Evaluating the effectiveness of bedaquiline for treating multidrug-resistant tuberculosis in children and adolescents
A Phase 2, Open-label, Multicenter, Single-arm Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Anti-mycobacterial Activity of TMC207 in Combination With a Background Regimen (BR) of Multidrug Resistant Tuberculosis (MDR-TB) Medications for the Treatment of Children and Adolescents 0 Months to <18 Years of Age Who Have Confirmed or Probable Pulmonary MDR-TB
This study is testing if bedaquiline can safely help children and teenagers with multidrug-resistant tuberculosis feel better when used with other TB medications.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 0 Months to 18 Years |
| Sex | All |
| Sponsor | Janssen Research & Development, LLC Industry-sponsored |
| Locations | 11 sites (Maputo and 10 other locations) |
| Trial ID | NCT02354014 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety, tolerability, pharmacokinetics, and anti-mycobacterial activity of bedaquiline (TMC207) in children and adolescents diagnosed with pulmonary multidrug-resistant tuberculosis (MDR-TB). It is an open-label, multicenter Phase 2 study that includes a screening phase, a 24-week treatment phase where participants receive bedaquiline alongside a background regimen of MDR-TB medications, and a 96-week follow-up phase. The study will enroll participants in four age-based cohorts, with safety monitored throughout the trial to guide subsequent cohort enrollment and dosing.
Who should consider this trial
Good fit: Ideal candidates for this study are children and adolescents aged from birth to less than 18 years who have confirmed or probable pulmonary multidrug-resistant tuberculosis.
Not a fit: Patients who are not diagnosed with multidrug-resistant tuberculosis or are outside the specified age range may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide an effective treatment option for children and adolescents suffering from multidrug-resistant tuberculosis.
How similar studies have performed: Other studies have shown promise in using bedaquiline for treating multidrug-resistant tuberculosis, indicating a potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participant must be a boy or girl, aged from birth (0 months) to less than (\<) 18 years at screening. Participants in Cohort 4 who are \<6 months of age at screening, gestational age at birth had to be greater than or equal to (\>=) 37 weeks * Participant must weigh \>3 kilogram (kg) at baseline and be within the 5th and 95th percentiles (inclusive) for the participant's age, based on the World Health Organization (WHO) child growth standards; Body Mass Index (BMI) for age. In Cohorts 3 and 4, weight for height/length may be used instead of BMI for age according to the local standard of care. Per WHO guidance, for participants aged \< 2 years in Cohort 4, length will be used to calculate the BMI instead of height * For Cohorts 1 and 2 only: Heterosexually active girls may participate if they are of non-childbearing potential, or if they are using effective birth control methods and are willing to continue practicing birth control methods throughout Multidrug Resistant Tuberculosis (MDR-TB) treatment and for 6 months after stopping TMC207 treatment, or if they are non-heterosexually active or willing to practice sexual abstinence throughout MDR-TB treatment * For Cohorts 1 and 2 only: Boys who engage in sexual activity that could lead to pregnancy of the female partner must use at minimum a male condom throughout MDR-TB treatment and for 3 months after stopping TMC207 treatment * Participant must have confirmed or probable (clinically diagnosed or presumed) pulmonary and/or non-severe extrapulmonary MDR-TB, including pre-extensively drug-resistant TB (pre- extensively drug resistant \[XDR\]-TB) or XDR-TB infection, based on the case definitions of pediatric pulmonary and non-severe extrapulmonary TB as described in the International (WHO) guidelines and in accordance with the local standard of care * Participants must be starting the initial MDR-TB treatment at Day 1 or have started an MDR-TB treatment within 12 weeks of Day 1 and are willing to modify it if necessary to an acceptable MDR-TB regimen for use with TMC207 * Participant must be willing to permanently discontinue RMP from at least 7 days before the baseline visit * Participant or legally acceptable representative must consent/assent to human Immunodeficiency virus (HIV) testing of the participant. The mother must also consent to testing of her own HIV status, if the potential participant is a child aged \<2 years, or if the participant is \>= 2 years old and being breastfed or was breastfed within the last 8 weeks before screening, unless the mother had HIV test performed within 1 month prior to screening and documentation of HIV status can be provided. When documented HIV-positive status is available prior to screening for participants in Cohort 4 or their mother, HIV testing for the participant and mother is not required Exclusion Criteria: * Participant has a clinically significant active medical condition or the presence of any concomitant severe illness or rapidly deteriorating health condition, including immune deficiency (except HIV infection), which in the opinion of the investigator would prevent appropriate participation in the study, or that would make implementation of the protocol or interpretation of the study results difficult, or otherwise make the subject a poor candidate for a clinical study * Participant is a girl who is pregnant, or breast-feeding, or planning to become pregnant while enrolled in this study or within 6 months after stopping TMC207 treatment * Participant has known or presumed forms of extrapulmonary TB, other than: Lymphadenopathy (peripheral nodes or isolated mediastinal mass without significant airway compression); Pleural effusion or pleural fibrotic lesions * Participant has a significant cardiac arrhythmia that requires medication or risk factors for Torsade de Pointes, example heart failure, hypokalemia, known personal or family history of Long QT Syndrome, and untreated hypothyroidism
Where this trial is running
Maputo and 10 other locations
- Hospital Geral da Polana Caniço — Maputo, Mozambique (Recruiting)
- De La Salle Health Sciences Institute- DLSUMC — Dasmariñas, Philippines (Recruiting)
- Lung Center Of The Philippines — Quezon City, Philippines (Completed)
- Silang Specialists Medical Center — San Vincent Silang, Philippines (Recruiting)
- First Moscow State Medical University n.a. I.M. Sechenov — Moscow, Russia (Completed)
- THINK: Tuberculosis & HIV Investigative Network — Durban, South Africa (Completed)
- Sizwe Tropical Diseases Hospital — Johannesburg, South Africa (Recruiting)
- Wits Health Consortium — Port Elizabeth, South Africa (Recruiting)
- Desmond Tutu TB Centre — Stellenboch, South Africa (Recruiting)
- Makerere University Lung Institute — Kampala, Uganda (Terminated)
- State Institute Of Phthisiology And Pulmonology N.A. F.G. Yanovskiy Of Ams Ukraine — Kiev, Ukraine (Completed)
Study contacts
- Study coordinator: Study Contact
- Email: Participate-In-This-Study1@its.jnj.com
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.