Trial Outcomes & Findings for 3-year, Open-Label Study Evaluating Safety, Tolerability, and Efficacy of Lorecivivint in Knee Osteoarthritis (NCT NCT04931667)

NCT ID: NCT04931667

Last Updated: 2026-03-31

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

72 participants

Primary outcome timeframe

Three Months after Day 1 Injection

Results posted on

2026-03-31

Participant Flow

Participant milestones

Participant milestones
Measure
0.07 mg Lorecivivint
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 (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.
Overall Study
STARTED
72
Overall Study
COMPLETED
0
Overall Study
NOT COMPLETED
72

Reasons for withdrawal

Reasons for withdrawal
Measure
0.07 mg Lorecivivint
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 (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.
Overall Study
Lost to Follow-up
2
Overall Study
Study Terminated by Sponsor
63
Overall Study
Withdrawal by Subject
7

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
0.07 mg Lorecivivint
n=72 Participants
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 (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.
Age, Continuous
61.5 years
STANDARD_DEVIATION 9.0 • n=4 Participants
Sex: Female, Male
Female
42 Participants
n=4 Participants
Sex: Female, Male
Male
30 Participants
n=4 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
12 Participants
n=4 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
60 Participants
n=4 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=4 Participants
Race (NIH/OMB)
American Indian or Alaska Native
1 Participants
n=4 Participants
Race (NIH/OMB)
Asian
1 Participants
n=4 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
1 Participants
n=4 Participants
Race (NIH/OMB)
Black or African American
11 Participants
n=4 Participants
Race (NIH/OMB)
White
58 Participants
n=4 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=4 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=4 Participants
Symptomatic OA Laterality by Investigator Assessment
Bilateral
58 Participants
n=4 Participants
Symptomatic OA Laterality by Investigator Assessment
Unilateral, Right
10 Participants
n=4 Participants
Symptomatic OA Laterality by Investigator Assessment
Unilateral, Left
4 Participants
n=4 Participants

PRIMARY outcome

Timeframe: Three Months after Day 1 Injection

Population: Efficacy analyses were not performed due to the early termination of the study (administrative reasons). Given the impact of early termination on data completeness, efficacy of LOR for the treatment of knee OA was not formally assessed. Data collected on those subjects completing Month 3 visit are summarized without any imputation for missing data.

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.

Outcome measures

Outcome measures
Measure
0.07 mg Lorecivivint
n=13 Participants
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 (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.
Change From Baseline in Pain Numeric Rating Scale (NRS) Score for the Left Knee
-0.9 units on a scale
Interval -6.0 to 4.0

PRIMARY outcome

Timeframe: Three Months after Day 1 Injection

Population: Efficacy analyses were not performed due to the early termination of the study (administrative reasons). Given the impact of early termination on data completeness, efficacy of LOR for the treatment of knee OA was not formally assessed. Data collected on those subjects completing Month 3 visit are summarized without any imputation for missing data.

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.

Outcome measures

Outcome measures
Measure
0.07 mg Lorecivivint
n=13 Participants
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 (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.
Change From Baseline in Pain Numeric Rating Scale (NRS) Score for the Right Knee
-1.2 units on a scale
Interval -5.0 to 3.0

SECONDARY outcome

Timeframe: Three Months after Day 1 Injection

Population: Efficacy analyses were not performed due to the early termination of the study (administrative reasons). Given the impact of early termination on data completeness, efficacy of LOR for the treatment of knee OA was not formally assessed. Data collected on those subjects completing Month 3 visit are summarized.

Change from baseline OA function in the target knee as assessed by Western Ontario and McMaster Universities (WOMAC) Osteoarthritis Index NRS3.1 physical function subscore (WOMAC Function) in left knee. The WOMAC is a widely-used, proprietary outcome measurement tool used to evaluate the condition of subjects with OA of the knee and hip, including pain, stiffness, and physical functioning of a target joint. Each question is measured on a scale from 0 (lowest pain/lowest stiffness/highest function) to 10 (highest pain/highest stiffness/lowest function). The WOMAC Function subscore ranges from 0 to 170 (raw) and will be scaled 0 to 100 for reporting. Change was calculated as three-month visit minus baseline; a negative value indicates improvement while a positive value indicates worsening.

Outcome measures

Outcome measures
Measure
0.07 mg Lorecivivint
n=13 Participants
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 (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.
Change From Baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Physical Function Subscore (WOMAC Function) in Left Knee
-13.4 units on a scale
Interval -58.2 to 20.0

SECONDARY outcome

Timeframe: Three Months after Day 1 Injection

Population: Efficacy analyses were not performed due to the early termination of the study (administrative reasons). Given the impact of early termination on data completeness, efficacy of LOR for the treatment of knee OA was not formally assessed. Data collected on those subjects completing Month 3 visit are summarized.

