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

A Phase 1, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, PK and PD of DISC-0974 in Healthy Subjects Following Dose-Escalating Single Intravenous or Subcutaneous Administration

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

Study Overview

• Status: Completed
• Conditions: Healthy Volunteer
• Intervention / Treatment: Drug: DISC-0974; Drug: Placebo

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 42
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Texas
    • San Antonio, Texas, United States, 78217
      • Worldwide Clinical Trials

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Healthy Volunteer: Single Ascending Dose of DISC-0974; Single Intravenous (IV) or Subcutaneous (SC) ascending dose in healthy volunteers	Drug: DISC-0974; DISC-0974 is administered (IV infusion or Subcutaneous Injection) as a single dose
Experimental: Healthy Volunteer: Single Ascending Dose of Placebo; Single Intravenous (IV) or Subcutaneous (SC) ascending dose in healthy volunteers	Drug: Placebo; Placebo is administered (IV infusion or Subcutaneous Injection) as a single dose

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of adverse events	Up to 71 days of monitoring
Incidence of abnormal laboratory test results	Up to 71 days of monitoring
Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)	Up to 71 days of monitoring
Incidence of treatment-emergent clinically abnormal physical exam	Up to 71 days of monitoring

Secondary Outcome Measures

Outcome Measure	Time Frame
Plasma maximum measured drug concentration (Cmax)	Up to 71 days of testing
Time of maximum concentration (Tmax)	Up to 71 days of testing
Area under the concentration-time curve (AUC)	Up to 71 days of testing
Plasma half-life (T½)	Up to 71 days of testing

Collaborators and Investigators

• Sponsor: Disc Medicine, Inc
• Investigators: Study Director: William Savage, MD, PhD, Disc Medicine

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): August 10, 2021
• Primary Completion (Actual): June 30, 2022
• Study Completion (Actual): June 30, 2022

Study Registration Dates

• First Submitted: July 20, 2021
• First Submitted That Met QC Criteria: August 7, 2021
• First Posted (Actual): August 11, 2021

Study Record Updates

• Last Update Posted (Actual): July 19, 2022
• Last Update Submitted That Met QC Criteria: July 18, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Other Study ID Numbers: DISC-0974-101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No