Now recruiting patients (clinicaltrials.gov)
Gene Therapy & Bone Marrow Transplant Trials:
1. Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Age: Children and Adults
Type of SCID: X-linked SCID
Collaborators/Locations: Children’s Hospital Boston – Boston, MA, Cincinnati Children’s Medical Center – Cincinnati, OH, and Mattel Children’s Hospital (UCLA) – Los Angeles, CA
ClinicalTrials.gov Identifier: NCT01129544
Contacts:
Dr. David A Williams, MD 617-919- 2697 dawilliams@tch.harvard.edu
Colleen H. Dansereau, RN 617-919-7008 colleen.dansereau@childrens.harvard.edu
Principal Investigator: Sung-Yun Pai, MD Boston Children’s Hospital
2. Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
Age: Children ages 2 to 20 years old
Type of SCID: X-linked SCID
Location: National Institute of Health – Bethesda, Maryland
ClinicalTrials.gov Identifier: NCT01306019
Contacts:
Contact: Nana Kwatemaa, R.N. (301) 451-7820 email: nkwatemaa@niaid.nih.gov
Contact: Suk S De Ravin, M.D. (301) 496-6772 email: sderavin@mail.nih.gov
Principal Investigator: Suk S De Ravin, M.D. National Institute of Allergy and Infectious Diseases (NIAID)
3. Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
Age: Children up to 24 months of age
Type of SCID: X-linked SCID
Location: St. Jude Children’s Research Hospital Memphis and TN, University of California – San Francisco – San Francisco, CA,
ClinicalTrials.gov Identifier: NCT01512888
Contacts:
Contact: Brian Sorrentino, MD 901-595-2727 brian.sorrentino@stjude.org
Contact: Ewelina Mamcarz, MD 901-595-8343 ewelina.mamcarz@stjude.org
Principal Investigator: Brian Sorrentino, MD St. Jude Children’s Research Hospital
4. Immune Disorder HSCT Protocol
Age: up to 21 Years (Child, Adult)
Immune Disorder: Severe Combined Immune Deficiency
Location: Washington University – St. Louis, MS and Methodist Healthcare – San Antonio, TX
ClinicalTrials.gov Identifiers: NCT01821781
Contacts:
Contact: Jeffrey Bednarski, MD 314-454-6018 Bednarski_J@kids.wustl.edu
Contact: Lisa Murray, CCRP 314-454-4240 Murray_L@kids.wustl.edu
Contact: Troy Quigg, DO, MS Troy.Quigg@mhshealth.com
5. Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases (BOLT+BMT)
Age: 5 Years to 40 Years (Child, Adult)
Immune Disorder: Severe Combined Immune Deficiency
Location: Children’s Hospital of Pittsburg – Pittsburg, PA
ClinicalTrials.gov Identifiers: NCT01852370
Contacts:
Contact: Paul Szabolcs, MD 412-692-5427 paul.szabolcs@chp.edu
Contact: Shawna McIntyre, BSN, RN 412-692-5552 mcintyresm@upmc.edu
Principal Investigator: Paul Szabolcs, MD Division of BMT and Cellular Therapy, Children’s Hospital of Pittsburgh of UPMC
6. Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
Age: up to 50 Years (Child, Adult)
Immune Disorder: Severe Combined Immune Deficiency
Location: Masonic Cancer Center, University of Minnesota – Minneapolis, MN
ClinicalTrials.gov Identifiers: NCT01652092
Contacts:
Contact: Angela R. Smith, M.D. 612-626-2778 smith719@umn.edu
7. Autologous Cryopreserved CD34+ Hematopoietic Cells Transduced With EFS-ADA Lentivirus for ADA SCID
Age: up to 17 Years (Child 30 days old to < 18 y.o.)
Type of SCID: ADA
Location: University of California, Los Angeles – Los Angeles, CA
ClinicalTrials.gov Identifiers: NCT02999984
Contacts:
Contact: Theodore B Moore, M.D.
(310) 825-6708
tbmoore@mednet.ucla.edu
Contact: Satiro De Oliveira, MD
t(310) 825-6708
sdeoliveira@mednet.ucla.edu
Study Director: Donald B. Kohn, MD University of California, Los Angeles
8. A Study of the Use of a Monoclonal Antibody to Allow Hematopoietic Stem Cell Engraftment in Children with SCID without Chemotherapy
Age: 3 months of age and older
Type of SCID: Includes all sub types of SCID except for Omenn syndrome, Leaky SCID, Reticular dysgenesis, Adenosine deaminase deficiency and Purine nucleoside phosphorylase deficiency
Collaborators/Locations: Lucile Packard Children’s Hospital at Stanford University – CA and Benioff Children’s Hospital at the University of California San Francisco – San Francisco, CA
ClinicalTrials.gov identifier: NCT02963064
Contacts:
Dr. Rajni Agarwal 650-724-7173 rajnia@stanford.edu)
Dr. Christopher Dvorak 415-476-2188 christopher.dvorak@ucsf.edu
Biological Intervention:
1. EZN-2279 in Patients With ADA-SCID
Age: Child, Adult, Senior
Type of SCID: ADA
Location: Children’s Hospital Los Angeles – Los Angeles, CA, University of California San Francisco – San Francisco, CA, Women’s and Children’s Hospital of Buffalo – Buffalo, and NY, Albert Einstein College of Medicine – The Bronx, NY
ClinicalTrials.gov Identifier: NCT01420627
Contacts:
Contact: Greg Balagot 323-361-8569 gbalagot@chla.edu
Contact: Heidi Shannon 415-502-6057 heidi.shannon@ucsf.edu
Contact: Helena McClenahan hmcclenahan@upa.chob.edu
Contact: Gayle Krenik 718-405-8830 gkreinik@montefiore.org
