A Phase 1 interventional study of Amphotericin B Liposomal 50 MG and Placebo in Invasive Pulmonary Aspergillosis, sponsored by Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal. Completed at 1 site in Spain. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-06-15.
Sponsored by Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal · Phase 1, Interventional, and Treatment
The investigators aim to assess the safety and efficacy of nebulized liposomal amphotericin B (ALN) as a complementary therapy to the usual systemic treatment in patients with invasive pulmonary aspergillosis and the utility of a non-routine test as a surrogate marker of efficacy. To this end, a 3-year phase I, prospective, randomized and controlled clinical trial will be carried out in a single center, in patients with proven or probable pulmonary aspergillosis receiving routine systemic treatment. Participants will be randomized ( 1: 1) to receive ALN, 25 mg or nebulizer injection water 3 times a week, for 6 weeks. The primary objective is the safety of ALN in this scenario, including clinical tolerance and pharmacokinetic studies. Secondary objectives are presented as: a) clinical efficacy, using the following criteria: complete response, partial response, stability and progression or death, on week 12; b) microbiological efficacy, using culture, galactomannan, BDGlucan and Aspergillus PCR in induced sputum on week +6; and c) to explore the utility of the SUV ("standardized uptake value") index in PET-CT performed on week +6 in relation to a baseline PET-CT as a surrogate marker of response. The administration of ALN and placebo will be carried out by eFlowR vibrating membrane electronic nebulizers. To carry out the study, the following visits will be made: baseline, week 1,2,3,4,5,6 (efficacy and safety evaluation), 9 and 12 (overall evaluation).
Phase of the clinical trial Phase I trial (pilot study), prospective, randomised, controlled trial using a drug under conditions of use not approved for the form of administration.
It is a Phase I study since the primary objective includes tolerance analysis, safety and pharmacokinetic and distribution studies of nebulized amphotericin, but when administered to patients it also carries out the preliminary assessment of the safety and efficacy of the treatment.
Primary and secondary variables
Primary variable:
Reduction of >20% of post-nebulized FVC and FEV1 in relation to values obtained before nebulization.
In each visit made during the nebulized therapy period (week 1 visit in view of week 6) the following parameters will be objectified: before and after nebulization:
Secondary variables:
201 studies on the registry are indexed under Aspergillosis; 22 are open to participants now.
This study's enrollment of 13 is below the median of 50 across 106 interventional studies indexed under Aspergillosis.
Browse Aspergillosis studies →Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal is the lead sponsor of 82 studies on the registry; 21 are open to participants now.
Counted across the registry records on this site, refreshed daily.
In the absence of proven aspergillosis, patients who meet all 3 conditions below are considered likely and also eligible for inclusion:
Exclusion Criteria:
Amphotericin B.
Drug: Amphotericin B Liposomal 50 MG
Sterile water for injection.
Other: Placebo
Amphotericin B 50 mg powder for infusion diluted in 12 ml of sterile water. It will be administered by nebulized route 25 mg (6 ml), 3 times a week, for 6 weeks.
Also known as: Ambisome
Sterile water for injection. It will be administered by nebulized route: 6 ml, 3 times a week for 6 weeks.
Reduction of >20% of FVC postnebulization compared to values before amphotericin nebulization
FVC will be measured with the Micro Spirometer (Micro Medical Ltd; Rochester UK) 3 times before and after nebulization and the higher value will be recorded.
Time frame: During amphotericin treatment: week 1 to week 6
Reduction of >20% of FEV1 postnebulization compared to values before amphotericin nebulization
FEV1 will be measured with the Micro Spirometer (Micro Medical Ltd; Rochester UK) 3 times before and after nebulization and the higher value will be recorded.
Time frame: During amphotericin treatment: week 1 to week 6
Fr Resp/min (Breathing rate per minute)
Change in Fr Resp/min values postnebulization compared to values before nebulization
Time frame: During amphotericin treatment: week 1 to week 6
Sat 02 (%) (Percentage of oxygen saturation)
Change in Sat 02 (%) values postnebulization compared to values before nebulization
Time frame: During amphotericin treatment: week 1 to week 6
Fr Card/min (Heart rate per minute)
Change in Card/min values postnebulization compared to values before nebulization
Time frame: During amphotericin treatment: week 1 to week 6
Number of events observed at pulmonary level.
Symptomatic cough, bronchospasm (auscultation), dyspnea or shortness of breath, need for bronchodilator treatment.
Time frame: During amphotericin treatment: week 1 to week 6
Pharmacokinetics. Concentrations of amphotericin
Amphotericin concentrations in bronchoalveolar lavage and in plasma.
Time frame: Week 6
Radiological response
Evaluated by PET-TC
Time frame: Week 6
Microbiological response.
Evaluated in bronchoalveolar lavage (BAL)
Time frame: Week 6
Plan to share: Yes — Identifiing candidates prospectively according inclusion and exclusion criteria
Supporting information: Study protocol, Sap, Icf, Csr, Analytic code
No publications or documents are linked to this record.
This study is completed, as verified in Jan 2021. You cannot join it, but the record below documents what was studied.
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Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal