Evaluation of the Safety, Tolerability, Immunogenicity, Pharmacokinetics and Pharmacodynamics of Intravenous ATYR1923 in Healthy Volunteers.

A Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Safety, Tolerability, Immunogenicity, Pharmacokinetics and Pharmacodynamics of Single Doses of Intravenous ATYR1923 in Healthy Volunteers

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12617001446358

Enrollment

Registered

2017-10-12

Start date

2017-11-16

Completion date

2018-03-23

Last updated

2018-06-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

This study is a first-in-human, randomized, double-blind, placebo-controlled, study to evaluate safety, tolerability, immunogenicity, pharmacokinetics and pharmacodynamics of single ascending doses of IV administered ATYR1923 at doses up to 5 mg/kg in healthy volunteers. This study will include 6 separate cohorts with ascending doses.

Interventions

The intervention is a single infusion, given once, There is no requirement to implement strategies to improve adherence. Any deviations from the protocol mandated infusion requirements will be documented. Single Ascending Dose (SAD) Cohort 1 - Single dose of 0.03mg/kg IV administered ATYR1923/placebo over a 60 minute period Cohort 2 - Single dose of 0.1mg/kg IV administered ATYR1923/placebo over a 60 minute period Cohort 3 - Single dose of 0.3mg/kg IV administered ATYR1923/placebo over a 60 minute period Cohort 4 - Single dose of 1.0mg/kg IV administered ATYR1923/placebo over a 60 minute period Cohort 5 - Single dose of 3.0mg/kg IV administered ATYR1923/placebo over a 60 minute period Cohort 6 - Single dose of 5.0mg/kg IV administered ATYR1923/placebo over a 60 minute period

Study design

Allocation

Randomised controlled trial

Intervention model

Other

Primary purpose

Treatment

Masking

Blinded (masking used) (Subject, Caregiver, Outcomes Assessor)

Eligibility

Sex/Gender

All

Age

18 Years to 55 Years

Healthy volunteers

Yes

Inclusion criteria

1. Willing and able to provide written informed consent 2. Healthy male or female subjects, aged greater than or equal to 18 and less than or equal to 55 years inclusive at time of informed consent. 3. Body Mass Index (BMI) greater than or equal to 19.0 to less than or equal to 30.0 kg/m2 and weight greater than or equal to 55 kg and less than or equal to 100kg. 4. Female subjects may be of childbearing potential or of non-childbearing potential (either surgically sterilized or at least 1 year post-menopausal as confirmed by amenorrhea duration of at least 12 months and appropriate levels of serum follicle-stimulating hormone [FSH]). Female subjects of childbearing potential must be non-pregnant and non-lactating, have a negative serum pregnancy test at screening and a negative urine pregnancy test at Day -1 prior to first study drug infusion. Additionally, female subjects of childbearing potential must be willing to use adequate contraception from screening until 90 days after the last follow-up visit. 5. Male subjects, if not infertile or surgically sterilized, must agree to use adequate contraception and not donate sperm from Day -1 until 90 days after the last follow-up visit 6. Adequate venous access. 7. Be able to communicate well with the Investigator and site personnel, and agree to comply with all study procedures and requirements.

Exclusion criteria

1. Exposure to any prescription medications (small molecules, biologics including vaccines) or, systemically administered over the counter drugs, dietary supplements or herbal remedies, within 30 days or 5 half-lives (if known), whichever is longer, prior to Day -1. An exception is made for hormonal contraceptives and a limited amount of paracetamol for the treatment of headache or any other pain. 2. Any condition which (while not requiring regular use of medication) is likely to require intermittent (as required) therapeutic intervention (e.g., seizure disorder, seasonal allergies, asthma) at any point during the study. 3. History of significant drug allergies or a history of anaphylactic reaction. 4. History of any clinically significant condition (including and not limited to the gastrointestinal, renal, hepatic, cardiovascular, respiratory, or neurological systems) and/ or other major disease or malignancy, as determined by the Investigator. 5. Major surgery within 3 months prior to Day -1 or anticipated surgery during the study. 6. Any condition that necessitated hospitalization within the 3 months prior to Day -1 or is likely to require so during the study. 7. Blood/plasma donation or blood loss greater than or equal to 470 ml within 3 months prior to Day -1. 8. Participation in another clinical study of an investigational agent within 3 months (small molecules) / 6 months (biologics) or 5 half-lives of the agent (if known), whichever is longer. 9. History of, or positive results of screening for: hepatitis B (hepatitis B surface antigen [HBsAg]), hepatitis C (anti-hepatitis C virus [HCV] antibodies) or human immunodeficiency virus (HIV) (antibodies to HIV types 1 and 2). 10. History of drug or alcohol abuse within 12 months prior to Day -1, or evidence of such abuse on laboratory assays at screening. Drug or alcohol abuse includes heavy smoking (> 1 pack per day), frequent use of recreational drug products, and an average intake of more than 24 units of alcohol per week (1 unit of alcohol equals approximately 250 mL of beer, 100 mL of wine or 35 mL of spirits). 11. History of alcohol consumption in the 3 days prior to Day -1, or inability to abstain from alcohol consumption during the study. 12. Clinically significant abnormalities in the screening physical examination, vital signs, electrocardiogram (ECG) or clinical laboratory tests, as determined by the Investigator. Repeat testing may be performed at the Investigator’s discretion. 13. Jo-1 positivity during Screening or past history of Jo-1 positivity. 14. Unable to refrain from strenuous activity for a period of 4 days before Day -1 and for the duration of the study. 15. Unlikely to comply with the clinical study protocol; e.g., uncooperative attitude, inability to return for follow-up visits, and improbability of completing the study. 16. Any other condition, which in the opinion of the Investigator precludes the subject’s participation in the clinical study. 17. Significant and/or acute illness within 5 days prior to (the first) drug administration that may impact safety assessments, in the opinion of the Investigator. 18. An employee of the Contract Research Organization (CRO) or the Sponsor.

Outcome results