Amphotericin B for Non-HIV Cryptococcal Meningitis Patients

NCT ID: NCT06178627

Last Updated: 2023-12-21

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

ENROLLING_BY_INVITATION

Clinical Phase

PHASE4

Total Enrollment

250 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-13

Study Completion Date

2026-08-31

Brief Summary

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Cryptococcus neoformans and C. gatti are important causes of central nervous system (CNS) infections with significant mortality, remaining a great public health challenge worldwide. Commonly seen as an opportunistic infection in adults with HIV/AIDS, cryptococcal meningitis (CM) accounts for 15% of HIV-related mortality globally \[1\]. In addition, a growing number of non-HIV CM patients have been observed in recent years with fatality approaching 30% in some areas \[2,3\]. It occurs in both those with natural or iatrogenic immunosuppression, as well as the apparently immunocompetent individuals. Approximately 65-70% of non-HIV CM patients were without any predisposing factors, particularly in the East Asia \[4,5\]. With the increasing number of hematopoietic stem cell transplantation, solid organ transplantation recipients and administration of immunosuppressive and corticosteroids agents, this illness will assume even greater public health significance.

Current Infectious Disease Society of America (IDSA) guideline suggest the use of combination antifungal therapy: normal dose amphotericin (0.7-1mg/kg/day) combined with flucytosine for a minimum of 4 weeks, followed by fluconazole (600-800 mg/day) for a minimum of 10 weeks in total for HIV patients \[6\]. However, for non-HIV and immunocompetent patients, the treatment remains controversial. IDSA guideline recommended that the treatment of non-HIV patients could refer to the treatment of HIV patients. That is, amphotericin B combined with flucytosine is still administered in the induction period. However, as amphotericin B have nonspecific effect on ergosterol, it has strong side effects (hepatorenal toxicity, electrolyte disorder, anemia, ventricular fibrillation, etc.). Therefore, the dose of amphotericin B may not be appropriate for Asian patients due to the different drug metabolism and pharmacokinetic. In the prospective studies of Bennett\[7\] and Dismuke\[8\], low dose amphotericin B (0.3 mg/kg/d) combined with flucytosine achieved response rates of 66% and 85% at 6 weeks, respectively. A similar conclusion was also extracted from a large multicenter retrospective study that low dose amphotericin B (\<0.7 mg/kg/d) combined with flucytosine for a minimum of 2 weeks, followed by fluconazole could achieve a response rate of 84%, indicating that the efficacy of low dose amphotericin B (\< 0.7 mg/kg/d) may be equivalent with normal dose in non-HIV patients. Therefore, we plan to conduct a prospective, multicenter, open-label randomized controlled study to compare the efficacy and safety of normal dose amphotericin B (0.7 mg/kg/ d) and low dose amphotericin B (0.5 mg/kg/d) in the initial antifungal treatment for non-HIV cryptococcal meningitis patients.

Detailed Description

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Conditions

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Cryptococcal Meningitis Antifungal Agents

Keywords

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Cryptococcal Meningitis Antifungal agents amphotericin B non-HIV

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Normal dose amphotericin B (0.7 mg/kg/d)

Study regimen 1: amphotericin B 0.7 mg/kg/day i.v. plus flucytosine for 4 weeks

Group Type ACTIVE_COMPARATOR

Amphotericin B 0.7 mg/kg/day i.v. combined with flucytosine four times per day orally for the first 4 weeks.

Intervention Type DRUG

Different amphotericin B dosage

Low dose amphotericin B (0.5 mg/kg/d)

Amphotericin B 0.5 mg/kg/day i.v. plus flucytosine for 4 weeks

Group Type EXPERIMENTAL

Amphotericin B 0.5 mg/kg/day i.v. combined with flucytosine four times per day orally for the first 4 weeks.

Intervention Type DRUG

Different amphotericin B dosage

Interventions

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Amphotericin B 0.5 mg/kg/day i.v. combined with flucytosine four times per day orally for the first 4 weeks.

Different amphotericin B dosage

Intervention Type DRUG

Amphotericin B 0.7 mg/kg/day i.v. combined with flucytosine four times per day orally for the first 4 weeks.

