Efficacy and Safety of XT-150 in Osteoarthritis of the Knee

NCT04124042 | Completed Phase 2 Results FDA Drug

• 1 other identifier: XT-150-2-0204
• Study Type: interventional
• Target: 289
• Locations: 2 countries
• Sites: 6

Brief Summary

This is a Phase 2 safety and efficacy study of XT-150 in adult participants experiencing moderate to severe pain due to osteoarthritis of the knee.

Conditions

Osteoarthritis, Knee

Keywords

Inflammation; Pain; Interleukin; Gene Therapy; plasmid DNA

Outcome Measures

Primary Outcomes (4)

• Stage A: Number of Participants Achieving 30% Improvement From Baseline in Western Ontario and McMasters Arthritis Index (WOMAC) Pain Score

  The Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) pain score will be obtained from the Knee injury and Osteoarthritis Outcome Score (KOOS) questionnaire which is a validated, commonly used instrument to assess the participant's opinion about their knee and associated problems. Each item is answered on a 5-point Likert scale. The score for pain category ranges from 0 (no pain) to 20 (maximum pain); higher score indicates worse outcomes. Baseline is defined as the Day 0 value.

  Day 180

• Stage A: Change From Baseline in WOMAC Pain Score at Day 180

  The Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) pain score will be obtained from the Knee injury and Osteoarthritis Outcome Score (KOOS) questionnaire which is a validated, commonly used instrument to assess the participant's opinion about their knee and associated problems. Each item is answered on a 5-point Likert scale. The score for pain category ranges from 0 (no pain) to 20 (maximum pain); higher score indicates worse outcomes. Baseline is defined as the Day 0 value.

  Day 180

• Stage A: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Adverse events were collected from the time of informed consent through the last study visit on Day 360 (1 year). Treatment Emergent Adverse Events (TEAEs) occurred from the time of study drug treatment on Day 0 through end of study (Day 360) or early termination. This analysis reports any AEs/SAEs that occurred prior to the second dose.

  Prior to second dose (Up to Day 180-Day 330)

• Stage B: Number of Participants With AEs and SAEs

  Adverse events were collected from the time of informed consent through the last study visit on Day 360 (1 year). Treatment Emergent Adverse Events occurred from the time of study drug treatment on Day 0 through end of study (Day 360) or early termination. This analysis reports any AEs/SAEs that occurred after the second dose.

  Post Second Dose (Day 180-Day 330 through Day 360)

Secondary Outcomes (5)

• Stage B: Change From Baseline in WOMAC Pain Score at Day 360

  Day 360

• Stage B: Change From Baseline in WOMAC Function Score

  At Day 360

• Stage A: Change From Baseline in Brief Pain Inventory (BPI) of Interference Score

  Day 180

• Stage A: Change From Baseline in Patients Overall Assessment (POA)

  Day 180

• Stage A and B: Number of Participants With Presence of Anti-interleukin (IL)-10 Antibody

  Up to Day 360

Study Arms (6)

Stage A: 0.15 mg/mL XT-150, Stage B: 0.15 mg/mL XT-150

EXPERIMENTAL

Low dose active in Stage A and Stage B

Biological: XT-150

Stage A: 0.15 mg/mL XT-150, Stage B: 0.45 mg/mL XT-150

EXPERIMENTAL

Low dose active in Stage A, high dose active in Stage B

Biological: XT-150

Stage A: 0.45 mg/mL XT-150, Stage B: 0.15 mg/mL XT-150

EXPERIMENTAL

High dose active in Stage A, low dose active in Stage B

Biological: XT-150

Stage A: 0.45 mg/mL XT-150, Stage B: 0.45 mg/mL XT-150

EXPERIMENTAL

High dose active in Stage A and Stage B

Biological: XT-150

Stage A: Placebo, Stage B: 0.15 mg/mL XT-150

PLACEBO COMPARATOR

Inactive comparator in Stage A, low dose active in Stage B

Biological: XT-150; Drug: Placebo

Stage A: Placebo, Stage B: 0.45 mg/mL XT-150

PLACEBO COMPARATOR

Inactive comparator in Stage A, high dose active in Stage B

Biological: XT-150; Drug: Placebo

Interventions

XT-150 BIOLOGICAL

plasmid DNA

Stage A: 0.15 mg/mL XT-150, Stage B: 0.15 mg/mL XT-150; Stage A: 0.15 mg/mL XT-150, Stage B: 0.45 mg/mL XT-150; Stage A: 0.45 mg/mL XT-150, Stage B: 0.15 mg/mL XT-150; Stage A: 0.45 mg/mL XT-150, Stage B: 0.45 mg/mL XT-150; Stage A: Placebo, Stage B: 0.15 mg/mL XT-150; Stage A: Placebo, Stage B: 0.45 mg/mL XT-150

