Wednesday, June 26, 2024 6/26/2024

Meaningful Outcomes and Multi-Site Readiness for Clinical Trials in Juvenile Neuronal Ceroid Lipofuscinosis

Award Number: U01NS101946
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: COOPERATIVE AGREEMENT
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

Group Awards By Issue Date FY or Funding FY:

View Award Abstract

The neuronal ceroid lipofuscinosis (NCL) therapeutic pipeline is rapidly expanding. The NCLs are rare, devastating, neurodegenerative lysosomal storage disorders that mainly affect children. Juvenile NCL (CLN3 disease) is the most prevalent form, with a complex set of multi-domain signs and symptoms that gradually progress over 20 years and culminate in premature death. Currently, there are no approved disease-modifying therapies. This proposal will foster collaboration between international leaders in NCL research, the University of Rochester (Rochester, New York) and the University of Hamburg (Hamburg, Germany). This multi-center, multi-national partnership mirrors the future model for clinical trial implementation and lays the foundation for development of novel therapeutics for CLN3 disease. The proposal will address critical challenges for the design and implementation of clinical trials in CLN3 disease: optimizing sensitive clinical assessments that measure how patients feel and function, validating early readouts of efficacy for go-no go decision making in phase 2 trials, and preparing for rigorous and consistent disease evaluation across multiple clinical trial sites. In parallel, our aims are: 1) to refine approaches to systematic and comprehensive assessment of CLN3 disease in order to best quantify progression in future trials, 2) to validate quantitative brain neuroimaging measurements as biomarkers of disease progression for phase 2 trials, and 3) to prepare for reproducible multi-site use of clinical outcome assessment tools. During the award period, we will engage the Food and Drug Administration in clinical outcome assessment and biomarker qualification processes to ensure optimal preparation for future regulatory review processes for emerging CLN3 disease therapeutic trials.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2024 ( Subtotal = $610,733 )
2024	2024	UNIVERSITY OF ROCHESTER	910 GENESEE ST	ROCHESTER	NY	14611	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	5	4/9/2024	NON-COMPETING CONTINUATION	$44,074
2024	2024	UNIVERSITY OF ROCHESTER	910 GENESEE ST	ROCHESTER	NY	14611	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	5	3/6/2024	NON-COMPETING CONTINUATION	$566,659
														Subtotal = $610,733

Issue Date FY: 2023 ( Subtotal = $707,937 )
2023	2023	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	4	2/24/2023	NON-COMPETING CONTINUATION	$707,937
2023	2021	UNIVERSITY OF ROCHESTER	910 GENESEE ST	ROCHESTER	NY	14611	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	3	9/19/2023	NON-COMPETING CONTINUATION	$0
														Subtotal = $707,937

Issue Date FY: 2022 ( Subtotal = $0 )
2022	2021	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	2/21/2022	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2021 ( Subtotal = $745,729 )
2021	2021	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	3	1/27/2021	NON-COMPETING CONTINUATION	$745,729
2021	2020	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	11/13/2020	NON-COMPETING CONTINUATION	$0
														Subtotal = $745,729

Issue Date FY: 2020 ( Subtotal = $901,782 )
2020	2020	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	4/28/2020	NON-COMPETING CONTINUATION	$747,799
2020	2020	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	002	2	7/21/2020	SUPPLEMENT FOR EXPANSION	$153,983
2020	2020	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	2	6/15/2020	NON-COMPETING CONTINUATION	$0
2020	2019	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	003	1	7/29/2020	NEW	$0
														Subtotal = $901,782

Issue Date FY: 2019 ( Subtotal = $765,450 )
2019	2019	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	1	3/15/2019	NEW	$0
2019	2019	UNIVERSITY OF ROCHESTER	500 JOSEPH C WILSON BLVD	ROCHESTER	NY	14627	MONROE	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	3/14/2019	NEW	$765,450
														Subtotal = $765,450

Grand Total All Awards = $3,731,631

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Meaningful Outcomes and Multi-Site Readiness for Clinical Trials in Juvenile Neuronal Ceroid Lipofuscinosis

Award Number: U01NS101946

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: COOPERATIVE AGREEMENT

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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