285. Fanconi anemia Clinical trials / Disease details
Clinical trials : 62 / Drugs : 93 - (DrugBank : 30) / Drug target genes : 30 - Drug target pathways : 144
| 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 | NCT04248439 (ClinicalTrials.gov) | July 15, 2020 | 24/1/2020 | Gene Therapy for Fanconi Anemia, Complementation Group A | A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A | Fanconi Anemia Complementation Group A | Biological: RP-L102 | Rocket Pharmaceuticals Inc. | NULL | Recruiting | 1 Year | N/A | All | 5 | Phase 2 | United States |
| 2 | EUCTR2018-002502-31-GB (EUCTR) | 07/02/2020 | 08/10/2019 | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A - FANCOLEN-II | Fanconi anemia (subtype A) MedDRA version: 20.0;Level: LLT;Classification code 10055206;Term: Fanconi's anemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: RP-L102 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 2 | Spain;United Kingdom | ||
| 3 | NCT04069533 (ClinicalTrials.gov) | November 28, 2019 | 23/8/2019 | Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients With Fanconi Anemia Subtype A | Fanconi Anemia Complementation Group A | Biological: RP-L102 | Rocket Pharmaceuticals Inc. | NULL | Recruiting | 1 Year | 17 Years | All | 5 | Phase 2 | Spain |
| 4 | NCT03814408 (ClinicalTrials.gov) | January 11, 2019 | 14/1/2019 | A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A | A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A | Fanconi Anemia Complementation Group A | Biological: RP-L102 | Rocket Pharmaceuticals Inc. | NULL | Active, not recruiting | 1 Year | 12 Years | All | 2 | Phase 1 | United States |
| 5 | EUCTR2018-002502-31-ES (EUCTR) | 31/10/2018 | 02/08/2018 | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A - FANCOLEN-II | Fanconi anemia (subtype A) MedDRA version: 20.0;Level: LLT;Classification code 10055206;Term: Fanconi's anemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: RP-L102 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 2 | Spain |