A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)

NCT06349473 | Recruiting Phase 1 FDA Drug

• 1 other identifier: SR604-1
• Study Type: interventional
• Target: 31
• Locations: 2 countries
• Sites: 10

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604 in participants with Hemophilia A or Hemophilia B, or Factor VII (FVII) deficiency, with or without inhibitors (Part B).

Conditions

Healthy Participants; Hemophilia A; Hemophilia B; Factor VII Deficiency

Keywords

Single ascending dose; Multiple ascending dose; Factor IX; Factor VIII; Factor VII

Outcome Measures

Primary Outcomes (2)

• Parts A and B: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

  Safety and tolerability of a single ascending SC dose of SR604 in healthy participants and multiple ascending SC doses of SR604 in participants with Hemophilia A or Hemophilia B or FVII deficiency will be evaluated.

  Part A: From Baseline (Day 1) up to Day 57; Part B: From Baseline (Day 1) up to 3 months

• Parts A and B: Number of Participants with Clinical Abnormal Changes in Coagulations Markers

  Safety and tolerability of a single ascending SC dose of SR604 in healthy participants and multiple ascending SC doses of SR604 in participants with Hemophilia A or Hemophilia B or FVII deficiency will be evaluated.

  Part A: From Baseline (Day 1) till Day 57; Part B: From Baseline (Day 1) till Day 90

Secondary Outcomes (12)

• Part A: Area Under the Serum Concentration-time Curve from time Zero to the Last Quantifiable Time Point (AUC[0-t])

  From Baseline (Day 1) up to Day 57

• Part A: Area Under the Serum Concentration-time Curve from time Zero Extrapolated to Infinity (AUC[0-inf])

  From Baseline (Day 1) up to Day 57

• Parts A and B: Maximum Concentration (Cmax) of SR604

  Part A: From Baseline (Day 1) up to Day 57; Part B: From Baseline (Day 1) up to Day 90

• Parts A and B: Time to Maximum Concentration (tmax) of SR604

  Part A: From Baseline (Day 1) up to Day 57; Part B: From Baseline (Day 1) up to Day 90

• Parts A and B: Terminal Half-life (T1/2) of SR604

  Part A: From Baseline (Day 1) up to Day 57; Part B: From Baseline (Day 1) up to Day 90

Study Arms (8)

Part A: Cohort 1A (SR604 Dose 1)

EXPERIMENTAL

Participants will receive single subcutaneous (SC) dose of SR604 dose 1 or matching placebo to SR604 on Day 1.

Drug: SR604; Drug: Placebo

Part A: Cohort 2A (SR604 Dose 2)

EXPERIMENTAL

Participants will receive single SC dose of SR604 dose 2 or matching placebo to SR604 on Day 1.

Drug: SR604; Drug: Placebo

Part A: Cohort 3A (SR604 Dose 3)

EXPERIMENTAL

Participants will receive single SC dose of SR604 dose 3 or matching placebo to SR604 on Day 1.

Drug: SR604; Drug: Placebo

Part A: Cohort 4A (SR604 Dose 4)

EXPERIMENTAL

Participants will receive single SC dose of SR604 dose 4 or matching placebo to SR604 on Day 1.

Drug: SR604; Drug: Placebo

Part B: Cohort 1B (SR604 Dose 5)

EXPERIMENTAL

Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 5 as multiple SC injections every 4-weeks.

Drug: SR604

Part B: Cohort 2B (SR604 Dose 6)

EXPERIMENTAL

Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 6 as multiple SC injections every 4-weeks.

Drug: SR604

Part B: Cohort 3B (SR604 Dose 7)

EXPERIMENTAL

Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 7 as multiple SC injections every 4-weeks.

Drug: SR604

Part B: Cohort 4B (SR604 Dose 8)

EXPERIMENTAL

Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 8 as multiple SC injections every 4-weeks.

Drug: SR604

Interventions

SR604 DRUG

SR604 will be administered as SC injection.

Part A: Cohort 1A (SR604 Dose 1); Part A: Cohort 2A (SR604 Dose 2); Part A: Cohort 3A (SR604 Dose 3); Part A: Cohort 4A (SR604 Dose 4); Part B: Cohort 1B (SR604 Dose 5); Part B: Cohort 2B (SR604 Dose 6); Part B: Cohort 3B (SR604 Dose 7); Part B: Cohort 4B (SR604 Dose 8)

Placebo DRUG

Placebo will be administered as single SC injection.

Part A: Cohort 1A (SR604 Dose 1); Part A: Cohort 2A (SR604 Dose 2); Part A: Cohort 3A (SR604 Dose 3); Part A: Cohort 4A (SR604 Dose 4)

Eligibility Criteria

• Age: 18 Years - 60 Years
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Part A:
• Male participants aged 18 to 55 years, inclusive.
• Body mass index between 18 and 30 kilograms per meter square (kg/m\^2), inclusive, and weighs greater than or equal to (\>=) 50 kilograms (kg), less than or equal to (\<=) 90 kg.
• No clinically significant findings on medical examination, including physical examination, 12-lead electrocardiogram, and clinical laboratory tests.
• Sexually active men must commit to use an effective method of birth control while taking the study intervention and for 90 days after the dose of study intervention.
• Part B:
• Male and female participants (only female participants with congenital FVII deficiency) aged 18 to 60 years, inclusive.
• Participants must have one of the following bleeding disorders: Severe hemophilia A (\<1% Factor VIII \[FVIII\]); or Severe and/or moderately severe Hemophilia B (≤ 2% Factor IX \[FIX\]); or Severe FVII deficiency (\<10% FVII activity). Participants with severe FVII deficiency must satisfy with either of following criteria:
• Participants with history of \>2 bleeding events in the last 12 months require on-demand treatment with recombinant factor VIIa (rFVIIa) or plasma-derived FVII concentrates (pd-FVII) or fresh frozen plasma (FFP) for bleeding control.
• Participants on prophylaxis treatment with rFVIIa or pd-FVII or FFP regardless of bleeding history.
• Participants with Hemophilia A or Hemophilia B must satisfy either of the following criteria:
• Participants not on prophylaxis must have a documented ABR of 6 in 12 months before screening.
• Participants on prophylaxis must have a documented ABR of ≥ 2 in 12 months before screening.
• Intolerant to current treatment regimen.
• Medical records documenting a minimum of 2 years of bleeding event history.

You may not qualify if:

• Part A:
• Participant has clinically significant history or evidence of cardiovascular, respiratory (including all chronic lung diseases), hepatic, renal, gastrointestinal, endocrine, neurological, immunological, bleeding, or psychiatric disorder(s).
• Participant has a mean pulse less than (\<) 40 or greater than (\>) 90 beats per minute (bpm), mean systolic blod pressure (BP) \< 90 millimeter of mercury (mmHg) or \> 140 mmHg, or mean diastolic BP \< 50 mmHg or \> 90 mmHg at the screening visit.
• Participant has a mean corrected QT corrected for heart rate by Fridericia's formula (QTcF) of \> 450 msec at the Screening Visit.
• Participant has had injury, trauma, and/or major surgery within 3 months before Screening, or is planned to undergo surgery during the study.
• Participant has received vaccination within 14 days before the dose of study intervention or has a vaccination planned during the study.
• History of one or more of the following in participants and/or family members:
• Factor V (FV) Leiden mutation.
• Activated protein C (APC) resistant.
• Protein C (PC) or protein S (PS) deficiency.
• Prothrombin 20210 mutation;
• Antithrombin III (ATIII) deficiency.
• History of clinically significant intracranial hemorrhage, pneumonia, chronic liver disease, liver or kidney transplants, or malignant diseases.
• Any medical condition (eg, diabetes, obesity.) which, in the Investigator's opinion, could compromise participant safety, interfere with study intervention metabolism, or put the study outcome at undue risk. Any condition for which, in the opinion of the Investigator, participation would not be in the best interest of the participant or could prevent, limit or confound protocol-specified assessments.
• Participants with a history of all types of thrombosis, including any arterial and/or venous thrombosis, superficial thrombophlebitis, or embolism. Additionally, participants with a history of thrombotic microangiopathy, stroke, and transient ischemic attack (TIA), or abnormal findings in any prior laboratory thrombophilia evaluation will be excluded.

Sponsors & Collaborators

• Equilibra Bioscience LLC: lead

Study Sites (10)

California Clinical Trials Medical Group (CCTMG)

Glendale, California, 91206, United States

COMPLETED

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Rush University Medical Center

Chicago, Illinois, 60612, United States

NOT YET RECRUITING

LA Center for Bleeding and Clotting Disorders - Metairie

Metairie, Louisiana, 70001, United States

RECRUITING

University of Michigan Hospitals - Michigan Medicine

Ann Arbor, Michigan, 48109, United States

RECRUITING

Washington University School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

Brody School of Medicine at East Carolina University

Greenville, North Carolina, 27834, United States

NOT YET RECRUITING

Penn State Milton S Hershey Medical Center Pediatrics

Hershey, Pennsylvania, 17033, United States

NOT YET RECRUITING

Perelman Center for Advanced Medicine (PCAM)- Penn Blood Disorders Program

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

McMaster University Medical Centre, Hamilton Health Sciences

Hamilton, Ontario, L8N 3Z5, Canada

RECRUITING

MeSH Terms

Conditions

Hemophilia A; Hemophilia B; Factor VII Deficiency

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Central Study Contacts

Clinical Trials

CONTACT

925-490-0278; inf@equilibrabioscience.com

Study Design

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

Study Record Dates

• First Submitted: March 20, 2024
• First Posted: April 5, 2024
• Study Start: May 10, 2024
• Primary Completion (Estimated): September 28, 2026
• Study Completion (Estimated): September 28, 2026
• Last Updated: March 10, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (9); CA (1)