Leprosy, MultibacillaryPhase 2NCT03384641
A Study to Evaluate the Efficacy and Safety of Bedaquiline (TMC207) in Participants With Multibacillary Leprosy
A Phase 2 study testing Bedaquiline 200 mg in people with Leprosy, Multibacillary, run by Janssen Research & Development, LLC at 2 sites in Brazil.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Leprosy, Multibacillary
- What is being tested?
- Bedaquiline 200 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 2 sitesBrazil
- Study dates
- 26 Sep 2018to 9 Jan 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
Brazil2 sites
Instituto Lauro de Souza Lima
Bauru, 17034-971
Fundacao Hospitalar de Dermatologia Tropical e Venereologia Alfredo da Matta - FUHAM
Manaus, 69065-130
About this study
Official title: An Open-Label Study to Evaluate the Efficacy and Safety of TMC207 in Subjects With Multibacillary Leprosy
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.
- Conditions
- Leprosy, Multibacillary
- Being tested
- Bedaquiline 200 mg
- Medical subject headings
- Leprosy, Multibacillary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bedaquiline 200 mgDrug
Also called TMC207
Participants will receive bedaquiline 200 mg (2*100 mg tablets) once daily for 2 weeks followed by 100 mg tablet tiw for 6 weeks with at least 48 hours between doses.
| Study group | Role | What they receive |
|---|---|---|
| Bedaquiline | Experimental | Participants will receive bedaquiline 200 (milligram) mg (2*100 mg tablets) once daily for 2 weeks followed by 100 mg tablet three times a week (tiw) for 6 weeks with at least 48 hours between doses. Receives: Drug: Bedaquiline 200 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in the Odds of Mycobacterium leprae (M. leprae) Growth in Mouse Footpads Following 8 Weeks of Treatment with Bedaquiline
Change from baseline in the odds of M. leprae growth in mouse footpads will be evaluated. M. leprae bacilli will be inoculated in footpads of mice, according to the method of Shepard. M. leprae growth will be determined by technologists trained and experienced in mouse footpad procedures, at 1 year after infection (or at mouse death or humane endpoint, if it occurs greater than or equal to [>=] 6 months after footpad inoculation). The number of footpads with positive growth (>=10^5 M. leprae) will be counted and used to determine the odds of bacterial growth.
Measured: Baseline up to Week 8
Secondary outcomes (2)
Number of Participants with Adverse Events (AEs)
An adverse event is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product.
Measured: Up to 124 Weeks
Number of Participants with AEs by Severity
Severity of adverse events will be graded by using division of microbiology and infectious diseases (DMID) adult toxicity scale to estimate grade of severity. Severity scale ranges from Grade 1 (Mild) to Grade 4 (Life-threatening). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, and Grade 4= Life-threatening.
Measured: Up to 124 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Dec 2017
- Study start26 Sep 2018
- Primary completion13 Jan 2023
- Study completion9 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Leprosy, MultibacillaryStudies of the same condition, open and under way.
- More trials run by Janssen Research & Development, LLCThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Janssen Research & Development, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Janssen Research & Development, LLC Clinical Trial, Study director, Janssen Research & Development, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03384641
- Sponsor's protocol ID
- CR108416
- Other IDs
- TMC207LEP2001
- Status last verified
- Feb 2024
- First submitted
- 20 Dec 2017
- Last updated
- 2 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Barreto J, Sammarco Rosa P, Adams L, Aguilar Z, Bakare N, Chaplan SR, Akli RD, Ernault E, Kulke S, Lounis N, Millington D, Palmer JA, Remmerie B, Wang M, Young S, Truman R, Rebello PFB. Bedaquiline Monotherapy for Multibacillary Leprosy. N Engl J Med. 2024 Dec 12;391(23):2212-2218. doi: 10.1056/NEJMoa2312928.
PubMed 39665652
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Evaluate the Efficacy and Safety of TMC207 in Subjects With Multibacillary Leprosy
- ClinicalTrials.gov ID
- NCT03384641
- Acronym / sponsor ID
- CR108416
- Conditions
- Leprosy, Multibacillary
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Janssen Research & Development, LLCIndustry (pharma, biotech, devices)
- Study start
- 26 Sep 2018 (actual)
- Primary completion
- 13 Jan 2023 (actual)
- Study completion
- 9 Jan 2024 (actual)
- First posted
- 27 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bedaquiline | Experimental | Drug: Bedaquiline 200 mgParticipants will receive bedaquiline 200 (milligram) mg (2*100 mg tablets) once daily for 2 weeks followed by 100 mg tablet three times a week (tiw) for 6 weeks with at least 48 hours between doses. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bedaquiline 200 mgAlso: TMC207Participants will receive bedaquiline 200 mg (2*100 mg tablets) once daily for 2 weeks followed by 100 mg tablet tiw for 6 weeks with at least 48 hours between doses. | Drug | Bedaquiline |
Primary outcome
Change from Baseline in the Odds of Mycobacterium leprae (M. leprae) Growth in Mouse Footpads Following 8 Weeks of Treatment with Bedaquiline
Time frame: Baseline up to Week 8
Change from baseline in the odds of M. leprae growth in mouse footpads will be evaluated. M. leprae bacilli will be inoculated in footpads of mice, according to the method of Shepard. M. leprae growth will be determined by technologists trained and experienced in mouse footpad procedures, at 1 year after infection (or at mouse death or humane endpoint, if it occurs greater than or equal to [>=] 6 months after footpad inoculation). The number of footpads with positive growth (>=10^5 M. leprae) will be counted and used to determine the odds of bacterial growth.
2 secondary outcomes
Number of Participants with Adverse Events (AEs)
Time frame: Up to 124 Weeks
Number of Participants with AEs by Severity
Time frame: Up to 124 Weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2024.
- Derived from this study: PubMed 39665652Barreto J, Sammarco Rosa P, Adams L, Aguilar Z, Bakare N, Chaplan SR, Akli RD, Ernault E, Kulke S, Lounis N, Millington D, Palmer JA, Remmerie B, Wang M, Young S, Truman R, Rebello PFB. Bedaquiline Monotherapy for Multibacillary Leprosy. N Engl J Med. 2024 Dec 12;391(23):2212-2218. doi: 10.1056/NEJMoa2312928.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Janssen Research & Development, LLC Clinical Trial study director
Janssen Research & Development, LLC
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bedaquiline 200 mg in people with Leprosy, Multibacillary, run by Janssen Research & Development, LLC at 2 sites in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in the Odds of Mycobacterium leprae (M. leprae) Growth in Mouse Footpads Following 8 Weeks of Treatment with Bedaquiline (time frame: Baseline up to Week 8). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03384641, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Feb 2024.