Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990



Status:Active, not recruiting
Healthy:No
Age Range:Any
Updated:11/23/2018
Start Date:June 2014
End Date:August 2019

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Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990 (RDCRN PIDTC-6904)

Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems
both with the immune system and with easy bruising and bleeding. The immune abnormalities
cause patients with WAS to be very susceptible to infections. Depending on the specific type
of primary immune deficiency diseases, there are effective treatments, including antibiotics,
cellular therapy and gene therapy, but studies of large numbers of patients are needed to
determine the full range of causes, natural history, or the best methods of treatment for
long term success.

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.


Inclusion Criteria:

- WAS participants will be defined as males who have:

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.

Exclusion Criteria:

- As this is a natural history study, for both the Longitudinal Analysis and the
Cross-Sectional Analysis we will not exclude any patients due to race or age who fit
the inclusion criteria.
We found this trial at
39
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1720 2nd Ave S
Birmingham, Alabama 35233
(205) 934-4011 
Principal Investigator: Fred Goldman, MD
Phone: 205-939-5855
University of Alabama at Birmingham The University of Alabama at Birmingham (UAB) traces its roots...
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Principal Investigator: Jacob Bleesing, MD
Phone: 513-636-0992
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Principal Investigator: Rolla Abu-Arja, MD
Phone: 614-722-3582
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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5323 Harry Hines Blvd
Dallas, Texas 75235
(214) 648-3111
Principal Investigator: Victor Aquino, MD
Phone: 214-648-8800
Univ of Texas, Southwestern Med Ctr of Dallas The story of UT Southwestern Medical Center...
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Denver, Colorado 80218
Principal Investigator: Ralph Quinones, MD
Phone: 720-777-8465
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Neena Kapoor, MD
Phone: 323-361-2217
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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262 Danny Thomas Pl
Memphis, Tennessee 38105
(901) 495-3300
Principal Investigator: Ewelina Mamcarz, MD
Phone: 901-595-8343
St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Jennifer Heimall, MD
Phone: 215-590-2549
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Principal Investigator: Hey Chong, MD
Phone: 412-692-5103
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: Evan Shereck, MD
Phone: 503-494-0829
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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7700 Floyd Curl Dr
San Antonio, Texas 78229
(210) 575-7000
Principal Investigator: Troy Quigg, MD
Phone: 210-575-7348
Methodist Children's Hospital of South Texas Methodist Children
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500 S State St
Ann Arbor, Michigan 48109
(734) 764-1817
Principal Investigator: Mark Vander Lugt, MD
Phone: 737-936-9814
University of Michigan The University of Michigan was founded in 1817 as one of the...
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Atlanta, Georgia 30322
Principal Investigator: Elizabeth Stenger, MD
Phone: 404-785-1272
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Boston, Massachusetts 02115
Principal Investigator: Sung-Yun Pai, MD
Phone: 617-919-2508
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Calgary, Alberta
Principal Investigator: Nicola Wright, MD
Phone: 403-955-3035
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Morris Kletzel, MD
Phone: 773-880-4598
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Cleveland, Ohio 44106
Principal Investigator: Jingesh Dalal, MD
Phone: 216-844-3345
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
Principal Investigator: Suhag Parikh, MD
Phone: 919-668-1121
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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30 Prospect Ave
Hackensack, New Jersey 07601
(201) 996-2000
Principal Investigator: Alfred Gillio, MD
Phone: 201-996-5645
Hackensack University Medical Center Hackensack University Medical Center, part of the Hackensack University Health Network,...
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Houston, Texas
Principal Investigator: William Shearer, MD
Phone: 832-824-1339
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Los Angeles, California 90095
(310) 825-4321
Principal Investigator: Ted Moore, MD
Phone: 310-825-6708
UCLA UCLA's primary purpose as a public research university is the creation, dissemination, preservation and...
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Madison, Wisconsin 53705
Principal Investigator: Kenneth DeSantes, MD
Phone: 608-263-8563
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Milwaukee, Wisconsin 53226
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Minneapolis, Minnesota 55455
Principal Investigator: Angie Smith, MD
Phone: 612-626-2778
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New Orleans, Louisiana 70118
Principal Investigator: Lolie Yu, MD
Phone: 504-896-9740
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1275 York Ave
New York, New York 10021
(212) 639-2000
Principal Investigator: Susan Prockop, MD
Phone: 212-639-6715
Memorial Sloan Kettering Cancer Center Memorial Sloan Kettering Cancer Center — the world's oldest and...
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Principal Investigator: Holly Miller, MD
Phone: 602-933-0920
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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Rochester, Minnesota 55905
Principal Investigator: Richard Bram, MD
Phone: 507-284-2695
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Rochester, New York 14642
Principal Investigator: Jeffrey Andolina, MD
Phone: 585-276-3229
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Saint Louis, Missouri 63104
Principal Investigator: Alan Knutsen, MD
Phone: 314-268-2700
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Saint Louis, Missouri 63110
Principal Investigator: Shalini Shenoy, MD
Phone: 314-454-6018
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Salt Lake City, Utah 84113
Principal Investigator: David Shyr, MD
Phone: 801-662-4736
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San Francisco, California 94143
Principal Investigator: Morton Cowan, MD
Phone: 415-502-0203
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Seattle, Washington 98109
Principal Investigator: Lauri Burroughs, MD
Phone: 206-667-2396
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Valhalla, New York 10595
Principal Investigator: Allyson Flower, MD
Phone: 914-493-7997
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Washington, District of Columbia 20010
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Wilmington, Delaware 19899
Principal Investigator: Emi Caywood, MD
Phone: 302-651-5500
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