Completed Gene Therapy Trials Tracked By U.S. National Library Of Medicine As Of December 2020

A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies.

In a clinical trial, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants’ behavior, such as diet. Clinical trials may compare a new medical approach to a standard one that is already available, to a placebo that contains no active ingredients, or to no intervention. Some clinical trials compare interventions that are already available to each other. When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful, or no different than available alternatives (including no intervention). The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants. For example, investigators may give a drug or treatment to participants who have high blood pressure to see whether their blood pressure decreases.

Clinical trials used in drug development are sometimes described by phase. These phases are defined by the Food and Drug Administration (FDA).

In an observational study, investigators assess health outcomes in groups of participants according to a research plan or protocol. Participants may receive interventions (which can include medical products such as drugs or devices) or procedures as part of their routine medical care, but participants are not assigned to specific interventions by the investigator (as in a clinical trial). For example, investigators may observe a group of older adults to learn more about the effects of different lifestyles on cardiac health.

Here’s the link for studies inluded under the search term, “gene therapy.”

RankTitleStatusStudy ResultsConditionsInterventionsLocationsURL
1Gene Therapy of Pancreatic Ductal AdenocarcinomaCompletedNo Results AvailablePancreatic AdenocarcinomaGenetic: Gene Therapy product CYL-02 = plasmid DNA pre-complexed to linear polyethylenimine encoding sst2 + dck::umk genesToulouse Universitary Hospital (Rangueil), Department of Gastroenterology At Rangueil Hospital, Toulouse, France
2EndocardialVascularEndothelialGrowth Factor D(VEGF-D)Gene Therapy for the Treatment of Severe Coronary Heart DiseaseCompletedNo Results AvailableAngina Pectoris|Myocardial InfarctionBiological: VEGF-D gene transferKuopio University Hospital, Kuopio, Finland
3ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCIDCompletedHas ResultsImmunologic Deficiency SyndromesGenetic: Gene Therapy|Drug: Busulfan|Drug: PEG-ADAInvestigational Site, Jerusalem, Israel|Investigational Site, Milano, Lombardia, Italy
4Gene Therapy for ADA-SCIDCompletedNo Results AvailableSevere Combined Immunodeficiency SyndromeGenetic: gene transduced PBL and/or gene transduced HSC
5Liver Transplantation With ADV-TK Gene Therapy Improves Survival in Patients With Advanced Hepatocellular CarcinomaCompletedNo Results AvailableHepatocellular Carcinoma|Liver TransplantationGenetic: ADV-TK (adenovirus-thymidine kinase enzyme) gene therapyBeijing Chao Yang Hospital, Beijing, Beijing, China
6Gene Therapy for Gyrate AtrophyCompletedNo Results AvailableGyrate AtrophyProcedure: Gene therapyNational Eye Institute (NEI), Bethesda, Maryland, United States
7Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2CCompletedNo Results AvailableLimb Girdle Muscular Dystrophy Type 2C|Gamma-sarcoglycanopathyBiological: AAV1-gamma-sarcoglycan vector injectionHôpital Pitié-Salpêtrière, Paris, France
8Gene Therapy for Chronic Granulomatous Diseases – Long-term Follow-upCompletedNo Results AvailableChronic Granulomatous Disease|Communicable DiseaseDrug: Gene Therapy Method for CGD|Device: Isolex 300i Magnetic Cell SelectorNational Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States
9Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial HypercholesterolemiaCompletedNo Results AvailableFamilial HypercholesterolemiaProcedure: gene therapy
10Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCACompletedNo Results AvailableLimb Girdle Muscular Dystrophy Type 2D (LGMD2D)Drug: scAAVrh74.tMCK.hSGCANationwide Children’s Hospital, Columbus, Ohio, United States
Completed Gene Therapy Trials Tracked By U.S. National Library Of Medicine As Of December 2020 was last modified: December 14th, 2020 by Staff