Beta-Lactam Containing Regimen for the Shortening of Buruli Ulcer Disease Therapy

NCT ID: NCT05169554

Last Updated: 2024-05-16

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

PHASE2

Total Enrollment

140 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-12-01

Study Completion Date

2026-05-31

Brief Summary

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Buruli ulcer (BU) is a skin Neglected Tropical Disease (NTD) that is caused by Mycobacterium ulcerans. It affects skin, soft tissues and bones causing long-term morbidity, stigma and disability. The greatest burden falls on children in sub-Saharan Africa. Treating BU requires 8-weeks with daily rifampicin and clarithromycin, wound care, and sometimes tissue grafting and surgery. Healing can take up to one year. Compliance is challenging due to socioeconomic determinants and may pose an unbearable financial burden to the household.

Recent studies led by members of this Consortium demonstrated that beta-lactams combined with rifampicin and clarithromycin are synergistic against M. ulcerans in vitro. Amoxicillin/clavulanate is oral, suitable for treatment in adults and children, and readily available with an established clinical pedigree. Its inclusion in a triple oral BU therapy has the potential of improving healing and shortening BU therapy.

The investigators propose a single blinded, randomized, controlled open label non-inferiority phase II, multi-centre trial in Benin with participants stratified according to BU category lesions and randomized in two oral regimens: (i) Standard \[RC8\]: rifampicin plus clarithromycin (RC) therapy for 8 weeks; and (ii) Investigational \[RCA4\]: standard (RC) plus amoxicillin/clavulanate (A) for 4 weeks. At least, a total of 140 patients will be recruited (70 per treatment arm), of which at least 132 will be PCR-confirmed. The primary efficacy outcome will be lesion healing without recurrence and without excision surgery 12 months after start of treatment (i.e. cure). A clinical expert panel assessing the need of excision surgery in both treatment arms will be blinded for treatment allocation in order to make objectives comparisons. Decision for excision surgery will be delayed to 14 weeks after initiation of antibiotic treatment. Secondary clinical efficacy outcomes include recurrence, treatment discontinuation and compliance rates, and the incidence of adverse effects, among others. In addition, two sub-studies will be performed: a pharmacokinetic (PK) analysis and a bacterial clearance study.

If successful, this study will create a new paradigm for BU treatment, which could inform changes in WHO policy and practice. This trial may also provide information on treatment shortening strategies for other mycobacterial infections, such as tuberculosis or leprosy.

Detailed Description

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Conditions

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Buruli Ulcer

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

BLMs4BU study is a Phase II, randomized, controlled open label non-inferiority, multi-centre trial, with two treatment arms:

* Standard \[RC8\]: rifampicin plus clarithromycin (RC) therapy for 8 weeks; and
* Investigational \[RCA4\]: standard (RC) plus amoxicillin/clavulanate (A) for 4 weeks.

Randomization will be stratified by lesion category categories I, II or III, using a fixed block size within each strata.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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RC8, Rifampicin plus Clarithromycin for 8 weeks

Rifampicin plus Clarithromycin (RC) therapy for 8 weeks

Group Type ACTIVE_COMPARATOR

Standard [RC8]: rifampicin plus clarithromycin (RC) therapy for 8 weeks.

Intervention Type DRUG

Treatment will be rifampicin (600 mg, daily) and clarithromycin (500 mg, twice daily) for 8 weeks. On the posology, dosage for rifampicin and clarithromycin will be standardized according to patient body weight following WHO guidelines. In general, for a 60 kg adult dosage will be RIF, 10 mg/kg once daily and CLA, 7.5 mg/kg twice daily.

RCA4, Rifampicin plus Clarithromycin plus Amoxicillin/clavulanate for 4 weeks.

Rifampicin plus Clarithromycin (RC) plus Amoxicillin/clavulanate (A) for 4 weeks.

Group Type EXPERIMENTAL

Investigational [RCA4]: standard (RC) plus amoxicillin/clavulanate (A) for 4 weeks.

Intervention Type DRUG

On the posology, dosage for rifampicin and clarithromycin will be standardized according to patient body weight following WHO guidelines. In general, for a 60 kg adult dosage will be RIF, 10 mg/kg once daily and CLA, 7.5 mg/kg twice daily.

Dosages for amoxicillin/clavulanate are calculated according to manufacturer indications:

Dose of amoxicillin/clavulanate 1000/125 mg twice daily, which makes a total of 2000/250 mg/day, for patients over 40 kg, and 22.5/5.6 mg/kg twice daily, which makes a total of 45/11.25 mg/kg/day, for those equal and below 40 kg. For children, posology will be adapted to the age of the patient according to drug manufacturer indications. Frequency of AMX/CLV administration will match that of CLA, twice daily.

