Osteo Arthritis KneeEarly Phase 1EXO-OA01NCT06431152
Intra-articular Injection of UC-MSC Exosome in Knee Osteoarthritis
A Early Phase 1 study testing UC-MSC sEV in people with Osteo Arthritis Knee, run by Universidad de los Andes, Chile at 1 site in Chile.
Trial summary
The study aim to evaluate safety of exosomes (sEVs) from allogeneic mesenchymal stromal cells delivered by an intra-articular injection in the knee of patients with mild to moderate symptomatic osteoarthritis. The sEVs will be produced in a GMP-facility. Read more about this study
- Who is it for?
- Ages 30 to 70 years
- Conditions the study is for
- Osteo Arthritis Knee
- What is being tested?
- UC-MSC sEV
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChile
- Study dates
- 1 Jun 2024to 31 Dec 2025
- Phase
- Early 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 30 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 30 to 70 years.
- Kellgren-Lawrence grade II - III knee OA (Rosenberg view x-ray)
- VAS for pain ≥ 40 mm, without surgical indication in the affected knee.
- In case of bilateral involvement, the most affected knee will be treated. The contralateral knee should be asymptomatic or present a VAS ≤ 20 mm.
- Stable knee with normal physical examination.
- Signed Informed Consent
Who cannot join
- Symptomatic bilateral knee OA
- BMI > 30 kg/m2
- Joint instability at physical examination.
- Mechanical meniscal tear on physical examination.
- Associated conditions: active local or systemic infection, neoplasia, immunosuppression, pregnancy, anticoagulant therapy, coagulation disorders, inflammatory joint disease (autoimmune, by crystal or other), joint prosthesis, symptomatic spine or hip disease.
- Recent use of intra-articular (last 6 months) or oral (last month) steroid therapy.
- Recent use of intra-articular hyaluronic acid therapy (last 6 months)
- Subchondral bone fracture.
Trial locations and contacts
1 site in 1 country.
Chile1 site, 1 recruiting
- Recruiting
Clinica Universidad de los Andes
Santiago, Las Condes, 2501
Bernardita Hurtado, 226182071, bhurtadodeme@clinicauandes.cl
Consuelo Covarrubias, 226182071, ccovarrubias@uandes.cl
About this study
Official title: Administration of sEV Derived From UC-MSC in Patients With Osteoarthritis of the Knee: Safety Determination in a Pilot Dose-escalation Study
The study aim to evaluate safety of exosomes (sEVs) from allogeneic mesenchymal stromal cells delivered by an intra-articular injection in the knee of patients with mild to moderate symptomatic osteoarthritis. The sEVs will be produced in a GMP-facility. The investigators expect to enroll 12 patients in this phase 1 trial open label dose-escalation pilot and the follow-up will be up to 12 months.
Read the detailed description
The clinical investigation will represent a Phase 1 trial focusing on small extracellular vesicles derived from mesenchymal stem cell (MSC-sEV) in patients with symptomatic Kellgren II-III knee OA. The phase 1 component of the study will be an open-label dose escalation pilot study in which three cohorts of subjects with OA will receive increasing doses of UC-MSC-sEV administered as a single intra-articular (IA) injection. Each cohort will comprise four participants. Eligible study subjects will be enrolled at the "Clinica Universidad de los Andes".
The small extracellular vesicles derived from umbilical cord mesenchymal stem cell (UC-MSC-sEV) will be prepared in the "Clinica Universidad de los Andes" GMP facility. The sEV-based therapeutic for clinical use will be manufactured in compliance with standardized procedures based on Good Manufacture Practice (GMP) regulations and all quality controls aforementioned. The sEV therapeutic will be transported to the patient administration site under controlled conditions, ensuring maintenance of a temperature range between 2-8°C. The sEV injection is expected to be administered within the first 6 h of product manufacture.
The primary study endpoints of this trial will focus on the safety, feasibility, and toxicity of the sEV-based product. The phase I will examine (1) the incidence of immediate post-infiltration adverse reactions in patients; (2) the occurrence of synovitis post-infiltration in patients at 24 and 48 hours, as well as on days 7 and 15; (3) the frequency of post-infiltration pain reported by patients at 24 and 48 h, and on days 7 and 15; and (4) the prevalence of adverse events related to sEV therapy occurring beyond IA infiltration at 24 and 48 h, and on days 7 and 15, as well as at months 2, 4, 6, 8, 10, and 12. The secondary study endpoint will be determine the optimal dose for phase II trials. The criteria that will be considered are (1) Safety profile at infiltration at 24 and 48 h, and on days 7 and 15, as well as at months 2, 4, 6, 8, 10, and 12; (2) changes in WOMAC scores at months 2, 4, 6, 8, 10, and 12; and (3) alterations in the Visual Analog Scale (VAS) pain scores at months 2, 4, 6, 8, 10, and 12.
- Conditions
- Osteo Arthritis Knee
- Being tested
- UC-MSC sEV
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UC-MSC sEVBiological
Also called Cellistem®sEV-OA
Small extracellular vesicles derived from allogenic mesenchymal stromal cells, single dose
| Study group | Role | What they receive |
|---|---|---|
| Low-Dose | Experimental | Intra-articular knee injection of exosomes (2 x 10e9 particles/dose) derived from allogeneic mesenchymal stromal cells. Single dose.
4 patients Receives: Biological: UC-MSC sEV |
| Medial-Dose | Experimental | Intra-articular knee injection of exosomes (6 x 10e9 particles/dose) derived from allogeneic mesenchymal stromal cells. Single dose.
4 patients Receives: Biological: UC-MSC sEV |
| High-Dose | Experimental | Intra-articular knee injection of exosomes (2 x 10e10 particles/dose) derived from allogeneic mesenchymal stromal cells. Single dose.
4 patients Receives: Biological: UC-MSC sEV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Event
Occurrence of any adverse reactions within 12 months of treatment
Measured: 12 month
Secondary outcomes (5)
Incidence of injection-related pain according to Visual Analogue Scale (VAS) (0-100mm)
Pain measured by Visual Analogue Scale (VAS) (0-100mm) after first week of treatment
Measured: 1-2 weeks
Incidence of injection-related synovitis according to effusion grading scale of knee joint
Synovitis measured by effusion grading scale (zero to 3+) after first week of treatment
Measured: 1-2 weeks
Pain change
Change in Visual Analogue Scale (VAS) (0-100mm) score every 2 months
Measured: 12 months
Disability change
Change in WOMAC subscale (Western Ontario and McMaster Universities Osteoarthritis IndexWomac) related to function (C) every 2 months Pain (0-20), Stiffness (0-8), functional capacity (0-68).
Measured: 12 months
Percentage of responders
According to OMERACT-OARSI Criteria Index Response after 52 weeks
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2024
- Study start1 Jun 2024 (estimated)
- Primary completion30 Jun 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Osteo Arthritis 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Universidad de los Andes, Chile
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06431152
- Sponsor's protocol ID
- EXO-OA01
- Acronym
- EXO-OA01
- Status last verified
- May 2024
- First submitted
- 17 May 2024
- Last updated
- 29 May 2024
- Results posted
- No
- Data monitoring committee
- No
- 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 29 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Administration of sEV Derived From UC-MSC in Patients With Osteoarthritis of the Knee: Safety Determination in a Pilot Dose-escalation Study
- ClinicalTrials.gov ID
- NCT06431152
- Acronym / sponsor ID
- EXO-OA01 / EXO-OA01
- Conditions
- Osteo Arthritis Knee
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Universidad de los Andes, ChileUniversity, hospital or other
- Study start
- 1 Jun 2024 (estimated)
- Primary completion
- 30 Jun 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 28 May 2024 (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 study aim to evaluate safety of exosomes (sEVs) from allogeneic mesenchymal stromal cells delivered by an intra-articular injection in the knee of patients with mild to moderate symptomatic osteoarthritis. The sEVs will be produced in a GMP-facility. The investigators expect to enroll 12 patients in this phase 1 trial open label dose-escalation pilot and the follow-up will be up to 12 months.
Detailed description
The clinical investigation will represent a Phase 1 trial focusing on small extracellular vesicles derived from mesenchymal stem cell (MSC-sEV) in patients with symptomatic Kellgren II-III knee OA. The phase 1 component of the study will be an open-label dose escalation pilot study in which three cohorts of subjects with OA will receive increasing doses of UC-MSC-sEV administered as a single intra-articular (IA) injection. Each cohort will comprise four participants. Eligible study subjects will be enrolled at the "Clinica Universidad de los Andes".
