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
Contacts:
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
Contacts:
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 and TN, University of California – San Francisco – San Francisco, CA,
ClinicalTrials.gov Identifier: NCT01512888
Contacts:
| Contact: Stephen Gottschalk, MD | 901-595-2166 | stephen.gottschalk@stjude.org |
Contact: Ewelina Mamcarz, MD 901-595-8343 ewelina.mamcarz@stjude.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
Contacts:
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
Contacts:
Contact: Paul Szabolcs, MD 412-692-5427 paul.szabolcs@chp.edu
Contact: Shawna McIntyre, BSN, RN 412-692-5552 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
Contacts:
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
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:
Contact: Bobby Gaspar, Professor +44 (0)207 905 2319 H.Gaspar@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
Contacts:
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
Contacts:
| Contact: US GSK Clinical Trials Call Center | 877-379-3718 | GSKClinicalSupportHD@gsk.com |
