Trial Outcomes & Findings for AMZ001 for the Treatment of Knee Osteoarthritis Symptoms (NCT NCT03691844)

Seven trial sites in Denmark (1 site), Czech Republic (3 sites) and the USA (3 sites). Trial initiation: 04-Oct-2018 Trial completion: 09-Jul-2019

AMZ001 for the Treatment of Knee Osteoarthritis Symptoms

Change from baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) pain sub-score (questions 1-5; score 0 \[no pain\]-50 \[extreme pain\]) on target knee. The WOMAC scores were normalized to a 0-100 point scale for data analysis, where higher scores indicate greater pain, stiffness or difficulty in performing daily tasks.

Change from baseline in Western Ontario McMasters Universities Osteoarthritis Index (WOMAC) total score and WOMAC function (degree of difficulty experienced in performing daily activities - 17 questions score 0-170) and stiffness (the degree and timing of joint stiffness - 2 questions score 0-20). The WOMAC scores were normalized to a 0-100 point scale for data analysis, where 0 is no pain and higher scores indicate greater pain, stiffness or difficulty in performing daily tasks.

Change from baseline in Western Ontario McMasters Universities Osteoarthritis Index (WOMAC) pain weight-bearing score (questions 1,2, \& 5; score 0-30) and non-weight-bearing score (questions 3\&4; score 0-20) on target knee. The WOMAC scores were normalized to a 0-100 point scale for data analysis, where 0 is no pain and higher scores indicate greater pain, stiffness or difficulty in performing daily tasks.

Change from baseline in Intermittent and Constant Osteoarthritis Pain (ICOAP) scores (score 0 \[no pain\]- 4 \[extreme pain\]). ICOAP scores were normalized to a 0-100 point scale, where 0 is no pain and higher scores indicate greater pain.

Change in baseline in physical function assessed by the chair-stand test. The test measures the maximum number of chair stand repetitions possible in a 30-second period with out using the arms. Where 0 is no repetition completed and higher numbers corresponds to greater repetitions and physical function.

Outcome Measures in Rheumatology- Osteoarthritis Research Society International (OMERACT-OARSI) response involves changes that are deemed to be clinically relevant in three domains: pain, function, and PGA (Patient Global Assessment). For each of these domains, ranges are defined for absolute and percent changes from baseline that correspond to "high improvement" and "moderate improvement". OMERACT-OARSI response is defined as either high improvement in at least 1 of WOMAC pain and function scores OR moderate improvement in at least 2 of WOMAC pain scores, WOMAC function score or Patient Global Assessment (PGA)

Total dose of rescue medication calculated as the average gram use/day, based on pill counts.

Time between baseline and first use of rescue medication.

Change from baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) pain sub-score (questions 1-5; score 0 \[no pain\]-50 \[extreme pain\]) on target knee. The WOMAC scores were normalized to a 0-100 point scale for data analysis, where 0 is no pain and higher scores indicate greater pain, stiffness or difficulty in performing daily tasks.

Change from baseline in Intermittent and Constant Osteoarthritis Pain (ICOAP) scores (score 0 \[no pain\]- 4 \[extreme pain\]). ICOAP scores were normalized to a 0-100 point scale, where 0 is no pain and higher scores indicate greater pain.

Changes from baseline in Western Ontario and McMasters Universities Osteoarthritis Index (WOMAC) pain weight-bearing score (questions 1,2, \& 5; score 0-30) and non-weight-bearing score (questions 3\&4; score 0-20) on target knee. The WOMAC scores were normalized to a 0-100 point scale for data analysis, where 0 is no pain and higher scores indicate greater pain, stiffness or difficulty in performing daily tasks.

Change in baseline assessed by the chair-stand test. The test measures the maximum number of chair stand repetitions possible in a 30-second period with out using the arms. Where 0 is no repetition completed and higher numbers corresponds to greater repetitions and physical function.

Change from baseline in Western Ontario McMasters Universities Osteoarthritis Index (WOMAC) total score and WOMAC function (degree of difficulty experienced in performing daily activities - 17 questions score 0-170) and stiffness (the degree and timing of joint stiffness - 2 questions score 0-20). The WOMAC scores were normalized to a 0-100 point scale for data analysis, where 0 is no pain and higher scores indicate greater pain, stiffness or difficulty in performing daily tasks.

Change from baseline in Impact of OA daily living assessed using Patient Global Assessment (PGA) score. PGA is scored on a 11-point scale from 0 (none) to 10 (extreme), where higher scores represents a higher level of disease activity or worse health.

Change from baseline in work productivity and activity assessed by the Work Productivity and Active Impairment (WPAI scores 0-100% in four different categories: absenteeism, presenteeism, work productivity loss, and activity impairment). Outcomes are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity.

The EuroQol-5 Domain (EQ-5D) is a standardized generic measure of health-related quality of life. The visual analog scale (VAS) is scored on a 0-100 scale, where 0 is "the worst health you can imagine" and 100, "the best health you can imagine".

Nature, incidence and severity of AEs.

Skin tolerability assessment, incidence of erythema at the application site. Grading scheme from 0-4 (0, normal skin, no erythema; 4, blister formation and/or necrosis).

Change from baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) pain sub-score (questions 1-5; score 0 \[no pain\]-50 \[extreme pain\]) on target knee. The WOMAC scores were normalized to a 0-100 point scale for data analysis. The WOMAC scores were normalized to a 0-100 point scale for data analysis, where 0 is no pain and higher scores indicate greater pain, stiffness or difficulty in performing daily tasks.

Change from baseline in Western Ontario McMasters Universities Osteoarthritis Index (WOMAC) total score and WOMAC function (degree of difficulty experienced in performing daily activities - 17 questions score 0-170) and stiffness (the degree and timing of joint stiffness - 2 questions score 0-20) . The WOMAC scores were normalized to a 0-100 point scale for data analysis, where 0 is no pain and higher scores indicate greater pain, stiffness or difficulty in performing daily tasks.