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

NCT05802264 | Recruiting Phase 1 DMC

• 1 other identifier: CM001001
• Study Type: interventional
• Target: 108
• Locations: 2 countries
• Sites: 5

Brief 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

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• 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

  up to 10 weeks

Secondary Outcomes (12)

• Pharmacokinetics (PK) Profile - SAD Cmax

  1 day

• Pharmacokinetics (PK) Profile - SAD Tmax

  1 day

• Pharmacokinetics (PK) Profile - SAD AUC0-24

  1 day

• Pharmacokinetics (PK) Profile - SAD AUClast

  1 day

• Pharmacokinetics (PK) Profile - SAD AUCinf

  1 day

Other Outcomes (14)

• ppFEV1 - Subjects with CF

  Up to 42 days

• LCI - Subjects with CF

  Up to 42 days

• Questionnaire - Subjects with CF

  Up to 42 days

Study Arms (3)

Part A Healthy Volunteer

EXPERIMENTAL

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

Part B Healthy Volunteer

EXPERIMENTAL

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

Part C People with Cystic Fibrosis

EXPERIMENTAL

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

ABCI COMBINATION_PRODUCT

Subjects will receive ABCI via oral inhalation

Also known as: Amphotericin B Cystetic for Inhalation

Part A Healthy Volunteer; Part B Healthy Volunteer; Part C People with Cystic Fibrosis

Placebo COMBINATION_PRODUCT

Subjects will receive ABCI via oral inhalation

Part A Healthy Volunteer; Part B Healthy Volunteer

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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%

You may not qualify if:

• 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.

Sponsors & Collaborators

• Cystetic Medicines, Inc.: lead
• DevPro Biopharma: collaborator

Study Sites (5)

Canberra Hospital

Canberra, Australian Capital Territory, 2605, Australia

RECRUITING

Westmead Hospital

Westmead, New South Wales, 2145, Australia

RECRUITING

The Prince Charles Hospital

Brisbane, Queensland, 4032, Australia

RECRUITING

Monash Medical Centre

Clayton, Victoria, 3168, Australia

RECRUITING

New Zealand Clinical Research

Christchurch, New Zealand

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Inhalation

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Respiratory Mechanics; Respiration; Respiratory Physiological Phenomena; Circulatory and Respiratory Physiological Phenomena

Study Officials

• Martin Burke, MD, PhD

  Founder of cystetic Medicines

  STUDY CHAIR

Central Study Contacts

Martin Burke, MD, PhD

CONTACT

217-244-8726; mburke@cysteticmedicines.com

Daniele Tompkins, MA

CONTACT

973-983-3700; dtompkins@devprobiopharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 28, 2023
• First Posted: April 6, 2023
• Study Start: March 21, 2023
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: February 12, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

AU (4); NZ (1)