Different amphotericin B dosage

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Age more than 18 years
2. HIV antibody negative
3. Cryptococcal meningitis defined as a syndrome consistent with CM and one or more of: 1) positive CSF India ink (budding encapsulated yeasts), 2) C.neoformans cultured from CSF or blood, 3) positive cryptococcal antigen Lateral Flow Antigen Test (LFA) in CSF, 4) positive brain tissue representing Cryptococcus
4. Having no severe immunocompromised conditions
5. Informed consent to participate given by patient or acceptable representative

Exclusion Criteria

1. Previously cryptococcal disease
2. Currently receiving treatment for cryptococcal meningitis and having received ≥72 hours of anti-cryptococcal meningitis therapy in 96 hours
3. Creatinine clearance lower than 80 ml/min
4. Liver dysfunction (defined as ALT or AST \> 2×ULN and bilirubin \> 1.5×ULN, or ALT or AST \> 3×ULN, or bilirubin \> 2×ULN)
5. Liver cirrhosis or chronic liver failure
6. Pregnancy or breast-feeding
7. Known allergy to study drugs
8. Failure to consent - the patient, or if they are incapacitated, their responsible relative, declines to enter the study
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Huashan Hospital

OTHER

Sponsor Role lead

Responsible Party

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Li-Ping Zhu

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Huashan Hospital

Shanghai, Shanghai Municipality, China

Site Status

The Fourth People's Hospital of Nanning

Nanning, , China

Site Status

Countries

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China

References

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Lortholary O, Dromer F, Mathoulin-Pelissier S, Fitting C, Improvisi L, Cavaillon JM, Dupont B; French Cryptococcosis Study Group. Immune mediators in cerebrospinal fluid during cryptococcosis are influenced by meningeal involvement and human immunodeficiency virus serostatus. J Infect Dis. 2001 Jan 15;183(2):294-302. doi: 10.1086/317937. Epub 2000 Dec 8.

Reference Type BACKGROUND
PMID: 11110651 (View on PubMed)

Jiang YK, Wu JQ, Zhao HZ, Wang X, Wang RY, Zhou LH, Yip CW, Huang LP, Cheng JH, Chen YH, Li H, Zhu LP, Weng XH. Genetic influence of Toll-like receptors on non-HIV cryptococcal meningitis: An observational cohort study. EBioMedicine. 2018 Nov;37:401-409. doi: 10.1016/j.ebiom.2018.10.045. Epub 2018 Oct 23.

Reference Type BACKGROUND
PMID: 30366814 (View on PubMed)

Haddow LJ, Colebunders R, Meintjes G, Lawn SD, Elliott JH, Manabe YC, Bohjanen PR, Sungkanuparph S, Easterbrook PJ, French MA, Boulware DR; International Network for the Study of HIV-associated IRIS (INSHI). Cryptococcal immune reconstitution inflammatory syndrome in HIV-1-infected individuals: proposed clinical case definitions. Lancet Infect Dis. 2010 Nov;10(11):791-802. doi: 10.1016/S1473-3099(10)70170-5.

Reference Type BACKGROUND
PMID: 21029993 (View on PubMed)

Chau TT, Mai NH, Phu NH, Nghia HD, Chuong LV, Sinh DX, Duong VA, Diep PT, Campbell JI, Baker S, Hien TT, Lalloo DG, Farrar JJ, Day JN. A prospective descriptive study of cryptococcal meningitis in HIV uninfected patients in Vietnam - high prevalence of Cryptococcus neoformans var grubii in the absence of underlying disease. BMC Infect Dis. 2010 Jul 9;10:199. doi: 10.1186/1471-2334-10-199.

Reference Type BACKGROUND
PMID: 20618932 (View on PubMed)

Thwaites GE, Nguyen DB, Nguyen HD, Hoang TQ, Do TT, Nguyen TC, Nguyen QH, Nguyen TT, Nguyen NH, Nguyen TN, Nguyen NL, Nguyen HD, Vu NT, Cao HH, Tran TH, Pham PM, Nguyen TD, Stepniewska K, White NJ, Tran TH, Farrar JJ. Dexamethasone for the treatment of tuberculous meningitis in adolescents and adults. N Engl J Med. 2004 Oct 21;351(17):1741-51. doi: 10.1056/NEJMoa040573.

