Table 3.

Clinical trials.

References	Objectives	NCT #	Participants	Study design	Disease models	Delivery methods	Targets	Effects
Ha C-et al. (59)	Evaluate the dose-response of TissueGeneC in OA patients	NCT02341391	Adults with KL grade IV knee OA	Clinical Phase I, single center, open-label, dose-escalation	Human OA patients	TissueGeneC	TGFβ1	No treatment-related serious adverse events; swelling, effusion and minor reactions are dose-dependent; a dose-dependent trend efficacy
Cherian et al. (60)	Evaluate efficacy of treating grade III OA patients with genetically engineered allogenic human chondrocytes expressing TGF-β1	NCT01221441	Adults with KL grade III knee OA	Clinical phase II: multi-center, double-blinded, placebo-controlled, randomized	Human OA patients	Retrovirally transduced allogenic human chondrocytes (TissueGene-C)	TGFβ1	Improved keen function and pain as assessed by IKDC and VAS; less analgesic use
Ha et al. (61)	Evaluate the efficacy and safety of a cell-mediated therapy in OA patients	NCT02341378	Adults with KL grade IV knee OA	Clinical phase IIa, multicenter, single -blind	Human OA patients	GEC-TGF-β1 (TissueGeneC)	TGFβ1	Improved symptoms, activity levels and knee function; Significant improvement in stiffness, motor function and pain demonstrated via the IKDC, WOMAC and VAS scores
Kim et al. (62)	Evaluate the efficacy and safety of a cell-mediated therapy in OA patients	NCT02072070	Adults with KL grade III knee OA	Clinical Phase III, multicenter, double-blind	Human OA patients	TissueGeneC	TGFβ1	Decreased serum CTX-1 and urine CTX-II levels over 1 year in TG-C than placebo-treated patients; significant improvements in function and pain in OA patients; frequent adverse events were edema, arthralgia, joint swelling and injection site pain
NA	Post Marketing Surveillance on Safety and Effectiveness Evaluation of INVOSSA	NCT03412864	Adults with KL grade III knee OA	Post marketing surveillance study	Human OA patients	INVOSSA	TGFβ1	No outcome data
NA	Preliminary Evaluation of Safety, Tolerability, and Efficacy of XT-150	NCT03282149 NCT03477487 NCT03769662	Adults for whom replacement knee surgery is recommended	Clinical Phase I, single center, dose escalation	Human OA patients	hIL-10var pDNA (XT-150)	IL-10	No outcome data
NA	Evaluate the efficacy and safety of XT-150 in patients experiencing moderate to severe pain due to OA of the knee	NCT04124042	Adults with WOMAC pain score ≥ 8	Clinical phase II: multi-center, double-blinded, placebo-controlled, randomized	Human OA patients	hIL-10var pDNA (XT-150)	IL-10	No outcome data
Sellon et al. (63) (reviewed by Evans C 2022) (64)	Evaluate safety of three different doses of sc-rAAV2.5IL-1Ra	NCT02790723	Adults with moderate OA of the knee	Clinical Phase I, single center, open-label, dose-escalation	Human OA patients	Sc-rAAV2.5IL-1Ra	IL-1Ra	No outcome data
Kelley et al. (65) (reviewed by Evans C 2022) (64)	Evaluate the Safety and Tolerability of FX201	NCT04119687	Adults with KL grade II, III or IV knee OA	Clinical Phase I, single center, open-label, dose-escalation	Human OA patients	HDAd-IL-1Ra (FX201)	IL-1Ra	No outcome data