RecruitingNCT05802264ABCIUpdated Feb 12, 2025

Study to Assess Amphotericin B Cystetic for Inhalation (ABCI) Doses in Healthy Volunteers & People with Cystic Fibrosis

A Phase 1 interventional study of ABCI and Placebo in Cystic Fibrosis, sponsored by Cystetic Medicines, Inc.. Recruiting at 5 sites in 2 countries. Open to participants aged 16 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-02-12.

Sponsored by Cystetic Medicines, Inc. · Phase 1, Interventional, and Treatment

From the registry’s dates

Primary completion was expected by Dec 2025, 10 months ago, but the record still lists the study as recruiting.
Started Mar 2023; still recruiting 3 years 6 months later.

Phase: Phase 1
Study type: Interventional
Enrollment: 108
Allocation: Randomized
Ages: 16 Years and older
Sex: All

Study summary

This is a 3-part, single-ascending dose Phase 1a randomized, double-blind, placebo-controlled study in healthy volunteers (Part A) and multiple-ascending dose Phase 1a randomized, double-blind, placebo-controlled study in healthy volunteers (Part B), and a Phase 1b open-label study in subjects with CF (Part C) to assess the safety, tolerability, PK, and preliminary efficacy of ABCI. Subjects will be evaluated for eligibility during Screening within 30 days prior to Day 1 (Randomization; Visit 3). In Parts A and B, eligible healthy volunteers may be enrolled in the study and randomly allocated to treatment with ABCI or placebo as described below. In Part C, eligible subjects with CF may be enrolled in the study and receive treatment with ABCI as described below. Approximately 72 healthy subjects total will be randomized to 9 cohorts (48 subjects in 6 cohorts in Part A, 24 subjects in 3 cohorts in Part B) and approximately 36 subjects with CF will receive the low dose, medium dose (2 sentinel subjects), or high dose of ABCI in Part C.

Conditions studied

Cystic Fibrosis

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In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's planned enrollment of 108 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

This is the only study on the registry with Cystetic Medicines, Inc. as lead sponsor.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 16 Years and older
Sexes eligible: All
Accepts healthy volunteers: Yes

Inclusion criteria

Part A and Part B: Each subject must meet the following criteria to be enrolled in Part A and Part B of this study.

Subject has signed, dated, and received a copy of the IRB/IEC-approved written ICF.
Subject is male or female aged ≥18 to ≤55 years.
Subject has a BMI between 18 and 32 kg/m2
Subject has an FEV1 of >90% of predicted normal value
Subject has normal or clinically acceptable physical examination, vital signs, clinical laboratory values, and ECG at Screening.
Female subjects must be of non-childbearing potential or male/female subjects of childbearing potential agree to use highly effective contraception/preventive exposure measures

Part C: Each subject must meet the following criteria to be enrolled in Part C of this study.

Subject has signed, dated, and received a copy of the IRB/IEC-approved written ICF.
Age 16 years or older
Confirmed diagnosis of CF, including sweat chloride >60 mM.
Subject is either: Being treated with an approved CFTR modulator for at least 28 days prior to Screening, or Not being treated with a CFTR modulator
FEV1:
For subjects on CFTR modulators: FEV1 ≥40% and ≤90%
For subjects not on CFTR modulators: FEV1 ≥40% and ≤100%
Stable CF disease and treatment regiment
Female subjects must be of non-childbearing potential or male/female subjects of childbearing potential agree to use highly effective contraception/preventive exposure measures

Exclusion criteria

Exclusion Criteria:

Part A and Part B: Any subject who meets any of these criteria must be excluded from Part A and Part B of this study:

Subject has history or evidence of any clinically significant pulmonary condition
Subject has history or evidence of any clinically significant diseases or conditions
Subject has history of malignancy of any type
Subject has an active COVID-19 infection within 4 weeks
Subject is positive for human immunodeficiency virus antibodies, hepatitis B surface antigen, or hepatitis C antibodies, or has a positive QuantiFERON®-tuberculosis Gold (QFT-G) test for tuberculosis at Screening
Subject has a self-reported lower respiratory tract infection within 6 weeks
Subject has evidence of any active or suspected bacterial, viral, fungal or parasitic infections within the past 4 weeks
A subject who is an active smoker or a former smoker
Subject has history of alcohol or drug abuse in the past year
Subject has tested positive for drugs (including cannabis), nicotine/cotinine, and/or alcohol use at Screening, subject has consumed alcohol within 24 hours prior to Visit 3
Subject has participated in any clinical study or had been treated with any investigational drugs within 28 days or 5 half-lives
Female subject who is pregnant or breastfeeding.
Subject has any episode of paradoxical bronchospasm in the past 12 months.
Subject has pacemaker; is not in sinus rhythm; has a corrected QT interval (QTc; using Fridericia's [QTcF] formula) of >450 ms (for males) and >470 ms (for females); or has a left bundle branch block or bifascicular block.
Subject has a pulse \<40 or >100 bpm; systolic blood pressure >140 mmHg, or diastolic blood pressure >90 mmHg at Screening
Subject has Type I or II diabetes requiring medication.
Subject has received any vaccine within 30 days prior to Day 1.
Subject has received any of the following immunosuppressant therapies within 6 months prior to Screening: imatinib, ambrisentan, azathioprine, cyclophosphamide, cyclosporine A, bosentan, or methotrexate.
Subject has received any antibody or therapeutic biologic product during the 6 months prior to Screening.
Subject has received any oral, intravenous, or intramuscular steroid within 4 weeks prior to Screening. Intrathecal or intraarticular steroids are permitted.
A subject who is not vaccinated with the COVID-19 vaccine with appropriate window from last dose of vaccine to Screening per local guidelines, policies, and availability within 30 days prior to Day 1.

Part C: Any subject who meets any of these criteria must be excluded from Part C of this study:

History of any illness or any clinical condition that might confound the results of the study or pose an additional risk in administering study drug(s) to the subject.
Any of the following abnormal laboratory tests: Hemoglobin, Total bilirubin, liver enzymes or creatine clearance
An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for sinopulmonary disease within 28 days before the screening visit.
An acute illness not related to CF within 14 days before the first dose of study drug.
Subject has an active COVID-19 infection within 4 weeks prior to screening.
Ongoing or prior participation in a study of an investigational treatment within 28 days or 5 terminal half-lives (whichever is longer) before screening.
Female subject who is pregnant or breastfeeding.

Please refer to study protocol for the complete inclusion/exclusion criteria list.

Study design

Phase: Phase 1
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment: 108 participants (estimated)

Study arms

Experimental
Part A Healthy Volunteer
Subjects will be assigned to one of six planned dose cohorts and receive single doses of ABCI (0.5mg, 1.0mg, 2.0mg, 4.0mg, 6.0mg, 10.0mg). In each cohort, six subjects will receive ABCI and 2 will receive placebo
Combination Product: ABCI · Combination Product: Placebo
Experimental
Part B Healthy Volunteer
Subjects will be assigned to one of three planned dose cohorts and receive a loading dose and multiple ascending doses of ABCI (loading dose 1.5mg/0.5mg daily, loading dose 6.0mg/2.0 daily, loading dose 10.0mg/4.0mg daily). In each cohort, six subjects will receive ABCI and 2 will receive placebo.
Combination Product: ABCI · Combination Product: Placebo
Experimental
Part C People with Cystic Fibrosis
Subjects will be assigned to one of two planned dose cohorts of ABCI (loading dose 1.5 mg/0.5 mg daily, loading dose 6.0mg/2.0mg daily, loading dose 10.0mg/4.0mg daily) for a total of 28 days of open-label study drug administration. Up to 36subjects with CF, including 2 sentinels subjects not on cystic fibrosis transmembrane conductance regulator (CFTR) modulators will be enrolled. The 2 sentinel subjects will receive the medium dose/regimen. If the medium dose/regimen is tolerated, the remaining subjects with CF may receive the low, medium, high dose/regimen of ABCI and may be either on CFTR modulators or not on CFTR modulators. It is anticipated that approximately 24 subjects will be enrolled as follows: 8 high, 8 medium, and 8 low dose/regimen.
Combination Product: ABCI

