARE RELEVANT TO THE FDA SCIENTIFIC MISSION AND PUBLIC HEALTH ARE ALSO AVAILABLE.

93.103 - Food and Drug Administration Research

FDA Office of Acquisition and Grant Services (OAGS) [HHS - FDA]

Center for Tobacco Products (CTP) [HHS - FDA]

Minneapolis, Minnesota 554550356 United States

Single Zip Code

Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01) Clinical Trials Not Required (RFA-FD-22-001)

Amendment Since initial award the End Date has been extended from 08/31/26 to 08/31/27 and the total obligations have increased 395% from $1,200,000 to $5,935,878.