Interventions

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Standard [RC8]: rifampicin plus clarithromycin (RC) therapy for 8 weeks.

Treatment will be rifampicin (600 mg, daily) and clarithromycin (500 mg, twice daily) for 8 weeks. On the posology, dosage for rifampicin and clarithromycin will be standardized according to patient body weight following WHO guidelines. In general, for a 60 kg adult dosage will be RIF, 10 mg/kg once daily and CLA, 7.5 mg/kg twice daily.

Intervention Type DRUG

Investigational [RCA4]: standard (RC) plus amoxicillin/clavulanate (A) for 4 weeks.

On the posology, dosage for rifampicin and clarithromycin will be standardized according to patient body weight following WHO guidelines. In general, for a 60 kg adult dosage will be RIF, 10 mg/kg once daily and CLA, 7.5 mg/kg twice daily.

Dosages for amoxicillin/clavulanate are calculated according to manufacturer indications:

Dose of amoxicillin/clavulanate 1000/125 mg twice daily, which makes a total of 2000/250 mg/day, for patients over 40 kg, and 22.5/5.6 mg/kg twice daily, which makes a total of 45/11.25 mg/kg/day, for those equal and below 40 kg. For children, posology will be adapted to the age of the patient according to drug manufacturer indications. Frequency of AMX/CLV administration will match that of CLA, twice daily.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

All patients (both genders) with a new very likely or likely (WHO scoring criteria) clinical diagnosis of BU (all categories: I, II, III) and normal electrocardiogram (ECG) at baseline giving informed consent will be included in the study, as agreed by study site treatment team led by the lead clinicians.

Exclusion Criteria

* Children \< 5 years and adults \>70 years.
* Children in foster care.
* Patients weighing less than 11 kilograms.
* Pregnancy positive (urine test: beta-HCG positive).
* Previous treatment of Buruli ulcer, tuberculosis or leprosy with at least one of the study drugs.
* Patients with diagnose leprosy or tuberculosis disease.
* Hypersensitivity to at least one of the study drugs or to any of the excipients.
* History of a severe immediate hypersensitivity reaction (e.g. anaphylaxis) to another beta-lactam agent (e.g. a cephalosporin, carbapenem or monobactam).
* History of jaundice/hepatic impairment due to amoxicillin/clavulanic acid or rifampicin.
* Patients with history of treatment with macrolide or quinolone antibiotics, anti-tuberculosis medication, or immuno-modulatory drugs including corticosteroids within one month.
* Patients currently receiving treatment with any drugs likely to interact with the study medications, i.e. anticoagulants, cyclosporine, phenytoin or phenobarbitone. Users of oral contraceptives should be notified that such contraceptive is less reliable if taken with rifampicin; additional (mechanical) contraceptive methods will be discussed with the study participant (Appendix 5).
* Patients with HIV co-infection.
* Patients with QTc prolongation \>450 ms on ECG or on other medication known to prolong the QTc interval. In this case, if suspected of BU disease, patients will be offered 8-weeks rifampicin plus streptomycin therapy.
* Patients unable to take oral medication or having gastrointestinal disease likely to interfere with drug absorption.
* Patients with history or having current clinical signs of ascites, jaundice, myasthenia gravis, renal dysfunction (known or suspected), diabetes mellitus, and severe immune compromise, or evidence of tuberculosis, or leprosy; terminal illness (e.g., metastasized cancer), haematological malignancy, chronic liver disease, abnormal liver function test and coronary artery disease or any other condition that would preclude enrolment into the study in the study physician's opinion.
* Evidence of a clinically significant (as judged by the Investigator) condition or abnormality (other than the indication being studied) that might compromise safety or the interpretation of trial efficacy or safety endpoints
* Patients with known or suspected bowel strictures who cannot tolerate clarithromycin.
* Patients with a mental health condition that is likely to interfere with compliance with the study protocol in the opinion of the study physician.
* Patients (or parent/legal representative) who are not willing to give informed consent or withdrawal of consent.
Minimum Eligible Age

5 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Universidad de Zaragoza

OTHER

Sponsor Role collaborator

Fondation Raoul Follereau

UNKNOWN

Sponsor Role collaborator

Université d'Abomey-Calavi

OTHER

Sponsor Role collaborator

Instituto de Salud Carlos III

OTHER_GOV

Sponsor Role collaborator

Fundación Anesvad

UNKNOWN

Sponsor Role collaborator

Tres Cantos Open Lab Foundation

UNKNOWN

Sponsor Role collaborator

GlaxoSmithKline

INDUSTRY

Sponsor Role collaborator

Fundacion Agencia Aragonesa para la Investigacion y Desarrollo (ARAID)

