Clinical Trials for SCID


Clinical Trials for the Treatment of SCID

Now recruiting patients (clinicaltrials.gov)

For the most recent information on clinical trials, please visit www.clinicaltrials.gov.

Gene Therapy & Bone Marrow Transplant Clinical Trials:

1.  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 , TN, University of California – San Francisco – San Francisco, CA, and Seattle Children’s Research Institute Seattle, Washington

ClinicalTrials.gov Identifier:  NCT01512888

Contact: Stephen Gottschalk, MD   901-595-2166 Stephen.gottschalk@stjude.org

Contact: Mort Cowan, MD    415-476-2656    mort-cowan@ucsf.edu 

Contact: Aleksandra Petrovic, MD   206-987-7450 aleksandra.petrovic@seattlechildrens.org

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 40 years old (Child/Adult)

Type of SCID:  X-linked SCID

Location:  National Institute of Health – Bethesda, Maryland

ClinicalTrials.gov Identifier:  NCT01306019

Contact: Lee C England, P.A.-C  (240) 858-3649  lee.england@nih.gov

Contact: Suk S De Ravin, M.D. (301) 496-6772   sderavin@mail.nih.gov

Principal Investigator: Suk S De Ravin, M.D. National Institute of Allergy and Infectious Diseases (NIAID)

3.  A Study of the Use of a Monoclonal Antibody (AMG 191) 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

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 and Memorial Sloan Kettering Cancer Center New York, NY

ClinicalTrials.gov identifier:  NCT02963064

Contacts:

Dr. Rajni Agarwal                  650-724-7173          rajnia@stanford.edu

Dr. Christopher  Dvorak      415-476-2188           christopher.dvorak@ucsf.edu

Dr. Susan Prockop                 212-639-6715          prockops@mskcc.org

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

Age:  up to 5 years (Child)

Type of SCID:  X-linked SCID

Locations:  Mattel Children’s Hospital – UCLA – Los Angeles, CA, Boston Children’s Hospital – Boston, MA, Great Ormond Street Hospital – London, United Kingdom

ClinicalTrials.gov Identifier NCT03311503

Contact:  Colleen Dansereau      617-919-7008  colleen.dansereau@childrens.harvard.edu

Principal Investigator:  Sung-Yun Pai, MD  Boston Children’s Hospital

5.  A Study of the Use of a Monoclonal Antibody (AMG 191) 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

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 and Memorial Sloan Kettering Cancer Center New York, NY

ClinicalTrials.gov identifier:  NCT02963064

Contacts:

Dr. Rajni Agarwal                  650-724-7173          rajnia@stanford.edu

Dr. Christopher  Dvorak      415-476-2188           christopher.dvorak@ucsf.edu

Dr. Susan Prockop                 212-639-6715          prockops@mskcc.org

6.  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

ClinicalTrials.gov Identifier:  NCT03538899

Contact: Morton Cowan, MD     415-476-2188    Mort.Cowan@ucsf.edu
Contact: Jennifer Puck, MD       415 502-2090    Jennifer.Puck@ucsf.edu

7.  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

ClinicalTrials.gov Identifier: NCT03217617

Contact: XiaoDong Shi, M.D./P.H.D    +86-13911601076    xsusan28@sina.com
Contact: Lixiao Shi, M.M.    +86-18810963129    13780524314@163.com

Contact: Jie Zheng, MD/PhD    +86-13683284467    cutezjie@163.com

Contact: Lung-Ji Chang, PhD    86-075586725195    c@szgimi.org

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

8. Gene Transfer for ADA-SCID Using an Improved Lentiviral Vector (TYF-ADA)

Age:  1 Month and Older (Child/Adult/Older Adult)

Type of SCID:  ADA 

Location:  Shenzhen, Guangdong, China – Shenzhen Geno-immune Medical Institute 

ClinicalTrials.gov Identifier NCT03645460

Contact: Lung-Ji Chang, Ph.D    86-0755-86725195  c@szgimi.org

9.  Lentiviral Gene Therapy for X-SCID

Age:  8 Weeks to 5 Years (Child)

