A Study Evaluating the Safety, Tolerability, and Activity of ICM-203 in Subjects With Knee Osteoarthritis.

NCT05454566 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: ICM 20-1003
• Study Type: interventional
• Target: 18
• Locations: 0 countries
• Sites: N/A

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 into the knee of subjects with mild to moderate knee osteoarthritis (OA). Approximately 6 to 18 subjects will be enrolled into 3 successive dose-escalating groups in a 3+3 study design, whereby 3 study subjects in each group will be dosed sequentially with ICM-203 and 3 additional subjects will be dosed at the same dose level if a dose limiting toxicity (DLT) occurs in any of the first 3 subjects.

Conditions

Osteoarthritis, Knee

Keywords

adeno-associated virus; gene therapy; ICM-203; osteoarthritis

Outcome Measures

Primary Outcomes (6)

• Treatment-Emergent Adverse Events (TEAEs)

  Incidence and Severity of Treatment-Emergent Adverse Events following administration of study drug

  Up to Week 52

• Knee pain

  Evaluation of change from baseline in knee pain as measured using a Numerical Rating Scale (NRS) ranging from 0 (no pain) to 10 (worst pain imaginable)

  Up to Week 52

• Knee function

  Evaluation of change from baseline in knee function as measured using the Knee Injury and Osteoarthritis Outcome Score (KOOS) function in daily living subscale ranging from 0 (extreme problems) to 100 (no problems)

  Up to Week 52

• Analgesic use

  Evaluation of change from baseline in in use of analgesics, such as acetaminophen and NSAIDs

  Up to Week 52

• Magnetic Resonance Imaging Osteoarthritis Knee Score (MOAKS)

  Evaluation of change from baseline in MOAKS, focusing on articular cartilage and effusion-synovitis

  Up to Week 52

• Joint space width

  Evaluation of change from baseline in Joint space width in mm as measured on knee radiograph

  Up to Week 52

Secondary Outcomes (3)

• Humoral response to AAV5.2 capsid

  Up to Week 52

• Cellular immune response to AAV5.2 capsid

  Up to Week 52

• Systemic biodistribution of ICM-203

  Up to Week 52

Study Arms (3)

Group 1: ICM-203 (Low dose)

EXPERIMENTAL

3 to 6 subjects will receive a single intra-articular injection of ICM-203 at 6x10e12 vg into the target knee at Day 1

Genetic: ICM-203

Group 2: ICM-203 (Medium dose)

EXPERIMENTAL

3 to 6 subjects will receive a single intra-articular injection of ICM-203 at 2x10e13 vg into the target knee at Day 1

Genetic: ICM-203

Group 3: ICM-203 (High dose)

EXPERIMENTAL

3 to 6 subjects will receive a single intra-articular injection of ICM-203 at 6x10e13 vg into the target knee at Day 1

Genetic: ICM-203

Interventions

ICM-203 GENETIC

Intra-articular injection

Group 1: ICM-203 (Low dose); Group 2: ICM-203 (Medium dose); Group 3: ICM-203 (High dose)

Eligibility Criteria

• Age: 50 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Body mass index (BMI) of 18.0 to 40.0 kg/m2, inclusive, at the time of screening.
• Kellgren-Lawrence grade 2 or grade 3 OA of target knee.
• Target knee pain between 4 and 9, inclusive, on an NRS ranging from 0 (no pain) to 10 (worst pain imaginable), at the time of screening.
• KOOS function in daily living score \>25, a measure of knee function ranging from 0 (extreme problems) to 100 (no problems), at the time of screening .
• A stable treatment regimen for symptomatic relief of OA, including NSAIDs, for 4 weeks prior to screening.

You may not qualify if:

• 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.
• Injection of steroid, hyaluronate or other agent, into the target knee less than 90 days prior to day 1.
• Major injury to the target knee, such as torn ligament or severe sprain, within 12 months of screening.
• Disability so severe that the subject cannot comply with the study requirements, including knee symptoms that result in significant difficulty or inability to walk.
• Surgery on the target knee within 180 days prior to day 1.
• Total knee arthroplasty or other knee surgery planned in the next 12 months.
• Active joint infection or other concurrent medical or psychiatric condition that, in the opinion of the Investigator, would make the subject unsuitable for the study.

Sponsors & Collaborators

• ICM Co. Ltd.: lead

MeSH Terms

Conditions

Osteoarthritis, Knee; Osteoarthritis

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Study Officials

• Alison Heald, MD

  ICM Co. Ltd.

  STUDY DIRECTOR

Central Study Contacts

Alison Heald, MD

CONTACT

+1 206 465 3912; alison.heald@icm-bio.com

Youngna Yum, PhD

CONTACT

+82 2 324 8534; youngna.yum@icm-bio.com

Study Design

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

Model Details: * Group 1: ICM-203 6x10e12 vg * Group 2: ICM-203 2x10e13 vg * Group 3: ICM-203 6x10e13 vg

Study Record Dates

• First Submitted: July 4, 2022
• First Posted: July 12, 2022
• Study Start (Estimated): November 15, 2026
• Primary Completion (Estimated): May 15, 2027
• Study Completion (Estimated): November 15, 2028
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share