Interventions

Combination productABCI
Subjects will receive ABCI via oral inhalation
Also known as: Amphotericin B Cystetic for Inhalation
Combination productPlacebo
Subjects will receive ABCI via oral inhalation

What researchers measure

Primary outcomes

Adverse Events (AEs), and Serious Adverse Events (SAEs)
The safety and tolerability of ABCI following oral inhalation of single and multiple ascending doses in healthy subjects (Parts A and B), and in people with Cystic Fibrosis (Part C) will be assessed
Time frame: up to 10 weeks

Secondary outcomes

Pharmacokinetics (PK) Profile - SAD Cmax
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: Observed maximum concentration (Cmax)
Time frame: 1 day
Pharmacokinetics (PK) Profile - SAD Tmax
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: time to reach maximum concentration (Tmax)
Time frame: 1 day
Pharmacokinetics (PK) Profile - SAD AUC0-24
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: Area under the concentration-time curve from time 0 to 24 hours post-dose (AUC0-24)
Time frame: 1 day
Pharmacokinetics (PK) Profile - SAD AUClast
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: Area under the concentration-time curve from the time of dosing to the last measurable concentration (AUClast)
Time frame: 1 day
Pharmacokinetics (PK) Profile - SAD AUCinf
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: Area under the concentration-time curve from the time of dosing extrapolated to infinity (AUCinf)
Time frame: 1 day
Pharmacokinetics (PK) Profile - SAD AUCtau
Pharmacokinetics Characteristics in Single Ascending Dose HV Subjects: Area under the concentration- concentration-time curve over the dosing interval (AUCtau)
Time frame: Up to 28 days
Pharmacokinetics (PK) Profile - MAD Cmax
Pharmacokinetics Characteristics in Multiple Ascending Dose HV Subjects: Observed maximum concentration (Cmax)
Time frame: Up to 28 days
Pharmacokinetics (PK) Profile - MAD Tmax
Pharmacokinetics Characteristics in Multiple Ascending Dose HV Subjects: time to reach maximum concentration (Tmax)
Time frame: Up to 28 days
Pharmacokinetics (PK) Profile - MAD AUC0-24
Pharmacokinetics Characteristics in Multiple Ascending Dose HV Subjects: Area under the concentration-time curve from time 0 to 24 hours post-dose (AUC0-24)
Time frame: Up to 28 days
Pharmacokinetics (PK) Profile - MAD Plasma AmB assessments
Pharmacokinetics Characteristics in Multiple Ascending Dose HV Subjects: Plasma AmB assessments
Time frame: Up to 84 days
Pharmacokinetics (PK) Profile - MAD AmB concentrations in BAL fluid
Pharmacokinetics Characteristics in Multiple Ascending Dose HV Subjects: AmB concentrations in BAL fluid after study drug administration
Time frame: Up to 29 days
AmB concentrations - Subjects with CF
Cumulative effect on pre-dose AmB concentrations through Day 29 and assessment of washout through Day 42
Time frame: Through 42 days