Type of SCID:  X-SCID

Location:  Great Ormond Street Hospital for Children NHS Foundation Trust – London, United Kingdom

ClinicalTrials.gov Identifier NCT03601286

Contact: Claire Booth, MBBS, MRCPCH, MSc, PhD  c.booth@ucl.ac.uk

Contact: Fiona Shepheard, BSc    0207 762 6058 f.shepheard@ucl.ac.uk

10.  Gene Therapy for X-SCID

Age:  Up to 18 Years (Child/Adult)

Type of SCID:  X-linked SCID

Location:  Children’s Hospital of Chongqing Medical University – Chongqing, China

ClinicalTrials.gov Identifier NCT04286815

Contact: Xiaodong Zhao, PHD  18623070626  zhaoxd530@aliyun.com

Contact: Qiling Xu, MD   18581059910      272864835@qq.com

11.  Conditioning SCID Infants Diagnosed Early (CSIDE)

Age:  Up to 2 Years (Child)

Type of SCID:  IL2RG, JAK3, RAG1 or RAG2

Location:  20 Study Locations in the United States

ClinicalTrials.gov Identifier NCT 03619551

Contact: Jenny Vogel         763-406-8691    jvogel@nmdp.org

Contact: Liz Gourdine       323-361-6652   CSIDE@chla.usc.edu

12. 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

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

13.  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

Contact: Angela R. Smith, M.D.    612-626-2778    smith719@umn.edu

14.  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

ClinicalTrials.gov Identifier: NCT03597594

Contact: Ewelina Mamcarz, MD    866-278-5833    referralinfo@stjude.org

15.  Efficacy and Safety of Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Age:  Up to 17 Year (Child)

Type of SCID:  ADA

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

ClinicalTrials.gov Identifier NCT03765632

Contact: Claire Booth, Dr  +44 (0)207 905 2198    c.booth@ucl.ac.uk

 

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

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

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

ClinicalTrials.gov Identifier: NCT03478670

Contact: US GSK Clinical Trials Call Center  877-379-3718    GSKClinicalSupportHD@gsk.com

3.  Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogenic Hematopoietic Stem Cell Transplantation in SCID Patients

Age:  2 Years to 18 Years (Child/Adult)

Type of SCID:  All Types 

Location:  Unité d’Immunologie Hématologie Rhumatologie Pédiatrique (UIHR), Paris, France

ClinicalTrials.gov Identifier:  NCT03879876

Contact: Marina CAVAZZANA, MD, PhD    33 1 44 49 50 68   m.cavazzana@aphp.fr

4.  Registry Study of Revcovi Treatment in Patients With ADA-SCID

Age:  Up to 65 Years   (Child, Adult, Older Adult)

Type of SCID:  ADA-SCID

Location:  University of South Florida Allergy Immunology Clinic – Saint Petersburg, FL, UBMD Pediatrics Outpatient Center – Buffalo, NY, UPMC Children’s Hospital of Pittsburgh – Pittsburg, PA, Le Bonheur Children’s Hospital – Memphis, TN

ClinicalTrial.gov Identifier NCT03878069\

Contact: Jolan Walter, MD, PhD    727-553-1258    jolanwalter@usf.edu

Contact: Maryssa Ellison        mellison@usf.edu

Contact: Heather Lehman, MD    716-323-0130  hkm@buffalo.edu

Contact: Helena McClenahan  hmcclenahan@upa.chob.edu

Contact: Hey Chong, MD    412-692-7785  hey.chong@chp.edu

Contact: Jay Lieberman, MD    901-287-7337  jlieber1@uthsc.edu

5.  Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

Age:  18 Years and Older (Adult/Older Adult)

Type of SCID:  ADA-SCID

Location:  Ospedale San Raffaele – Telethon Institute for Gene Therapy (OSR-TIGET) Milan, Italy

ClinicalTrials.gov Identifier NCT03232203

Contact: Orchard Clinical Trials +44 (0) 20 3808 8286 medinfo@orchard-tx.com

Study Director:  Clinical Trials  Orchard Therapeutics