Clinical trial NCT03384641: A Study to Evaluate the Efficacy and Safety of Bedaquiline (TMC207) in Participants With Multibacillary Leprosy
Official title: An Open-Label Study to Evaluate the Efficacy and Safety of TMC207 in Subjects With Multibacillary Leprosy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Leprosy, Multibacillary
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2017-12-27
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the efficacy of an 8-week bedaquiline monotherapy regimen in participants with treatment-naive, multibacillary (MB) leprosy.


Eligibility criteria: Inclusion Criteria:

* Participant has: a) multibacillary (MB) leprosy, defined as 6 or more skin lesions or extensive confluent lesions or diffuse skin involvement, and is either borderline lepromatous or polar lepromatous, as determined using Ridley and Jopling classification system; and b) has a bacteriological index of \>=4+ from the lesion biopsy obtained at screening, and a bacteriological index of \>= 1+ from each of 4 slit skin smear assessments taken at screening
* Otherwise healthy on the basis of physical examination, medical history, vital signs, and 12-lead electrocardiogram (ECG) performed at screening. If there are abnormalities, they must be consistent with the underlying illness in the study population. This determination must be recorded in the participant's source documents and initialed by the investigator
* Otherwise healthy on the basis of clinical laboratory tests performed at screening. If the results of the serum chemistry panel, including liver enzymes, other specific tests, blood coagulation, hematology, or urinalysis are outside the normal reference ranges, the participant may be included only if the investigator judges the abnormalities or deviations from normal to be not clinically significant or to be appropriate and reasonable for the population under study. This determination must be recorded in the participant's source documents and initialed by the investigator
* a man must agree not to donate sperm throughout treatment with TMC207 and for 3 months after treatment is stopped
* Willing and able to adhere to the prohibitions, restrictions, and long-term follow up requirements specified in this protocol
* If a woman is of childbearing potential, must be practising a highly effective method of birth control (failure rate of \<1% per year when used consistently and correctly) before entry, and must also agree to use a barrier contraceptive method (that is, male or female condom, diaphragm or cervical cap) plus spermicide. participant must also agree to continue to use a highly effective method of contraception plus a barrier method throughout treatment with TMC207 and until 6 months after treatment is stopped

Exclusion Criteria:

* Has experienced or is experiencing a lepra reaction requiring treatment with a prohibited therapy
* Has a contraindication limiting the implementation of a medically accepted MB leprosy regimen
* a) Has ever received pharmacotherapy for leprosy; b) has ever been treated with a drug that is a component of the primary World Health Organization (WHO) regimen for the treatment of MB leprosy (example, dapsone, rifampicin, clofazimine). Short-term (\<=2 weeks) pharmacotherapy with any antibiotic that could be used as a second line treatment for leprosy (eg, of the macrolide, quinolone, or tetracycline class) is acceptable as long as the last administration occurred \>=4 weeks before first dose of study drug (TMC207)
* Has a concomitant infection that requires an additional systemic antimicrobial agent
* Has tuberculosis (TB), as determined by medical history and chest x-ray
* Is a woman who is pregnant, or breast-feeding, or planning to become pregnant while enrolled in this study or within 6 months after the last dose of study drug

Primary outcomes:
measure: Change from Baseline in the Odds of Mycobacterium leprae (M. leprae) Growth in Mouse Footpads Following 8 Weeks of Treatment with Bedaquiline
timeFrame: Baseline up to Week 8
Secondary outcomes:
measure: Number of Participants with Adverse Events (AEs)
timeFrame: Up to 124 Weeks
measure: Number of Participants with AEs by Severity
timeFrame: Up to 124 Weeks
Other outcomes:
Locations:
city: Bauru
country: Brazil
facility: Instituto Lauro de Souza Lima
city: Manaus
country: Brazil
facility: Fundacao Hospitalar de Dermatologia Tropical e Venereologia Alfredo da Matta - FUHAM
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: