Efficacy, Safety and Immunogenicity Evaluation of MTBVAC in Newborns in Sub-Saharan Africa

NCT ID: NCT04975178

Last Updated: 2025-02-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

7120 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-10-17

Study Completion Date

2029-02-28

Brief Summary

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The objective of this project is to demonstrate safety, immunogenicity and improved efficacy of the new live attenuated M. tuberculosis vaccine called MTBVAC in a Phase 3 efficacy trial in HIV-uninfected infants born to HIV-infected and HIV-uninfected mothers as compared to standard of care BCG vaccination. The proposal builds upon a group of TB vaccine development partners in Europe and sub-Saharan Africa established in a previous EDCTP-supported project. It creates an expanded consortium of clinical trial partners for the optimal implementation of a large infant efficacy trial of MTBVAC in high TB incidence settings. New capacity for efficacy trials in infants will be a valuable resource for the TB vaccine development community. The proposal will create a network of institutions in three TB endemic African countries with enhanced laboratory capacity to conduct TB vaccine immunology studies and to bio-bank samples to discover immune correlates of vaccine-mediated protection.

Detailed Description

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A new effective tuberculosis (TB) vaccine is essential to achieve World Health Organization End TB goals and eliminate TB by 2050. The optimal long-term strategy would be a combination of serial mass campaigns in adults, coupled with universal newborn vaccination. Newborns are the only human population without prior mycobacterial exposure in TB endemic countries and we hypothesize that live attenuated mycobacterial vaccines will offer better protection to this naïve population compared to adults.

The objective of this project is to demonstrate safety, immunogenicity and improved efficacy of the new live attenuated M. tuberculosis vaccine called MTBVAC in a Phase 3 efficacy trial in HIV-uninfected infants born to HIV-infected and HIV-uninfected mothers as compared to standard of care BCG vaccination. The proposal builds upon a group of TB vaccine development partners in Europe and sub-Saharan Africa established in a previous EDCTP-supported project. It creates an expanded consortium of clinical trial partners for the optimal implementation of a large infant efficacy trial of MTBVAC in high TB incidence settings. New capacity for efficacy trials in infants will be a valuable resource for the TB vaccine development community. The proposal will create a network of institutions in three TB endemic African countries with enhanced laboratory capacity to conduct TB vaccine immunology studies and to bio-bank samples to discover immune correlates of vaccine-mediated protection.

MTBVAC is a novel TB vaccine candidate based on an attenuated M. tuberculosis clinical isolate of the Euro-American lineage. Attenuation is based on two independent, stable genetic deletions of the genes phoP and fadD26 coding for two major virulence factors, the transcription factor PhoP and the cell-wall lipids PDIM, respectively. The hypothesis is that MTBVAC will provide improved protection, as individuals latently infected with live M.tuberculosis have an 80% lower chance of developing TB, and as MTBVAC contains most of the genes deleted from BCG and presents a wider collection of antigens to the host immune system. Preclinical studies in different animal models indicated that MTBVAC is safe and is able to induce an improved protection compared to BCG.

Phase 1 studies showed that MTBVAC was safe and immunogenic in naïve adults and newborns, and evoked an immune response that exceeded the magnitude of BCG-induced immune responses. Larger dose-defining Phase 2a studies in newborns and in adults at extended dose-ranges to confirm these findings will be finalised in early 2021, and allow selection of a vaccine dose to progress into the proposed multi-centre efficacy trial in infants.

Conditions

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Tuberculosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

The study is designed to include the following:

1. Safety population (all study participants randomized to receive BCG or MTBVAC in a 1:1 fashion): All participants from the 4 South African sites (approx. 7000); all 120 participants from Madagascar \& Senegal.
2. Reactogenicity population:

\- First 1000 from the 4 South African sites and all 120 in Madagascar and Senegal.
3. Immunogenicity population (subset of the safety population - total 460):

\- First 60 HU and 25 HEU participants from the South African sites (total 340) and all 120 from Madagascar \& Senegal.
4. Efficacy population (subset of the safety population - all participants from the South African sites):

* First 1000, including 240 HU and 100 HEU newborns.
* Additional 6000 (not part of reactogenicity \& immunogenicity population). Senegal \& Madagascar participants are excluded from the efficacy population. DSMB will periodically review cumulative safety and reactogenicity data.
Primary Study Purpose

