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

ICM Co. Ltd.

Status and phase: Begins enrollment in 4 months; Phase 2; Phase 1

Conditions: Osteoarthritis, Knee

Treatments: Genetic: ICM-203

Study type: Interventional

Funder types: Industry

Identifiers

NCT05454566

ICM 20-1003

Details and patient eligibility

About

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.

Enrollment: 18 estimated patients

Sex: All

Ages: 50 to 80 years old

Volunteers: No Healthy Volunteers

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), at the time of screening.
4. 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 .
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 or psychiatric condition that, in the opinion of the Investigator, would make the subject unsuitable for the study.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

---

18 participants in 3 patient groups

Group 1: ICM-203 (Low dose)

Experimental group

Description:

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

Treatment:

Genetic: ICM-203

Group 2: ICM-203 (Medium dose)

Experimental group

Description:

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

Treatment:

Genetic: ICM-203

Group 3: ICM-203 (High dose)

Experimental group

Description:

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

Treatment:

Genetic: ICM-203