Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis

NCT06479863 | Recruiting Early Phase 1 DMC FDA Drug

• 1 other identifier: R3918-OT-2383
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

Brief Summary

To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM

Conditions

Sporadic Inclusion Body Myositis (sIBM); Idiopathic Inflammatory Myopathies

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  change from screening visit through week 104.

Secondary Outcomes (7)

• Manual Muscle Testing

  Change from screening visit through week 104

• Hand Grip Strength

  Change from screening visit through week 104

• Quadriceps Strength Test

  Change from screening visit through week 104

• Health Assessment Questionnaire Disability Index (HAQ/HAQ-DI)

  Change from screening visit through week 104

• 6 Minute Walk Test

  Change from screening visit through week 104

Study Arms (1)

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.

Combination Product: Pozelimab/Cemdisiran

Interventions

Pozelimab/Cemdisiran COMBINATION_PRODUCT

Patients to receive Pozelimab 200mg/Cemdisiran 200 mg SC injections every 4 weeks for 104 weeks

INJECTION

Eligibility Criteria

• Age: 45 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. 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.
• \. 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.
• \. 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
• Transdermal
• Progestogen-only hormonal contraception associated with inhibition of ovulation:
• Oral
• Injectable
• Implantable
• Intrauterine device
• Intrauterine hormone-releasing system
• Bilateral tubal occlusion
• Vasectomized partner

You may not qualify if:

• \. Other neurological conditions (e.g., hemiplegia post-stroke, Parkinson's Disease) or musculoskeletal conditions (e.g., severe osteoarthritis) causing mobility impairment.
• \. 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.
• \. 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.
• \. Known contraindication to meningococcal (group ACWY conjugate and group B vaccines) and pneumococcal vaccines.
• \. 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).
• \. 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.
• \. 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

Sponsors & Collaborators

• Austin Neuromuscular Center: lead
• Regeneron Pharmaceuticals: collaborator

Study Sites (2)

Austin Neuromuscular Center

Austin, Texas, 78759, United States

RECRUITING

Austin Neuromuscular Center

Austin, Texas, 78759, United States

RECRUITING

MeSH Terms

Conditions

Myositis, Inclusion Body; Myositis

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Study Officials

• Yessar Hussain, MD

  Austin Neuromuscular Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yessar Hussain, MD

CONTACT

5129200140; research@austinneuromuscle.com

Emil Hussain, Manager

CONTACT

5129200140; emil@austinneuromuscle.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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

Study Record Dates

• First Submitted: May 24, 2024
• First Posted: June 28, 2024
• Study Start: August 8, 2024
• Primary Completion (Estimated): May 30, 2027
• Study Completion (Estimated): August 30, 2027
• Last Updated: January 16, 2026

Record last verified: 2026-01

Locations

US (2)