Clinical Trials for SCID

Clinical Trials for the Treatment of SCID

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Gene Therapy & Bone Marrow Transplant Clinical Trials:

1.  Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Age:  up to 18 years (Child/Adult)

Type of SCID:  X-SCID

Collaborators/Locations:  Boston Children’s Hospital – Boston, MA Identifier NCT03311503

Contact:  Sung-Yun Pai, MD    617-919-2191

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 Identifier:  NCT01306019

Contact: Nana Kwatemaa, R.N. (301) 451-7820 email:

Contact: Suk S De Ravin, M.D. (301) 496-6772 email:

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

Sponsor:  St. Jude Children’s Research Hospital

Location:  St. Jude Children’s  Research Hospital Memphis , TN, University of California – San Francisco – San Francisco, CA, and Seattle Children’s Research Institute Seattle, Washington Identifier:  NCT01512888

Contact: Stephen Gottschalk, MD   901-595-2166

Contact: Ewelina Mamcarz, MD 901-595-8343

Contact: Mort Cowan, MD    415-476-2656 

Contact: Aleksandra Petrovic, MD    206-987-7450

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 Identifiers:  NCT01821781

Contact: Jeffrey Bednarski, MD    314-454-6018
Contact: Lisa Murray, CCRP    314-454-4240
Contact: Troy Quigg, DO, MS

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 Identifiers:  NCT01852370

Contact: Paul Szabolcs, MD 412-692-5427

Contact: Shawna McIntyre, BSN, RN 412-692-5552

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 Identifiers:  NCT01652092

Contact: Angela R. Smith, M.D.    612-626-2778

7.  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 (Child, Adult, Older Adult)

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 identifier:  NCT02963064


Dr. Rajni Agarwal                  650-724-7173

Dr. Christopher  Dvorak      415-476-2188 

8.  Lentiviral Gene Therapy for Adenosine Deaminase (ADA) Deficiency

Age:  Up to 5 years

Type of SCID:  ADA

Location:  Great Ormond Street Hospital for Children NHS Trust         London, United Kingdom Identifier: NCT01380990

Contact:  Bobby Gaspar, Professor    +44 (0)207 905 2319

9.  Autologous Gene Therapy for Artemis-Deficient SCID

Age:  2 Months and older   (Child, Adult, Older Adult)

Sex:  All

Type of SCID:  Artemis (ART-SCID)

Location: University of California, San Francisco (UCSF) Children’s Hospital
San Francisco, California, United States, 94143 Identifier:  NCT03538899

Contact:  Contact: Morton Cowan, MD   415-476-2188
Contact: Jennifer Puck, MD                       415 502-2090

10.  Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector (TY{-IL-2RG)

Age:  1 Month to 10 Years   (Child)

Sex:  Male

Type of SCID:  X-SCID

Location:  China, Beijing – Capital Institute of Pediatrics affiliated Children’s hospital
Beijing, China, Beijing Children’s Hospital Beijing, Beijing, China, and Shenzhen Geno-immune Medical Institute Shenzhen, Guangdong, China Identifier: NCT03217617

Contact: XiaoDong Shi, M.D./P.H.D    +86-13911601076
Contact: Lixiao Shi, M.M.    +86-18810963129

Contact: Jie Zheng, MD/PhD    +86-13683284467

Contact: Lung-Ji Chang, PhD    86-075586725195

Principal Investigator:  Lung-Ji Chang, Ph.D   Shenzhen Geno-Immune Medical Institute

Study Director:  Xiao-Dong Shi, M.D./Ph. D  Capital Institute of Pediatrics affiliated Children’s hospital

Study Director:  Jie Zheng, M.D./Ph. D  Beijing Children’s Hospital

11.  Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)

Age:  2 Months and older   (Child, Adult, Older Adult)

Sex:  All

Location:  St. Jude Children’s Research Hospital  Memphis, Tennessee Identifier: NCT03597594

Contact: Ewelina Mamcarz, MD    866-278-5833

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 Identifier:  NCT01420627

Contact: Greg Balagot    323-361-8569

Contact: Heidi Shannon    415-502-6057

Contact: Helena McClenahan

Contact: Gayle Krenik    718-405-8830

2.  Phase IV, GSK2696273, Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Age:  Child, Adult, Senior

Type of SCID:  ADA

Location:  Italy Identifier: NCT03478670

Contact: US GSK Clinical Trials Call Center  877-379-3718