A Phase 1 study to evaluate the safety and pharmacokinetics of single and multiple doses of SION-719 in healthy participants

Phase 1

• Conditions: Cystic Fibrosis; Human Genetics and Inherited Disorders - Cystic fibrosis; Respiratory - Other respiratory disorders / diseases

• Registration Number: ACTRN12624000739516
• Lead Sponsor: Sionna Therapeutics, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-06-14
• Last Update Posted: 24 June 2024

Recruitment & Eligibility

• Status: ot yet recruiting
• Sex: All
• Target Recruitment: 96

Inclusion Criteria

Participants must meet all of the following criteria to be included in the study:
1.Healthy male or female adult participants aged 18 to 55 years, inclusive, at the time of consent.
2.Weight of at least 45 kg (99 lbs) and body mass index between 18.0 and 32.0 kg/m2, inclusive, at Screening.
3.Participant is willing to abstain from alcohol, caffeine, smoking, and nicotine-containing products for 72 hours prior to Day -1 through the duration of the study. Participant is willing to abstain from eating cruciferous vegetables, charcoal-grilled meats, and poppy seeds for 48 hours prior to dosing throughout the duration of the study
4.Participant has read, understood and voluntarily provided written informed consent
5.Participant has an understanding, ability, and willingness to fully comply with study procedures and restrictions.
6.Female participants (sex assigned at birth) must be of non-childbearing potential or willing to comply with acceptable highly effective contraceptive requirements (including negative pregnancy tests). Male participants (sex assigned at birth) must be infertile or willing to comply with acceptable highly effective contraceptive requirements.

Exclusion Criteria

Participants who meet any of the following criteria must be excluded from the study:
1.Participant has clinically significant current or recurrent illness, such as cardiovascular (including but not limited to known structural cardiac abnormalities, family history of long QT syndrome, or cardiac syncope or recurrent, idiopathic syncope), neurologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, endocrine, or psychiatric disease or disorder, or other abnormality which may affect safety or clinical laboratory evaluations.
2.Participant has a history of malignancy, except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of recurrence for at least 3 years.
3.Participant has clinically significant abnormalities on ECG, physical examination, or vital sign assessment at Screening or Day -1.
4.Participant has any single reading of QTcF > 470 ms (females) or > 450 ms (males) at Screening or Day -1.
5.Chronic or habitual alcohol or tobacco use or use of recreational drugs in the opinion of the Investigator.
6.Participant is positive for drug screen at Screening or Day -1. Of note, the drug screen does not include cannabis, cotinine or alcohol testing at Screening but does include this testing at study Check-in (Day -1).
7.Participant has taken any prescription or over the counter medications within 14 days (or 5 half-lives of the medication, whichever is longer) prior to dosing or requires the use of these medications during the study, including herbal or homeopathic preparations (excluding standard doses of vitamin/mineral supplements and occasional acetaminophen or ibuprofen, which are allowed).
8.Participant has abnormalities at Screening or clinically significant abnormalities, in the opinion of the Investigator, at Day -1 on safety laboratory tests including serum chemistry, haematology, coagulation tests, and urinalysis.
a.Participant must have an estimated glomerular filtration rate >90 mL/min/1.73 m2 using the CKD-EPI 2021 formula and based on individual body surface area at Screening and Day -1.
b.Participant must have ALT, AST, alkaline phosphatase, and direct bilirubin less than or equal to the ULN at Screening and Day -1.
9.Participant has a positive test for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCV Ab), or human immunodeficiency virus antibody (HIV Ab) at Screening.
10.Participant has a positive test for Coronavirus Disease 2019 (COVID-19) during the Screening Period (including Day -1).
11.Participant has used any medication listed on the Flockhart table that is a substrate, inhibitor, or inducer of CYP3A4, or a substrate of CYP1A2, CYP2B6, CYP2C8, CYP2C19 or CYP2D6 within 28 days or 10 half-lives (whichever is longer) prior to the planned first study drug administration. Additionally, participants must not have consumed other substances known to be potent inhibitors or inducers of CYPP450 such as Seville orange, grapefruit, or cranberry juice-containing products and herbal supplements such as St. John’s Wort within 14 days before the planned first study drug administration.
12.Participant has used any other prescription or over-the-counter medication that the Investigator judges is likely to interfere with the study or pose an additional risk in participating, within 14 days or 5 half-lives (whichever is longer), or has received any vaccinations within 14 days prior to the planned first study drug

Study & Design

• Study Type: Interventional
• Study Design: Not specified