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

Last updated: November 12, 2024
Sponsor: Austin Neuromuscular Center
Overall Status: Active - Recruiting

Phase

1

Condition

Idiopathic Inflammatory Myopathies

Myositis

Treatment

Pozelimab/Cemdisiran

Clinical Study ID

NCT06479863
R3918-OT-2383
  • Ages 45-75
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Age ≥45 years to 75 years of age 2. Diagnosed with sIBM based on the ENMC IBMResearch Diagnostic Criteria 3. Willing and able to comply with clinic visitsand study-related procedures. 4. Provide informed consent signed by the studypatient or legally acceptable representative.

  2. Able to understand and complete study-related questionnaires. 6. Able toambulate at least 20 ft/6 meters with or without assistive device Once arisingfrom the chair, the participant may use any walking device, i.e. walker/frame,cane, crutches, or braces. They cannot be supported by another person andcannot use furniture or walls for support.

  3. If female, the subject must be: (a) surgically sterilized via hysterectomy,bilateral oophorectomy, or bilateral tubal ligation. or (b) of childbearingpotential and using a birth control method, such as:

•Combined (estrogen and progestogen-containing) hormonal. contraception associatedwith inhibition of ovulation: o Oral

  • Intravaginal

  • Transdermal

  • Progestogen-only hormonal contraception associated with inhibition ofovulation:

  • 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) Thesubject must agree to continue using her selected method of birth controlwith her sexual partner during the study and for 120 days after studycompletion.

  1. If male, the subject must have had a vasectomy or must use a reliablemethod of birth control with their partner or maintain totalabstinence from sexual intercourse. The subject must agree tocontinue using his selected method of birth control with his sexualpartner during the study and for 120 days after the study completion.

Exclusion

Exclusion Criteria:

  1. Other neurological conditions (e.g., hemiplegia post-stroke, Parkinson'sDisease) or musculoskeletal conditions (e.g., severe osteoarthritis) causingmobility impairment.

  2. Any known active malignancy 3. Significant illness or history of significantillness that, in the investigator's opinion, may adversely affect the patient'sparticipation in this study.

  3. Any previous treatment with a complement inhibitor 5. History of meningococcaldisease 6. No documented meningococcal and pneumococcal vaccination within 5years prior to screening visit unless vaccination will be administered duringthe screening period and prior to initiation of study treatment.

  4. Known contraindication to meningococcal (group ACWY conjugate and group Bvaccines) and pneumococcal vaccines.

  5. 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).

  6. 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 theexperimental drug.

  7. Participation in another concurrent interventional clinical trial. 14. Subjectswith functional and anatomic asplenia 15. Subjects with positive tests forhepatitis B, hepatitis C, or HIV at screening 16. Presence of active or latenttuberculosis 17. Presence of any other form of myositis or myopathy or myositisoverlap with other autoimmune diseases 18. Presence of other autoimmune orautoinflammatory diseases

Study Design

Total Participants: 10
Treatment Group(s): 1
Primary Treatment: Pozelimab/Cemdisiran
Phase: 1
Study Start date:
August 08, 2024
Estimated Completion Date:
August 30, 2027

Study 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.

Connect with a study center

  • Austin Neuromuscular Center

    Austin, Texas 78759
    United States

    Active - Recruiting

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