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

NCT04999527 | Completed Phase 1 FDA Drug

• 1 other identifier: DISC-0974-101
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Healthy Volunteer

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (4)

• 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

Study Arms (2)

Healthy Volunteer: Single Ascending Dose of DISC-0974

EXPERIMENTAL

Single Intravenous (IV) or Subcutaneous (SC) ascending dose in healthy volunteers

Drug: DISC-0974

Healthy Volunteer: Single Ascending Dose of Placebo

EXPERIMENTAL

Single Intravenous (IV) or Subcutaneous (SC) ascending dose in healthy volunteers

Drug: Placebo

Interventions

DISC-0974 DRUG

DISC-0974 is administered (IV infusion or Subcutaneous Injection) as a single dose

Healthy Volunteer: Single Ascending Dose of DISC-0974

Placebo DRUG

Placebo is administered (IV infusion or Subcutaneous Injection) as a single dose

Healthy Volunteer: Single Ascending Dose of Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Disc Medicine, Inc: lead

Study Sites (1)

Worldwide Clinical Trials

San Antonio, Texas, 78217, United States

Location

Related Publications (1)

• Novikov N, Buch A, Yang H, Andruk M, Liu G, Wu M, Howell H, MacDonald B, Savage W. First-in-Human Phase 1 Study Evaluating the Safety, Pharmacokinetics, and Pharmacodynamics of DISC-0974, an Anti-Hemojuvelin Antibody, in Healthy Participants. J Clin Pharmacol. 2024 Aug;64(8):953-962. doi: 10.1002/jcph.2432. Epub 2024 Mar 21.

  PMID: 38515275 DERIVED

Related Links

• Related Info

Study Officials

• William Savage, MD, PhD

  Disc Medicine

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 20, 2021
• First Posted: August 11, 2021
• Study Start: August 10, 2021
• Primary Completion: June 30, 2022
• Study Completion: June 30, 2022
• Last Updated: July 19, 2022

Record last verified: 2022-07

Locations

US (1)