Osteoarthritis, KneePhase 1AAVIL-1RaNCT02790723
Safety of Intra-Articular Sc-rAAV2.5IL-1Ra in Subjects With Moderate Knee OA
A Phase 1 study testing sc-rAAV2.5IL-1Ra in people with Osteoarthritis, Knee, run by Mayo Clinic at 1 site in United States.
Trial summary
This study seeks to determine the safety of three different doses of sc-rAAV2.5IL-1Ra delivered by intra-articular injection into one knee joint of patients with moderate OA of the knee. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Osteoarthritis, Knee
- What is being tested?
- sc-rAAV2.5IL-1Ra
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Jun 2019to 17 Mar 2022
- Phase
- Phase 1
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18-80 years
- Gender: both males and females
- Target disease: Moderate OA of the knee
- Persistent symptoms, despite standard NSAID
- Absence of clinically significant abnormal values for the following:
- Complete blood count
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- Prothrombin Time, Activated partial thromboplastin time
- Blood chemistry (Glucose, Na, K, Cl, CO2, BUN, creatinine, Ca, PO4, magnesium, uric acid)
- Liver function tests (amylase, total bilirubin, alkaline phosphate, GGT, AST, ALT, total protein, albumin)
- Able and willing to return to the Mayo Clinic for follow-up visits, as required by this study
- Able undergo MRI of the knee
- Subjects should be able to give appropriate consent.
- Potential subjects should have failed a three-month trial of a minimum of two conservative therapies before being considered for this trial. These conservative therapies include: activity modification, weight loss, physical therapy, and anti-inflammatory or injection therapy.
Who cannot join
- Pregnant, or currently breast-feeding
- Ongoing infectious disease including HIV, HTLV, hepatitis, syphilis or tuberculosis positive
- Individuals who have OA as part of another syndrome (e.g. Ehler's Danlos, Stickler syndrome, etc.)
- Systemic, rheumatic or inflammatory disease of the knee or chondrocalcinosis, hemochromatosis, inflammatory arthritis, arthropathy of the knee associated with juxta-articular Paget's disease of the femur or tibia, ochronosis, hemophilic arthropathy, infectious arthritis, Charcot's knee joint, villonodular synovitis, and synovial chondromatosis.
- Clinically significant cardiovascular, renal, hepatic, endocrine disease, diabetes, cancer, autoimmune diseases; a serious infection or major operation within 30 days of enrollment; a history of untreated psychiatric disease or recent history of alcoholism or drug addiction.
- Currently taking immunosuppressant medications
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- Anticipated major surgery during the study period.
- Individuals involved in another interventional protocol, or have been treated under one within the last 3 months.
- Intra-articular therapy in the index knee within the previous 3 months.
- Surgery to the target knee within 6 months prior to screening.
- Surgery to other weight bearing joints if it will interfere with knee assessments
- Prior articular transplant procedures
- Orthopedic hardware or implantable devices in the index knee
- Prior reconstruction surgery to the target knee within 12 months
- X-ray findings of acute fractures
- Known severe loss of bone density, and/or severe bone or joint deformity in the target knee.
- Significant target knee infection or overlying skin disorder/infection within the previous 6 months prior to study enrollment
- Require cane or other assistive device for walking
- Symptomatic OA of the hips, spine or ankle if it would interfere with the evaluation of the target knee,
- History of documented nerve damage in the affected limb, or vascular insufficiency,
- Condition requiring use of systemic steroids,
- Coagulation disorder.
- Patients with unstable knees
- Temperature above 99.5o F.
- Identification as a member of a vulnerable population.
- BMI greater than 40.
- History of allergy to local anesthetics
- Currently taking anti-rheumatic disease medication (including methotrexate or other antimetabolites) within the 3 months prior to entry in the study.
- Any illness or condition which, in the investigators' judgement will interfere with the patient's ability to comply with the protocol, compromise patient safety, or interfere with the interpretation of the study results
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I Study Evaluating the Safety of Intra-Articular Sc-rAAV2.5IL-1Ra in Subjects With Moderate Osteoarthritis of the Knee
This study seeks to determine the safety of three different doses of sc-rAAV2.5IL-1Ra delivered by intra-articular injection into one knee joint of patients with moderate OA of the knee.
- Conditions
- Osteoarthritis, Knee
- Being tested
- sc-rAAV2.5IL-1Ra
- Medical subject headings
- Osteoarthritis, Knee
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sc-rAAV2.5IL-1RaDrug
Also called AAVIL-1Ra, IL-1Ra
Self-complementing, recombinant AAV carrying IL-1Ra cDNA produced by the Research Institute at Nationwide Children's Hospital.
| Study group | Role | What they receive |
|---|---|---|
| Low Dose Group | Experimental | Subjects will receive a single intra-articular 2mL injection consisting of 10{11} viral genomes. Receives: Drug: sc-rAAV2.5IL-1Ra |
| Medium Dose Group | Experimental | Subjects will receive a single intra-articular 2mL injection consisting of 10{12} viral genomes. Receives: Drug: sc-rAAV2.5IL-1Ra |
| High Dose Group | Experimental | Subjects will receive a single intra-articular 2mL injection consisting of 10{13} viral genomes. Receives: Drug: sc-rAAV2.5IL-1Ra |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects experiencing severe adverse events.
Assess the local and systemic safety of three different doses of sc-rAAV2.5IL-1Ra delivered by intra-articular injection into one knee joint of patients with moderate OA of the knee.
Measured: 53 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2016 (estimated)
- Study start5 Jun 2019
- Primary completion17 Mar 2022
- Study completion17 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Osteoarthritis, KneeStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Christopher H. Evans, Ph.D., Mayo Clinic
- Investigators
- Christopher H. Evans, Ph.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02790723
- Sponsor's protocol ID
- 15-007542
- Acronym
- AAVIL-1Ra
- Status last verified
- Apr 2022
- First submitted
- 3 May 2016
- Last updated
- 15 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Evans CH, Ghivizzani SC, Robbins PD. Osteoarthritis Gene Therapy: A 30-Year Journey From Concept to Clinical Trials. J Orthop Res. 2026 Feb;44(2):e70141. doi: 10.1002/jor.70141.
PubMed 41607103Evans CH, Ghivizzani SC, Robbins PD. The 2024 OREF Clinical Research Award: Progress Toward a Gene Therapy for Arthritis. J Am Acad Orthop Surg. 2024 Dec 1;32(23):1052-1060. doi: 10.5435/JAAOS-D-24-00831. Epub 2024 Oct 17.
PubMed 392840302022 AMSSM Rising With Research Presentations. Clin J Sport Med. 2022 Mar 1;32(2):168-176. doi: 10.1097/JSM.0000000000001014. No abstract available.
PubMed 352347442021 AMSSM Oral Research Poster Presentations. Clin J Sport Med. 2021 Mar 1;31(2):185-221. doi: 10.1097/JSM.0000000000000914. No abstract available.
PubMed 33647918Mehta S, He T, Bajpayee AG. Recent advances in targeted drug delivery for treatment of osteoarthritis. Curr Opin Rheumatol. 2021 Jan;33(1):94-109. doi: 10.1097/BOR.0000000000000761.
PubMed 33229973
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study Evaluating the Safety of Intra-Articular Sc-rAAV2.5IL-1Ra in Subjects With Moderate Osteoarthritis of the Knee
- ClinicalTrials.gov ID
- NCT02790723
- Acronym / sponsor ID
- AAVIL-1Ra / 15-007542
- Conditions
- Osteoarthritis, Knee
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 5 Jun 2019 (actual)
- Primary completion
- 17 Mar 2022 (actual)
- Study completion
- 17 Mar 2022 (actual)
- First posted
- 6 Jun 2016 (estimated)
- 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.
