Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990
NCT ID: NCT02064933
Last Updated: 2020-08-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
305 participants
OBSERVATIONAL
2014-02-02
2019-05-01
Brief Summary
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This multicenter study combines retrospective, prospective and cross-sectional analyses of the transplant experiences for patients with WAS who have already received HCT since 1990, or who will undergo Hematopoietic cell transplant (HCT) during the study period. The retrospective and prospective portions of the study will address the impact of a number of pre and post-transplant factors on post-transplant disease correction and ultimate benefit from HCT and the cross-sectional portion of the study will assess the benefit of HCT 2 years post-HCT in consenting surviving patients.
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Detailed Description
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Conditions
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Study Design
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COHORT
OTHER
Study Groups
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Retrospective Cohort (Longitudinal Analysis)
Participants with WAS treated at consortium centers since 1990 who have received transplant (Stratum A) or gene therapy (Stratum B)
No interventions assigned to this group
Prospective Cohort (Longitudinal Analysis)
Participants treated at consortium centers since 1990 who will receive transplant (Stratum A) or gene therapy (Stratum B)
No interventions assigned to this group
Cross-Sectional Cohort (Cross-sectional Analysis)
Living participants with WAS who have received transplant (Stratum A) or gene therapy (Stratum B) at Consortium Centers 1990-Present And \>= 2 Years Post-Transplant
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
1. thrombocytopenia (\< 100K) AND EITHER molecular diagnosis of WAS OR reduced WASP expression; OR
2. thrombocytopenia (\< 100K) AND positive family history consistent with WAS diagnosis; OR
3. chronic thrombocytopenia (\< 100K for minimum of 3 months) AND low mean platelet volume (MPV below normal range for age) AND EITHER recurrent and/or severe infections requiring treatment and/or eczema OR lack of antibody response to polysaccharide antigens or low IgM.
* Longitudinal Analysis (Retrospective and Prospective)
1. Stratum A. Participants with WAS who have or will Receive HCT
* Participants with WAS who have received an HCT since January 1, 1990
2. Stratum B. Participants with WAS who have or will Receive Gene Transfer
* Participants in which the intention is to treat with gene transfer with autologous modified cells
* Cross-Sectional Analysis (Strata A and B) 1. Participants with WAS who are surviving and at least 2 years after the most recent HCT or gene therapy.
MALE
No
Sponsors
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Primary Immune Deficiency Treatment Consortium (PIDTC)
OTHER
Rare Diseases Clinical Research Network
NETWORK
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Responsible Party
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Principal Investigators
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Lauri M. Burroughs, MD
Role: STUDY_CHAIR
Fred Hutchinson Cancer Center
David J. Rawlings, MD
Role: STUDY_CHAIR
Department of Pediatrics, University of Washington-Seattle Children's Hospital
Luigi D. Notarangelo, MD
Role: STUDY_CHAIR
National Institute of Allergy and Infectious Diseases, NIH
Alexandra H. Filipovich, MD
Role: STUDY_CHAIR
Children's Hospital Medical Center, Cincinnati
Locations
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Department of Pediatrics, University of Alabama at Birmingham
Birmingham, Alabama, United States
Phoenix Children's Hospital
Phoenix, Arizona, United States
Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California
Los Angeles, California, United States
Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles,
Los Angeles, California, United States
Lucile Salter Packard Children's Hospital at Stanford
Palo Alto, California, United States
University of California, San Francisco Benioff Children's Hospital
San Francisco, California, United States
Children's Hospital Denver, University of Colorado
Denver, Colorado, United States
Nemours Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
Children's National Hospital-George Washington University School of Medicine and Health Sciences
Washington D.C., District of Columbia, United States
Blood and Marrow Transplant Program, Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States
Aflac Cancer and Blood Disorders Center, Emory/Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Center for Cancer and Blood Disorders, Children's Hospital/Louisiana State University
New Orleans, Louisiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Department of Pediatrics, C. S. Mott Children's Hospital, University of Michigan
Ann Arbor, Michigan, United States
Division of Pediatric Blood and Marrow Transplantation, University of Minnesota
Minneapolis, Minnesota, United States
Mayo Clinic Children's Center
Rochester, Minnesota, United States
Cardinal Glennon Children's Hospital, Saint Louis University
St Louis, Missouri, United States
Saint Louis Children's Hospital, Washington University
St Louis, Missouri, United States
Institute for Pediatric Cancer and Blood Disorders, Hackensack University Medical Center
Hackensack, New Jersey, United States
Department of Pediatrics, Memorial Sloan Kettering Cancer Center
New York, New York, United States
Department of Pediatrics, Golisano Children's Hospital, University of Rochester
Rochester, New York, United States
Maria Fareri Children's Hospital, New York Medical College
Valhalla, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center, University of Cincinnati
Cincinnati, Ohio, United States
Rainbow Babies and Children's Hospital
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Division of Pediatric Hematology/Oncology, Oregon Health and Science University
Portland, Oregon, United States
Children's Hospital of Philadelphia, University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Pittsburgh Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
Pediatrics, University of Texas Southwestern Medical Center
Dallas, Texas, United States
Baylor College of Medicine Section of Immunology, Allergy, and Retrovirology, Texas Children's Hospital
Houston, Texas, United States
Texas Transplant Institute, Methodist Children's Hospital
San Antonio, Texas, United States
Primary Children's Hospital, University of Utah
Salt Lake City, Utah, United States
Fred Hutchinson Cancer Research Center and University of Washington-Seattle Children's Hospital
Seattle, Washington, United States
American Family Children's Hospital, University of Wisconsin
Madison, Wisconsin, United States
Children's Hospital of Wisconsin-Milwaukee
Milwaukee, Wisconsin, United States
Alberta Children's Hospital
Calgary, Alberta, Canada
Children's & Women's Health Centre of British Columbia
Vancouver, British Columbia, Canada
Cancer Care Manitoba, University of Manitoba
Winnipeg, Manitoba, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
CHU Sainte-Justine, Department of Pediatrics, University of Montreal
Montreal, Quebec, Canada
Countries
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References
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Griffith LM, Cowan MJ, Kohn DB, Notarangelo LD, Puck JM, Schultz KR, Buckley RH, Eapen M, Kamani NR, O'Reilly RJ, Parkman R, Roifman CM, Sullivan KE, Filipovich AH, Fleisher TA, Shearer WT. Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. J Allergy Clin Immunol. 2008 Dec;122(6):1087-96. doi: 10.1016/j.jaci.2008.09.045. Epub 2008 Nov 6.
Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC; workshop participants. Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol. 2014 Feb;133(2):335-47. doi: 10.1016/j.jaci.2013.07.052. Epub 2013 Oct 15.
Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Shearer WT, Burroughs LM, Torgerson TR, Decaluwe H, Haddad E; workshop participants. Primary Immune Deficiency Treatment Consortium (PIDTC) update. J Allergy Clin Immunol. 2016 Aug;138(2):375-85. doi: 10.1016/j.jaci.2016.01.051. Epub 2016 Apr 22.
Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O'Reilly RJ. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014 Jul 31;371(5):434-46. doi: 10.1056/NEJMoa1401177.
Haddad E, Allakhverdi Z, Griffith LM, Cowan MJ, Notarangelo LD. Survey on retransplantation criteria for patients with severe combined immunodeficiency. J Allergy Clin Immunol. 2014 Feb;133(2):597-9. doi: 10.1016/j.jaci.2013.10.022. Epub 2013 Dec 10. No abstract available.
Shearer WT, Dunn E, Notarangelo LD, Dvorak CC, Puck JM, Logan BR, Griffith LM, Kohn DB, O'Reilly RJ, Fleisher TA, Pai SY, Martinez CA, Buckley RH, Cowan MJ. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. J Allergy Clin Immunol. 2014 Apr;133(4):1092-8. doi: 10.1016/j.jaci.2013.09.044. Epub 2013 Nov 28.
Dvorak CC, Cowan MJ, Logan BR, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Shearer WT, O'Reilly RJ, Fleisher TA, Pai SY, Hanson IC, Pulsipher MA, Fuleihan R, Filipovich A, Goldman F, Kapoor N, Small T, Smith A, Chan KW, Cuvelier G, Heimall J, Knutsen A, Loechelt B, Moore T, Buckley RH. The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. J Clin Immunol. 2013 Oct;33(7):1156-64. doi: 10.1007/s10875-013-9917-y. Epub 2013 Jul 2.
Griffith LM, Cowan MJ, Notarangelo LD, Puck JM, Buckley RH, Candotti F, Conley ME, Fleisher TA, Gaspar HB, Kohn DB, Ochs HD, O'Reilly RJ, Rizzo JD, Roifman CM, Small TN, Shearer WT; Workshop Participants. Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management. J Allergy Clin Immunol. 2009 Dec;124(6):1152-60.e12. doi: 10.1016/j.jaci.2009.10.022.
Burroughs LM, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, Bleesing JJ, Edwards S, Dvorak CC, Chaudhury S, Prockop SE, Quinones R, Goldman FD, Quigg TC, Chandrakasan S, Smith AR, Parikh S, Davila Saldana BJ, Thakar MS, Phelan R, Shenoy S, Forbes LR, Martinez C, Chellapandian D, Shereck E, Miller HK, Kapoor N, Barnum JL, Chong H, Shyr DC, Chen K, Abu-Arja R, Shah AJ, Weinacht KG, Moore TB, Joshi A, DeSantes KB, Gillio AP, Cuvelier GDE, Keller MD, Rozmus J, Torgerson T, Pulsipher MA, Haddad E, Sullivan KE, Logan BR, Kohn DB, Puck JM, Notarangelo LD, Pai SY, Rawlings DJ, Cowan MJ. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood. 2020 Jun 4;135(23):2094-2105. doi: 10.1182/blood.2019002939.
Related Links
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Primary Immune Deficiency Treatment Consortium (PIDTC), Rare Clinical Diseases Research Network, National Institutes of Health
Division of Allergy, Immunology, and Transplantation (DAIT)
National Institute of Allergy and Infectious Diseases (NIAID)
Other Identifiers
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DAIT RDCRN PIDTC-6904
Identifier Type: -
Identifier Source: org_study_id
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