PREVENTION

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
MTBVAC and BCG vaccine will be prepared, and allocation concealed by the site pharmacist. All other site staff will be blinded to vaccine allocation, throughout the follow-up period. Data pertaining to the MTBVAC vaccine and to BCG control will be collected in an observer-blinded manner. Blinding will be maintained throughout the vaccination and follow-up portions of the vaccine trial. No set of individual codes will be held at Biofabri's Headquarters. Biofabri's Headquarters will be able to access the individual randomization code from the SATVI Pharmacy randomization register. The code will be broken by the Site's Pharmacist (Study Contact for Emergency Code Break) only in the case of medical events that the investigator/physician in charge of the participant feels cannot be treated without knowing the identity of the study vaccine(s). The Site's Pharmacist is responsible for unblinding the treatment assignment in accordance with specified time frames for expedited reporting of SAEs.

Study Groups

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MTBVAC

Both MTBVAC and BCG vaccines are administered by intradermal route in the left deltoid region. One 0.05 mL reconstituted dose of MTBVAC will be defined based on the phase IIa results.

MTBVAC is manufactured by Biofabri. MTBVAC is formulated (1.5 - 8.5 x104 CFU/dose, 1.5 - 8.5 x105 CFU/dose or 1.5 - 8.5 x106 CFU/dose (to be selected) and presented as a lyophilised pellet in 20 dose vials (0.05 mL/dose, after reconstitution with sterile water for injection). MTBVAC vaccine will be released and distributed by BIOFABRI, and imported to the sites following approval by the local regulatory authority. MTBVAC vaccine must be stored at +2°C to +8°C. Reconstituted MTBVAC vaccine must be stored at +2ºC to +8ºC and administered as soon as possible, within 4 hours of reconstitution. A single vaccine vial will be used for each participant.

Group Type EXPERIMENTAL

MTBVAC

Intervention Type BIOLOGICAL

MTBVAC is a novel TB vaccine candidate based on an attenuated M. tuberculosis clinical isolate of the Euro-American lineage. Attenuation is based on two independent, stable genetic deletions of the genes phoP and fadD26 coding for two major virulence factors, the transcription factor PhoP and the cell-wall lipids PDIM, respectively. We hypothesize that MTBVAC will provide improved protection, as individuals latently infected with live M.tuberculosis have an 80% lower chance of developing TB, and as MTBVAC contains most of the genes deleted from BCG and presents a wider collection of antigens to the host immune system. Preclinical studies in different animal models indicated that MTBVAC is safe and is able to induce an improved protection compared to BCG.

BCG

BCG is a live attenuated M. bovis strain developed 100 years ago and is used as a preventive vaccine against tuberculosis. It is administered at birth.

One 0.05 mL reconstituted dose of BCG contains 2.5 x 105 CFU. The control vaccine will be the BCG vaccine available and recommended in South Africa at time of the trial.

BCG vaccine produced by AJ Biologics (formerly Staten Serum Institute) is the only BCG vaccine (Danish strain) currently licensed for routine use in South Africa. The recommended BCG injection volume for newborn infants (0.05 mL, after reconstitution with BCG diluent) contains approximately 2.5 x 105 CFU (range 1-4 x 105 CFU). BCG vaccine vials should be stored in the site pharmacy at 2-8ºC.

Group Type ACTIVE_COMPARATOR

BCG

Intervention Type BIOLOGICAL

BCG is a live attenuated M. bovis strain developed 100 years ago and is used as a preventive vaccine against tuberculosis. It is administered at birth.

Interventions

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MTBVAC

MTBVAC is a novel TB vaccine candidate based on an attenuated M. tuberculosis clinical isolate of the Euro-American lineage. Attenuation is based on two independent, stable genetic deletions of the genes phoP and fadD26 coding for two major virulence factors, the transcription factor PhoP and the cell-wall lipids PDIM, respectively. We hypothesize that MTBVAC will provide improved protection, as individuals latently infected with live M.tuberculosis have an 80% lower chance of developing TB, and as MTBVAC contains most of the genes deleted from BCG and presents a wider collection of antigens to the host immune system. Preclinical studies in different animal models indicated that MTBVAC is safe and is able to induce an improved protection compared to BCG.

