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Phase 2 Trial of Gamunex ( Intravenous Gammaglobulin) for Sickle Cell Acute Pain

Award Number: R01FD005341
ORGANIZATION: FOOD AND DRUG ADMINISTRATION
OPDIV: FDA
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/10/2015
PERIOD OF PERFORMANCE END DATE: 06/30/2023

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Phase 2 Trial of Gamunex ( Intravenous Gammaglobulin) for Sickle Cell Acute Pain - Protocol Title: Phase 2 trial of Gamunex (Intravenous Gamma globulin) for Sickle Cell Acute Pain Principal Investigator: Deepa Manwani Co-Investigators: Paul S. Frenette, Patricia A. Shi, Hillel Cohen, Catherine Driscoll, Veronica Carullo ABSTRACT Sickle cell anemia is one of the most common hematologic diseases in the world. The most common clinical manifestation of this disease is the recurrence of vaso-occlusive episodes (“crises”) for which there is currently no therapy other than supportive care and analgesics. Studies using a mouse model of sickle cell disease (SCD) suggest that leukocytes (WBCs) that are adherent to the vessel wall of post-capillary and collecting venules play a key role in vascular occlusion by interacting with sickle eythrocytes (RBCs)1-3. In search for the molecular mechanisms responsible for these interactions, it was found that intravenous immune globulin (IVIG) had a profound effect on the interactions among RBCs, WBCs, and the endothelium, improved blood flow in venules of the cremaster muscle and, most importantly, impacted on the overall survival of sickle cell mice.4,5 A pilot phase I double blind placebo controlled randomized, dose escalation study demonstrated that while doses up to 800mg/kg can be safely administered to sickle cell disease patients during acute pain events, a 400mg/kg dose provided optimal biomarker and clinical efficacy with decreased infusion associated adverse events. We therefore want to evaluate intravenous immune globulin (IVIG) at the 400mg/kg dose in a phase II clinical study in sickle cell disease patients that are admitted to Montefiore Medical Center for vaso-occlusive crisis (VOC). Hypothesis: 1) IVIG at 400mg/kg is well tolerated by patients with sickle cell disease admitted with VOC. 2) IVIG can be effective in ameliorating VOC in patients with sickle cell disease. Specific Aims: 1) To evaluate the effect of a single dose of 400mg/kg of IV Gamunex on time to resolution of VOC in subjects 8-21years of age hospitalized for sickle cell VOC in a randomized, double blind placebo controlled Phase 2 trial. 2) To further evaluate safety of a single dose of 400mg/kg of IV Gamunex in subjects 8-21years of age hospitalized for sickle cell VOC 3) To evaluate biomarkers of adhesion and inflammation and secondary clinical end points (e.g. total opioid use, length of hospitalization and change in LDH) in subjects enrolled on this study).


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 = -$2,344 )
2024	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	000	5	4/27/2024	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	-$2,344
														Subtotal = -$2,344

Issue Date FY: 2022 ( Subtotal = $0 )
2022	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE, INC.	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	000	4	7/7/2022	NON-COMPETING CONTINUATION	$0
2022	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	001	5	8/15/2022	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$0
														Subtotal = $0

Issue Date FY: 2021 ( Subtotal = $0 )
2021	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	000	5	7/12/2021	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$0
														Subtotal = $0

Issue Date FY: 2020 ( Subtotal = $0 )
2020	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	000	5	9/8/2020	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$0
														Subtotal = $0

Issue Date FY: 2019 ( Subtotal = $0 )
2019	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE, INC.	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	003	4	8/9/2019	NON-COMPETING CONTINUATION	-$172,118
2019	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	002	5	8/7/2019	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$286,059
2019	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE, INC.	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	001	4	8/7/2019	NON-COMPETING CONTINUATION	-$113,941
2019	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE, INC.	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	000	4	7/3/2019	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2018 ( Subtotal = $400,000 )
2018	2018	ALBERT EINSTEIN COLLEGE OF MEDICINE, INC.	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	000	4	7/20/2018	NON-COMPETING CONTINUATION	$400,000
														Subtotal = $400,000

Issue Date FY: 2017 ( Subtotal = $400,000 )
2017	2017	ALBERT EINSTEIN COLLEGE OF MEDICINE, INC.	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	004	3	8/3/2017	NON-COMPETING CONTINUATION	$400,000
2017	2016	ALBERT EINSTEIN COLLEGE OF MEDICINE, INC.	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	001	2	5/17/2017	NON-COMPETING CONTINUATION	$400,000
2017	2016	ALBERT EINSTEIN COLLEGE OF MEDICINE	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	000	2	5/11/2017	NON-COMPETING CONTINUATION	-$400,000
2017	2015	ALBERT EINSTEIN COLLEGE OF MEDICINE	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	002	1	5/26/2017	NEW	-$400,000
2017	2015	ALBERT EINSTEIN COLLEGE OF MEDICINE	1300 MORRIS PARK AVE	BRONX	NY	10461	BRONX	USA	Food and Drug Administration Research	003	1	6/2/2017	NEW	$400,000
														Subtotal = $400,000

Issue Date FY: 2016 ( Subtotal = $400,000 )
2016	2016	ALBERT EINSTEIN COLLEGE OF MEDICINE OF YESHIVA UNIV	1300 MORRIS PARK AVENUE	BRONIX	NY	10461		USA	Food and Drug Administration Research	000	2	8/11/2016	NON-COMPETING CONTINUATION	$400,000
														Subtotal = $400,000

Issue Date FY: 2015 ( Subtotal = $400,000 )
2015	2015	ALBERT EINSTEIN COLLEGE OF MEDICINE OF YESHIVA UNIV	1300 MORRIS PARK AVENUE	BRONIX	NY	10461		USA	Food and Drug Administration Research	000	1	9/4/2015	NEW	$400,000
														Subtotal = $400,000

Grand Total All Awards = $1,597,656

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Phase 2 Trial of Gamunex ( Intravenous Gammaglobulin) for Sickle Cell Acute Pain

Award Number: R01FD005341

ORGANIZATION: FOOD AND DRUG ADMINISTRATION

OPDIV: FDA

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 09/10/2015

PERIOD OF PERFORMANCE END DATE: 06/30/2023

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