Study to Evaluate the Safety, Tolerability, PK and PD of DISC-0974 in Healthy Subjects

Disc Medicine

Status and phase

Completed

Phase 1

Conditions

Healthy Volunteer

Treatments

Drug: Placebo

Drug: DISC-0974

Study type

Interventional

Funder types

Industry

Identifiers

NCT04999527

DISC-0974-101

Details and patient eligibility

About

This Phase 1 study of DISC-0974 will assess the safety, tolerability, pharmacokinetics (PK) and Pharmacodynamics (PD) of DISC-0974 in adult healthy volunteers.

Full description

Enrolled participants in this study will receive a single dose of DISC-0974 or a placebo. Samples will be collected to measure how DISC-0974 is processed by the body and how the body responds when exposed to DISC-0974. Participants will receive a single study drug dose on Day 1; and follow-up for 10 weeks (71 days). If an undetectable DISC-0974 blood concentration is observed, the duration of follow-up will be shortened to 7 days thereafter, as feasible.

Enrollment

42 patients

Sex

All

Ages

18 to 65 years old

Volunteers

Accepts Healthy Volunteers

Inclusion criteria

Male or female (HV only) adults aged 18 to 65 years, inclusive at the time of consent.
Body weight ≥50 kg and body mass index (BMI) between 18 and 33, inclusive, at Screening.
Systolic blood pressure ≤140 mmHg and diastolic blood pressure ≤90 at Screening.
No clinically significant abnormalities as determined by medical history, by results of physical examination, vital signs, ECG and lab tests at Screening.
QTcF <450 msec at Screening.
Estimated glomerular filtration rate >60 mL/min/1.73m2 based on the 4 parameter MDRD (Modification of Diet in Renal Disease) equation at Screening
TSAT ≤30% at Screening, as determined in a fasting morning blood sample (06:00 to 11:00 hours).
Hematologic parameters (red blood cell count [RBC], hemoglobin, hematocrit, platelet count, mean corpuscular volume, mean corpuscular hemoglobin concentration), serum iron, and TIBC within normal range and serum ferritin (within normal range and ≥30 ng/mL) at Screening.
If a male with female sexual partner(s) of childbearing potential, must agree to use acceptable methods of birth control during the study and through the End of Study (EOS) visit
If female, postmenopausal, as defined with at least 12 months natural, spontaneous amenorrhea, or at least 6 weeks following surgical menopause
Able to understand and provide written informed consent and comply with protocol requirements

Exclusion criteria

History of anemia or hematologic disorder within 1 year of Screening
History of splenectomy
Diagnosis or first-degree relative with a diagnosis of hemochromatosis
History of diabetes, cardiovascular, hepatic, renal, or malabsorptive disease
Vegan or iron-deficient diet within 3 months of Screening
Blood transfusion within 1 year of Screening
Whole blood donation within 6 months of Screening or plasma donation within 30 days of Screening.
A history of alcohol or illicit drug use disorder within 3 years of Screening, as assessed by the Investigator
Use of any tobacco- and/or nicotine-containing containing products, including e-cigarettes, vaping products, and nicotine replacement products, within 3 months of Screening
Use of multivitamin or iron supplements within 30 days prior to Screening
ALT or aspartate aminotransferase (AST) level above the normal range at Screening
Positive urine pregnancy test at Screening or Baseline (Day -1).
Positive serologic test for Hepatitis B surface antigen, Hepatitis C antibody, or human immunodeficiency virus (HIV) at Screening.
Positive urine screen for drugs of abuse or alcohol test on admission to the study center
Use of any systemic prescription medication within 14 days of Screening, non-iron containing dietary supplements, or non-prescription drugs within 7 days of dosing.
History of a major surgical procedure within 60 days of Screening or planned surgical procedure within 90 days of dosing.
A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug.
History or presence or any illness that, in the opinion of the Investigator, would interfere with the ability to provide informed consent or comply with study instructions.