Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- IBM Physical Functioning Assessment (sIFA)
研究概览
简要总结
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 45 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1. Age ≥45 years to 75 years of age
- •Diagnosed with sIBM based on the ENMC IBM Research Diagnostic Criteria
- •Willing and able to comply with clinic visits and study-related procedures.
- •Provide informed consent signed by the study patient or legally acceptable representative.
- •5. Able to understand and complete study-related questionnaires.
- •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:
- •Intravaginal
- •Transdermal
- •Progestogen-only hormonal contraception associated with inhibition of ovulation:
- •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.
排除标准
- •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
- •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
- •History of meningococcal disease
- •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.
- •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.
- •Pregnant, planning to become pregnant, or lactating female subjects.
- •Treatment with an experimental drug within 30 days or 5 half-lives of the experimental drug.
- •13. Participation in another concurrent interventional clinical trial.
- •Subjects with functional and anatomic asplenia
- •Subjects with positive tests for hepatitis B, hepatitis C, or HIV at screening
- •Presence of active or latent tuberculosis
- •Presence of any other form of myositis or myopathy or myositis overlap with other autoimmune diseases
- •Presence of other autoimmune or autoinflammatory diseases
研究组 & 干预措施
INJECTION
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.
干预措施: Pozelimab/Cemdisiran (Combination Product)
结局指标
主要结局
IBM Physical Functioning Assessment (sIFA)
时间窗: 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.
IBM-Functional Rating Scale (IBM-FRS)
时间窗: 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.
次要结局
- Health Assessment Questionnaire Disability Index (HAQ/HAQ-DI)(Change from screening visit through week 104)
- Timed up and go (TUG)(Change from screening visit through week 104)
- 6 Minute Walk Test(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)
- Manual Muscle Testing(Change from screening visit through week 104)
- 4-point Hopkins Falls Grading Scale (HFGS)(Change from screening visit through week 104)