A Phase 1 Single and Multiple Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Healthy Participants (Part A) and the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SR604 in Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency (Part B)

Status: Recruiting
Location: See all (10) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 60
Healthy Volunteers: t
View:

∙ 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.

• Willing to undergo a weaning period from prior treatment or prophylaxis for Hemophilia A or Hemophilia B or FVII deficiency.

• 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 SR604.

• Women of childbearing potential must have a negative pregnancy test at the Screening Visit and agree to follow the contraception guidance during the intervention period and for at least 90 days after the last dose of SR604.

Locations
United States
California
California Clinical Trials Medical Group (CCTMG)
COMPLETED
Glendale
Children's Hospital Los Angeles
RECRUITING
Los Angeles
Illinois
Rush University Medical Center
NOT_YET_RECRUITING
Chicago
Louisiana
LA Center for Bleeding and Clotting Disorders - Metairie
RECRUITING
Metairie
Michigan
University of Michigan Hospitals - Michigan Medicine
RECRUITING
Ann Arbor
Missouri
Washington University School of Medicine
RECRUITING
St Louis
North Carolina
Brody School of Medicine at East Carolina University
NOT_YET_RECRUITING
Greenville
Pennsylvania
Penn State Milton S Hershey Medical Center Pediatrics
NOT_YET_RECRUITING
Hershey
Perelman Center for Advanced Medicine (PCAM)- Penn Blood Disorders Program
RECRUITING
Philadelphia
Other Locations
Canada
McMaster University Medical Centre, Hamilton Health Sciences
RECRUITING
Hamilton
Contact Information
Primary
Clinical Trials
inf@equilibrabioscience.com
925-490-0278
Time Frame
Start Date: 2024-05-10
Estimated Completion Date: 2026-04-27
Participants
Target number of participants: 36
Treatments
Experimental: Part A: Cohort 1A (SR604 Dose 1)
Participants will receive single subcutaneous (SC) dose of SR604 dose 1 or matching placebo to SR604 on Day 1.
Experimental: Part A: Cohort 2A (SR604 Dose 2)
Participants will receive single SC dose of SR604 dose 2 or matching placebo to SR604 on Day 1.
Experimental: Part A: Cohort 3A (SR604 Dose 3)
Participants will receive single SC dose of SR604 dose 3 or matching placebo to SR604 on Day 1.
Experimental: Part A: Cohort 4A (SR604 Dose 4)
Participants will receive single SC dose of SR604 dose 4 or matching placebo to SR604 on Day 1.
Experimental: Part B: Cohort 1B (SR604 Dose 5)
Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 5 as multiple SC injections every 4-weeks.
Experimental: Part B: Cohort 2B (SR604 Dose 6)
Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 6 as multiple SC injections every 4-weeks.
Experimental: Part B: Cohort 3B (SR604 Dose 7)
Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 7 as multiple SC injections every 4-weeks.
Experimental: Part B: Cohort 4B (SR604 Dose 8)
Participants with Hemophilia A or Hemophilia B or FVII deficiency will receive SR604 dose 8 as multiple SC injections every 4-weeks.
Sponsors
Leads: Equilibra Bioscience LLC

This content was sourced from clinicaltrials.gov