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