Sporadic Inclusion Body Myositis (sIBM)Early Phase 1NCT06479863
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
A Early Phase 1 study testing Pozelimab/Cemdisiran in people with Sporadic Inclusion Body Myositis (sIBM) and Idiopathic Inflammatory Myopathies, run by Austin Neuromuscular Center at 2 sites in United States.
Trial summary
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM Read more about this study
- Who is it for?
- Ages 45 to 75 years
- Conditions the study is for
- Sporadic Inclusion Body Myositis (sIBM), Idiopathic Inflammatory Myopathies
- What is being tested?
- Pozelimab/Cemdisiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesUnited States
- Study dates
- 8 Aug 2024to 30 Aug 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 45 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1. Age ≥45 years to 75 years of age 2. Diagnosed with sIBM based on the ENMC IBM Research Diagnostic Criteria 3. Willing and able to comply with clinic visits and study-related procedures. 4. Provide informed consent signed by the study patient or legally acceptable representative.
- 5. Able to understand and complete study-related questionnaires. 6. Able to ambulate at least 20 ft/6 meters with or without assistive device Once arising from the chair, the participant may use any walking device, i.e. walker/frame, cane, crutches, or braces. They cannot be supported by another person and cannot use furniture or walls for support.
- 7. If female, the subject must be: (a) surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation. or (b) of childbearing potential and using a birth control method, such as:
- •Combined (estrogen and progestogen-containing) hormonal. contraception associated with inhibition of ovulation:
- o Oral
- Intravaginal
Show all 17Show fewer
- Transdermal
- Progestogen-only hormonal contraception associated with inhibition of ovulation:
- Oral
- Injectable
- Implantable
- Intrauterine device
- Intrauterine hormone-releasing system
- Bilateral tubal occlusion
- Vasectomized partner
- Sexual abstinence or of non-childbearing potential (i.e., no menses for ≥12 consecutive months without any other underlying medical cause) The subject must agree to continue using her selected method of birth control with her sexual partner during the study and for 120 days after study completion.
- 8. If male, the subject must have had a vasectomy or must use a reliable method of birth control with their partner or maintain total abstinence from sexual intercourse. The subject must agree to continue using his selected method of birth control with his sexual partner during the study and for 120 days after the study completion.
Who cannot join
- 1. Other neurological conditions (e.g., hemiplegia post-stroke, Parkinson's Disease) or musculoskeletal conditions (e.g., severe osteoarthritis) causing mobility impairment.
- 2. Any known active malignancy 3. Significant illness or history of significant illness that, in the investigator's opinion, may adversely affect the patient's participation in this study.
- 4. Any previous treatment with a complement inhibitor 5. History of meningococcal disease 6. No documented meningococcal and pneumococcal vaccination within 5 years prior to screening visit unless vaccination will be administered during the screening period and prior to initiation of study treatment.
- 7. Known contraindication to meningococcal (group ACWY conjugate and group B vaccines) and pneumococcal vaccines.
- 8. Subject unwilling to receive meningococcal and pneumococcal vaccinations. 9. Patients with screening serum ALT levels >3×ULN and/or total bilirubin >2×ULN (unless bilirubin elevation is due to suspected Gilbert's syndrome).
- 10. Active or recent systemic infection within 2 weeks prior to Baseline. 11. Pregnant, planning to become pregnant, or lactating female subjects. 12. Treatment with an experimental drug within 30 days or 5 half-lives of the experimental drug.
- 13. Participation in another concurrent interventional clinical trial. 14. Subjects with functional and anatomic asplenia 15. Subjects with positive tests for hepatitis B, hepatitis C, or HIV at screening 16. Presence of active or latent tuberculosis 17. Presence of any other form of myositis or myopathy or myositis overlap with other autoimmune diseases 18. Presence of other autoimmune or autoinflammatory diseases
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Austin Neuromuscular Center
Austin, Texas, 78759
Emil Hussain, Manager, 5129200140 ext. 212, emil@austinneuromuscle.com
Marisol Barrientos, Superviser, 5129200140 ext. 214, marisol@austinneuromuscle.com
- Recruiting
Austin Neuromuscular Center
Austin, Texas, 78759
Emil Hussain, 5129200140 ext. 212, emil@austinneuromuscle.com
Yessar hussain, MD
About this study
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
Read the detailed description
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.
- Being tested
- Pozelimab/Cemdisiran
- Medical subject headings
- Myositis, Inclusion Body, Myositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pozelimab/CemdisiranCombination product
Patients to receive Pozelimab 200mg/Cemdisiran 200 mg SC injections every 4 weeks for 104 weeks
| Study group | Role | What they receive |
|---|---|---|
| INJECTION | Other | 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. Receives: Combination Product: Pozelimab/Cemdisiran |
What is being measured
The primary outcome is the main question the study is designed to answer.
IBM-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.
Measured: 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.
Measured: change from screening visit through week 104.
