A Phase 2 Multicentre, Open-label Study to Evaluate the Efficacy, Safety and Tolerability, and Pharmacokinetics of 14 -28 Days Telacebec Treatment in Adult Participants With Buruli Ulcer.
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 4
- 主要终点
- Rate of complete lesion healing by 52 weeks from treatment initiation.
研究概览
简要总结
The goal of this interventional clinical trial is to demonstrate the complete BU lesion healing rate by 52 weeks after treatment initiation, without relapse and/or curative intent excision surgery, after 10-28 days of telacebec (T) treatment. Males and females age 18 and older will be included.
• Participants will attend visits every 2 weeks during treatment and thereafter every 2 weeks until week 24 Thereafter they will be followed by visits at weeks 30, 40, and 52. From week 10 to week 52, if the lesion has healed, follow-up visits may be remote.
详细描述
This is an open label single arm (telacebec), phase 2 multi-centre, clinical trial. Eligible participants with clinically diagnosed BU, confirmed by polymerase chain reaction (PCR) or culture presence of MU infection will receive telacebec 300 mg orally once daily for 14-28 days with food (protocol V2.0 - 6.0) or telacebec 100 mg orally once daily for 10 days with food (protocol V7.0).
Participants who meet entry criteria and give consent will attend a baseline enrolment visit (Day 1), then visits every week for 2 weeks until week 24, followed by visits at weeks 30, 40, and 52.
BU lesion management will be provided to all trial participants. In case of participant early withdrawal from the trial during or after the treatment period, participants will be treated per the investigational site and/or country BU treatment guidelines.
A Data Review Committee (DRC) will review efficacy and safety data. Enrolment and enrolled participants will continue the study whilst the DRC reviews are ongoing.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Major Inclusion Criteria:
- •Clinical diagnosis of BU WHO categories, single or multiples:
- •Category I- a single lesion < 5 cm Category II - a single lesion with cross-sectional diameter ≤ 10 cm Category III - where multiple and all lesions are ≤ 10 cm cross-sectional diameter
- •Positive PCR or culture for confirmation of presence of mycobacterium ulcerans (MU).
- •Aged 18 years and older, except for those included in exclusion below.
排除标准
- •Participants with the following known or suspected medical conditions/criteria:
- •Any non BU related condition where participation in the study, as judged by the Investigator, could compromise the well-being of the participant or prevent, limit or confound protocol specified treatment and assessments.
- •History or current ascites, jaundice, myasthenia gravis, clinically significant renal dysfunction [estimated glomerular filtration rate (eGFR) < 30 mls/min],
- •Compromised immune status (e.g. uncontrolled HIV-infection)
- •History of previous BU in the previous 12 months (except current infection)
- •Participants who fulfill both of the following criteria:
- •Aged ≥ 60 years
- •Weight ≥ 85 kg
- •Planned/expected to require curative intent excision surgery, defined as excision of lesion which may include surrounding macroscopically healthy tissue with the aim of helping to sterilize the wound rather than improve wound healing alone, for their BU during the entire study period. Simple removal of necrotic slough and skin grafting is considered normal wound care and allowed.
研究组 & 干预措施
300 mg Telacebec per day
Telacebec will be administered orally with food or within 30 minutes after food once daily for 14-28 consecutive days.
干预措施: Telacebec (Drug)
结局指标
主要结局
Rate of complete lesion healing by 52 weeks from treatment initiation.
时间窗: 52 weeks from treatment initiation
Ulcerated Lesions: Healing and re-epithelialisation of ulcerated area with stable scar formation. Non-ulcerated Lesions: resolution of induration of skin. Complete healing rate will be summarized as percentage with corresponding Clopper-Pearson 95% confidence interval.
次要结局
- Median time to healing(52 weeks from treatment initiation)
- Treatment failure rate within 52 weeks from treatment initiation(52 weeks from treatment initiation)
- Median time to healing(52 weeks from treatment initiation)
- Safety and Tolerability(Time of first dose to 28 days after last dose of telacebec.)
- Recurrence rate within 52 weeks from treatment initiation(52 weeks from treatment initiation)