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

NCT ID: NCT04124042

Last Updated: 2025-03-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 289 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-02-14
• Study Completion Date: 2022-04-26

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.

Detailed Description

In this Phase 2 study, Baseline (Day 0) confirmation of study eligibility will be completed the day before or day of study drug administration.

Study drug will be administered by intra-articular (IA) injection into the joint space of the index knee (knee selected for treatment).

Up to 270 participants will be randomly enrolled into 1 of 6 treatment sequences (45 participants/ group). Treatment Groups:

1. 0.15 mg/mL XT-150 (1mL), 0.15 mg/mL XT-150 (1mL)

2. 0.15 mg/mL XT-150 (1mL), 0.45 mg/mL XT-150 (1mL)

3. 0.45 mg/mL XT-150 (1mL), 0.15 mg/mL XT-150 (1mL)

4. 0.45 mg/mL XT-150 (1mL), 0.45 mg/mL XT-150 (1mL)

5. Placebo (1mL), 0.15 mg/mL XT-150 (1mL)

6. Placebo (1mL), 0.45 mg/mL XT-150 (1mL)

The study will be conducted in 2 stages, A and B. Participants will be randomized at Day 0 to a treatment regimen, one treatment assignment for Stage A and one treatment assignment for Stage B:

Stage A (Up to Day 180): Participants will receive placebo, 0.15 mg/mL XT-150 or 0.45 mg/mL XT-150 to the index knee at Day 0.

Stage B (Day 180 to Day 360): Participants will have the option to receive a pre-randomized dose (XT-150 0.15 mg/mL or 0.45 mg/mL) to the index knee anytime between Day 180 and Day 330.

Final assessments will be 12 months after the first IA dose.

Conditions

Osteoarthritis, Knee

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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

Low dose active in Stage A and Stage B

Group Type: EXPERIMENTAL

XT-150

Intervention Type: BIOLOGICAL

plasmid DNA

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

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

Group Type: EXPERIMENTAL

XT-150

Intervention Type: BIOLOGICAL

plasmid DNA

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

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

Group Type: EXPERIMENTAL

XT-150

Intervention Type: BIOLOGICAL

plasmid DNA

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

High dose active in Stage A and Stage B

Group Type: EXPERIMENTAL

XT-150

Intervention Type: BIOLOGICAL

plasmid DNA

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

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

Group Type: PLACEBO_COMPARATOR

XT-150

Intervention Type: BIOLOGICAL

plasmid DNA

Placebo

Intervention Type: DRUG

Placebo is a sterile phosphate-buffered saline

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

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

Group Type: PLACEBO_COMPARATOR

XT-150

Intervention Type: BIOLOGICAL

plasmid DNA

Placebo

Intervention Type: DRUG

Placebo is a sterile phosphate-buffered saline

Interventions

XT-150

plasmid DNA

Intervention Type: BIOLOGICAL

Placebo

Placebo is a sterile phosphate-buffered saline

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Symptomatic disease due to osteoarthritis, defined as a WOMAC Pain score ≥ 8 (worst possible = 20)

2. Focused Analgesia Selection Test will be used to determine whether patients can report pain with sufficient consistency to enter the clinical trial

3. Males and females between 45 and 85 years of age, inclusive

4. Kellgren-Lawrence grading of 2 or 3 within the last 6 months

5. Stable analgesic regimen during the 4 weeks prior to enrollment

6. In the judgment of the Investigator, acceptable general medical condition

7. Life expectancy >6 months

8. 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

9. Have suitable knee joint anatomy for intra-articular injection

10. Willing and able to return for the follow-up (FU) visits

11. Able to read and understand study instructions, and willing and able to comply with all study procedures

Exclusion Criteria

1. Hypersensitivity, allergy, or significant reaction to any ingredient of the study drug, including double-stranded DNA, mannose, and sucrose

2. Previously received XT-150 injection(s)

3. Scheduled partial or complete knee replacement within 6 months; participant agrees not to schedule a knee replacement during Stage A of the study

4. History of knee arthroplasty on the Index Knee, i.e., selected for study injection(s)

5. History of rheumatoid arthritis or other inflammatory disease

6. History of immunosuppressive therapy; systemic steroids in the last 3 months

7. Received knee injection with hyaluronic acid or stem-cells in the last 6 months

8. Knee injection of glucocorticoid in the last 3 months

9. Current treatment with systemic immunosuppressive (systemic corticosteroid therapy or other strong immunosuppressant)

10. Currently receiving systemic chemotherapy or radiation therapy for malignancy

11. 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)

12. 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

13. Positive serology for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus

14. Significant neuropsychiatric conditions; dementia, major depression, or altered mental state that in the opinion of the Investigator will interfere with study participation

15. Current treatment with systemic antibiotics or antivirals (EXCEPTION: topical treatments)

16. Current anticoagulant or anti-platelet treatment (e.g., warfarin, heparins, factor X inhibitors, clopidogrel, prasugrel, ticagrelor, or dipyridamole). Low-dose (≤ 325 mg/day) aspirin is permitted

17. Known or suspected history of active alcohol or intravenous/oral drug abuse within 1 year before the screening visit

18. Use of any investigational drug or device within 3 months before enrollment or current participation in a trial that included intervention with a drug or device; or currently participating in an investigational drug or device study

19. Any condition that, in the opinion of the Investigator, could compromise the safety of the participant, the participant's ability to communicate with the study staff, or the quality of the data

• Minimum Eligible Age: 45 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Xalud Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

eStudySite

La Mesa, California, United States

Neurovations (Napa Pain Institute)

Napa, California, United States

Source Healthcare

Santa Monica, California, United States

Carolinas Clinical Research Institute

Winston-Salem, North Carolina, United States

University of Adelaide in collaboration with CMAX Clinical Research Pty Ltd

Adelaide, South Australia, Australia

Alfred Health

Melbourne, Victoria, Australia

Countries

United States; Australia

References

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.

Reference Type: DERIVED

PMID: 40826764 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

XT-150-2-0204

Identifier Type: -

Identifier Source: org_study_id