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Santiago Ramón-García

Role: STUDY_DIRECTOR

Fundacion Agencia Aragonesa para la Investigacion y Desarrollo (ARAID)

Locations

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Centre de Dépistage et de Traitement de l'Ulcère de Buruli (CDTUB) (Centers for Detection and Treatment of Buruli ulcer), Allada

Allada, , Benin

Site Status RECRUITING

Centre de Dépistage et de Traitement de l'Ulcère de Buruli (CDTUB) (Centers for Detection and Treatment of Buruli ulcer), Lalo

Lalo, , Benin

Site Status RECRUITING

Centre de Dépistage et de Traitement de l'Ulcère de Buruli (CDTUB) (Centers for Detection and Treatment of Buruli ulcer), Pobè

Pobè, , Benin

Site Status RECRUITING

Countries

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Benin

Central Contacts

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Christian Johnson

Role: CONTACT

0022996221132

Facility Contacts

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Gilbert Ayelo

Role: primary

Godwin Kpoton

Role: primary

Ronald Gnimavo

Role: primary

References

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O'Brien DP, Athan E, Blasdell K, De Barro P. Tackling the worsening epidemic of Buruli ulcer in Australia in an information void: time for an urgent scientific response. Med J Aust. 2018 Apr 16;208(7):287-289. doi: 10.5694/mja17.00879. No abstract available.

Reference Type BACKGROUND
PMID: 29642808 (View on PubMed)

Revill WD, Morrow RH, Pike MC, Ateng J. A controlled trial of the treatment of Mycobacterium ulcerans infection with clofazimine. Lancet. 1973 Oct 20;2(7834):873-7. doi: 10.1016/s0140-6736(73)92005-9. No abstract available.

Reference Type BACKGROUND
PMID: 4126917 (View on PubMed)

Espey DK, Djomand G, Diomande I, Dosso M, Saki MZ, Kanga JM, Spiegel RA, Marston BJ, Gorelkin L, Meyers WM, Portaels F, Deming MS, Horsburgh CR Jr. A pilot study of treatment of Buruli ulcer with rifampin and dapsone. Int J Infect Dis. 2002 Mar;6(1):60-5. doi: 10.1016/s1201-9712(02)90138-4.

Reference Type BACKGROUND
PMID: 12044304 (View on PubMed)

Guarner J. Buruli Ulcer: Review of a Neglected Skin Mycobacterial Disease. J Clin Microbiol. 2018 Mar 26;56(4):e01507-17. doi: 10.1128/JCM.01507-17. Print 2018 Apr.

Reference Type BACKGROUND
PMID: 29343539 (View on PubMed)

Thangaraj HS, Adjei O, Allen BW, Portaels F, Evans MR, Banerjee DK, Wansbrough-Jones MH. In vitro activity of ciprofloxacin, sparfloxacin, ofloxacin, amikacin and rifampicin against Ghanaian isolates of Mycobacterium ulcerans. J Antimicrob Chemother. 2000 Feb;45(2):231-3. doi: 10.1093/jac/45.2.231.

Reference Type BACKGROUND
PMID: 10660507 (View on PubMed)

World Health Organization. Treatment of Mycobacterium ulcerans infection. 2012

Reference Type BACKGROUND

Boeree MJ, Diacon AH, Dawson R, Narunsky K, du Bois J, Venter A, Phillips PP, Gillespie SH, McHugh TD, Hoelscher M, Heinrich N, Rehal S, van Soolingen D, van Ingen J, Magis-Escurra C, Burger D, Plemper van Balen G, Aarnoutse RE; PanACEA Consortium. A dose-ranging trial to optimize the dose of rifampin in the treatment of tuberculosis. Am J Respir Crit Care Med. 2015 May 1;191(9):1058-65. doi: 10.1164/rccm.201407-1264OC.

Reference Type BACKGROUND
PMID: 25654354 (View on PubMed)

Hu Y, Liu A, Ortega-Muro F, Alameda-Martin L, Mitchison D, Coates A. High-dose rifampicin kills persisters, shortens treatment duration, and reduces relapse rate in vitro and in vivo. Front Microbiol. 2015 Jun 23;6:641. doi: 10.3389/fmicb.2015.00641. eCollection 2015.

Reference Type BACKGROUND
PMID: 26157437 (View on PubMed)

Omansen TF, Stienstra Y, van der Werf TS. Treatment for Buruli ulcer: the long and winding road to antimicrobials-first. Cochrane Database Syst Rev. 2018 Dec 17;12(12):ED000128. doi: 10.1002/14651858.ED000128. No abstract available.