Intervention Type BIOLOGICAL

BCG

BCG is a live attenuated M. bovis strain developed 100 years ago and is used as a preventive vaccine against tuberculosis. It is administered at birth.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Male or female newborns within seven days of birth.
* Written informed maternal consent, including permission to access maternal antenatal, postnatal, and infant medical records.
* Infant participants and their caregivers available for trial follow-up and display the willingness and capacity to comply with trial procedures.
* Newborns must be in good general health during pregnancy and delivery, as assessed by medical history and targeted physical examination.
* Birth weight ≥ 2450 grams.
* Apgar score at 5 minutes ≥ 7.
* A maternal HIV test result (rapid test, enzyme-linked immunosorbent assay (ELISA), or Polymerase chain reaction (PCR)) taken within 30 days of delivery, or within seven days post-partum must be available and documented if HIV uninfected. If the mother is HIV infected, then she must be on antiretroviral (ARV) therapy as per in-country guidelines with a viral load of \<50 copies/mL (within six months of labour).
* Estimated gestational age ≥ 37 weeks.
* Mother has not participated in a clinical trial within three months prior to the infant's birth.
* Mother has never participated in a TB vaccine trial before.
* Infant may not participate in any other clinical trials.

Exclusion Criteria

Receipt of BCG vaccination prior to enrolment.

* Significant antenatal, intrapartum, or postpartum complications that may affect the health of the newborn.
* Skin condition, bruising or birth mark at the intended injection site.
* Maternal HIV test (rapid test, ELISA, or PCR) result not available.
* HIV exposed Newborn's HIV PCR result positive or not available.
* Maternal history of TB during pregnancy.
* History of close/household contact with a TB patient, antenatal or postnatal, whether maternal, other family member or another household member who has not yet completed TB treatment.
* Clinically suspected neonatal sepsis.
* Any severe congenital malformation.
* History or evidence of any systemic disease on examination, or any illness that in the opinion of the Investigator may interfere with the evaluation of the safety and immunogenicity of the vaccine. Neonatal jaundice not considered clinically significant is not an exclusion.
Minimum Eligible Age

5 Minutes

Maximum Eligible Age

7 Days

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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TuBerculosis Vaccine Initiative

OTHER

Sponsor Role collaborator

University of Cape Town

OTHER

Sponsor Role collaborator

Institut Pasteur de Madagascar

OTHER

Sponsor Role collaborator

Biomedical Research Center EPLS

OTHER

Sponsor Role collaborator

Universidad de Zaragoza

OTHER

Sponsor Role collaborator

University of Stellenbosch

OTHER

Sponsor Role collaborator

University of KwaZulu

OTHER

Sponsor Role collaborator

Wits Health Consortium (Pty) Ltd

OTHER

Sponsor Role collaborator

Biofabri, S.L

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Mark Hatherill

Role: PRINCIPAL_INVESTIGATOR

University of Cape Town, Faculty of Health Sciences

Locations

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South African Tuberculosis Initiative, Brewelskloof Hospital

Worcester, Western Cape, South Africa

Site Status RECRUITING

Countries

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South Africa

Central Contacts

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Ingrid Murillo Jelsbak

Role: CONTACT

+34 986 33 04 00 ext. 307

Andrea García Silva

Role: CONTACT

+34 986 33 04 00 ext. 309

Facility Contacts

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Michele Tameris, MD

Role: primary

+27 23 346 5400

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Related Links

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https://www.who.int/publications/i/item/9789240061729

WHO, Global Tuberculosis Report 2022. \[Online\] https://www.who.int/publications /i/item/9789240061729 \[accessed on 27 June 2023\].

https://cdn.who.int/media/docs/default-source/immunization/product-and-delivery-research/framework_working_document_july_2023.pdf?sfvrsn=224b6151_3&download=true

WHO preferred products characteristics for TB vaccines 2018. \[Online\] https://apps.who.int/iris/ bitstream/handle/10665/273089/ WHO-IVB-18.06-eng.pdf?ua=1 \[accessed on 05 May 2021\].

https://rsc.niaid.nih.gov/sites/default/files/daidsgradingcorrectedv21.pdf

Division of AIDS (DAIDS) Table for Grading the Severity of Adult and Pediatric Adverse Events, Corrected Version 2.1, July 2017. \[Online\] https://rsc.niaid.nih.gov/clinical-research-sites/daids-adverse-event-grading-tables \[Accessed on 05 May 2021\].

Other Identifiers

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MTBVACN3

Identifier Type: -

Identifier Source: org_study_id

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