A Single Center, Open Label Pilot Study to Evaluate the Safety and Efficacy of CC-11050 in Nepalese Patients With Erythema Nodosum Leprosum
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 2
- 主要终点
- Number of participants with moderate to severe ENL with severity changes as assessed by the ENL Severity Scale.
研究概览
简要总结
This study will be a single center, Phase 2, open-label trial to evaluate the safety and efficacy of 200mg CC-11050 administered twice daily taken with food for patients with moderate to severe ENL. The study will be performed in two steps: 1) to evaluate immediate effect in safety and efficacy of drug in 10 males with new or new recurrent episode ENL and, if found to be safe and effective by the DSMB and 2) if allowed by the DSMB, and approved by relevant study stakeholders, an additional 40 ENL patients will be enrolled for up to 52 weeks of treatment. A safety analysis will be conducted on all patients who have received at least one dose of study drug, and will include the frequency of all adverse events and laboratory abnormalities as well as frequency of dose interruptions, dose reductions and treatment discontinuation.
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Study participants must satisfy the following criteria to be enrolled in the study:
- •Must be 18 -65 years old, weight > 35kg for women and >40kg for men at the time of signing the informed consent.
- •Understand and voluntarily sign an informed consent document prior to any study related assessments/procedures are conducted.
- •Have signs or symptoms of new or new episode ENL. In Step one, participants must be male. In Step 2, participants can be either male or female.
- •Able to adhere to the study schedule and other protocol requirements.
排除标准
- •The presence of any of the following will exclude a participant from enrollment:
- •Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the participants from participating in the study.
- •Patients on continuous rifampicin will be excluded, however, If rifampicin is given only once a month as part of a multi-drug regimen, a minimum of 2-week washout period is required prior to administration of CC-11050
- •Any condition including the presence of laboratory abnormalities, which places the participant at unacceptable risk if he were to participate in the study.
- •Any condition that confounds the ability to interpret data from the study (ie, HIV, chronic HepB, chronic HepC or TB patients under active treatment). Resolved TB patients will not be excluded from the study.
- •Stable and well managed patients with Diabetes and hypertension will not be excluded from the study.
- •Use of systemic corticosteroid or immunosuppressant therapy within 7 days of study medication initiation
- •Pregnant or nursing females.
研究组 & 干预措施
CC-11050 treatment
200mg CC-11050 will be administered twice daily as a pill taken with food (at breakfast and evening meal) for participants with moderate to severe ENL for 10 days, then up to 28 days (during Step 1) and then up to 1 year (during Step 2).
干预措施: CC-11050 (Drug)
结局指标
主要结局
Number of participants with moderate to severe ENL with severity changes as assessed by the ENL Severity Scale.
时间窗: 10 days, 28 days and monthly up to 1 year of treatment
Changes in ENL Severity Scale Score (Walker et al., PLoS Negl Trop Dis 11(7): e0005716, https://doi.org/10.1371/journal.pntd.0005716) as compared to baseline and any previous time points. Ten clinical categories contribute 0-3 points toward the total score of 0-30. An ENL patient with a total scale score of less than 9 is considered to have mild ENL, while scores \>9 are considered moderate to severe ENL. A minimum score of 9 is required to indicate sufficient ENL symptoms from which post-intervention reduction in score(s) can be determined meaningful improvement.
次要结局
- Number of participants with moderate to severe ENL with decreased duration of episode(s) as assessed by standard ENL episode definition.(10 days, 28 days, monthly up to 1 year of treatment and up to 1 year followup after treatment cessation.)
- Number of participants with moderate to severe ENL as assessed by urinalysis.(Enrolment, day 10, day 17, day 28, monthly up to 1 year of treatment, and 1 month post-cessation of treatment.)
- Number of participants with moderate to severe ENL with decreased bacterial index (BI) as assessed by slit skin smear microscopy.(Enrolment and after 1 year treatment.)
- Number of participants with moderate to severe ENL as assessed by standard blood panel and basic metabolic testing.(Enrolment, day 10, day 17, day 28, monthly up to 1 year of treatment, and 1 month post-cessation of treatment.)
- Number of participants with moderate to severe ENL as assessed by serological indicators associated with ENL.(Enrolment, day 10, day 17, day 28, monthly up to 1 year of treatment, and 1 month post-cessation of treatment.)
- Number of patients with moderate to severe ENL with improved peripheral neuropathy as assessed by standardized monofilament and voluntary muscle tests.(enrolment, 10 days, 28 days, biweekly (as needed), up to 1 year treatment, up to 1 year followup after treatment cessation)
- Number of participants with moderate to severe ENL with decreased bacterial index (BI) as assessed skin histopathology.(Enrolment and after 1 year treatment.)
- Number of participants with moderate to severe ENL with improved quality of life as assessed by the World Health Organization Quality of Life tool.(Enrolment, 28 days, monthly up to 1 year of treatment and up to 1 year followup after treatment cessation.)