One-Shot gene therapy could ease knee arthritis pain for years

NCT ID NCT06884865

Symptom relief Sponsor: Pacira Pharmaceuticals, Inc Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 26, 2026 · Updated 4 times

Summary

This Phase 2 trial tests a single injection of a gene therapy called PCRX-201 in 135 adults aged 45–80 with painful knee osteoarthritis. Participants receive one of two doses or a placebo, then are monitored for safety and pain relief over 5 years. The goal is to see if the injection is safe and can reduce knee pain and improve joint function.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Enekinragene inzadenovec (PCRX-201) injection
What this could lead to: If successful, this could lead to a new, long-lasting treatment option for knee osteoarthritis pain that may reduce the need for frequent injections or surgery.
What could go wrong: This is an early Phase 2 trial with only 135 participants, so results may not apply to everyone. The treatment is a gene therapy, and long-term safety and effectiveness are still unknown.

Why investors are watching

Pacira is testing PCRX-201, a single knee injection for osteoarthritis pain, in a small phase 2 trial with 135 patients. For a small company, this readout matters because it will show whether the treatment is safe and whether either dose reduces knee pain and improves function over a year. A clear signal could validate the drug's potential and support further development.

If it works: If the trial shows PCRX-201 is safe and meaningfully eases knee pain, Pacira could advance the drug toward later-stage testing. That would give the company a new product candidate in a large patient population.

If it fails: Phase 2 trials often fail to show benefit, and a negative or unclear result could set back the program and raise doubts about the drug's future. Delays in enrollment or follow-up could also slow the timeline.

AI-written from the trial record. Speculative, and not investment advice.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 135 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2025

Expected to finish

Jul 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

45 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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 received 2 or more 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 (Part A only) For Part B, participants must have active synovitis in the index knee as determined by contrast-enhanced MRI. * Subjects must exhibit American College of Rheumatology Criteria (clinical and radiological) for osteoarthritis as follows: 1. Knee pain 2. 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 (Part A) or K-L Grade 2 or 3 (Part B) in the index knee based on X-rays performed during Screening and confirmed by trained radiographers at a central facility before enrollment * Subjects need to show the presence of moderate or severe synovitis based on 11-point synovitis score using contrast-enhanced MRI (Part A). For Part B, participants need to show the presence of synovitis ≥5 on the Guermazi synovitis scoring scale using contrast-enhanced MRI to be enrolled. Exclusion Criteria: * 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 with X-ray or MRI exclusionary events * 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 * For Part B, subjects with any surgery related tot he index knee. Other protocol-defined criteria apply

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Conditions

The condition(s) this trial relates to.

osteoarthritis osteoarthritis, knee

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Osteoarthritis (OA) of the knee

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

27 sites. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

AARA Clinical Research - Arizona Arthritis & Rheumatology Associates - Phoenix
NOT_YET_RECRUITING

Phoenix, Arizona, 85032, United States
Accurate Clinical Research, Inc - Houston
NOT_YET_RECRUITING

Houston, Texas, 77034, United States
Accurate Clinical Research, Inc. - Lake Charles
NOT_YET_RECRUITING

Lake Charles, Louisiana, 70605, United States
Advanced Clinical Research Center, LLC dba TriWest Research Associates, LLC
NOT_YET_RECRUITING

Chula Vista, California, 91910, United States
Alabama Clinical Therapeutics, LLC
WITHDRAWN

Birmingham, Alabama, 35235, United States
Alliance Clinical West Hills (Focus Clinical Research)
WITHDRAWN

West Hills, California, 91307, United States
Altoona Center for Clinical Research
ACTIVE_NOT_RECRUITING

Duncansville, Pennsylvania, 16635, United States
Arrow Clinical Trials
RECRUITING

Daytona Beach, Florida, 32117, United States
Beach Physicians Medical Group Inc.
RECRUITING

Huntington Beach, California, 92647, United States
Best Clinical Trials, LLC
NOT_YET_RECRUITING

New Orleans, Louisiana, 70115, United States
Campbell Clinic
NOT_YET_RECRUITING

Collierville, Tennessee, 38017, United States
Clinical Trials of South Carolina - Charleston
ACTIVE_NOT_RECRUITING

Charleston, South Carolina, 29406, United States
Clinical Trials of South Carolina - Columbia
ACTIVE_NOT_RECRUITING

Columbia, South Carolina, 29206, United States
Conquest Research
ACTIVE_NOT_RECRUITING

Winter Park, Florida, 32789, United States
Discovery Health Research Center - Plantation, FL
NOT_YET_RECRUITING

Plantation, Florida, 33317, United States
Epic Clinical Research
ACTIVE_NOT_RECRUITING

Lewisville, Texas, 75057, United States
Excel Clinical Research
ACTIVE_NOT_RECRUITING

Las Vegas, Nevada, 89109, United States
Flourish Research - Birmingham
NOT_YET_RECRUITING

Birmingham, Alabama, 35205, United States
Gulfcoast Research Institute
NOT_YET_RECRUITING

Sarasota, Florida, 34232, United States
Helios CR, Inc.
NOT_YET_RECRUITING

Dallas, Texas, 75231, United States
Horizon Clinical Research
ACTIVE_NOT_RECRUITING

La Mesa, California, 91942, United States
Journey Research Inc.
ACTIVE_NOT_RECRUITING

Oldsmar, Florida, 34677-4681, United States
LifeBridge Health
NOT_YET_RECRUITING

Baltimore, Maryland, 21215, United States
M3 Wake Research - Wilmington, NC
NOT_YET_RECRUITING

Wilmington, North Carolina, 28412, United States
META Medical Research Institute
NOT_YET_RECRUITING

Dayton, Ohio, 45432, United States
Medvin Clinical Research - Covina
NOT_YET_RECRUITING

Covina, California, 91722, United States
Medvin Clinical Research - Whittier
NOT_YET_RECRUITING

Whittier, California, 90602, United States
Michigan Orthopaedic and Spine Surgeons
NOT_YET_RECRUITING

Rochester Hills, Michigan, 48307, United States
NY Scientific
NOT_YET_RECRUITING

Brooklyn, New York, 11235-3919, United States
New York University (NYU) Center for Musculoskeletal Care
NOT_YET_RECRUITING

New York, New York, 10016, United States
OrthoArizona
NOT_YET_RECRUITING

Phoenix, Arizona, 85018, United States
Osteoporosis Medical Center
ACTIVE_NOT_RECRUITING

Beverly Hills, California, 90211, United States
Palm Beach Research Center
ACTIVE_NOT_RECRUITING

West Palm Beach, Florida, 33409, United States
Physicians Research Collaboration
RECRUITING

Lincoln, Nebraska, 68516, United States
Pinnacle Trials, Inc.
NOT_YET_RECRUITING

Atlanta, Georgia, 30329, United States
Rush Medical College - Division of Rheumatology
NOT_YET_RECRUITING

Chicago, Illinois, 60612, United States
Source Healthcare
NOT_YET_RECRUITING

Santa Monica, California, 90403, United States
University Orthopedics Center
NOT_YET_RECRUITING

Altoona, Pennsylvania, 16602-4472, United States
West Clinical Research
NOT_YET_RECRUITING

Morehead City, North Carolina, 28557, United States
Zenos Clinical Research
ACTIVE_NOT_RECRUITING

Dallas, Texas, 75230, United States

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