This study seeks to determine the safety of three different doses of sc-rAAV2.5IL-1Ra delivered by intra-articular injection into one knee joint of patients with moderate OA of the knee.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low Dose Group | Experimental | Drug: sc-rAAV2.5IL-1RaSubjects will receive a single intra-articular 2mL injection consisting of 10{11} viral genomes. |
| Medium Dose Group | Experimental | Drug: sc-rAAV2.5IL-1RaSubjects will receive a single intra-articular 2mL injection consisting of 10{12} viral genomes. |
| High Dose Group | Experimental | Drug: sc-rAAV2.5IL-1RaSubjects will receive a single intra-articular 2mL injection consisting of 10{13} viral genomes. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Age 18-80 years
- Gender: both males and females
- Target disease: Moderate OA of the knee
- Persistent symptoms, despite standard NSAID
- Absence of clinically significant abnormal values for the following:
Key exclusion criteria (29 listed)
- Pregnant, or currently breast-feeding
- Ongoing infectious disease including HIV, HTLV, hepatitis, syphilis or tuberculosis positive
- Individuals who have OA as part of another syndrome (e.g. Ehler's Danlos, Stickler syndrome, etc.)
- Systemic, rheumatic or inflammatory disease of the knee or chondrocalcinosis, hemochromatosis, inflammatory arthritis, arthropathy of the knee associated with juxta-articular Paget's disease of the femur or tibia, ochronosis, hemophilic arthropathy, infectious arthritis, Charcot's knee joint, villonodular synovitis, and synovial chondromatosis.
- Clinically significant cardiovascular, renal, hepatic, endocrine disease, diabetes, cancer, autoimmune diseases; a serious infection or major operation within 30 days of enrollment; a history of untreated psychiatric disease or recent history of alcoholism or drug addiction.
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 |
|---|---|---|
| sc-rAAV2.5IL-1RaAlso: AAVIL-1Ra, IL-1RaSelf-complementing, recombinant AAV carrying IL-1Ra cDNA produced by the Research Institute at Nationwide Children's Hospital. | Drug | High Dose Group, Low Dose Group, Medium Dose Group |
Primary outcome
Number of subjects experiencing severe adverse events.
Time frame: 53 weeks
Assess the local and systemic safety of three different doses of sc-rAAV2.5IL-1Ra delivered by intra-articular injection into one knee joint of patients with moderate OA of the knee.
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 15 Apr 2022.
- Derived from this study: PubMed 41607103Evans CH, Ghivizzani SC, Robbins PD. Osteoarthritis Gene Therapy: A 30-Year Journey From Concept to Clinical Trials. J Orthop Res. 2026 Feb;44(2):e70141. doi: 10.1002/jor.70141.
- Derived from this study: PubMed 39284030Evans CH, Ghivizzani SC, Robbins PD. The 2024 OREF Clinical Research Award: Progress Toward a Gene Therapy for Arthritis. J Am Acad Orthop Surg. 2024 Dec 1;32(23):1052-1060. doi: 10.5435/JAAOS-D-24-00831. Epub 2024 Oct 17.
- Derived from this study: PubMed 352347442022 AMSSM Rising With Research Presentations. Clin J Sport Med. 2022 Mar 1;32(2):168-176. doi: 10.1097/JSM.0000000000001014. No abstract available.
- Derived from this study: PubMed 336479182021 AMSSM Oral Research Poster Presentations. Clin J Sport Med. 2021 Mar 1;31(2):185-221. doi: 10.1097/JSM.0000000000000914. No abstract available.
- Derived from this study: PubMed 33229973Mehta S, He T, Bajpayee AG. Recent advances in targeted drug delivery for treatment of osteoarthritis. Curr Opin Rheumatol. 2021 Jan;33(1):94-109. doi: 10.1097/BOR.0000000000000761.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Christopher H. Evans, Ph.D. principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing sc-rAAV2.5IL-1Ra in people with Osteoarthritis, Knee, run by Mayo Clinic at 1 site in United States.
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 13 inclusion and 29 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 15 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 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.
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?
Number of subjects experiencing severe adverse events. (time frame: 53 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02790723, which also lists 5 linked publications.
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 15 Apr 2022; AzTrials last refreshed its copy on 8 Nov 2026.