A Study Evaluating the Safety, Tolerability, and Range of Biologically Active Doses of ICM-203 in Mild to Moderate Knee Osteoarthritis

NCT ID: NCT04875754

Last Updated: 2025-05-01

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-03-17
• Study Completion Date: 2026-12-31

Brief Summary

The purpose of this study is to determine the safety, tolerability, and activity of ICM-203, a recombinant adeno-associated viral (AAV) vector that expresses a therapeutic gene that promotes cartilage formation, reduces joint inflammation and pain, as well as improves joint physical function, by injecting escalating doses of ICM-203 or matching placebo into the knee of subjects with mild to moderate knee osteoarthritis (OA).

Conditions

Osteoarthritis, Knee

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Group 1: ICM-203 (Low dose) vs Placebo

8 subjects will be randomized to receive a single intra-articular injection of ICM-203 at 6x10e12 vg (n=6) or placebo (n=2) into the target knee at Day 1

Group Type: EXPERIMENTAL

ICM-203

Intervention Type: GENETIC

Intra-articular injection

Placebo

Intervention Type: DRUG

Intra-articular injection

Group 2: ICM-203 (Medium dose) vs Placebo

4 subjects will be randomized to receive a single intra-articular injection of ICM-203 at 2x10e13 vg (n=3) or placebo (n=1) into the target knee at Day 1

Group Type: EXPERIMENTAL

ICM-203

Intervention Type: GENETIC

Intra-articular injection

Placebo

Intervention Type: DRUG

Intra-articular injection

Group 3: ICM-203 (High dose) vs Placebo (Optional)

4 subjects will be randomized to receive a single intra-articular injection of ICM-203 at 6x10e13 vg (n=3) or placebo (n=1) into the target knee at Day 1

Group Type: EXPERIMENTAL

ICM-203

Intervention Type: GENETIC

Intra-articular injection

Placebo

Intervention Type: DRUG

Intra-articular injection

Interventions

ICM-203

Intra-articular injection

Intervention Type: GENETIC

Placebo

Intra-articular injection

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Body Mass Index (BMI) of 18.0 to 40.0 kg/m2, inclusive, at the time of screening.

2. Kellgren-Lawrence grade 2 or grade 3 OA of target knee.

3. Target knee pain between 4 and 9, inclusive, on an NRS ranging from 0 (no pain) to 10 (worst pain imaginable).

4. KOOS function in daily living score >25, a measure of knee function ranging from 0 (extreme problems) to 100 (no problems).

5. A stable treatment regimen for symptomatic relief of OA, including NSAIDs, for 4 weeks prior to screening.

Exclusion Criteria

1. History of rheumatoid arthritis, psoriatic arthritis, gout, pseudogout, autoimmune OA, chondrocalcinosis, hemochromatosis, villonodular synovitis, and synovial chondromatosis or other disorder that in the opinion of the Investigator could cause inflammation of the knee.

2. Injection of steroid, hyaluronate or other agent, into the target knee less than 90 days prior to day 1.

3. Major injury to the target knee, such as torn ligament or severe sprain, within 12 months of screening.

4. Disability so severe that the subject cannot comply with the study requirements, including knee symptoms that result in significant difficulty or inability to walk.

5. Surgery on the target knee within 180 days prior to day 1

6. Total knee arthroplasty or other knee surgery planned in the next 12 months.

7. Active joint infection or other concurrent medical (diabetes, uncontrolled hypertension, severe osteoporosis, glaucoma) or psychiatric condition that, in the opinion of the Investigator, would make the subject unsuitable for the study.

• Minimum Eligible Age: 50 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ICM Biotech Australia Pty Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Alison Heald, MD

Role: STUDY_DIRECTOR

ICM Co. Ltd.

Locations

Royal Adelaide Hospital

Adelaide, South Australia, Australia

Barwon Health

Geelong, Victoria, Australia

Countries

Australia

Other Identifiers

ICM 20-1001

Identifier Type: -

Identifier Source: org_study_id