7 completed, 1 not yet recruiting, 1 recruiting. 9 Phase 1, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 9 | Completed 7, Not yet recruiting 1, Recruiting 1 |
| Phase 2 | 4 | Completed 3, Recruiting 1 |
Follow Genetic Engineering: new trials by email Literature Review of Genetic Engineering trials Research Report on Genetic Engineering
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase - GM1 Gangliosidoses, GM1 Gangliosidosis, Type I | Phase 1 | Not yet recruiting | Tippi Mackenzie | 5 | 2027-01-01 | 2026-08-31 |
| HSV-tk + Valacyclovir + SBRT + Chemotherapy for Recurrent GBM ADV/HSV-tk (gene therapy) - Glioblastoma Multiforme, Astrocytoma, Grade III | Phase 1 / Phase 2 | Recruiting | David Baskin MD | 62 | 2017-12-11 | 2026-03-30 |
| Gene Therapy for X-linked Severe Combined Immunodeficiency Gene transfer - X-linked Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Completed | Assistance Publique - Hôpitaux de Paris | 5 | 2010-12-01 | 2026-03-11 |
| Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease rAAV1-CMV-GAA (study agent) Administration, RMST - Pompe Disease | Phase 1 / Phase 2 | Completed | University of Florida | 9 | 2010-09-01 | 2018-09-14 |
| A Study of a Retroviral Replicating Vector Combined With a Prodrug Administered to Patients With Recurrent Malignant Glioma Toca 511 vector, Toca FC - Glioblastoma, Anaplastic Astrocytoma | Phase 1 | Completed | Tocagen Inc. | 54 | 2010-07-01 | 2018-05-21 |
| Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector Gene transfer - Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Completed | David Williams | 8 | 2010-04-01 | 2026-04-21 |
| Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B Gene Transfer, scAAV2/8-LP1-hFIXco - Hemophilia B | Phase 1 | Completed | St. Jude Children's Research Hospital | 14 | 2010-02-22 | 2026-04-02 |
| Effect of AdhAQP1 on Salivary Flow in Patients Treated With Radiation for Head and Neck Cancer Gene Transfer, AdhAQP1 - Parotid Salivary Dysfunction | Phase 1 | Completed | National Institute of Dental and Craniofacial Research (NIDCR) | 17 | 2006-09-01 | 2018-09-11 |
| Gene Modified Allogeneic Neuroblastoma Cells For Treatment of Relapsed/Refractory Neuroblastoma Interleukin-2 - Neuroblastoma | Phase 1 | Completed | St. Jude Children's Research Hospital | 24 | 1998-08-01 | 2008-06-03 |
Astrocytoma (2) · Glioblastoma (2) · X-Linked Combined Immunodeficiency Diseases (2) · Adenoviridae Infections (1) · Brain Neoplasms (1) · Gangliosidosis, GM1 (1) · Glioma (1) · Glycogen Storage Disease (1) · Glycogen Storage Disease Type II (1) · Head and Neck Neoplasms (1) · Hemophilia B (1) · Infections (1) · Neuroblastoma (1) · Oligodendroglioma (1) · Severe Combined Immunodeficiency (1)
St. Jude Children's Research Hospital (2) · Assistance Publique - Hôpitaux de Paris (1) · David Baskin MD (1) · David Williams (1) · National Institute of Dental and Craniofacial Research (NIDCR) (1) · Tippi Mackenzie (1) · Tocagen Inc. (1) · University of Florida (1)
United States (7) · France (1) · United Kingdom (1)
Alain Fischer, MD, PhD (1) · Asha Das, MD (1) · Barry J Byrne, MD, PhD (1) · Blake M Warner, D.D.S. (1) · David S Baskin, MD (1) · Gregory A Hale, MD (1) · Jennifer Whangbo, MD (1) · Manish Aghi, MD, NS (1) · Shelley Collins, MD (1) · Ulrike Reiss, MD (1)
PMID 25295500, PMID 30261899, PMID 19892587, PMID 22149959, PMID 22794786, PMID 23570273, PMID 25409372, PMID 27996967, PMID 40499172
2008 (1), P01HL059412 (1), R01HL094396 (1)