Secondary outcomes (7)
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
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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).
Measured: Change from screening visit through week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2024
- Study start8 Aug 2024
- Primary completion30 May 2027 (estimated)
- Study completion30 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sporadic Inclusion Body Myositis (sIBM)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- The primary objective of the study is to evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM, The primary outcome measures are: IBM-Functional Rating Scale (IBM-FRS) change from screening visit through week 104. (2)IBM Physical Functioning Assessment (sIFA) change from screening visit through week 104
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Austin Neuromuscular Center
- Sponsor type
- University, hospital or other
- Collaborators
- Regeneron Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Yessar Hussain, MD, Principal investigator, Austin Neuromuscular Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06479863
- Sponsor's protocol ID
- R3918-OT-2383
- Status last verified
- Jan 2026
- First submitted
- 24 May 2024
- Last updated
- 16 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
- ClinicalTrials.gov ID
- NCT06479863
- Acronym / sponsor ID
- R3918-OT-2383
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Austin Neuromuscular CenterUniversity, hospital or other
- Collaborators
- Regeneron Pharmaceuticals
- Study start
- 8 Aug 2024 (actual)
- Primary completion
- 30 May 2027 (estimated)
- Study completion
- 30 Aug 2027 (estimated)
- First posted
- 28 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The primary objective of the study is to evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM, The primary outcome measures are: IBM-Functional Rating Scale (IBM-FRS) change from screening visit through week 104. (2)IBM Physical Functioning Assessment (sIFA) change from screening visit through week 104
| Arm | Type | Receives |
|---|---|---|
| INJECTION | Other | Combination Product: Pozelimab/Cemdisiranpatients 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. |
Who may qualify
- Ages
- 45 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 1. Age ≥45 years to 75 years of age 2. Diagnosed with sIBM based on the ENMC IBM Research Diagnostic Criteria 3. Willing and able to comply with clinic visits and study-related procedures. 4. Provide informed consent signed by the study patient or legally acceptable representative.
- 5. Able to understand and complete study-related questionnaires. 6. Able to ambulate at least 20 ft/6 meters with or without assistive device Once arising from the chair, the participant may use any walking device, i.e. walker/frame, cane, crutches, or braces. They cannot be supported by another person and cannot use furniture or walls for support.
- 7. If female, the subject must be: (a) surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation. or (b) of childbearing potential and using a birth control method, such as:
- •Combined (estrogen and progestogen-containing) hormonal. contraception associated with inhibition of ovulation:
- o Oral
Key exclusion criteria (7 listed)
- 1. Other neurological conditions (e.g., hemiplegia post-stroke, Parkinson's Disease) or musculoskeletal conditions (e.g., severe osteoarthritis) causing mobility impairment.
- 2. Any known active malignancy 3. Significant illness or history of significant illness that, in the investigator's opinion, may adversely affect the patient's participation in this study.
- 4. Any previous treatment with a complement inhibitor 5. History of meningococcal disease 6. No documented meningococcal and pneumococcal vaccination within 5 years prior to screening visit unless vaccination will be administered during the screening period and prior to initiation of study treatment.
- 7. Known contraindication to meningococcal (group ACWY conjugate and group B vaccines) and pneumococcal vaccines.
- 8. Subject unwilling to receive meningococcal and pneumococcal vaccinations. 9. Patients with screening serum ALT levels >3×ULN and/or total bilirubin >2×ULN (unless bilirubin elevation is due to suspected Gilbert's syndrome).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pozelimab/CemdisiranPatients to receive Pozelimab 200mg/Cemdisiran 200 mg SC injections every 4 weeks for 104 weeks | Combination product | INJECTION |
Primary outcome
IBM-Functional Rating Scale (IBM-FRS)
Time frame: change from screening visit through week 104.
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.
IBM Physical Functioning Assessment (sIFA)
Time frame: change from screening visit through week 104.
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.
7 secondary outcomes
Manual Muscle Testing
Time frame: Change from screening visit through week 104
Hand Grip Strength
Time frame: Change from screening visit through week 104
Quadriceps Strength Test
Time frame: Change from screening visit through week 104
Health Assessment Questionnaire Disability Index (HAQ/HAQ-DI)
Time frame: Change from screening visit through week 104
6 Minute Walk Test
Time frame: Change from screening visit through week 104
Timed up and go (TUG)
Time frame: Change from screening visit through week 104
4-point Hopkins Falls Grading Scale (HFGS)
Time frame: Change from screening visit through week 104
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2026.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Yessar Hussain, MD principal investigator
Austin Neuromuscular Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Pozelimab/Cemdisiran in people with Sporadic Inclusion Body Myositis (sIBM) and Idiopathic Inflammatory Myopathies, run by Austin Neuromuscular Center at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yessar Hussain, MD (5129200140, research@austinneuromuscle.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
IBM-Functional Rating Scale (IBM-FRS) (time frame: change from screening visit through week 104.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jan 2026.