To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
This is a pilot study of 10 patients to receive Pozelimab 200mg/Cemdisiran 200 mg SC injections every 4 weeks for 104 weeks. SC injections will be administered by the study staff and the patient will be monitored for 30 minutes after the initial first injection. During the remainder of the study, injections can be administered at home by the patient or caregiver after injection training provided by the study staff. Designated persons will be observed to confirm their ability to perform the injections. The dosing window of the study treatment is within ±7 days from the scheduled dose date.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Patients to receive Pozelimab 200mg/Cemdisiran 200 mg SC injections every 4 weeks for 104 weeks
Austin Neuromuscular Center
Austin, Texas, United States
RECRUITINGAustin Neuromuscular Center
Austin, Texas, United States
RECRUITINGIBM-Functional Rating Scale (IBM-FRS)
The IBM-FRS is a 10-item interviewer-administered patient-reported outcomes (PRO) measure that assesses physical function in patients with IBM. The IBMFRS includes items assessing lower body physical function, general physical function, the ability to perform certain activities of daily living, fine motor skills, and swallowing. On a Likert scale ranging from 0 (unable to perform activity) to 4 (normal), patients are asked to rate their difficulty in performing tasks relative to each concept before disease onset. Scores on the IBMFRS range from 0 to 40, with higher scores indicating better functioning.
Time frame: change from screening visit through week 104.
IBM Physical Functioning Assessment (sIFA)
The sIFA is an 11-item self-administered PRO measure assessing physical function in patients with sIBM. This questionnaire will be completed by the patient on a numeric rating scale ranging from 0 (no difficulty) to 10 (unable to do), with a total score ranging from 0 to 100, where 100 indicates severe limitations. P atients will be asked to rate their difficulty with performing tasks involving lower and upper body physical function, general physical function, fine motor skills, and swallowing in the past 7 days.
Time frame: change from screening visit through week 104.
Manual Muscle Testing
This tool assesses muscle strength using a 0 - 10-point scale. A set of 7 designated muscles is tested bilaterally plus axial (neck flexor) testing so that the potential MMT-8 score ranges from 0 - 150, where a higher score indicates better functioning. The designated muscles tested will be: * Neck flexors * Deltoid middle * Biceps brachii * Gluteus maximus or Iliopsoas * Gluteus Medius * Quadriceps * Wrist extensors * Ankle dorsiflexors
Time frame: Change from screening visit through week 104
Hand Grip Strength
In this test, strength is measured in both the right and left hands separately with a hand grip dynamometer. Consistent angles are maintained for each patient across the shoulder, elbow, and wrist joints. Forearm and hand placement are standardized for each participant and the forearm is supported by a flat surface. Muscle strength for this test will be assessed in kilograms (kg).
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: Change from screening visit through week 104
Quadriceps Strength Test
In this test, strength is measured in both the right and left quadriceps with a dynamometer. Consistent 90-degree angles are maintained for each patient across the hip and knee joints in sitting position. Muscle strength for this test will be assessed in kilograms (kg).
Time frame: Change from screening visit through week 104
Health Assessment Questionnaire Disability Index (HAQ/HAQ-DI)
This tool consists of 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities, each containing 2 to 3 questions. Scoring includes 0 (without any difficulty), 1 (with some difficulty), 2 (with much difficulty), and 3 (unable to perform). The score for each section is determined by its highest score. The HAQ-DI considers the subject's use of aids, devices, or assistance in the scoring algorithm for a disability category. If either "aids/devices" and/or "assistance from another person" are checked for a disability category, the score for this category is set to "2" (much difficulty), if the original score was "0" (no difficulty) or "1" (some difficulty). The HAQ-DI is then calculated by summing the adjusted categories scores and dividing by the number of categories answered. Scoring conventions are based on the Standard Disability Index of HAQ/HAQ-DI, using the 20 response items. A higher score indicates greater disability.
Time frame: Change from screening visit through week 104
6 Minute Walk Test
. Originally developed for patients with respiratory disease, this is a widely used physical function test with well-established validity and reliability in patients with respiratory and cardiovascular diseases (Holland, 2014). The 6MWT has also been used as an outcome measure in sIBM studies (Mendell, 2017) (Rose, 2013). This test will measure the distance a patient walks in meters (M) in 6 minutes.
Time frame: Change from screening visit through week 104
Timed up and go (TUG)
In this test, the amount of time it takes the patient to stand from a seated position, walk 3 meters, turn, return to the chair, and then sit down is measured. This test will be measured in seconds (secs).
Time frame: Change from screening visit through week 104
4-point Hopkins Falls Grading Scale (HFGS)
HFGS is a 4-item interviewer-administered scale which assesses the nature and severity of a fall event. Using illustrations, falls are divided into four grades: Grade 1 - near fall; Grade 2 - fall where the patient does not receive medical attention; Grade 3 - fall where the patient receives medical attention but is not admitted to the hospital; and Grade 4 - fall where the patient is admitted to the hospital. HFGS exhibited good face and content validity and had an intraclass correlation coefficient of 0.998 (Davalos-Bichara, 2013).
Time frame: Change from screening visit through week 104