NIH – Treatments for Lewy Body Dementias–Exploratory Clinical Trial (U01 Clinical Trial Required)

November 6, 2020 by


This Funding Opportunity Announcement invites applications from investigators seeking to conduct exploratory clinical trials designed to test new treatments for patients with Lewy Body Dementia (LBD). Applicants may propose to conduct either Phase I or Phase II clinical trials depending on the developmental stage of the potential therapeutic, but all trials must include patients with LBD. Proposed therapies may include novel medications or devices, or existing treatments that are potentially beneficial but not currently approved for use in patients with LBD. Treatments intended to prevent or delay disease progression in LBD patients, as well as therapies to alleviate existing motor or non-motor clinical symptoms, are of interest.


In 2011, the National Alzheimer’s Project Act (NAPA) allocated resources “to prevent and effectively treat Alzheimer’s by 2025.” Since then, the National Institute on Aging (NIA) and the National Institute of Neurological Disorders and Stroke (NINDS) have held multiple research summits to assess the needs and opportunities relevant to this goal for Alzheimer’s Disease (AD) and Alzheimer’s Disease Related Dementias (ADRD). In particular, the NINDS has convened expert panels in 20132016, and 2019 that were tasked with recommending research priorities for advancing the state-of-the-science for the Lewy Body Dementias (LBD), among other types of non-Alzheimers disease dementias. The expert panel tasked with making research recommendations for LBD has repeatedly identified the need for clinical trials as the highest priority recommendation at all three summits.

The LBDs, which include Dementia with Lewy Bodies (DLB) and Parkinson’s Disease Dementia (PDD) are considered to be the second most common form of dementia. No treatments exist that prevent LBD disease onset or substantially slow disease progression. Several symptomatic therapies that are approved to treat diseases that present similarly (e.g., Alzheimer’s disease, Parkinson’s disease) have been tested for their ability to improve motor and/or non-motor symptoms in patients with LBD. For the most part, these trials have involved small sample sizes and/or shown conflicting results, so many of these potential therapies are not widely adopted, and most medications tested remain off-label for use in LBD patients.

Additional factors make the conduct of clinical trials in this specific patient population difficult. Despite the publication of consensus-based diagnostic criteria, the diagnosis of DLB or PDD can be missed or significantly delayed due to symptom overlap with other disorders. LBD patients frequently experience medication side effects, often due to the need for conflicting pharmacologic approaches to treat their symptom profiles (e.g., dopamine agonists for motor symptoms, dopamine antagonists for psychiatric symptoms). Finally, cognitive impairment and psychosis, both common in patients with LBD, complicate clinical trial efforts to ensure compliance with a treatment regimen, timely follow-up, and accurate assessment of treatment efficacy.

Key Dates:

Open Date (Earliest Submission Date): February 02, 2021
Letter of Intent Due Date(s): February 2, 2021
Application Due Date(s): March 2, 2021

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Filed Under: Funding Opportunities