Other outcomes

ppFEV1 - Subjects with CF
Absolute change in percent-predicted morning pre-dose forced expiratory volume in 1 second (ppFEV1) from baseline to Day 29 and from Day 29 to Day 42
Time frame: Up to 42 days
LCI - Subjects with CF
Absolute change in Lung Clearance Index (LCI) (where available)
Time frame: Up to 42 days
Questionnaire - Subjects with CF
Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ-R) in Subjects with Cystic Fibrosis: Revised (CFQ-R) respiratory domain score from baseline to Day 29 and to Day 42 where scores range from 0 to 100, with higher scores indicating better health.
Time frame: Up to 42 days
ppFVC - Subjects with CF
Absolute change in percent-predicted morning pre-dose forced vital capacity (ppFVC) from baseline to Day 29 and from Day 29 to Day 42
Time frame: Up to 42 days
FVC - Subjects with CF
Absolute change in morning pre-dose FVC from baseline to Day 29 and from Day 29 to Day 42 (mLs)
Time frame: Up to 42 days
FEV1 - Subjects with CF
Absolute change in morning pre-dose FEV1 from baseline to Day 29 and from Day 29 to Day 42 (mLs)
Time frame: Up to 42 days
DLCO - Subjects with CF
Absolute change in diffusing capacity of the lungs for carbon monoxide (DLCO \[expressed as percent-predicted corrected for hemoglobin\]) from baseline to Day 29
Time frame: Up to 29 days
Body weight - Subjects with CF
Absolute change in body weight from baseline to Day 29 and from Day 29 to Day 42
Time frame: Up to 42 days
% solids in sputum - Subjects with CF
Absolute change in % solids in sputum from baseline (optional)
Time frame: Day 29
FRI biomarkers - Subjects with CF
Change from baseline in Functional Respiratory Imaging (FRI) biomarkers, including but not limited to airway wall volume, mucus plug volume, and blood vessel volume (where available)
Time frame: Up to 28 days
IVIVC - chloride secretion - Subjects with CF
Change in chloride secretion in response to AmB in vitro in primary cultured nasal epithelial cells
Time frame: Up to 42 days
IVIVC - FEV1 - Subjects with CF
Comparison of change from baseline FEV1 (ppFEV1 and absolute FEV1) (Day 29) and change in chloride secretion in response to AmB in vitro in primary cultured nasal epithelial cells
Time frame: Up to 42 days
IVIVC - ASL pH - Subjects with CF
Change in ASL pH in response to AmB in vitro in primary cultured nasal epithelial cells
Time frame: Up to 42 days
IVIVC - FEV1 & ASL pH - Subjects with CF
Comparison of change from baseline FEV1 (ppFEV1 and absolute FEV1) (Day 29) and ASL pH in response to AmB in vitro in primary cultured nasal epithelial cells
Time frame: Up to 29 days

Study locations

5 of 5 sites recruiting

Canberra Hospital
Canberra, Australian Capital Territory 2605, Australia
- Yashneel Prasad, MD · Contact
- Joelle Bourke · Contact
Recruiting
Westmead Hospital
Westmead, New South Wales 2145, Australia
- Jimmy Chien, MD · Contact
- Tracey Burns · Contact
Recruiting
The Prince Charles Hospital
Brisbane, Queensland 4032, Australia
- Ieuan Evans, MD · Contact
- Michelle Wood · Contact
Recruiting
Monash Medical Centre
Clayton, Victoria 3168, Australia
- Christopher Daley, MD · Contact
- Corinne Van Asha · Contact
Recruiting
New Zealand Clinical Research
Christchurch, New Zealand
- Cory Sellwood, MD · Contact
- David Lee · Contact
Recruiting

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Feb 12, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT05802264
Lead sponsor: Cystetic Medicines, Inc.
Collaborators: DevPro Biopharma
Responsible party: Sponsor
First posted: Apr 6, 2023
Start date: Mar 21, 2023
Primary completion: Dec 2025 (estimated)
Completion: Dec 2025 (estimated)
Last update: Feb 12, 2025

Study contacts

Martin Burke, MD, PhD

Contact

mburke@cysteticmedicines.com

217-244-8726

Daniele Tompkins, MA

Contact

dtompkins@devprobiopharma.com

973-983-3700 ext. 205

Martin Burke, MD, PhD

study chair · Founder of cystetic Medicines

Oversight

Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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