65. Primary immunodeficiency Clinical trials / Disease details
Clinical trials : 500 / Drugs : 614 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 217
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03601286 (ClinicalTrials.gov) | December 21, 2018 | 22/2/2018 | Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency | Phase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan | Severe Combined Immunodeficiency, X-Linked | Drug: Lentiviral vector transduced CD34+ cells | Great Ormond Street Hospital for Children NHS Foundation Trust | NULL | Recruiting | 8 Weeks | 5 Years | Male | 5 | Phase 1 | United Kingdom |
| 2 | EUCTR2018-000673-68-GB (EUCTR) | 09/10/2018 | 27/07/2018 | A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined Immunodeficiency | Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 | Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. Children with SCID lack virtually all immune protection from pathogens. They are prone to repeated and persistent infections that can be very serious or life threatening. MedDRA version: 20.0;Level: LLT;Classification code 10069566;Term: Severe combined immunodeficiency syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells INN or Proposed INN: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 3 | NCT02022696 (ClinicalTrials.gov) | December 16, 2013 | 20/12/2013 | Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector | Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector | Adenosine Deaminase Deficiency;ADA-SCID | Genetic: Lentiviral Gene Transfer | National Human Genome Research Institute (NHGRI) | UCLA@@@Duke University Medical Center;Duke Univ. Medical Center;National Cancer Institute (NCI);National Institutes of Health Clinical Center (CC) | Completed | 1 Year | 65 Years | All | 1 | Phase 1 | United States |
| 4 | NCT01306019 (ClinicalTrials.gov) | September 25, 2012 | 26/2/2011 | Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-linked Severe Combined Immunodeficiency (XSCID) | Drug: Palifermin;Drug: Busulfan;Biological: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Suspended | 2 Years | 40 Years | Male | 19 | Phase 1/Phase 2 | United States |
| 5 | NCT03315078 (ClinicalTrials.gov) | April 2012 | 16/10/2017 | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-Linked Combined Immunodeficiency Diseases | Biological: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1a-h?c-OPT;Drug: Palifermin;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 2 Years | 40 Years | All | 13 | Phase 1/Phase 2 | United States |