Osteoarthritis, KneePhase 1/2NCT04875754
A Study Evaluating the Safety, Tolerability, and Range of Biologically Active Doses of ICM-203 in Mild to Moderate Knee Osteoarthritis
A Phase 1/2 study testing ICM-203 in people with Osteoarthritis, Knee, run by ICM Biotech Australia Pty Ltd. at 2 sites in Australia.
Trial 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… Read more about this study
- Who is it for?
- Ages 50 to 80 years
- Conditions the study is for
- Osteoarthritis, Knee
- What is being tested?
- ICM-203
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 2 sitesAustralia
- Study dates
- 17 Mar 2022to 20 Feb 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
- KOOS function in daily living score >25, a measure of knee function ranging from 0 (extreme problems) to 100 (no problems).
- A stable treatment regimen for symptomatic relief of OA, including NSAIDs, for 4 weeks prior to screening.
Who cannot join
- 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 (diabetes, uncontrolled hypertension, severe osteoporosis, glaucoma) or psychiatric condition that, in the opinion of the Investigator, would make the subject unsuitable for the study.
Trial locations and contacts
2 sites in 1 country.
Australia2 sites
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Barwon Health
Geelong, Victoria, 3220
About this study
Official title: A Phase 1/2a, Double-Blind, Placebo-Controlled Single Dose Escalation Study of Intra-Articular ICM 203 in Subjects With Kellgren-Lawrence Grade 2 or Grade 3 Osteoarthritis of the Knee
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
- Being tested
- ICM-203, Placebo
- Keywords
- adeno-associated virus, gene therapy, ICM-203, osteoarthritis
- Medical subject headings
- Osteoarthritis, Knee, Osteoarthritis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ICM-203Genetic
Intra-articular injection
PlaceboDrug
Intra-articular injection
| Study group | Role | What they receive |
|---|---|---|
| Group 1: ICM-203 (Low dose) vs Placebo | Experimental | 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 Receives: Genetic: ICM-203; Drug: Placebo |
| Group 2: ICM-203 (Medium dose) vs Placebo | Experimental | 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 Receives: Genetic: ICM-203; Drug: Placebo |
| Group 3: ICM-203 (High dose) vs Placebo (Optional) | Experimental | 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 Receives: Genetic: ICM-203; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-Emergent Adverse Events (TEAEs)
Incidence of Treatment-Emergent Adverse Events following administration of study drug
Measured: Up to Week 52
Severity of Treatment-Emergent Adverse Events (TEAEs)
Severity of Treatment-Emergent Adverse Events graded according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0, following administration of study drug
Measured: Up to Week 52
Secondary outcomes (8)
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)
Measured: Up to Week 52
Knee function
Evaluation of change from baseline in knee function as measured using the Function in Daily Living subscore of the Knee Injury and Osteoarthritis Outcome Score (KOOS)
Measured: Up to Week 52
Analgesic use
Evaluation of change from baseline in in use of analgesics, such as acetaminophen and NSAIDs
Measured: Up to Week 52
Magnetic Resonance Imaging Osteoarthritis Knee Score (MOAKS)
Evaluation of change from baseline in MOAKS, focusing on bone marrow lesions, articular cartilage, and effusion-synovitis
Measured: Up to Week 52
Joint space width
Evaluation of change from baseline in Joint space width in mm as measured on knee radiograph
Measured: Up to Week 52
Humoral response to AAV5.2 capsid
Evaluation of change from baseline in neutralizing antibody titers against AAV5.2 in serum
Measured: Up to Week 52
Cellular immune response to AAV5.2 capsid
Evaluation of change from baseline in T-cell responses to AAV5.2 capsid
Measured: Up to Week 52
Systemic biodistribution of ICM-203
Evaluation of presence of ICM-203 in peripheral blood after administration of study drug
Measured: Up to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2021
- Study start17 Mar 2022
- Primary completion20 Feb 2026
- Study completion20 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Osteoarthritis, KneeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- * Group 1: ICM-203 6x10e12 vg or Placebo * Group 2: ICM-203 2x10e13 vg or Placebo * Group 3: ICM-203 6x10e13 vg or Placebo (Optional)