The small extracellular vesicles derived from umbilical cord mesenchymal stem cell (UC-MSC-sEV) will be prepared in the "Clinica Universidad de los Andes" GMP facility. The sEV-based therapeutic for clinical use will be manufactured in compliance with standardized procedures based on Good Manufacture Practice (GMP) regulations and all quality controls aforementioned. The sEV therapeutic will be transported to the patient administration site under controlled conditions, ensuring maintenance of a temperature range between 2-8°C. The sEV injection is expected to be administered within the first 6 h of product manufacture.
The primary study endpoints of this trial will focus on the safety, feasibility, and toxicity of the sEV-based product. The phase I will examine (1) the incidence of immediate post-infiltration adverse reactions in patients; (2) the occurrence of synovitis post-infiltration in patients at 24 and 48 hours, as well as on days 7 and 15; (3) the frequency of post-infiltration pain reported by patients at 24 and 48 h, and on days 7 and 15; and (4) the prevalence of adverse events related to sEV therapy occurring beyond IA infiltration at 24 and 48 h, and on days 7 and 15, as well as at months 2, 4, 6, 8, 10, and 12. The secondary study endpoint will be determine the optimal dose for phase II trials. The criteria that will be considered are (1) Safety profile at infiltration at 24 and 48 h, and on days 7 and 15, as well as at months 2, 4, 6, 8, 10, and 12; (2) changes in WOMAC scores at months 2, 4, 6, 8, 10, and 12; and (3) alterations in the Visual Analog Scale (VAS) pain scores at months 2, 4, 6, 8, 10, and 12.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low-Dose | Experimental | Biological: UC-MSC sEVIntra-articular knee injection of exosomes (2 x 10e9 particles/dose) derived from allogeneic mesenchymal stromal cells. Single dose. 4 patients |
| Medial-Dose | Experimental | Biological: UC-MSC sEVIntra-articular knee injection of exosomes (6 x 10e9 particles/dose) derived from allogeneic mesenchymal stromal cells. Single dose. 4 patients |
| High-Dose | Experimental | Biological: UC-MSC sEVIntra-articular knee injection of exosomes (2 x 10e10 particles/dose) derived from allogeneic mesenchymal stromal cells. Single dose. 4 patients |
Who may qualify
- Ages
- 30 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age 30 to 70 years.
- Kellgren-Lawrence grade II - III knee OA (Rosenberg view x-ray)
- VAS for pain ≥ 40 mm, without surgical indication in the affected knee.
- In case of bilateral involvement, the most affected knee will be treated. The contralateral knee should be asymptomatic or present a VAS ≤ 20 mm.
- Stable knee with normal physical examination.
Key exclusion criteria (8 listed)
- Symptomatic bilateral knee OA
- BMI > 30 kg/m2
- Joint instability at physical examination.
- Mechanical meniscal tear on physical examination.
- Associated conditions: active local or systemic infection, neoplasia, immunosuppression, pregnancy, anticoagulant therapy, coagulation disorders, inflammatory joint disease (autoimmune, by crystal or other), joint prosthesis, symptomatic spine or hip disease.
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 |
|---|---|---|
| UC-MSC sEVAlso: Cellistem®sEV-OASmall extracellular vesicles derived from allogenic mesenchymal stromal cells, single dose | Biological | High-Dose, Low-Dose, Medial-Dose |
Primary outcome
Adverse Event
Time frame: 12 month
Occurrence of any adverse reactions within 12 months of treatment
5 secondary outcomes
Incidence of injection-related pain according to Visual Analogue Scale (VAS) (0-100mm)
Time frame: 1-2 weeks
Incidence of injection-related synovitis according to effusion grading scale of knee joint
Time frame: 1-2 weeks
Pain change
Time frame: 12 months
Disability change
Time frame: 12 months
Percentage of responders
Time frame: 52 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2024.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing UC-MSC sEV in people with Osteo Arthritis Knee, run by Universidad de los Andes, Chile at 1 site in Chile.
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 6 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 29 May 2024. 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 Chile. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jose Matas, MD (+56 2 26183347 ext. 3347, jmatas@clinicauandes.cl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Event (time frame: 12 month). 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 29 May 2024.