Table 1.
Characteristics of randomised, double-blind, parallel-group, placebo-controlled CTs included in the systematic review
| Registered number in EudraCT | Study name/Study design | Trade name/INN | Indication | Rare disease | Published papers of the RCT |
|---|---|---|---|---|---|
| 2012–001700-37 | CUPID-2b: phase 2b | AAV1/SERCA2a | Ischemic Cardiomyopathy (heart failure and reduced ejection fraction) | no | Greenberg, B., (2016)5; Greenberg, B. (2014)12 |
| 2011–004761-33 | phase 2b | pGM169/GL67A | Cystic fibrosis | yes | Alton, E. (2016)14 |
| 2006–001246-13 | TRIST: phase 3 | TroVax (Attenuated recombinant vaccinia virus containing the gene for human 5T4 oncofoetal antigen) | Locally advanced or metastatic renal clear cell adenocarcinoma | EU/3/06/429 | TroVax® Protocol TV3/001/06 Synopsis6; Amato, R. (2010)11 |
| 2004–002508-13 | phase 2 | Ad2/HIF-1α/VP16 | Peripheral Arterial Disease (PAD): Intermittent Claudication (IC) | no | Ad2/HIF-1α/VP16, Abbreviated Clinical Study Report: PADHIF007049; Creager, М. (2011)10 |
| 2005–004068-21 | phase 2 | no | n/a | ||
| 2016–002761-63 | phase 3 | VGX-3100 | HPV-16 and/or HPV-18 related high grade squamous intraepithelial lesions (HSIL) of the cervix | no | n/a |
| 2015–001265-11 | RESCUE: phase 3, Subjects Affected for 6 Months or Less | Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene (lenadogene nolparvovec) | Leber Hereditary Optic Neuropathy Due to the G11778A | EU/3/11/860 | n/a |
| 2015–001266-26 | REVERSE: phase 3, Subjects Affected for more than 6 Months and to 12 months | Mutation in the Mitochondrial NADH Dehydrogenase 4 Gene | EU/3/11/860 | n/a | |
| 2017–002187-40 | REFLECT: phase 3, in Subjects Affected for Up to One Year | EU/3/11/860 | n/a |
AAV1/SERCA2a: adeno-associated virus serotype 1 (AAV1); Sarcoplasmic/endoplasmic reticulum Ca2+-ATPase gene; pGM169/GL67A: Plasmid expressing the human CFTR gene complexed with cationic lipid 67 and helper lipids; CFTR: cystic fibrosis transmembrane conductance regulator; Ad2/HIF-1alfa/VP16: A recombinant adenovirus expressing a constitutively-active hybrid form of the Hypoxia-inducible factor-1α subunit; HPV-16/HPV-18: Human Papillomavirus Strains 16/18; NADH: Nicotinamide Adenine Dinucleotide – Hydrogen.