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- ICM Biotech Australia Pty Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Alison Heald, MD, Study director, ICM Co. Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04875754
- Sponsor's protocol ID
- ICM 20-1001
- Status last verified
- Sep 2026
- First submitted
- 26 Apr 2021
- Last updated
- 3 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2a, Double-Blind, Placebo-Controlled Single Dose Escalation Study of Intra-Articular ICM 203 in Subjects With Kellgren-Lawrence Grade 2 or Grade 3 Osteoarthritis of the Knee
- ClinicalTrials.gov ID
- NCT04875754
- Acronym / sponsor ID
- ICM 20-1001
- Conditions
- Osteoarthritis, Knee
- Keywords
- adeno-associated virus, gene therapy, ICM-203, osteoarthritis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- ICM Biotech Australia Pty Ltd.Industry (pharma, biotech, devices)
- Study start
- 17 Mar 2022 (actual)
- Primary completion
- 20 Feb 2026 (actual)
- Study completion
- 20 Feb 2026 (actual)
- First posted
- 6 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- * Group 1: ICM-203 6x10e12 vg or Placebo * Group 2: ICM-203 2x10e13 vg or Placebo * Group 3: ICM-203 6x10e13 vg or Placebo (Optional)
| Arm | Type | Receives |
|---|---|---|
| Group 1: ICM-203 (Low dose) vs Placebo | Experimental | Genetic: ICM-203, Drug: Placebo8 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 2: ICM-203 (Medium dose) vs Placebo | Experimental | Genetic: ICM-203, Drug: Placebo4 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 3: ICM-203 (High dose) vs Placebo (Optional) | Experimental | Genetic: ICM-203, Drug: Placebo4 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 |
Who may qualify
- Ages
- 50 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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).
- KOOS function in daily living score >25, a measure of knee function ranging from 0 (extreme problems) to 100 (no problems).
- A stable treatment regimen for symptomatic relief of OA, including NSAIDs, for 4 weeks prior to screening.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ICM-203Intra-articular injection | Genetic | Group 1: ICM-203 (Low dose) vs Placebo, Group 2: ICM-203 (Medium dose) vs Placebo, Group 3: ICM-203 (High dose) vs Placebo (Optional) |
| PlaceboIntra-articular injection | Drug | Group 1: ICM-203 (Low dose) vs Placebo, Group 2: ICM-203 (Medium dose) vs Placebo, Group 3: ICM-203 (High dose) vs Placebo (Optional) |
Primary outcome
Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to Week 52
Incidence of Treatment-Emergent Adverse Events following administration of study drug
Severity of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to Week 52
Severity of Treatment-Emergent Adverse Events graded according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0, following administration of study drug
8 secondary outcomes
Knee pain
Time frame: Up to Week 52
Knee function
Time frame: Up to Week 52
Analgesic use
Time frame: Up to Week 52
Magnetic Resonance Imaging Osteoarthritis Knee Score (MOAKS)
Time frame: Up to Week 52
Joint space width
Time frame: Up to Week 52
Humoral response to AAV5.2 capsid
Time frame: Up to Week 52
Cellular immune response to AAV5.2 capsid
Time frame: Up to Week 52
Systemic biodistribution of ICM-203
Time frame: Up to Week 52
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Osteoarthritis, Knee
Not yet recruiting
Opioid Reduction and Enhanced Recovery in Orthopaedic Surgery (OREOS)-Knee
Knee Arthroplasty, Total, Partial Knee Arthroplasty
Active, not recruiting
SakuraBead Used as Resorbable Embolic for Genicular Artery Embolization
Osteo Arthritis Knee, Joint Diseases
RecruitingPhase 2
Genicular Artery Embolization for Reducing Pain in Medically Refractory Symptomatic Knee Osteoarthritis
Knee Osteoarthritis, Knee Osteoarthritis (Knee OA)
Not yet recruiting
Comparison of High-Intensity and Conventional Physical Therapy in Musculoskeletal Disorders
Low Back Pain, Radicular Syndrome
Recruiting
The Effect of Different Exercise Training Programs on Muscle Oxygenation in Individuals With Knee Osteoarthritis
Osteoarthritis of Knee
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alison Heald, MD study director
ICM Co. Ltd.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ICM-203 in people with Osteoarthritis, Knee, run by ICM Biotech Australia Pty Ltd. at 2 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment-Emergent Adverse Events (TEAEs) (time frame: Up to Week 52). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2026.