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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)

RecruitingPhase 1Ages 18 Years - 60 YearsAllEquilibra Bioscience LLC
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In plain words

What the trial is, and whether it could suit you

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).

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
Key Inclusion Criteria: 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: 1. 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. 2. 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: 1. Participants not on prophylaxis must have a documented ABR of 6 in 12 months before screening. 2. Participants on prophylaxis must have a documented ABR of ≥ 2 in 12 months before screening. 3. 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. Key Exclusion Criteria: 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: 1. Factor V (FV) Leiden mutation. 2. Activated protein C (APC) resistant. 3. Protein C (PC) or protein S (PS) deficiency. 4. Prothrombin 20210 mutation; 5. 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. Part B: * 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 TIA, or abnormal findings in any prior laboratory thrombophilia evaluation will be excluded. * History of one or more of the following in participants and/or family members: 1. FV Leiden mutation. 2. APC resistant. 3. PC or PS deficiency. 4. Prothrombin 20210 mutation. 5. ATIII deficiency. * Impaired cardiac function or clinically significant cardiac disease, including any of the following: 1. Clinically significant and/or uncontrolled heart disease such as congestive heart failure requiring treatment (New York Heart Association Grade \>=2), left ventricular ejection fraction \< 50% as determined by multiple gated acquisition or echocardiogram, or clinically significant arrhythmia. 2. QTcF \> 450 ms ECG or congenital Long QT Syndrome at the Screening Visit. 3. Acute myocardial infarction or unstable angina pectoris \< 3 months prior to study entry. * Uncontrolled hypertension (systolic BP \> 150 mmHg and diastolic BP \> 100 mmHg), a history of hypertension crisis, or a history of hypertensive encephalopathy. * Participant with the following laboratory abnormalities: 1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 1.5 × upper limit of normal (ULN); 2. Total bilirubin ˃3.0 × ULN and direct bilirubin ˃1.5 × ULN (unless due to Gilbert
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What it tests

  • SR604
  • Placebo
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Where

10 sites
California Clinical Trials Medical Group (CCTMG)
Glendale, United States
Children's Hospital Los Angeles
Los Angeles, United States
Rush University Medical Center
Chicago, United States
LA Center for Bleeding and Clotting Disorders - Metairie
Metairie, United States
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, United States
Washington University School of Medicine
St Louis, United States
Brody School of Medicine at East Carolina University
Greenville, United States
Penn State Milton S Hershey Medical Center Pediatrics
Hershey, United States

…and 2 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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