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

NCT04119687 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: FX201-2019-001
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 6

Brief Summary

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.

Conditions

Osteoarthritis, Knee

Outcome Measures

Primary Outcomes (1)

• Primary Outcome Measure: Incidence of treatment emergent adverse events after a single intra-articular (IA) injection of FX201 (safety and tolerability)

  Incidence of treatment emergent adverse events after a single intra-articular (IA) injection of FX201

  Baseline to Week 104

Secondary Outcomes (1)

• Secondary Outcome Measure: Characterization of the systemic biodistribution of FX201 as seen through the percentage of patients positive for FX201 at Baseline, Day 1, Day 2, Week 1, Week 2, Week 3, and Week 4

  Baseline, Day 1, Day 2, Week 1, Week 2, Week 3, Week 4

Study Arms (3)

Low Dose FX201

EXPERIMENTAL

Single low dose FX201 injection

Biological: FX201

Mid Dose FX201

EXPERIMENTAL

Single mid dose FX201 injection

Biological: FX201

High Dose FX201

EXPERIMENTAL

Single high dose FX201 injection

Biological: FX201

Interventions

FX201 BIOLOGICAL

FX201, humantakinogene hadenovec, is a novel interleukin-1 receptor antagonist (IL-1Ra) gene therapy for IA administration that is being developed for the treatment of patients with OAK.

High Dose FX201; Low Dose FX201; Mid Dose FX201

Eligibility Criteria

• Age: 30 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written consent to participate in the study
• Male or female 30 to 80 years of age, inclusive, on the day of enrollment (Day 1)
• Body mass index (BMI) ≤ 40 kilograms per meters squared (kg/m2)
• Clinical diagnosis of OA of the knee on the American College of Rheumatology criteria (Altman, 1986) with radiologic evidence of OA (K-L Grade 2 or 3 for SAD Phase Cohort A and K-L Grade 2, 3 or 4 for SAD Phase Cohorts B and C and the Expansion Phase) at the index joint at the screening visit
• Moderate pain in the index joint
• Failed two or more types of conservative therapy for index knee osteoarthritis (e.g., structured exercise programs, topical or oral non-steroidal anti-inflammatory therapies); or failed one prior type of conservative therapy and at least one prior index knee IA treatment (corticosteroid or hyaluronic acid)
• Sexually active females and males agree to use highly effective methods of contraception

You may not qualify if:

• Reactive arthritis, rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, or arthritis associated with inflammatory bowel disease
• History of or current infection in the index knee joint
• Inability to undergo Magnetic Resonance Imaging (MRI) due to presence of surgical hardware or other foreign body in the index knee
• Unstable index knee joint (e.g., torn anterior cruciate ligament, etc.) within 12 months of Screening
• Any IA drug/biologic use within 6 months of Screening or 5 half-lives (whichever is longer) (e.g., IA corticosteroid, IA hyaluronic acid, platelet rich plasma injection, stem cells, prolotherapy and amniotic fluid injection, etc.)
• Cold or radiofrequency nerve ablation of the index knee within 12 months of Screening
• Arthroscopic or open surgery on the index knee within 12 months of Screening or planned/anticipated surgery on the index knee for the study period
• Anticipated major surgery during the study period
• ECG abnormality at Screening or Baseline judged clinically significant
• Use of immunomodulators, immunosuppressive, or chemotherapeutic agents within 5 years of Screening
• Use of any other investigational drug, biologic or device within 3 months of Screening
• Any systemic or local bacterial or viral infection requiring IV antibiotics or antivirals within 4 weeks of Screening or oral antibiotics or antivirals within 2 weeks of Screening
• Known allergy or sensitivity to methylprednisolone
• Any other clinically significant acute or chronic medical conditions (e.g., bleeding disorder) that, in the judgment of the Investigator, would preclude the use of an IA injection or that could compromise patient safety, limit the patient's ability to complete the study, and/or compromise the objectives of the study

Sponsors & Collaborators

• Pacira Pharmaceuticals, Inc: lead

Study Sites (6)

Arizona Research Center

Phoenix, Arizona, 85053, United States

Location

Gulfcoast Research Institute, LLC

Sarasota, Florida, 34232, United States

Location

Center for Pharmaceutical Research

Kansas City, Missouri, 64114, United States

Location

University Orthopedics Center

Altoona, Pennsylvania, 16602, United States

Location

Altoona Center for Clinical Research

Duncansville, Pennsylvania, 16635, United States

Location

Metroplex Clinical Research Center

Dallas, Texas, 75231, United States

Location

Related Publications (1)

• Mehta S, He T, Bajpayee AG. Recent advances in targeted drug delivery for treatment of osteoarthritis. Curr Opin Rheumatol. 2021 Jan;33(1):94-109. doi: 10.1097/BOR.0000000000000761.

  PMID: 33229973 DERIVED

MeSH Terms

Conditions

Osteoarthritis, Knee

Condition Hierarchy (Ancestors)

Osteoarthritis; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Study Officials

• Cecilia Barese, MD

  Pacira Pharmaceuticals, Inc

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: Open Label
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Part 1 - Open-Label, Single Ascending Dose Phase Part 2 - Open-Label, Expansion Phase to enroll additional patients in DMC-cleared dose levels by sequential assignment against a pre-defined randomized list

Study Record Dates

• First Submitted: October 3, 2019
• First Posted: October 8, 2019
• Study Start: March 2, 2020
• Primary Completion: November 28, 2023
• Study Completion (Estimated): November 28, 2026
• Last Updated: March 17, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)