Reference Type BACKGROUND
PMID: 15496623 (View on PubMed)

Beardsley J, Wolbers M, Kibengo FM, Ggayi AB, Kamali A, Cuc NT, Binh TQ, Chau NV, Farrar J, Merson L, Phuong L, Thwaites G, Van Kinh N, Thuy PT, Chierakul W, Siriboon S, Thiansukhon E, Onsanit S, Supphamongkholchaikul W, Chan AK, Heyderman R, Mwinjiwa E, van Oosterhout JJ, Imran D, Basri H, Mayxay M, Dance D, Phimmasone P, Rattanavong S, Lalloo DG, Day JN; CryptoDex Investigators. Adjunctive Dexamethasone in HIV-Associated Cryptococcal Meningitis. N Engl J Med. 2016 Feb 11;374(6):542-54. doi: 10.1056/NEJMoa1509024.

Reference Type BACKGROUND
PMID: 26863355 (View on PubMed)

Day JN, Chau TTH, Wolbers M, Mai PP, Dung NT, Mai NH, Phu NH, Nghia HD, Phong ND, Thai CQ, Thai LH, Chuong LV, Sinh DX, Duong VA, Hoang TN, Diep PT, Campbell JI, Sieu TPM, Baker SG, Chau NVV, Hien TT, Lalloo DG, Farrar JJ. Combination antifungal therapy for cryptococcal meningitis. N Engl J Med. 2013 Apr 4;368(14):1291-1302. doi: 10.1056/NEJMoa1110404.

Reference Type BACKGROUND
PMID: 23550668 (View on PubMed)

Zhao HZ, Wang RY, Wang X, Jiang YK, Zhou LH, Cheng JH, Huang LP, Harrison TS, Zhu LP. High dose fluconazole in salvage therapy for HIV-uninfected cryptococcal meningitis. BMC Infect Dis. 2018 Dec 12;18(1):643. doi: 10.1186/s12879-018-3460-7.

Reference Type BACKGROUND
PMID: 30541454 (View on PubMed)

Perfect JR, Dismukes WE, Dromer F, Goldman DL, Graybill JR, Hamill RJ, Harrison TS, Larsen RA, Lortholary O, Nguyen MH, Pappas PG, Powderly WG, Singh N, Sobel JD, Sorrell TC. Clinical practice guidelines for the management of cryptococcal disease: 2010 update by the infectious diseases society of america. Clin Infect Dis. 2010 Feb 1;50(3):291-322. doi: 10.1086/649858.

Reference Type BACKGROUND
PMID: 20047480 (View on PubMed)

Chen J, Varma A, Diaz MR, Litvintseva AP, Wollenberg KK, Kwon-Chung KJ. Cryptococcus neoformans strains and infection in apparently immunocompetent patients, China. Emerg Infect Dis. 2008 May;14(5):755-62. doi: 10.3201/eid1405.071312.

Reference Type BACKGROUND
PMID: 18439357 (View on PubMed)

Zhu LP, Wu JQ, Xu B, Ou XT, Zhang QQ, Weng XH. Cryptococcal meningitis in non-HIV-infected patients in a Chinese tertiary care hospital, 1997-2007. Med Mycol. 2010 Jun;48(4):570-9. doi: 10.3109/13693780903437876.

Reference Type BACKGROUND
PMID: 20392150 (View on PubMed)

Brizendine KD, Baddley JW, Pappas PG. Predictors of mortality and differences in clinical features among patients with Cryptococcosis according to immune status. PLoS One. 2013;8(3):e60431. doi: 10.1371/journal.pone.0060431. Epub 2013 Mar 26.

Reference Type BACKGROUND
PMID: 23555970 (View on PubMed)

Rajasingham R, Smith RM, Park BJ, Jarvis JN, Govender NP, Chiller TM, Denning DW, Loyse A, Boulware DR. Global burden of disease of HIV-associated cryptococcal meningitis: an updated analysis. Lancet Infect Dis. 2017 Aug;17(8):873-881. doi: 10.1016/S1473-3099(17)30243-8. Epub 2017 May 5.

Reference Type BACKGROUND
PMID: 28483415 (View on PubMed)

Other Identifiers

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Huashan Hospital ID

Identifier Type: -

Identifier Source: org_study_id