Clinical Trials for the Treatment of SCID
Now recruiting patients (clinicaltrials.gov)
For the most recent information on clinical trials or to verify if these trials are still recruiting, 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. 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
6. 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
7. 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
8. 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
9. 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
10. 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
11. 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
12. 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
13. 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
14. 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
| SCID Angels does not endorse or recommend any clinical study. The list on this page is provided for patients’ convenience and knowledge. | |