Clinical trial NCT06481163: Telacebec (T) Treatment in Adults With Buruli Ulcer (BU).
Official title: A Phase 2 Multicentre, Open-label Study to Evaluate the Efficacy, Safety and Tolerability, and Pharmacokinetics of 28 Days Telacebec Treatment in Adult Participants With Buruli Ulcer.
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Buruli Ulcer
Keywords: lesion
ulcer
Lead sponsor: Barwon Health
Sponsor class: OTHER_GOV
Collaborators: name: Global Alliance for TB Drug Development
First posted: 2024-07-01
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open Label
Primary purpose: TREATMENT

Brief summary: The goal of this interventional clinical trial is to determine if treatment with 4 weeks of telacebac (T) will completely heal lesions in participants with Buruli ulcer (BU) by 52 weeks after treatment initiation, without relapse and/or surgery. Males and females age 18 and older will be included.

* Participants will attend visits every week during treatment (4 weeks)
* Thereafter they will be followed every 2 weeks until week 24 followed by visits as weeks 30, 40 and 52.
* Dependent on site requirements, there may be follow up visits on site or remotely.

Detailed description: This is an open label single arm (telacebec), multi-centre, clinical trial. Eligible participants with clinically diagnosed World Health Organization (WHO) category I lesions and category II lesions ≤10 cm cross-sectional diameter and category III lesions where multiple and all lesions are ≤10 cm cross-sectional diameter, confirmed by polymerase chain reaction (PCR) or culture for presence of Mycobacterium ulcerans infection, will receive telacebec 300 mg orally once daily for 28 days with food.

Participants who meet entry criteria and give consent will attend a baseline enrolment visit (Day 1), then visits every week for 4 weeks during treatment (weeks 1, 2, 3 and 4). Thereafter they will be followed up every 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 be established to review efficacy and safety data. Enrolment and enrolled participants will continue the study whilst the DRC reviews are ongoing.

Eligibility criteria: Major Inclusion Criteria:

* Clinical diagnosis of BU WHO categories, single or multiples:

  1. I
  2. II with a cross-sectional diameter \< 10 cm
  3. III category III lesions where multiple and all are \< 10 cm cross-sectional diameter
* Positive PCR or culture for confirmation of presence of mycobacterium ulcerans (MU).

Major Exclusion Criteria:

* Participants with the following known or suspected medical conditions:

  1. 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.
  2. History or current ascites, jaundice, myasthenia gravis, clinically significant renal dysfunction \[estimated glomerular filtration rate (eGFR) \< 50 mls/min\], uncontrolled diabetes mellitus \[glycated heaemoglobin (HbA1C )\> 10%\], and severe immune compromise (e.g., immunosuppressive drugs after organ transplant)
  3. History of previous BU (except current infection)
* 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.

Primary outcomes:
measure: Rate of complete lesion healing by 52 weeks from treatment initiation.
timeFrame: 52 weeks from treatment initiation
Secondary outcomes:
measure: Complete lesion healing rate at 52 weeks from treatment initiation.
timeFrame: 52 weeks from treatment initiation
measure: Median time to healing
timeFrame: 52 weeks from treatment initiation
measure: Recurrence rate within 52 weeks from treatment initiation
timeFrame: 52 weeks from treatment initiation
measure: Treatment failure rate within 52 weeks from treatment initiation
timeFrame: 52 weeks from treatment initiation
measure: Paradoxical response rate with 52 weeks from treatment initiation
timeFrame: 52 weeks from treatment initiation
Other outcomes:
Locations:
city: Geelong
country: Australia
facility: Barwon Health
state: Victoria
city: Melbourne
country: Australia
facility: Royal Melbourne Hospital
Study officials:
affiliation: Barwon Health
name: Daniel O&amp;#39;Brien
role: PRINCIPAL_INVESTIGATOR
References: