Individuals with a past diagnosis of severe combined immune deficiency (including many cases of “leaky SCID”, Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant, gene therapy, or enzyme replacement in the past may be eligible for 6902. The purpose of 6902 is very similar to 6901, except 6902 is looking backwards at what has already been done in the past (compared to 6901 which is looking into the future). Over 800 patients with SCID are expected to be enrolled on 6902. This makes 6902 the largest study ever to describe outcomes for patients with SCID treated at many different hospitals around North America.
One of the most important components of the 6902 study is the “cross sectional” study. Patients who have received their treatments (BMT, gene therapy, enzyme replacement) many years ago are asked to come back to the hospital where they were treated. During this visit, additional research blood work is drawn and information is gathered regarding long-term transplant outcomes such as infections, graft-versus-host disease, autoimmune diseases, and quality of life. This will allow PIDTC researchers to better understand long-term outcomes from procedures that occurred many years ago (sometimes over 30 years ago) – something that is not possible at the present time with 6901. This will help researchers to best design new treatments and clinical trials in the future for children with SCID.
Participating Sites
Boston Children's Hospital
300 Longwood Avenue
Boston, Massachusetts, United States, 02115
https://www.childrenshospital.org/
Contact:
Craig
Platt
craig.platt@childrens.harvard.edu
Susan Prockop Malika KapadiaCardinal Glennon Children's Hospital
1465 S Grand Boulevard
Saint Louis, Missouri, United States, 63104
https://www.ssmhealth.com/locations/cardinal-glennon-childrens-hospital
Children's & Women's Health Centre of British Columbia
4500 Oak Street
Vancouver, British Columbia, Canada, V6H 3N1
http://www.bcwomens.ca/
Contact:
Stuart
Turvey
Children's Hospital/LSUHSC, New Orleans
200 Henry Clay Avenue, Suite 4109
New Orleans, Louisiana, United States, 70118
https://www.chnola.org
Contact:
Ken
Paris
Cincinnati Children's Hospital Medical Center
3333 Burnett Avenue
Cincinnati, Ohio, United States, 45229
https://www.cincinnatichildrens.org/
Contact:
Jack
Bleesing
Memorial Sloan Kettering Cancer Center
1275 York Avenue
New York, New York, United States, 10065
https://www.mskcc.org/
Contact:
Joseph
Oved
National Institute of Allergy and Infectious Diseases
5601 Fishers Lane, MSC 9806
Bethesda, Maryland, United States, 20892
https://www.niaid.nih.gov/
Contact:
Elizabeth M.
Kang,
MD
Oregon Health & Science University
3181 S.W. Sam Jackson Park Road
Portland, Oregon, United States, 97239-3098
https://www.ohsu.edu
Contact:
Evan
Shereck
St. Jude Children's Research Hospital
262 Danny Thomas Place
Memphis, Tennessee, United States, 38105
https://www.stjude.org/
Contact:
Ewelina
Mamcarz
University of Michigan Health System
500 S State Street
Ann Arbor, Michigan, United States, 48109
https://www.uofmhealth.org
Contact:
Gregory
Yanik
University of Rochester
500 Joseph C. Wilson Boulevard
Rochester, New York, United States, 14627
https://www.rochester.edu/
Contact:
Jeffrey
Andolina
jeffrey_andolina@urmc.rochester.edu
Katherine Tuttle Geoffrey Weinberg