A Study to Assess the Safety and Efficacy of a Single Dose of UBX0101 in Patients With Osteoarthritis of the Knee

• ClinicalTrials.gov Identifier: NCT04129944
• Recruitment Status: Completed
• First Posted: October 17, 2019
• Last Update Posted: September 3, 2020
• Sponsor: Unity Biotechnology, Inc.
• Information provided by (Responsible Party): Unity Biotechnology, Inc.

Tracking Information

• First Submitted Date: October 15, 2019
• First Posted Date: October 17, 2019
• Last Update Posted Date: September 3, 2020
• Actual Study Start Date: October 30, 2019
• Actual Primary Completion Date: May 20, 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 16, 2019)

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

• Original Primary Outcome Measures (submitted: October 15, 2019): Change from baseline to Week 12 of the WOMAC-A score in patients receiving a single dose of UBX0101 versus those receiving placebo [ Time Frame: Baseline to Week 12 ]

Current Secondary Outcome Measures (submitted: October 16, 2019)

• 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 ]
  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
• 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 ]
  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
• 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 AEs as well as change from baseline through Weeks 12 and 24 in vital signs and selected laboratory safety parameters (as deemed clinically appropriate) in patients receiving a single dose of UBX0101 versus those receiving placebo [ Time Frame: Baseline to Week 24 ]

Original Secondary Outcome Measures (submitted: October 15, 2019)

• Change from baseline to Week 12 of the WOMAC-C function subscale 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 intensity scores on the 11-point 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 AEs as well as change from baseline through Weeks 12 and 24 in vital signs and selected laboratory safety parameters (as deemed clinically appropriate) in patients receiving a single dose of UBX0101 versus those receiving placebo [ Time Frame: Baseline to Week 24 ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study to Assess the Safety and Efficacy of a Single Dose of UBX0101 in Patients With Osteoarthritis of the Knee
• Official Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Single-Dose Study of UBX0101 in Moderate to Severe, Painful Osteoarthritis of the Knee
• Brief 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).

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 Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment
• Condition: Osteoarthritis, Knee

Intervention

• Drug: UBX0101
  Investigational drug intra-articular injection
• Other: Placebo
  Placebo intra-articular injection

Study Arms

• Placebo Comparator: Placebo
  Intervention: Other: Placebo
• Experimental: UBX0101 0.5 mg
  Intervention: Drug: UBX0101
• Experimental: UBX0101 2.0 mg
  Intervention: Drug: UBX0101
• Experimental: UBX0101 4.0 mg
  Intervention: Drug: UBX0101

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: February 25, 2020): 183
• Original Estimated Enrollment (submitted: October 15, 2019): 180
• Actual Study Completion Date: August 7, 2020
• Actual Primary Completion Date: May 20, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

Key Inclusion Criteria:

• 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:

• 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.
• 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 40 Years to 85 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04129944
• Other Study ID Numbers: UBX0101-MUS-201
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Unity Biotechnology, Inc.
• Study Sponsor: Unity Biotechnology, Inc.
• Collaborators: Not Provided

Investigators

• Study Director: Medical Monitor; UNITY Biotechnology

• PRS Account: Unity Biotechnology, Inc.
• Verification Date: June 2020