Clinical Trials for the Treatment of SCID

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

 

 

 

 

 

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