Efficacy and Safety of GNSC-001 Intra-articular Injection for Knee Osteoarthritis (RAPHAEL)

September 15, 2026 updated by: Genascence Corporation

A Randomized, Double-blind, Placebo-controlled, Phase 2/3, Study to Evaluate the Efficacy and Safety of an Intraarticular Injection of GNSC-001 in Participants With Osteoarthritis of the Knee

The goal of this clinical trial is to learn if GNSC-001 works to treat knee osteoarthritis in adults. It will also learn about the safety of GNSC-001. The main questions it aims to answer are:

  1. Does GNSC-001 lower daily knee joint pain and improve function in participants?
  2. What medical problems do participants have when taking GNSC-001?

Researchers will compare GNSC-001 to a placebo (a look-alike substance that contains no drug) to see if GNSC-001 works to treat knee osteoarthritis.

Participants will:

  1. Receive a single injection of immune-modulation, and GNSC-001 or a placebo, in their knee
  2. Take an oral immune-modulating pill every day for the first 22 days
  3. Visit the clinic at least 9 times over 2 years for checkups and tests
  4. Keep a diary of their symptoms and the number of times they use a rescue medication

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

240

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

40 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key Inclusion Criteria:

  • OA in the target knee confirmed by radiograph and with a Kellgren-Lawrence (KL) score of 2 or 3
  • OA symptoms affecting the target knee present for at least 6 months
  • NRS pain score 5-9 (0-10 scale) in the target knee within the 24 hours prior to the time of consent
  • Synovitis and/or effusion detectable on imaging
  • Willing to stop use of any medication taken for knee OA pain for the duration of the study, including but not limited to nonsteroidal anti-inflammatory drugs (NSAIDs)
  • Able to provide written informed consent and comply with the requirements of the study

Key Exclusion Criteria:

  • Body mass index (BMI) > 38 kg/m2
  • NRS pain score in other (non-target) knee ≥ 4
  • Large effusion, substantial malalignment, structural compromise, prior partial or total joint replacement of target knee
  • Surgery on the target knee within the 6 months prior to screening or the anticipated need for treatment of either knee prior to the Month 12 Visit.
  • Current or history of inflammatory conditions (e.g., gout or rheumatoid arthritis), other joint disorders, septic arthritis, nerve damage or injury, chronic pain not associated with knee OA (e.g., fibromyalgia, hip OA), or otherwise immunocompromised
  • Active infection
  • Uncontrolled diabetes mellitus, glycosylated hemoglobin (HbA1c) >7.0% at screening
  • Initiation of GLP-1RA, or similar medication, within the last 12 months, or discontinued within last 6 months prior to Screening
  • Use of opioid medications (including tramadol or tapentadol) or medical/recreational cannabinoids (oral/inhaled) for > 5 days within the last 30 days
  • Use of medications for the treatment of chronic pain, immunosuppressant medications, or intra-articular injections into the target knee within 90 days of Day 1
  • Current use of anticoagulant therapy
  • Received live vaccine within 6 weeks of Day 1
  • Participation in another interventional clinical study with experimental therapy within 90 days prior to screening
  • Relies on a walking aids (e.g., cane, crutch, walker, wheelchair) or a personal assistant
  • Pregnant, breastfeeding, or intending to become pregnant during the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo

Single intra-articular injection into target knee
Experimental: GNSC-001
Drug: Gene Therapy GNSC-001

Single intra-articular injection into target knee

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in OA pain as assessed by the Weekly Average of Daily Pain (WADP) Numeric Rating Scale (NRS) at Week 52
Time Frame: Baseline and Week 52
Baseline and Week 52
Change from baseline in the function subscale of the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) at Week 52
Time Frame: Baseline and Week 52
Baseline and Week 52
Incidence and severity of treatment emergent adverse events (TEAEs), events of clinical interest (ECIs), and serious adverse events (SAEs)
Time Frame: From enrollment to the end of study at 104 weeks
From enrollment to the end of study at 104 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genascence Corporation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

July 1, 2027

Primary Completion (Estimated)

October 1, 2029

Study Completion (Estimated)

October 1, 2030

Study Registration Dates

First Submitted

September 15, 2026

First Submitted That Met QC Criteria

September 15, 2026

First Posted (Actual)

September 21, 2026

Study Record Updates

Last Update Posted (Actual)

September 21, 2026

Last Update Submitted That Met QC Criteria

September 15, 2026

Last Verified

September 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CPRO-25-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe