A Phase 2, monocentric, prospective, nonrandomized, open-label, experimental study to evaluate efficacy and safety of intrabronchial instillation of liposomal Amphotericin B in patients with simple pulmonary aspergilloma.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Pauls Stradins Clinical University Hospital

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08], Diseases [C] - Bacterial Infections and Mycoses [C01]

Decision date (initial)

2026-03-13

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Pauls Stradins Clnical University Hospital

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To evaluate the efficacy of intrabronchial instillation of liposomal Amphotericin B for the treatment of simple pulmonary aspergilloma.

Secondary objectives 4

1. To evaluate the proportion of patients with complete, partial and insufficient response to treatment
2. To evaluate the safety and tolerability of intrabronchial instillation of liposomal Amphotericin B for the treatment of simple pulmonary aspergilloma.
3. To evaluate the change in respiratory symptoms during the study treatment, using the COPD Assessment Test (CAT score)
4. To evaluate the frequency of disease progression to other forms of chronic pulmonary aspergillosis during the study treatment

Conditions and MedDRA coding

Simple aspergilloma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Diagnosis of (simple) pulmonary aspergilloma. Diagnosis of (simple) pulmonary aspergilloma is made in concordance with 2016 ESCMID/ERS guidelines for diagnosis and management of chronic pulmonary aspergillosis (Single pulmonary cavity containing a fungal ball, with serological or microbiological evidence implicating Aspergillus spp. in a non-immunocompromised patient with minor or no symptoms). The diagnosis is made based on the typical findings on chest CT scan (a rounded mass within a pre-existing pulmonary cavity, air crescent sign around the mass), combined with one of the following tests: • Positive Aspergillus Ag (Galactomannan) in bronchoalveolar lavage fluid and/or serum • Aspergillus Spp. culture positive in BAL fluid • Aspergillus-IgG antibody positive • Other diagnoses excluded - mainly pulmonary TB (sputum or BAL microscopy and cultures negative for M. tuberculosis)
2. Age 18 to 80 years
3. Surgical resection of aspergilloma not possible or not indicated, or the patient has refused surgical treatment
4. Systemic antifungal treatment not indicated or refused, or the patient has previously failed systemic antifungal treatment (currently not receiving)

Exclusion criteria 10

1. Not signed informed consent, refusal to participate in the study
2. Hypersensitivity to Amphotericin B class antifungal drugs
3. Systemic antifungal therapy indicated, or the patient is currently receiving such therapy
4. Surgical resection of pulmonary aspergilloma is indicated and is possible
5. Severe hemoptysis, requiring bronchial artery embolisation or surgical treatment
6. Pregnancy
7. More than 1 mycetoma on chest CT scan
8. Severe impairment in lung function, defined as FEV1 < 30 %
9. Contraindications to performing elective bronchoscopy: • Hemodynamically unstable patients • Hypoxaemia (SpO2 < 88% on room air) • Previously known chronic type II respiratory failure (pCO2 > 45 mmHg on arterial blood gas analysis) • Recent myocardial infarction (< 3 months)
10. Coagulation abnormalities: • Severe thrombocytopenia < 30 x10 9/L • Elevated INR > 2.50

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Relative change in lesion (aspergilloma) size, expressed as a percentage from baseline to post-treatment.

Secondary endpoints 5

1. The proportion of patients in each treatment response category based on changes in aspergilloma (mycetoma) size from baseline to Week 8 visit, categorized as complete response (complete absorption of the lesion, 100% reduction in size), partial response (≥ 50% reduction in lesion size) and insufficient response (< 50% reduction or no change in lesion size).
2. Proportion of patients with adverse events categorized by severity as mild, moderate or severe during treatment with intrabronchial instillation of liposomal Amphotericin B.
3. The proportion of patients with change shift on the COPD Assessment Test (CAT) score from baseline to post-treatment assessment visit.
4. The proportion of patients with disease progression during study treatment, defined as an increase in the size of aspergilloma (mycetoma) or progression to other forms of chronic pulmonary aspergillosis – chronic cavitary or fibrosing pulmonary aspergillosis, invasive pulmonary aspergillosis.
5. Proportion of patients with hemoptysis during study treatment, categorized by severity as mild, moderate and severe.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Pauls Stradins Clinical University Hospital

Sponsor organisation

Pauls Stradins Clinical University Hospital

Address

Pilsonu Iela 13

City

Riga

Postcode

1002

Country

Latvia

Scientific contact point

Organisation

Pauls Stradins Clinical University Hospital

Contact name

Paula Gavare

Public contact point

Organisation

Pauls Stradins Clinical University Hospital

Contact name

Paula Gavare

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 4 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications