Osteoarthritis, KneePhase 2NCT04129944
A Study to Assess the Safety and Efficacy of a Single Dose of UBX0101 in Patients With Osteoarthritis of the Knee
A Phase 2 study testing UBX0101 in people with Osteoarthritis, Knee, run by Unity Biotechnology, Inc. at 20 sites in United States.
Trial summary
A study to assess efficacy, safety, and tolerability of a single-dose intra-articular administration of UBX0101 in patients with moderate to severe painful knee osteoarthritis (OA). Read more about this study
- Who is it for?
- Ages 40 to 85 years
- Conditions the study is for
- Osteoarthritis, Knee
- What is being tested?
- UBX0101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 183 participants
- Where
- 20 sitesUnited States
- Study dates
- 30 Oct 2019to 7 Aug 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 40 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients who are ambulatory with a diagnosis of OA of the knee and who have baseline pain with a mean of ≥ 4 and ≤ 9 on the 11-point (0-10) average daily pain NRS for at least five of seven days during the Screening period.
- Kellgren-Lawrence grade of 1-4 on a weight-bearing radiograph of target knee.
- Patients aged ≥ 40 and ≤ 85 years.
- Patients are permitted but not required to use an oral NSAID, serotonin and norepinephrine reuptake inhibitors (SNRIs), tramadol, or acetaminophen, provided that they have been taking a stable dose and regimen of medication for at least 4 weeks prior to Screening.
Who cannot join
- Patients with any condition, including laboratory or imaging findings and findings in the medical history or in the pre-study assessments, that in the opinion of the Investigator or the Medical Monitor constitutes a risk or contraindication for participation in the study or that could interfere with the study objectives, conduct, or evaluation or prevent the patient from fully participating in all aspects of the study.
- Patients with a body mass index (BMI) ≥40 kg/m2 or a body habitus that precludes the MRI.
- Patients with fibromyalgia
- Systemic autoimmune disease with musculoskeletal involvement or any history of a systemic inflammatory arthritis
- Patients who have received IA treatment in the target knee with steroids or hyaluronic acid derivatives within the last 16 weeks prior to Screening, or with extended-release corticosteroid (e.g., Zilretta®) within the last 20 weeks
- Patients who are using a topical NSAID or topical analgesics on the target knee.
Show all 14Show fewer
- Patients who have used opioid analgesics (other than tramadol), marijuana or marijuana-derived products (e.g., cannabidiol), and topical capsaicin on the target knee within 8 weeks prior to Screening
- Patients with a history of traumatic knee injury to the target knee, including, but not limited to, patients with meniscal root tear, within 2 years of study entry.
- Patients who have undergone diagnostic arthroscopy to the target knee in the previous 6 months.
- Patients who have undergone arthroscopic surgery (including microfracture and meniscectomy) on the target knee in the last 2 years prior to the Screening visit or are anticipated to have arthroscopic surgery on either knee at any time during the study period.
- Patients with a history of previous total or partial knee arthroplasty.
- Patients with an effusion at the Screening visit, which, in the opinion of the Investigator following examination and discussions with the patient, requires drainage for symptom relief.
- Patients who have had regenerative joint procedures on any joint, including, but not limited to, platelet-rich plasma injections, stem cell transplantation, autologous chondrocyte transplantation, or mosaicplasty.
- Patients with secondary arthritis that involves the target knee or would confound assessments of knee OA
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
United States20 sites
Albuquerque Clinical Trials
Albuquerque, New Mexico, 87102
Hassman Research Institute
Berlin, New Jersey, 08009
Central Research Associates
Birmingham, Alabama, 35205
Chicago Clinical Research Institute
Chicago, Illinois, 60607
Altoona Center for Clinical Research
Duncansville, Pennsylvania, 16635
Drug Trials America
Hartsdale, New York, 10530
The Alliance for Multispecialty Research
Kansas City, Missouri, 64114
Biosolutions Clinical Research Center
La Mesa, California, 91942
Charter Research
Lady Lake, Florida, 32159
Alliance for Multispecialty Research-Lexington
Lexington, Kentucky, 40509
Well-Pharma Medical Research
Miami, Florida, 33143
Coastal Clinical Research, LLC.
Mobile, Alabama, 36608
Coastal Carolina Research Center
North Charleston, South Carolina, 29406
Rochester Clinical Research
Rochester, New York, 14609
Precision Clinical Research
Sunrise, Florida, 33351
Fiel Family and Sports Medicine
Tempe, Arizona, 85283
Premier Medical Associates
The Villages, Florida, 32159
Tucson Orthopaedic Institute
Tucson, Arizona, 85712
Diablo Clinical Research
Walnut Creek, California, 94598
The Alliance for Multispecialty Research
Wichita, Kansas, 67207
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Single-Dose Study of UBX0101 in Moderate to Severe, Painful Osteoarthritis of the Knee
A study to assess efficacy, safety, and tolerability of a single-dose intra-articular administration of UBX0101 in patients with moderate to severe painful knee osteoarthritis (OA).
Read the detailed description
This is a randomized, double-blind, placebo-controlled, single-dose, parallel-group study to assess the efficacy, safety, and tolerability of a single-dose intra-articular (IA) administration of UBX0101 in patients with moderate to severe painful knee osteoarthritis (OA).
Approximately 180 patients will be randomized (1:1:1:1) to one of four treatment groups (three dose levels of UBX0101 and Placebo; approximately 45 patients per group), all administered by IA route at Week 0. The four treatment groups will be enrolled concurrently.
The primary objective of the study is to evaluate the effect of IA administration of UBX0101 on the change from baseline to Week 12 of pain in the target knee.
- Conditions
- Osteoarthritis, Knee
- Being tested
- UBX0101, Placebo
- Keywords
- Osteoarthritis, Painful Osteoarthritis, Osteoarthritis, Knee, Senescence
- 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.
UBX0101Drug
Investigational drug intra-articular injection
PlaceboOther
Placebo intra-articular injection
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Other: Placebo |
| UBX0101 0.5 mg | Experimental | Not described Receives: Drug: UBX0101 |
| UBX0101 2.0 mg | Experimental | Not described Receives: Drug: UBX0101 |
| UBX0101 4.0 mg | Experimental | Not described Receives: Drug: UBX0101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to Week 12 of the Western Ontario and McMaster Universities Osteoarthritis Index Pain Subscale (WOMAC-A) Score in Patients Receiving a Single Dose of UBX0101 Versus Those Receiving Placebo
WOMAC-A is assessed by a Likert scale on a range from 0 (none) to 4 (extreme), with higher scores indicating higher levels of pain
Measured: Baseline to Week 12
Secondary outcomes (4)
Change From Baseline to Week 12 of the Western Ontario and McMaster Universities Osteoarthritis Index Function Subscale (WOMAC-C) Score in Patients Receiving a Single Dose of UBX0101 Versus Those Receiving Placebo
WOMAC-C is assessed by a Likert scale on a range from 0 (none) to 4 (extreme), with higher scores indicating higher levels of physical disability
Measured: Baseline to Week 12
Change From Baseline to Week 12 of the Weekly Mean of the Average Daily Pain (ADP) Intensity Scores on the 11-point Numeric Rating Scale (NRS) in Patients Receiving a Single Dose of UBX0101 Versus Those Receiving Placebo
ADP is assessed by NRS on a range from 0 (no pain) to 10 (worst pain imaginable), with higher scores indicating higher levels of pain
Measured: Baseline to Week 12
Change From Baseline (Over the Entire 24-week Period, Including Both the Primary Study Period and the 12-week Follow-up Period) to Week 24 for the WOMAC-A, NRS, and WOMAC-C Scores in Patients Receiving a Dose of UBX0101 Versus Those Receiving Placebo
WOMAC-A is assessed by a Likert scale on a range from 0 (none) to 4 (extreme), with higher scores indicating higher levels of pain WOMAC-C is assessed by a Likert scale on a range from 0 (none) to 4 (extreme), with higher scores indicating higher levels of physical disability.
