Study to Assess the Safety, Tolerability, PK and PD of ABX1100

Healthy Clinical Trial

Official title:

A Phase 1, Single Ascending Dose and Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ABX1100 in Normal Healthy Volunteers

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06109948
• Other study ID #: ABX1100-1001
• Status: Recruiting
• Phase: Early Phase 1
• Start date: October 19, 2023
• Est. completion date: September 30, 2024

Study information

• Verified date: May 2024
• Source: Aro Biotherapeutics
• Contact: Aro Study Information Center
• Phone: 215-544-1175
• Email: ABX1100[@]arobiotx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Study ABX1100-1001 is a first-in-human (FIH), phase 1 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of a single ascending dose (SAD) and multiple doses (MD) of ABX1100 administered intravenously to healthy participants.

• Part A features a SAD study with a double-blind, placebo-controlled, randomized design in NHVs involving 3 cohorts (A1-A3). This Part also includes a single dose, open-labeled cohort (A4) in NHVs which will commence after cohorts A1-3.

• Part B is a MD, double-blind, placebo-controlled, randomized design in NHVs. The MD Part B will commence after completion of Cohorts A1, A2 and A3 in the SAD Part A and SRC review of these 3 cohorts.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 38
• Est. completion date: September 30, 2024
• Est. primary completion date: August 31, 2024
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 50 Years

Eligibility

Inclusion Criteria:

• Body mass index (BMI) between 18.0 and 32.0 kg/m2, inclusive and weight between 50 and 90 kg, inclusive.

• Agree not to have a tattoo or body piercing until the end of the study.

• Agree not to receive COVID-19 vaccination from 7 days prior to first study drug administration until at least 7 days after the last study drug administration.

• Agree not to receive a vaccination (live attenuated vaccine) during the study and until 60 days after the study has ended (last study procedure).

• Willing to undergo needle muscle biopsies.

• Willing to avoid strenuous activities 48 hours before needle muscle biopsy and throughout the study.

• Female participants who are sexually active with a non-sterilized partner must be non-pregnant and non-lactating and agree to use a highly effective method of contraception.

• Males of childbearing potential must agree to use a highly effective method of contraception with female sexual partners of childbearing potential and not donate sperm during study participation and for 90 days after last administration of study drug ABX1100 or placebo.

Exclusion Criteria:

• Known history or presence of any clinically significant hepatic, renal/genitourinary, gastrointestinal, cardiovascular, cerebrovascular, pulmonary, endocrine, immunological, musculoskeletal, neurological, psychiatric, dermatological or hematological disease or condition.

• History of any inherited or acquired skeletal muscle diseases (for example, Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), Limb-Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy, Spinal Muscular Atrophy (SMA), Polymyositis, Rhabdomyolysis and Inclusion Body Myositis (IBM).

• History of any inherited or acquired cardiac disease including congestive heart failure, ischemic heart disease, or arrhythmias; an abnormal ECG.

• History of cancer within past 5 years, with the exception of treated or excised skin basal cell carcinoma.

• Any clinically significant illness, medical/surgical procedure, or trauma within 4 weeks prior to the screening visit

• Presence of any significant physical or organ abnormality.

• Major surgery within 6 months prior to the start of the study.

• Current smoker, recent history of smoking and/or use of any nicotine-containing products (within past 6 months).

• A known history or positive test result for hepatitis B surface antigen (HBsAg), hepatitis C antibody or human immunodeficiency virus (HIV) infection.

• Currently participating in another investigational trial or have received any investigational drug within the past 30 days.

Related Conditions & MeSH terms

• Healthy

Intervention

Drug:
• ABX1100 injection for IV infusion
Centyrin protein-siRNA conjugate
• Placebo injection for IV infusion
placebo saline injection

Locations

Country	Name	City	State
Canada	Study Site	Toronto

Sponsors (1)

Lead Sponsor	Collaborator
Aro Biotherapeutics

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects with treatment-emergent adverse events (TEAEs)	Adverse Events will be graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Adverse events (AEs) will be reported based on clinical laboratory tests, vital signs, physical examinations, electrocardiograms, and echocardiograms from the time informed consent is signed through 8 weeks after ABX1100 administration for Part A and through 12 weeks after ABX1100 administration for Part B. AEs will be considered to be treatment emergent (TEAE) if they occurred or worsened in severity after the first dose of study treatment.	up to 16 weeks
Secondary	Plasma pharmacokinetics as measured by Cmax	Plasma samples will be taken pre-dose, 10 min, end of infusion, 0.5, 1, 3, 6, 9, 12 and 24 hours after the start of ABX1100 administration	0-24 hours after ABX1100 administration
Secondary	Plasma pharmacokinetics as measured by Tmax	Plasma samples will be taken pre-dose, 10 min, end of infusion, 0.5, 1, 3, 6, 9, 12 and 24 hours after the start of ABX1100 administration	0-24 hours after ABX1100 administration
Secondary	Plasma pharmacokinetics as measured by AUC	Plasma samples will be taken pre-dose, 10 min, end of infusion, 0.5, 1, 3, 6, 9, 12 and 24 hours after the start of ABX1100 administration	0-24 hours after ABX1100 administration
Secondary	Immunogenicity of AXB1100 as measured by anti-ABX1100 antibodies in serum	Plasma samples will be taken pre-dose, Day 15 and Day 29 after ABX1100 administration (Part A); Plasma samples will be taken pre-dose, Day 15, Day 29 Day 43 and Day 57 after ABX1100 administration (Part B)	Up to 8 weeks