Rezafungin for treatment of chronic pulmonary aspergillosis (CPA) in adults with limited treatment options

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Mundipharma Research Limited

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Bacterial Infections and Mycoses [C01]

Trial duration

25 Jul 2025 → ongoing

Decision date (initial)

2025-07-30

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-519575-26-00

ClinicalTrials.gov

NCT06794554

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy, Others

To evaluate the effectiveness of 6 months’ rezafungin in the treatment of CPA in subjects with limited treatment options according to clinical and radiological response as measured by the St George's Respiratory Questionnaire (SGRQ), weight, and computed tomography (CT) imaging

Secondary objectives 3

1. To evaluate the effectiveness of 3- and 6-months’ rezafungin in the treatment of CPA in subjects with limited treatment options
2. To evaluate the safety and tolerability of rezafungin
3. [Exploratory Objective:] To evaluate the pharmacokinetics of rezafungin in subjects with CPA

Conditions and MedDRA coding

Chronic pulmonary aspergillosis (CPA)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Males or females ≥18 years of age (≥19 years in Republic of Korea), willing and able to provide written informed consent.
2. Patients with an established diagnosis of CPA according to ESCMID/ERS/ECMM criteria (2016) which must be present for ≥3 months, unable to receive systemic azole antifungal therapy, and where antifungal treatment is indicated according to the opinion of the investigator due to radiological changes consistent with CPA progression OR high burden of symptoms.
3. Female subjects of child-bearing potential must agree to use one highly effective contraceptive methods of birth control, or sexual abstinence, while participating in this study and for 30 days after the last dose of study drug. Male subjects must be vasectomized, abstain from heterosexual intercourse, or agree to use barrier contraception, unless the partner is >2 years post-menopausal or otherwise sterile, and agree not to donate sperm while participating in the study and for 120 days from the last dose of study drug.
4. [A full list of inclusion criteria is included in the study protocol.]

Exclusion criteria 7

1. Subjects with invasive aspergillosis or simple aspergilloma, known or suspected hypersensitivity to rezafungin, any echinocandin or any of their excipients, and current participation in another interventional treatment trial with an investigational agent.
2. Recent use of an investigational medicinal product (IMP) within 28 days or 5 half-lives of the IMP, whichever is greater, administration of any other echinocandin or IV antifungal treatment within 3 months of screening, administration of ≥15mg prednisolone daily (or equivalent corticosteroid) for at least 3 weeks within 4 weeks of screening, OR a peripheral neutrophil count <0.5x10[to the power of 9]/L.
3. Acute respiratory infections considered inadequately treated, subjects with active malignancy who are undergoing chemotherapy or radiation therapy.
4. Other conditions or laboratory abnormality that, in the opinion of the Investigator, would put the subject at unacceptable risk or interfere with the assessments included in the study.
5. Subjects with NCI-CTCAE Grade 2 or higher ataxia, tremor, motor neuropathy, or sensory neuropathy and/or a history of severe ataxia, tremor, or neuropathy or a diagnosis of multiple sclerosis or a movement disorder, therapy with a known severe neurotoxic medication or with a known moderate neurotoxic medication in a patient with ataxia, tremor, motor neuropathy, or sensory neuropathy of NCI-CTCAE Grade 1 or higher.
6. Pregnant or lactating females.
7. [A full list of exclusion criteria is included in the study protocol.]

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. [Clinical 1:] SGRQ - a patient reported outcome questionnaire (improvement, stable or deterioration of quality of life)
2. [Clinical 2:] Weight (weight gain / loss)
3. [Radiological:] CT thoracic scan to assess radiological improvement (CT changes assessed by a central imaging vendor)

Secondary endpoints 7

1. Change in Aspergillus IgG levels, weight, SGRQ score, Respiratory Symptom Score and EQ-5D-5L score at 3 and 6 months from baseline
2. Need for antibiotic treatment, change in antibiotic treatment or change in steroid dose
3. Absence of persistently positive respiratory sample for Aspergillus spp.
4. Change in frequency of echinocandin resistant isolates
5. Hospitalisations attributed to deterioration in any category
6. Safety and tolerability of up to 6 months’ treatment; this will be assessed by nature and severity of adverse events (AEs) and serious adverse events (SAEs), physical examinations and laboratory tests
7. [Exploratory endpoint:] Rezafungin plasma concentrations

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Mundipharma Research Limited

Sponsor organisation

Mundipharma Research Limited

Address

191 Science Park, Milton Road Milton Road

City

Cambridge

Postcode

CB4 0GW

Country

United Kingdom

Scientific contact point

Organisation

Mundipharma Research Limited

Contact name

Clinical Trial Information Desk

Public contact point

Organisation

Mundipharma Research Limited

Contact name

Clinical Trial Information Desk

Third parties 10

Locations

8 EU/EEA countries · 25 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 144 files

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

Application history

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