A Study to Assess Safety and Tolerability of PCRX-201 in Subjects With Painful Osteoarthritis of the Knee

NCT06884865 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 201-C-201
• Study Type: interventional
• Target: 135
• Locations: 1 country
• Sites: 17

Brief Summary

The goal of this clinical trial is to learn if a single knee injection of different doses (Dose A and Dose B) of an investigation product PCRX-201, following steroid pretreatment, is safe and tolerable in male of female patients 45 to 80 years old with osteoarthritis of the knee. It will also learn how the body reacts to the PCRX-201 injection and how effective the treatment is. Researchers will compare Dose A and Dose B of PCRX-201 versus placebo from Week 1 through Week 52 to assess adverse events. Researchers will also explore how PCRX-201 may impact knee pain and joint function over time. Participants will be asked to

• Visit the clinic for pretreatment and drug administration
• Visit the clinic for checkups and tests:
• Screening through Week 52: intially weekly, bi-weekly, later monthly
• Week 53 through Week 260 (Year 5): 8 visits throughout the period

Conditions

Osteoarthritis (OA) of the Knee

Keywords

gene therapy; Knee Pain Arthritis; Osteoarthritis of the Knee

Outcome Measures

Primary Outcomes (3)

• Incidence (number and percent) of treatment-emergent adverse events (TEAEs) for PCRX-201 Dose A and Dose B versus placebo from Week 1 through Week 52

  Week 1 through Week 52.

• Incidence (number and percent) of adverse events of special interest (AESIs) for PCRX-201 Dose A and Dose B versus placebo from Week 1 through Week 52

  Week 1 through Week 52.

• Incidence (number and percent) of serious adverse events (SAEs) for PCRX-201 Dose A and Dose B versus placebo from Week 1 through Week 52

  Week 1 through Week 52.

Secondary Outcomes (8)

• Real-time quantitative polymerase chain reaction (qPCR) measurement profile in plasma of PCRX-201 Dose A and Dose B from postdose through Week 8

  Days 1, 4, Weeks 2, 4, 8

• Real-time qPCR measurement profile of skin swab samples from Baseline through Week 4.

  Days 1, 4, Weeks 2, 4

• Assessment of the levels of neutralizing antibodies (NAbs), high-sensitivity C-reactive protein (hs-CRP), and biomarkers of inflammation (eg, interleukin [IL]- 1, cytokines [tumor necrosis factor alpha (TNFα), IL-1β, IL-6, IL-10], and chemokines [IL-8])

  Days 1, 4, Weeks 2, 3, 4,12, 16, 24, 38

• Absolute change from baseline in Average Daily Knee Pain (Numerical Rating Scale [NRS]) for PCRX-201 Dose A and Dose B versus placebo.

  Weeks 38 and 52

• Absolute change from baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Pain Score for PCRX-201 Dose A and Dose B versus placebo.

  Weeks 38 and 52

Study Arms (6)

Part A Dose A

EXPERIMENTAL

Single Intra-Articular injection

Biological: Enekinragene Inzadenovec (PCRX-201)

Part A, Placebo

PLACEBO COMPARATOR

Single Intra-Articular injection

Biological: Placebo

Part B Dose A

EXPERIMENTAL

Single Intra-Articular injection

Biological: Enekinragene Inzadenovec (PCRX-201)

Part B Dose B

EXPERIMENTAL

Single Intra-Articular injection

Biological: Enekinragene Inzadenovec (PCRX-201)

Part B Placebo

PLACEBO COMPARATOR

Part B Placebo

Biological: Placebo

Part A, Dose B

EXPERIMENTAL

Single Intra-Articular injection

Biological: Enekinragene Inzadenovec (PCRX-201)

Interventions

Enekinragene Inzadenovec (PCRX-201) BIOLOGICAL

Enekinragene Inzadenovec (PCRX-201) injection

Part A Dose A; Part A, Dose B; Part B Dose A; Part B Dose B

Placebo BIOLOGICAL

Placebo injection

Part A, Placebo; Part B Placebo

Eligibility Criteria

• Age: 45 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects must be willing and able to comply with the study procedures and visit schedule and able to follow verbal and written instructions.
• Subjects must be male or female and 45 to 80 years old, inclusive, at Screening.
• Subjects must exhibit symptoms associated with osteoarthritis of the index knee for ≥12 months before Screening (subject self-reporting is acceptable).
• Subjects must have index knee pain for \>15 days over the last month before Screening (subject self-reporting is acceptable).
• Subjects must have failed 2 or more therapies: Restricted physical activity as per Osteoarthritis Research Society International (OARSI) core level recommendation, ie, "structured land-based exercise" (this may include physical therapy), and failure of an additional type of conservative therapy for OA of the index knee, eg, nonselective NSAIDs or COX-2 inhibitors, in the past 12 months.
• Subjects must have a body mass index (BMI) ≤40 kg/m2 at Screening.
• Subjects must have an index knee examination indicating the index knee and the intended injection site area are free of any signs of local or joint infection at Baseline.
• Subjects must have an index knee Average Daily Knee Pain (NRS) between ≥5.0 and ≤9.0 at Screening and Baseline.
• Sexually active subjects of child-bearing potential (SOCBP) and their partners must agree to use effective birth control while in the study
• Subjects must have active synovitis in the index knee as determined by ultrasound Doppler.
• Subjects must exhibit American College of Rheumatology Criteria (clinical and radiological) for osteoarthritis as follows:
• Knee pain
• At least 1 of the following:
• i. Age \>50 years ii. Morning stiffness \<30 minutes iii. Crepitus on knee motion c. Osteophytes
• Subjects must have K-L Grade 2, 3, or 4 in the index knee based on X-rays performed during Screening and confirmed by trained radiographers at a central facility before enrollment

You may not qualify if:

• Subjects have any current or prior diagnosis of autoimmune connective tissue disorders, secondary OA conditions, benign synovial tumors, gout/pseudogout, reactive arthritis, RA, psoriatic arthritis, ankylosing spondylitis, or arthritis associated with inflammatory bowel disease.
• Subjects have any active systemic or local infection, including infection of the index knee
• Subjects are unable to undergo MRI with contrast MRI
• Subjects have an unstable index knee joint (eg, torn anterior cruciate ligament) within 12 months of Screening
• Subjects have used any approved or investigational IA drug/biologic in index knee within 6 months of Screening (eg, hyaluronic acid, platelet rich plasma, stem cells, prolotherapy, and amniotic fluid injection)
• Subjects are receiving or have received any gene therapy treatment (eg, IL-1Ra) in the past 3 years
• Subjects have used IA steroids ≤3 months before screening
• Other protocol-defined criteria apply

Sponsors & Collaborators

• Pacira Pharmaceuticals, Inc: lead

Study Sites (17)

Alabama Clinical Therapeutics, LLC

Birmingham, Alabama, 35235, United States

Location

Osteoporosis Medical Center

Beverly Hills, California, 90211, United States

Location

Beach Physicians Medical Group Inc.

Huntington Beach, California, 92647, United States

Location

Horizon Clinical Research

La Mesa, California, 91942, United States

Location

Alliance Clinical West Hills (Focus Clinical Research)

West Hills, California, 91307, United States

Location

Arrow Clinical Trials

Daytona Beach, Florida, 32117, United States

Location

Journey Research Inc.

Oldsmar, Florida, 34677-4681, United States

Location

Palm Beach Research Center

West Palm Beach, Florida, 33409, United States

Location

Conquest Research

Winter Park, Florida, 32789, United States

Location

Physicians Research Collaboration

Lincoln, Nebraska, 68516, United States

Location

Excel Clinical Research

Las Vegas, Nevada, 89109, United States

Location

NY Scientific

Brooklyn, New York, 11235-3919, United States

Location

Altoona Center for Clinical Research

Duncansville, Pennsylvania, 16635, United States

Location

Clinical Trials of South Carolina - Charleston

Charleston, South Carolina, 29406, United States

Location

Clinical Trials of South Carolina - Columbia

Columbia, South Carolina, 29206, United States

Location

Zenos Clinical Research

Dallas, Texas, 75230, United States

Location

Epic Clinical Research

Lewisville, Texas, 75057, United States

Location

MeSH Terms

Conditions

Osteoarthritis; Osteoarthritis, Knee

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Study Officials

• Warrren Rizzo, MD

  Pacira Pharmaceuticals, Inc

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: In Part A (manufacturing process 1), subjects are randomized in a 1:1:1 ratio to Dose A , Dose B , or placebo, each with a predose IA steroid of methylprednisolone acetate. In Part B (manufacturing process 2), subjects are randomized in a 1:1:1 ratio to Dose A, Dose B, or placebo, each with a pre-dose IA steroid of methylprednisolone acetate.

Study Record Dates

• First Submitted: March 13, 2025
• First Posted: March 19, 2025
• Study Start: February 26, 2025
• Primary Completion (Estimated): July 1, 2032
• Study Completion (Estimated): July 1, 2032
• Last Updated: March 24, 2026

Record last verified: 2026-03

Locations

US (17)