Change from baseline OA function in the target knee as assessed by Western Ontario and McMaster Universities (WOMAC) Osteoarthritis Index NRS3.1 physical function subscore (WOMAC Function) in right knee. The WOMAC is a widely-used, proprietary outcome measurement tool used to evaluate the condition of subjects with OA of the knee and hip, including pain, stiffness, and physical functioning of a target joint. Each question is measured on a scale from 0 (lowest pain/lowest stiffness/highest function) to 10 (highest pain/highest stiffness/lowest function). The WOMAC Function subscore ranges from 0 to 170 (raw) and will be scaled 0 to 100 for reporting. Change was calculated as three-month visit minus baseline; a negative value indicates improvement while a positive value indicates worsening.

Outcome measures

Outcome measures
Measure
0.07 mg Lorecivivint
n=13 Participants
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 (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.
Change From Baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Physical Function Subscore (WOMAC Function) in Right Knee
-13.5 units on a scale
Interval -46.9 to 5.9

SECONDARY outcome

Timeframe: Three Months after Day 1 Injection

Population: Efficacy analyses were not performed due to the early termination of the study (administrative reasons). Given the impact of early termination on data completeness, efficacy of LOR for the treatment of knee OA was not formally assessed. Data collected on those subjects completing Month 3 visit are summarized.

Change from baseline OA pain in the target knee as assessed by Western Ontario and McMaster Universities (WOMAC) Osteoarthritis Index NRS3.1 pain subscore (WOMAC Pain) in left knee. The WOMAC is a widely-used, proprietary outcome measurement tool used to evaluate the condition of subjects with OA of the knee and hip, including pain, stiffness, and physical functioning of a target joint. Each question is measured on a scale from 0 (lowest pain/lowest stiffness/highest function) to 10 (highest pain/highest stiffness/lowest function). The WOMAC Pain subscore ranges from 0 to 50 (raw) and will be scaled 0 to 100 for reporting. Change was calculated as three-month visit minus baseline; a negative value indicates improvement while a positive value indicates worsening.

Outcome measures

Outcome measures
Measure
0.07 mg Lorecivivint
n=13 Participants
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 (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.
Change From Baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Pain Subscore (WOMAC Pain) in Left Knee
-16.8 units on a scale
Interval -56.0 to -16.0

SECONDARY outcome

Timeframe: Three Months after Day 1 Injection

Population: Efficacy analyses were not performed due to the early termination of the study (administrative reasons). Given the impact of early termination on data completeness, efficacy of LOR for the treatment of knee OA was not formally assessed. Data collected on those subjects completing Month 3 visit are summarized.

Change from baseline OA pain in the target knee as assessed by Western Ontario and McMaster Universities (WOMAC) Osteoarthritis Index NRS3.1 pain subscore (WOMAC Pain) in right knee. The WOMAC is a widely-used, proprietary outcome measurement tool used to evaluate the condition of subjects with OA of the knee and hip, including pain, stiffness, and physical functioning of a target joint. Each question is measured on a scale from 0 (lowest pain/lowest stiffness/highest function) to 10 (highest pain/highest stiffness/lowest function). The WOMAC Pain subscore ranges from 0 to 50 (raw) and will be scaled 0 to 100 for reporting. Change was calculated as three-month visit minus baseline; a negative value indicates improvement while a positive value indicates worsening.

Outcome measures

Outcome measures
Measure
0.07 mg Lorecivivint
n=13 Participants
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 (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.
Change From Baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Pain Subscore (WOMAC Pain) in Right Knee
-13.7 units on a scale
Interval -36.0 to 8.0

Adverse Events

0.07 mg Lorecivivint

Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
0.07 mg Lorecivivint
n=72 participants at risk
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 (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.
Musculoskeletal and connective tissue disorders
Arthralgia
1.4%
1/72 • Number of events 1 • Adverse events were assessed at each in-person and phone visit from the time of study injection(s) on Day 1 through the end of the study (Day 116 / four months duration).
Infections and infestations
Bacterial Vaginosis
1.4%
1/72 • Number of events 1 • Adverse events were assessed at each in-person and phone visit from the time of study injection(s) on Day 1 through the end of the study (Day 116 / four months duration).

Additional Information

Christopher Swearingen, PhD, VP of Biometrics

Biosplice Therapeutics, Inc.

Phone: 858.926.2900

Results disclosure agreements

  • Principal investigator is a sponsor employee There IS an agreement between the Principal Investigator and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
  • Publication restrictions are in place

Restriction type: OTHER