Reference Type BACKGROUND
PMID: 30556580 (View on PubMed)

Ramon-Garcia S, Gonzalez Del Rio R, Villarejo AS, Sweet GD, Cunningham F, Barros D, Ballell L, Mendoza-Losana A, Ferrer-Bazaga S, Thompson CJ. Repurposing clinically approved cephalosporins for tuberculosis therapy. Sci Rep. 2016 Sep 28;6:34293. doi: 10.1038/srep34293.

Reference Type BACKGROUND
PMID: 27678056 (View on PubMed)

Arenaz-Callao MP, Gonzalez Del Rio R, Lucia Quintana A, Thompson CJ, Mendoza-Losana A, Ramon-Garcia S. Triple oral beta-lactam containing therapy for Buruli ulcer treatment shortening. PLoS Negl Trop Dis. 2019 Jan 28;13(1):e0007126. doi: 10.1371/journal.pntd.0007126. eCollection 2019 Jan.

Reference Type BACKGROUND
PMID: 30689630 (View on PubMed)

Rolinson GN. Forty years of beta-lactam research. J Antimicrob Chemother. 1998 Jun;41(6):589-603. doi: 10.1093/jac/41.6.589.

Reference Type BACKGROUND
PMID: 9687097 (View on PubMed)

Diacon AH, van der Merwe L, Barnard M, von Groote-Bidlingmaier F, Lange C, Garcia-Basteiro AL, Sevene E, Ballell L, Barros-Aguirre D. beta-Lactams against Tuberculosis--New Trick for an Old Dog? N Engl J Med. 2016 Jul 28;375(4):393-4. doi: 10.1056/NEJMc1513236. Epub 2016 Jul 13. No abstract available.

Reference Type BACKGROUND
PMID: 27433841 (View on PubMed)

Ma Z, Lienhardt C, McIlleron H, Nunn AJ, Wang X. Global tuberculosis drug development pipeline: the need and the reality. Lancet. 2010 Jun 12;375(9731):2100-9. doi: 10.1016/S0140-6736(10)60359-9. Epub 2010 May 18.

Reference Type BACKGROUND
PMID: 20488518 (View on PubMed)

Ramon-Garcia S, Ng C, Anderson H, Chao JD, Zheng X, Pfeifer T, Av-Gay Y, Roberge M, Thompson CJ. Synergistic drug combinations for tuberculosis therapy identified by a novel high-throughput screen. Antimicrob Agents Chemother. 2011 Aug;55(8):3861-9. doi: 10.1128/AAC.00474-11. Epub 2011 May 16.

Reference Type BACKGROUND
PMID: 21576426 (View on PubMed)

Sarpong-Duah M, Frimpong M, Beissner M, Saar M, Laing K, Sarpong F, Loglo AD, Abass KM, Frempong M, Sarfo FS, Bretzel G, Wansbrough-Jones M, Phillips RO. Clearance of viable Mycobacterium ulcerans from Buruli ulcer lesions during antibiotic treatment as determined by combined 16S rRNA reverse transcriptase /IS 2404 qPCR assay. PLoS Negl Trop Dis. 2017 Jul 3;11(7):e0005695. doi: 10.1371/journal.pntd.0005695. eCollection 2017 Jul.

Reference Type BACKGROUND
PMID: 28671942 (View on PubMed)

Kibadi K, Boelaert M, Fraga AG, Kayinua M, Longatto-Filho A, Minuku JB, Mputu-Yamba JB, Muyembe-Tamfum JJ, Pedrosa J, Roux JJ, Meyers WM, Portaels F. Response to treatment in a prospective cohort of patients with large ulcerated lesions suspected to be Buruli Ulcer (Mycobacterium ulcerans disease). PLoS Negl Trop Dis. 2010 Jul 6;4(7):e736. doi: 10.1371/journal.pntd.0000736.

Reference Type BACKGROUND
PMID: 20625556 (View on PubMed)

Sarfo FS, Phillips RO, Zhang J, Abass MK, Abotsi J, Amoako YA, Adu-Sarkodie Y, Robinson C, Wansbrough-Jones MH. Kinetics of mycolactone in human subcutaneous tissue during antibiotic therapy for Mycobacterium ulcerans disease. BMC Infect Dis. 2014 Apr 15;14:202. doi: 10.1186/1471-2334-14-202.

Reference Type BACKGROUND
PMID: 24731247 (View on PubMed)

Frimpong M, Agbavor B, Duah MS, Loglo A, Sarpong FN, Boakye-Appiah J, Abass KM, Dongyele M, Amofa G, Tuah W, Frempong M, Amoako YA, Wansbrough-Jones M, Phillips RO. Paradoxical reactions in Buruli ulcer after initiation of antibiotic therapy: Relationship to bacterial load. PLoS Negl Trop Dis. 2019 Aug 26;13(8):e0007689. doi: 10.1371/journal.pntd.0007689. eCollection 2019 Aug.

Reference Type BACKGROUND
PMID: 31449522 (View on PubMed)

Roltgen K, Pluschke G. Buruli Ulcer: History and Disease Burden. 2019 Apr 30. In: Pluschke G, Roltgen K, editors. Buruli Ulcer: Mycobacterium Ulcerans Disease [Internet]. Cham (CH): Springer; 2019. Available from http://www.ncbi.nlm.nih.gov/books/NBK553836/

Reference Type BACKGROUND
PMID: 32091710 (View on PubMed)

Omansen TF, van der Werf TS, Phillips RO. Antimicrobial Treatment of Mycobacterium ulcerans Infection. 2019 Apr 30. In: Pluschke G, Roltgen K, editors. Buruli Ulcer: Mycobacterium Ulcerans Disease [Internet]. Cham (CH): Springer; 2019. Available from http://www.ncbi.nlm.nih.gov/books/NBK553822/

Reference Type BACKGROUND
PMID: 32091697 (View on PubMed)

Phillips RO, Robert J, Abass KM, Thompson W, Sarfo FS, Wilson T, Sarpong G, Gateau T, Chauty A, Omollo R, Ochieng Otieno M, Egondi TW, Ampadu EO, Agossadou D, Marion E, Ganlonon L, Wansbrough-Jones M, Grosset J, Macdonald JM, Treadwell T, Saunderson P, Paintsil A, Lehman L, Frimpong M, Sarpong NF, Saizonou R, Tiendrebeogo A, Ohene SA, Stienstra Y, Asiedu KB, van der Werf TS; study team. Rifampicin and clarithromycin (extended release) versus rifampicin and streptomycin for limited Buruli ulcer lesions: a randomised, open-label, non-inferiority phase 3 trial. Lancet. 2020 Apr 18;395(10232):1259-1267. doi: 10.1016/S0140-6736(20)30047-7. Epub 2020 Mar 12.

Reference Type BACKGROUND
PMID: 32171422 (View on PubMed)

Wadagni AC, Barogui YT, Johnson RC, Sopoh GE, Affolabi D, van der Werf TS, de Zeeuw J, Kleinnijenhuis J, Stienstra Y. Delayed versus standard assessment for excision surgery in patients with Buruli ulcer in Benin: a randomised controlled trial. Lancet Infect Dis. 2018 Jun;18(6):650-656. doi: 10.1016/S1473-3099(18)30160-9. Epub 2018 Apr 5.

Reference Type BACKGROUND
PMID: 29605498 (View on PubMed)

Sato T. A further look at the Cochran-Mantel-Haenszel risk difference. Control Clin Trials. 1995 Oct;16(5):359-61. doi: 10.1016/0197-2456(95)00004-6. No abstract available.

Reference Type BACKGROUND
PMID: 8582154 (View on PubMed)

Saez-Lopez E, Millan-Placer AC, Lucia A, Ramon-Garcia S. Amoxicillin/clavulanate in combination with rifampicin/clarithromycin is bactericidal against Mycobacterium ulcerans. PLoS Negl Trop Dis. 2024 Apr 4;18(4):e0011867. doi: 10.1371/journal.pntd.0011867. eCollection 2024 Apr.

Reference Type DERIVED
PMID: 38573915 (View on PubMed)

Johnson RC, Saez-Lopez E, Anagonou ES, Kpoton GG, Ayelo AG, Gnimavo RS, Mignanwande FZ, Houezo JG, Sopoh GE, Addo J, Orford L, Vlasakakis G, Biswas N, Calderon F, Della Pasqua O, Gine-March A, Herrador Z, Mendoza-Losana A, Diez G, Cruz I, Ramon-Garcia S. Comparison of 8 weeks standard treatment (rifampicin plus clarithromycin) vs. 4 weeks standard plus amoxicillin/clavulanate treatment [RC8 vs. RCA4] to shorten Buruli ulcer disease therapy (the BLMs4BU trial): study protocol for a randomized controlled multi-centre trial in Benin. Trials. 2022 Jul 8;23(1):559. doi: 10.1186/s13063-022-06473-9.

Reference Type DERIVED
PMID: 35804454 (View on PubMed)

Other Identifiers

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TC281

Identifier Type: -

Identifier Source: org_study_id

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