Placebo DRUG

Placebo is a sterile phosphate-buffered saline

Stage A: Placebo, Stage B: 0.15 mg/mL XT-150; Stage A: Placebo, Stage B: 0.45 mg/mL XT-150

Eligibility Criteria

• Age: 45 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Symptomatic disease due to osteoarthritis, defined as a WOMAC Pain score ≥ 8 (worst possible = 20)
• Focused Analgesia Selection Test will be used to determine whether patients can report pain with sufficient consistency to enter the clinical trial
• Males and females between 45 and 85 years of age, inclusive
• Kellgren-Lawrence grading of 2 or 3 within the last 6 months
• Stable analgesic regimen during the 4 weeks prior to enrollment
• In the judgment of the Investigator, acceptable general medical condition
• Life expectancy \>6 months
• Male and female participants who are heterosexually active and not surgically sterile must agree to use effective contraception, including abstinence, for the duration of the study
• Have suitable knee joint anatomy for intra-articular injection
• Willing and able to return for the follow-up (FU) visits
• Able to read and understand study instructions, and willing and able to comply with all study procedures

You may not qualify if:

• Hypersensitivity, allergy, or significant reaction to any ingredient of the study drug, including double-stranded DNA, mannose, and sucrose
• Previously received XT-150 injection(s)
• Scheduled partial or complete knee replacement within 6 months; participant agrees not to schedule a knee replacement during Stage A of the study
• History of knee arthroplasty on the Index Knee, i.e., selected for study injection(s)
• History of rheumatoid arthritis or other inflammatory disease
• History of immunosuppressive therapy; systemic steroids in the last 3 months
• Received knee injection with hyaluronic acid or stem-cells in the last 6 months
• Knee injection of glucocorticoid in the last 3 months
• Current treatment with systemic immunosuppressive (systemic corticosteroid therapy or other strong immunosuppressant)
• Currently receiving systemic chemotherapy or radiation therapy for malignancy
• Clinically significant hepatic disease as indicated by clinical laboratory results ≥3 times the upper limit of normal for any liver function test (e.g., aspartate aminotransferase, alanine aminotransferase)
• Severe anemia (Grade 3; hemoglobin \<8.0 g/dL, \<4.9 mmol/L, \<80 g/L; transfusion indicated), Grade 1 white cell counts (lymphocytes \<LLN - 800/mm\^3; \<LLN - 0.8 x 109 /L, neutrophils \<LLN - 1500/mm\^3; \<LLN - 1.5 x 109 /L), LLN=Lower Limit Normal Range
• Positive serology for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus
• Significant neuropsychiatric conditions; dementia, major depression, or altered mental state that in the opinion of the Investigator will interfere with study participation
• Current treatment with systemic antibiotics or antivirals (EXCEPTION: topical treatments)

Sponsors & Collaborators

• Xalud Therapeutics, Inc.: lead

Study Sites (6)

eStudySite

La Mesa, California, 91942, United States

Location

Neurovations (Napa Pain Institute)

Napa, California, 94558, United States

Location

Source Healthcare

Santa Monica, California, 90403, United States

Location

Carolinas Clinical Research Institute

Winston-Salem, North Carolina, 27103, United States

Location

University of Adelaide in collaboration with CMAX Clinical Research Pty Ltd

Adelaide, South Australia, 5005, Australia

Location

Alfred Health

Melbourne, Victoria, 3004, Australia

Location

Related Publications (1)

• Chen L, Huang FL, Tang Q, Zhao ZK, Ye ZY, Liang JH. Targeted therapy for knee osteoarthritis: From basic to clinics. Medicine (Baltimore). 2025 Aug 15;104(33):e43686. doi: 10.1097/MD.0000000000043686.

  PMID: 40826764 DERIVED

MeSH Terms

Conditions

Osteoarthritis, Knee; Inflammation; Pain

Condition Hierarchy (Ancestors)

Osteoarthritis; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Neurologic Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Director of Medical Inquiries
• Organization: Xalud Therapeutics, Inc.

medical.information@xaludthera.com

212-301-6673

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 9, 2019
• First Posted: October 11, 2019
• Study Start: February 14, 2020
• Primary Completion: April 26, 2022
• Study Completion: April 26, 2022
• Last Updated: March 27, 2025
• Results First Posted: March 27, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (4); AU (2)