Now recruiting patients (clinicaltrials.gov)
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
ClinicalTrials.gov Identifier NCT03311503
Contact: Sung-Yun Pai, MD 617-919-2191 sung-yun.pai@childrens.harvard.edu
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
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 , 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: Ewelina Mamcarz, MD 901-595-8343 ewelina.mamcarz@stjude.org
Contact: Mort Cowan, MD 415-476-2656 mort-cowan@ucsf.edu
Contact: Aleksandra Petrovic, MD 206-987-7450 aleksandra.petrovic@seattlechildrens.org
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
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
Contact: Paul Szabolcs, MD 412-692-5427 paul.szabolcs@chp.edu
Contact: Shawna McIntyre, BSN, RN 412-692-5552 mailto: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
Contact: Angela R. Smith, M.D. 612-626-2778 smith719@umn.edu
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
ClinicalTrials.gov identifier: NCT02963064
Contacts:
Dr. Rajni Agarwal 650-724-7173 rajnia@stanford.edu
Dr. Christopher Dvorak 415-476-2188 christopher.dvorak@ucsf.edu
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
ClinicalTrials.gov Identifier: NCT01380990
Contact: Bobby Gaspar, Professor +44 (0)207 905 2319 H.Gaspar@ucl.ac.uk
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
ClinicalTrials.gov Identifier: NCT03538899
Contact: Contact: Morton Cowan, MD 415-476-2188 Mort.Cowan@ucsf.edu
Contact: Jennifer Puck, MD 415 502-2090 Jennifer.Puck@ucsf.edu
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
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
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
ClinicalTrials.gov Identifier: NCT03597594
Contact: Ewelina Mamcarz, MD 866-278-5833 referralinfo@stjude.org
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
