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

Sponsor
Cystetic Medicines, Inc.
Study ID
NCT05802264
Phase
PHASE1
Status
Recruiting
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Conditions

  • Cystic Fibrosis

Eligibility Criteria

Sex
ALL
Age
16 Years - N/A
Healthy Volunteers
Accepted

Interventions

  • ABCI — COMBINATION_PRODUCT
    Subjects will receive ABCI via oral inhalation
  • Placebo — COMBINATION_PRODUCT
    Subjects will receive ABCI via oral inhalation

Study Details

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.

Key Dates

First listed
Apr 6, 2023
Start date
Mar 21, 2023
Status verified
Feb 2025
Primary completion
Dec 31, 2025
Completion
Dec 31, 2025

Study Design

Enrollment
108 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

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
  • 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.
  • 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.

Primary Outcome Measure

Adverse Events (AEs), and Serious Adverse Events (SAEs) [ Time Frame: up to 10 weeks ]

Central Contacts

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