3-year, Open-Label Study Evaluating Safety, Tolerability, and Efficacy of Lorecivivint in Knee Osteoarthritis

NCT04931667 | Terminated Phase 3 Results FDA Drug

• 1 other identifier: SM04690-OA-17
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 4

Brief Summary

This study is designed as a 3-year, multicenter, open-label study evaluating the safety, tolerability, and efficacy of intra-articular (IA) Lorecivivint (LOR) in subjects with osteoarthritis of the knee in a real-world setting.

Conditions

Osteoarthritis of the Knee

Keywords

SM04690; Wnt pathway inhibitor; osteoarthritis; Biosplice Therapeutics, Inc.; Lorecivivint; DYRK inhibitor; CLK inhibitor; Samumed

Outcome Measures

Primary Outcomes (2)

• Change From Baseline in Pain Numeric Rating Scale (NRS) Score for the Left Knee

  Change from baseline in pain numeric rating scale (NRS) score for the left knee. The pain NRS is an 11-point scale \[0-10\] for subject self-reporting of average knee pain in the last 24 hours; 0 indicates no pain, and 10 represents the worst possible pain. Change was calculated as three-month visit minus baseline; a negative value indicates improvement while a positive value indicates worsening.

  Three Months after Day 1 Injection

• Change From Baseline in Pain Numeric Rating Scale (NRS) Score for the Right Knee

  Change from baseline in pain numeric rating scale (NRS) score for the right knee. The pain NRS is an 11-point scale \[0-10\] for subject self-reporting of average knee pain in the last 24 hours; 0 indicates no pain, and 10 represents the worst possible pain. Change was calculated as three-month visit minus baseline; a negative value indicates improvement while a positive value indicates worsening.

  Three Months after Day 1 Injection

Secondary Outcomes (4)

• Change From Baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Physical Function Subscore (WOMAC Function) in Left Knee

  Three Months after Day 1 Injection

• Change From Baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Physical Function Subscore (WOMAC Function) in Right Knee

  Three Months after Day 1 Injection

• Change From Baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Pain Subscore (WOMAC Pain) in Left Knee

  Three Months after Day 1 Injection

• Change From Baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Pain Subscore (WOMAC Pain) in Right Knee

  Three Months after Day 1 Injection

Study Arms (1)

0.07 mg Lorecivivint

EXPERIMENTAL

One intra-articular injection of 0.07 mg Lorecivivint in 2 ml vehicle in one or both knees as clinically indicated. Bilateral injection of 0.07 mg LOR are allowed. Lorecivivint: Healthcare professional-administered intra-articular injection

Drug: Lorecivivint

Interventions

Lorecivivint DRUG

Healthcare professional-administered intra-articular (IA) injection into one or both knees as clinically indicated. Bilateral injections of 0.07 mg of LOR are allowed. IA injection performed on Day 1. Additional IA injections of LOR to same knee (with at least a 6 month separation) between Day 1 and Month 33.

Also known as: SM04690

0.07 mg Lorecivivint

Eligibility Criteria

• Age: 40 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females between 40 and 80 years of age, inclusive, in general good health apart from their knee OA
• Femorotibial OA by standard American College of Rheumatology (ACR) criteria; knee OA is not to be secondary to any rheumatologic conditions (e.g., rheumatoid arthritis).
• Pain compatible with knee OA for at least 26 weeks prior to the Screening Visit
• Negative drug test for amphetamine, buprenorphine, cocaine, methadone, opiates, phencyclidine (PCP), propoxyphene, barbiturates, and benzodiazepine, unless any of these drugs are prescribed by a physician to treat a specific condition
• Full understanding of the requirements of the study and willingness to comply with all study visits and assessments
• Subjects must have read and understood the informed consent form (ICF), and must have signed and dated it prior to any study-related procedure being performed

You may not qualify if:

• Pregnant women, breastfeeding women, and women who are not post-menopausal (defined as 12 months with no menses without an alternative medical cause) or permanently surgically sterile (includes hysterectomy, bilateral salpingectomy, and bilateral oophorectomy) who have a positive or indeterminate pregnancy test result at Screening and Day 1
• Women who are not post-menopausal or permanently surgically sterile who are sexually active, and who are not willing to use birth control during the study period
• Any surgery (e.g., arthroscopy) in either knee within 26 weeks prior to Day 1
• Intra-articular (IA) injection into either knee with a therapeutic aim including, but not limited to, hyaluronic acid, platelet-rich plasma (PRP), and stem cell therapies within 26 weeks prior to Day 1 or IA glucocorticoids within 12 weeks prior to Day 1
• Any condition that, in the opinion of the Investigator, constitutes a risk or contraindication for participation in the study or that could interfere with the study objectives, conduct, or evaluation
• Any comorbid condition that could affect study endpoint assessments of the knee, including, but not limited to, rheumatoid arthritis, psoriatic arthritis, systemic lupus erythematosus, gout or pseudogout, and fibromyalgia
• Any contraindications for an IA injection in the knee(s) to be injected at Day 1 in the opinion of the Investigator

Sponsors & Collaborators

• Biosplice Therapeutics, Inc.: lead

Study Sites (4)

Research Site

Tucson, Arizona, 85712, United States

Location

Research Site

Winter Park, Florida, 32789, United States

Location

Research Site

Oak Brook, Illinois, 60523, United States

Location

Research Site

Dallas, Texas, 75231, United States

Location

MeSH Terms

Conditions

Osteoarthritis, Knee; Osteoarthritis

Interventions

lorecivivint

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Results Point of Contact

• Title: Christopher Swearingen, PhD, VP of Biometrics
• Organization: Biosplice Therapeutics, Inc.

info@biosplice.com

858.926.2900

Study Officials

• Yusuf Yazici, MD

  Biosplice Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 4, 2021
• First Posted: June 18, 2021
• Study Start: July 15, 2021
• Primary Completion: December 13, 2021
• Study Completion: December 13, 2021
• Last Updated: March 31, 2026
• Results First Posted: March 31, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)