Morton Cowan, Professor
University Of California San Francisco, 3333 California St., Ste 315, San Francisco, Ca 94143-0962
Keywords: Immune; clinical research site; clinical site
Project start date: 2009-09-12
Project end date: 2011-08-31
Budget end date: 31-AUG-2010
PFA/PA: RFA-OD-08-001
1U54AI082973-01_8803 (2009): $252518
Sponsored Links Excellgen http://Excellgen.com
PRIMARY IMMUNE DEFICIENCY TREATMENT CONSORTIUM ADMINISTRATIVE UNIT
Morton Cowan, Professor
University Of California San Francisco, 3333 California St., Ste 315, San Francisco, Ca 94143-0962
Abstract: responsibility for decision-making and administration (Figure 1). The Administrative Unit under the direction of Morton J. Cowan, PI, is responsible for the overall administration of the PIDTC. Dr. Cowan´s background and experience is provided in detail in Section V. Overall Clinical Research Program, Leadership and Resources of the proposal. The Administrative Unit will manage center contracts, monitor individual center activities through enrollment and participation on the Scientific Planning Committee, communication with center data managers, support of centers with respect to obtaining IRB approval and submission of data to the USIDNET, CIBMTR and DMCC, and arrangement of conference calls and travel to RDCRC meetings. As PI, Dr. Cowan will be responsible for the overall management of the consortium. He will direct the Administrative Unit of the PIDTC and lead the Steering Committee and the Scientific Planning Committee. He will adjudicate conflicts within the consortium and will ensure that the Advisory Committee and ORD/NIAID administrators are kept up to date with the progress of the PIDTC and he will work with NIAID and ORD administrators and other consortia directors to ensure that the goals of the consortia are met. He will also represent the PIDTC in the ASBMT, BMTCTN and PBMTC and with other groups outside the US and Canada, in particular, the Immunodeficiency Committee of the EBMT and the ESID and along with Project Pis and the co-Pi represent the PIDTC with the RDCRC administrators and other Pis
Keywords: Administrator; Advisory Committees; Canada; Clinical Research; Clinical Study; Communication; Conflict; Conflict (Psychology); Contracting Opportunities; Contracts; Data; Decision Making; Enrollment; Ensure; Ethics Committees, Research; Goals; IRBs; Immune; Immunodeficiency Disorder; Immunodeficiency Syndrome; Immunologic Deficiency Syndromes; Immunological Deficiency Syndromes; Individual; Institutional Review Boards; Lead; Leadership; Monitor; NIAID; National Institute of Allergy and Infectious Disease; Pb element; Programs (PT); Programs [Publication Type]; Research Ethics Committees; Research Resources; Resources; Task Forces; Travel; Work; adjudicate; conference; enroll; experience; heavy metal Pb; heavy metal lead; hypoimmunity; immune deficiency disorder; immunodeficiency; meetings; programs; symposium
Project start date: 2009-09-12
Project end date: 2011-08-31
Budget start date: 12-SEP-2009
Budget end date: 31-AUG-2010
PFA/PA: RFA-OD-08-001
1U54AI082973-01_8800 (2009): $325910
PRIMARY IMMUNE DEFICIENCY TREATMENT CONSORTIUM
Morton Cowan, Professor
University Of California San Francisco, 3333 California St., Ste 315, San Francisco, Ca 94143-0962
Grant 1U54AI082973-01 from National Institute Of Allergy And Infectious Diseases
Abstract: Primary immune deficiencies (PIDs) are rare, life-threatening inherited defects in the immune system. The focus of the PID Treatment Consortium (PIDTC) will be on three PIDs that can be cured with hematopoietic cell transplantation (HCT), enzyme replacement or gene therapy severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD). The objectives of the consortium are to characterize the long term outcomes and late effects in children with SCID, WAS and CGD who undergo HCT; to define the critical factors and biologic markers that influence the outcomes of children with SCID, WAS and CGD following HCT; to design and implement prospective clinical trials that improve care for children with PID; to prove the feasibility of newborn screening for SCID; and to provide training to physician scientists in the understanding and treatment of PIDs. Project 1 is a prospective study of SCID infants to identify early biomarkers and other disease- or HCT-related factors that affect engraftment, early immune reconstitution and survival. Project 2 is a cross-sectional retrospective study of SCID, exploring patient- and HCT-related factors that affect long term survival, immune reconstitution, late effects and quality of life. Project 3 addresses early and long-term