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Fragile X Clinic and Reseach Cooperative Consortium Agreement: component C

Award Number: U01DD001186
ORGANIZATION: NATIONAL CENTER ON BIRTH DEFECTS AND DEVELOPMENTAL DISABILITIES (NCBDDD)
OPDIV: CDC
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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¿ DESCRIPTION (provided by applicant): Fragile X syndrome (FXS) is the most common inherited cause of intellectual disability and autism spectrum disorder with an estimated frequency of about 1:4000-5000, affecting all racial and ethnic groups worldwide. Enormous progress in basic and translational FXS research has identified many neuronal targets and allowed early clinical trials of new treatments targeted to the underlying disease. A number of these agents have shown signal for benefit in open-label and early phase trials, but it has been challenging to meet primary behavioral outcomes in larger phase 2b and 3 trials, due to multiple complex issues including dosing age of subjects, length of treatment, placebo effects, and primary outcome off target for drug effect. A significant problem has been poor availability of biomarkers and well-validated cognitive and other objective outcome measures that do not rely on parent report. Further, very long-term treatment over years, a time frame over which placebo controls are not possible, will be necessary to show changes in the trajectory of disease. In order to know whether the disease trajectory has been changed, it will be CRITICAL to have detailed longitudinal phenotyping including cognitive, adaptive, language, motor, behavioral, social, and quality of life on a large cohort of individuals with FXS, in order to define the naturl history of the disease for future comparison in long-term intervention studies. The FORWARD longitudinal database tracks health, behavior and social issues including 3 standardized measures of behavior and social function in a large cohort of individuals with FXS but lacks good consistent cognitive and adaptive data. Given the infrastructure already present, the FORWARD project is the only currently plausible place to collect the necessary detailed longitudinal phenotyping. Thus, through this application, focused on both enhanced measurement and participation, we plan to collaborate with the other Component C FXS clinics to enhance the cohort size in which we will collect a comprehensive core battery of outcome measures yearly for a minimum of 3 years to begin to define the longitudinal trajectory of all aspects of the FXS phenotype (Aim 1). Additionally at our site alone we will collect pilot data longitudinally for two new outcome measures that address two areas of need in the field (Aim 2): objective direct measures of CNS function (auditory event-related potentials), and sensitive direct measures of communication function in very young and non-verbal individuals with FXS (Communication Complexity Scale). Finally, we will implement plans to improve our collection of this data from adults with FXS and from racial and ethnic groups according to the population percentages from the state of Illinois (Aim 3). Analysis plans for the data collected in this projet will be reviewed and modified by experts in FXS outcomes research and CDC partners. Results of analyses resulting from the data collection in this application will be disseminated to all stakeholders including the FXCRC, NFXF, FXS Community Support Network groups, FXS families, other researchers and CDC project partners.


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: 2022 ( Subtotal = -$4,553 )
2022	2020	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	02	6	1/3/2022	ADMINISTRATIVE SUPPLEMENT ( + OR - ) (DISCRETIONARY OR BLOCK AWARDS)	$0
2022	2019	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	01	5	1/20/2022	ADMINISTRATIVE SUPPLEMENT ( + OR - ) (DISCRETIONARY OR BLOCK AWARDS)	-$4,553
2022	2018	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	01	5	1/20/2022	ADMINISTRATIVE SUPPLEMENT ( + OR - ) (DISCRETIONARY OR BLOCK AWARDS)	$0
2022	2017	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	01	5	1/20/2022	ADMINISTRATIVE SUPPLEMENT ( + OR - ) (DISCRETIONARY OR BLOCK AWARDS)	$0
2022	2016	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	01	5	1/20/2022	ADMINISTRATIVE SUPPLEMENT ( + OR - ) (DISCRETIONARY OR BLOCK AWARDS)	$0
2022	2015	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	01	5	1/20/2022	ADMINISTRATIVE SUPPLEMENT ( + OR - ) (DISCRETIONARY OR BLOCK AWARDS)	$0
														Subtotal = -$4,553

Issue Date FY: 2021 ( Subtotal = $0 )
2021	2020	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	01	6	10/13/2020	ADMINISTRATIVE SUPPLEMENT ( + OR - ) (DISCRETIONARY OR BLOCK AWARDS)	$0
														Subtotal = $0

Issue Date FY: 2020 ( Subtotal = $349,999 )
2020	2020	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	00	6	8/3/2020	COMPETING CONTINUATION	$349,999
														Subtotal = $349,999

Issue Date FY: 2019 ( Subtotal = $99,946 )
2019	2019	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	00	5	8/16/2019	NON-COMPETING CONTINUATION	$99,946
2019	2018	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	01	4	10/5/2018	ADMINISTRATIVE SUPPLEMENT ( + OR - ) (DISCRETIONARY OR BLOCK AWARDS)	$0
														Subtotal = $99,946

Issue Date FY: 2018 ( Subtotal = $99,947 )
2018	2018	Rush University Medical Center	1653 W Congress Prkwy	Chicago	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	00	4	7/15/2018	NON-COMPETING CONTINUATION	$99,947
														Subtotal = $99,947

Issue Date FY: 2017 ( Subtotal = $99,946 )
2017	2017	RUSH UNIVERSITY MEDICAL CENTER	1653 W CONGRESS PRKWY	CHICAGO	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	003	3	8/1/2017	NON-COMPETING CONTINUATION	$99,946
2017	2016	RUSH UNIVERSITY MEDICAL CENTER	1653 W CONGRESS PRKWY	CHICAGO	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	001	2	1/27/2017	NON-COMPETING CONTINUATION	$0
2017	2016	RUSH UNIVERSITY MEDICAL CENTER	1653 W CONGRESS PRKWY	CHICAGO	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	002	2	3/10/2017	NON-COMPETING CONTINUATION	$0
2017	2016	RUSH UNIVERSITY MEDICAL CENTER	1653 W CONGRESS PRKWY	CHICAGO	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	000	2	10/26/2016	NON-COMPETING CONTINUATION	$0
														Subtotal = $99,946

Issue Date FY: 2016 ( Subtotal = $99,946 )
2016	2016	RUSH-PRESBYTERIAN-ST LUKE`S MEDICAL CENTER	1700 WEST VAN BUREN STREET	CHICAGO	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	002	2	7/29/2016	NON-COMPETING CONTINUATION	$99,946
2016	2015	RUSH UNIVERSITY MEDICAL CENTER	1653 WEST CONGRESS WAY	CHICAGO	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	001	1	1/20/2016	NEW	$0
2016	2015	RUSH UNIVERSITY MEDICAL CENTER	1653 WEST CONGRESS WAY	CHICAGO	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	000	1	12/17/2015	NEW	$0
														Subtotal = $99,946

Issue Date FY: 2015 ( Subtotal = $100,000 )
2015	2015	RUSH-PRESBYTERIAN-ST LUKE`S MEDICAL CENTER	1700 WEST VAN BUREN STREET	CHICAGO	IL	60612	COOK	USA	Rare Disorders: Research, Surveillance, Health Promotion, and Education	000	1	8/24/2015	NEW	$100,000
														Subtotal = $100,000

Grand Total All Awards = $845,231

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Fragile X Clinic and Reseach Cooperative Consortium Agreement: component C

Award Number: U01DD001186

ORGANIZATION: NATIONAL CENTER ON BIRTH DEFECTS AND DEVELOPMENTAL DISABILITIES (NCBDDD)

OPDIV: CDC

AWARD CLASS: DISCRETIONARY

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

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