Study to Evaluate the Safety and Tolerability of FX201 in Patients with Osteoarthritis of the Knee

NCTID	NCT04119687 (View at clinicaltrials.gov)
Description	This two-part study will be conducted in male and female patients, 30 - 80 years of age with painful OA of the index knee with Kellgren-Lawrence (K-L) Grade 2, 3 or 4. Part 1 - Single Ascending Dose (SAD) Phase: Up to three ascending doses of FX201 will be tested in cohorts of 5-8 patients. Each patient will only receive one injection of FX201. An independent Data Monitoring Committee (DMC) will review safety and guide the conduct of the study. Part 2 - Expansion Phase: Up to an additional 35 patients will be enrolled at each dose level reviewed by the DMC. Each patient will only receive one injection of FX201. (Show More)
Development Status	Active
Indication	Osteoarthritis, Knee
Disease Ontology Term	DOID:8398
Compound Name	PCRX-201
Compound Alias	Enekinragene inzadenovec, FX-201
Compound Description	AdV-NFKB-IL1RA
Sponsor	Pacira Pharmaceuticals, Inc
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	72
Results Posted	Not Available

Target Gene/Variant	IL1RA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intraarticular
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	Ad5
Editor Type	none
Dose 1	1.4E10 gc/knee
Dose 2	1.4E11 gc/knee
Dose 3	1.4E12 gc/knee
Dose 4
Dose 5

No.of Trial Sites	6
Locations	United States

Has US IND	True
FDA Designations	Regenerative Medicine Advanced Therapy
Recent Updates	FDA granted RMAT designation March 2024