outcomes following HCT in WAS and CGD, evaluating the degree of engraftment on outcome and identifying which patients with CGD are most likely to benefit from HCT. The Pilot Project Program will start with a Pilot Study of newborn screening for SCID. It will determine the efficacy of a novel test using newborn blood spots for early detection of SCID among Navajo Indians, who have a high incidence of SCID. The PIDTC encompasses 14 major centers that care for the majority of SCID, WAS and CGD patients in North America, bringing together for the first time physician/scientists with broad expertise in genetics, molecular biology, immunology, HCT, gene therapy and medical management. Parent advocacy groups will participate in PIDTC operations and oversight, subject recruitment, and dissemination of information resulting from our studies. These studies will resolve critical questions concerning HCT for these disorders and form the basis for future prospective clinical trials. RELEVANCE (See instructions) Primary immune deficiencies (PIDs) are rare, life-threatening inherited defects in the immune system. By forming this Consortium to compare different treatment approaches, improved survival and outcome should be afforded to future patients
Relevance: Primary immune deficiencies (PIDs) are rare, life-threatening inherited defects in the immune system. By forming this Consortium to compare different treatment approaches, improved survival and outcome should be afforded to future patients
Project start date: 2009-09-12
Project end date: 2011-08-31
Budget start date: 12-SEP-2009
Budget end date: 31-AUG-2010
PFA/PA: RFA-OD-08-001
1U54AI082973-01 (2009): $1250000
Grants awarded to Morton Cowan
PRIMARY IMMUNE DEFICIENCY TREATMENT CONSORTIUM
Morton Cowan
University Of California San Francisco, 3333 California St., Ste 315, San Francisco, Ca 94143-0962
Grant 5U54AI082973-02 from National Institute Of Allergy And Infectious Diseases
Abstract: Primary immune deficiencies (PIDs) are rare, life-threatening inherited defects in the immune system. The focus of the PID Treatment Consortium (PIDTC) will be on three PIDs that can be cured with hematopoietic cell transplantation (HCT), enzyme replacement or gene therapy severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD). The objectives of the consortium are to characterize the long term outcomes and late effects in children with SCID, WAS and CGD who undergo HCT; to define the critical factors and biologic markers that influence the outcomes of children with SCID, WAS and CGD following HCT; to design and implement prospective clinical trials that improve care for children with PID; to prove the feasibility of newborn screening for SCID; and to provide training to physician scientists in the understanding and treatment of PIDs. Project 1 is a prospective study of SCID infants to identify early biomarkers and other disease- or HCT-related factors that affect engraftment, early immune reconstitution and survival. Project 2 is a cross-sectional retrospective study of SCID, exploring patient- and HCT-related factors that affect long term survival, immune reconstitution, late effects and quality of life. Project 3 addresses early and long-term outcomes following HCT in WAS and CGD, evaluating the degree of engraftment on outcome and identifying which patients with CGD are most likely to benefit from HCT. The Pilot Project Program will start with a Pilot Study of newborn screening for SCID. It will determine the efficacy of a novel test using newborn blood spots for early detection of SCID among Navajo Indians, who have a high incidence of SCID. The PIDTC encompasses 14 major centers that care for the majority of SCID, WAS and CGD patients in North America, bringing together for the first time physician/scientists with broad expertise in genetics, molecular biology, immunology, HCT, gene therapy and medical management. Parent advocacy groups will participate in PIDTC operations and oversight, subject recruitment, and dissemination of information resulting from our studies. These studies will resolve critical questions concerning HCT for these disorders and form the basis for future prospective clinical trials. RELEVANCE (See instructions) Primary immune deficiencies (PIDs) are rare, life-threatening inherited defects in the immune system. By forming this Consortium to compare different treatment approaches, improved survival and outcome should be afforded to future patients
Project start date: 2009-09-12
Project end date: 2011-08-31
Budget start date: 1-SEP-2010
Budget end date: 31-AUG-2011
PFA/PA: RFA-OD-08-001
5U54AI082973-02 (2010): $1221118