Average Daily Pain (ADP) is assessed by Numerical Rating Score (NRS) on a range from 0 (no pain) to 10 (worst pain imaginable), with higher scores indicating higher levels of pain.
Measured: Baseline to Week 24
Incidence of Treatment Emergent Adverse Events (TEAEs)
Measured: Baseline to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2019
- Study start30 Oct 2019
- Primary completion20 May 2020
- Study completion7 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 183 participants (actual)
Sponsor and investigators
- Lead sponsor
- Unity Biotechnology, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, UNITY Biotechnology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04129944
- Sponsor's protocol ID
- UBX0101-MUS-201
- Status last verified
- Nov 2021
- First submitted
- 15 Oct 2019
- Last updated
- 29 Dec 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Hannani MT, Frederiksen P, Karsdal MA, Bager CL, Bihlet AR, Tunblad K, Oberg F, Collins JE, Kraus VB, Hunter DJ, Bacardit J, Bay-Jensen AC, Thudium CS. Therapeutic subtypes of knee osteoarthritis: differential treatment effects among predicted endotypes in past clinical trials. Arthritis Res Ther. 2026 May 7;28(1):133. doi: 10.1186/s13075-026-03825-7.
PubMed 42092979Diekman BO, Loeser RF. DNA Damage and Cellular Senescence in Osteoarthritis: An Unexpected Role for Interferon Regulatory Factor 1 in Chondrocyte DNA Repair. Arthritis Rheumatol. 2024 Jun;76(6):842-844. doi: 10.1002/art.42822. Epub 2024 Feb 27. No abstract available.
PubMed 38343350
Study at a glance
From the registry record, last updated by the sponsor on 29 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Single-Dose Study of UBX0101 in Moderate to Severe, Painful Osteoarthritis of the Knee
- ClinicalTrials.gov ID
- NCT04129944
- Acronym / sponsor ID
- UBX0101-MUS-201
- Conditions
- Osteoarthritis, Knee
- Keywords
- Osteoarthritis, Painful Osteoarthritis, Osteoarthritis, Knee, Senescence
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 183 participants (actual)
- Lead sponsor
- Unity Biotechnology, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Oct 2019 (actual)
- Primary completion
- 20 May 2020 (actual)
- Study completion
- 7 Aug 2020 (actual)
- First posted
- 17 Oct 2019 (actual)
- Results
- Posted 29 Dec 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study to assess efficacy, safety, and tolerability of a single-dose intra-articular administration of UBX0101 in patients with moderate to severe painful knee osteoarthritis (OA).
Detailed description
This is a randomized, double-blind, placebo-controlled, single-dose, parallel-group study to assess the efficacy, safety, and tolerability of a single-dose intra-articular (IA) administration of UBX0101 in patients with moderate to severe painful knee osteoarthritis (OA).
Approximately 180 patients will be randomized (1:1:1:1) to one of four treatment groups (three dose levels of UBX0101 and Placebo; approximately 45 patients per group), all administered by IA route at Week 0. The four treatment groups will be enrolled concurrently.
The primary objective of the study is to evaluate the effect of IA administration of UBX0101 on the change from baseline to Week 12 of pain in the target knee.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Other: Placebo |
| UBX0101 0.5 mg | Experimental | Drug: UBX0101 |
| UBX0101 2.0 mg | Experimental | Drug: UBX0101 |
| UBX0101 4.0 mg | Experimental | Drug: UBX0101 |
Who may qualify
- Ages
- 40 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients who are ambulatory with a diagnosis of OA of the knee and who have baseline pain with a mean of ≥ 4 and ≤ 9 on the 11-point (0-10) average daily pain NRS for at least five of seven days during the Screening period.
- Kellgren-Lawrence grade of 1-4 on a weight-bearing radiograph of target knee.
- Patients aged ≥ 40 and ≤ 85 years.
- Patients are permitted but not required to use an oral NSAID, serotonin and norepinephrine reuptake inhibitors (SNRIs), tramadol, or acetaminophen, provided that they have been taking a stable dose and regimen of medication for at least 4 weeks prior to Screening.
Key exclusion criteria (14 listed)
- Patients with any condition, including laboratory or imaging findings and findings in the medical history or in the pre-study assessments, that in the opinion of the Investigator or the Medical Monitor constitutes a risk or contraindication for participation in the study or that could interfere with the study objectives, conduct, or evaluation or prevent the patient from fully participating in all aspects of the study.
- Patients with a body mass index (BMI) ≥40 kg/m2 or a body habitus that precludes the MRI.
- Patients with fibromyalgia
- Systemic autoimmune disease with musculoskeletal involvement or any history of a systemic inflammatory arthritis
- Patients who have received IA treatment in the target knee with steroids or hyaluronic acid derivatives within the last 16 weeks prior to Screening, or with extended-release corticosteroid (e.g., Zilretta®) within the last 20 weeks
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 |
|---|---|---|
| UBX0101Investigational drug intra-articular injection | Drug | UBX0101 0.5 mg, UBX0101 2.0 mg, UBX0101 4.0 mg |
| PlaceboPlacebo intra-articular injection | Other | Placebo |
Primary outcome
Change From Baseline to Week 12 of the Western Ontario and McMaster Universities Osteoarthritis Index Pain Subscale (WOMAC-A) Score in Patients Receiving a Single Dose of UBX0101 Versus Those Receiving Placebo
Time frame: Baseline to Week 12
WOMAC-A is assessed by a Likert scale on a range from 0 (none) to 4 (extreme), with higher scores indicating higher levels of pain
4 secondary outcomes
Change From Baseline to Week 12 of the Western Ontario and McMaster Universities Osteoarthritis Index Function Subscale (WOMAC-C) Score in Patients Receiving a Single Dose of UBX0101 Versus Those Receiving Placebo
Time frame: Baseline to Week 12
Change From Baseline to Week 12 of the Weekly Mean of the Average Daily Pain (ADP) Intensity Scores on the 11-point Numeric Rating Scale (NRS) in Patients Receiving a Single Dose of UBX0101 Versus Those Receiving Placebo
Time frame: Baseline to Week 12
Change From Baseline (Over the Entire 24-week Period, Including Both the Primary Study Period and the 12-week Follow-up Period) to Week 24 for the WOMAC-A, NRS, and WOMAC-C Scores in Patients Receiving a Dose of UBX0101 Versus Those Receiving Placebo
Time frame: Baseline to Week 24
Incidence of Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline to Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Dec 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42092979Hannani MT, Frederiksen P, Karsdal MA, Bager CL, Bihlet AR, Tunblad K, Oberg F, Collins JE, Kraus VB, Hunter DJ, Bacardit J, Bay-Jensen AC, Thudium CS. Therapeutic subtypes of knee osteoarthritis: differential treatment effects among predicted endotypes in past clinical trials. Arthritis Res Ther. 2026 May 7;28(1):133. doi: 10.1186/s13075-026-03825-7.
- Derived from this study: PubMed 38343350Diekman BO, Loeser RF. DNA Damage and Cellular Senescence in Osteoarthritis: An Unexpected Role for Interferon Regulatory Factor 1 in Chondrocyte DNA Repair. Arthritis Rheumatol. 2024 Jun;76(6):842-844. doi: 10.1002/art.42822. Epub 2024 Feb 27. No abstract available.
- 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.
Medical Monitor study director
UNITY Biotechnology
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing UBX0101 in people with Osteoarthritis, Knee, run by Unity Biotechnology, Inc. at 20 sites 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 4 inclusion and 14 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 29 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites 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. Some participants in this study may receive a placebo. 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 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?
Yes. Results are posted in the registry (first posted 29 Dec 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline to Week 12 of the Western Ontario and McMaster Universities Osteoarthritis Index Pain Subscale (WOMAC-A) Score in Patients Receiving a Single Dose of UBX0101 Versus Those Receiving Placebo (time frame: Baseline to Week 12). 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 NCT04129944, which also lists 2 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 29 Dec 2021.