Evaluating the safety and effects of SR604 in healthy individuals and those with Hemophilia A or B
Official title: 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)
Run by Equilibra Bioscience LLC · NCT06349473 · Data updated 2026-11-12
This study is testing a new drug called SR604 to see if it's safe and effective for healthy people and those with Hemophilia A or B.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 60 Years |
| Sex | All |
| Enrollment | 31 (estimated) |
| Sponsor | Equilibra Bioscience LLC Industry-sponsored |
| Locations | 10 sites (Glendale, California and 9 other locations) |
| Status | Recruiting |
| Started | May 10, 2024 |
| Main results expected | September 28, 2026 |
| Study ends | September 28, 2026 |
| Registry record updated | July 2, 2026 |
| Trial ID | NCT06349473 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include healthy individuals aged 18-30 with a specific body mass index and individuals diagnosed with severe Hemophilia A or B who have not achieved adequate control with current treatments.
✕Probably not a fit
Patients with mild forms of Hemophilia or those whose bleeding is well-controlled on existing therapies may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This clinical trial aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of a new drug, SR604, in two groups of participants. Part A involves healthy participants receiving either SR604 or a placebo in a randomized manner, while Part B focuses on individuals with Hemophilia A or B, who will receive multiple doses of SR604. The study will monitor the effects and efficacy of the drug over approximately three months, providing valuable data on its potential use in treating bleeding disorders.
Why it matters
Potential benefit: If successful, this study could lead to a new treatment option for patients with Hemophilia A or B, improving their management and quality of life.
How similar studies have gone: Other studies have shown promise in similar approaches for treating hemophilia, but this specific drug and its effects are novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
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's syndrome). * Calculated creatinine clearance ˂ 60 mL/min using the 2021 Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula at the Screening Visit. * Participant has positive test result for human immunodeficiency virus (HIV) antibody. 1. If participants test positive for hepatitis B core antibody (HBcAb), additional tests including hepatitis B surface antibody, hepatitis B surface antigen (HBsAg), and hepatitis B viral deoxyribonucleic acid (DNA) polymerase chain reaction (PCR) will be conducted to determine if there is an active infection. Participants with active infection will be excluded from the study. 2. Participants who test positive for hepatitis C virus antibody will be required to have a negative result for hepatitis C viral ribonucleic acid (RNA) PCR before enrollment. Individuals with positive results for hepatitis C PCR will be excluded from the study. * Chronic liver disease (Child-Pugh class C hepatic impairment), or history of liver or kidney transplants. * Injury, trauma, and/or major surgery (mediastinoscopy, insertion of a central venous access device, and insertion of a feeding tube are not considered major surgery), major dental procedures (extractions, etc.) within 4 weeks of the first dose of SR604 or planned surgery during the study. * Active infection requiring systemic antibiotic or antiviral therapy or in a sepsis condition within 14 days prior to the first dose of SR604. * Any medical condition (eg, diabetes, obesity) which, in the Investigator's opinion, could compromise participant safety, interfere with SR604 metabolism, or put the study outcome at undue risk. * Female participants who are pregnant or are currently breastfeeding or planning to become pregnant while enrolled in this study or within 90 days after the last dose of SR604.
Where it's running
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Children's Hospital Los Angeles
Los Angeles, California, United States Recruiting -
Rush University Medical Center
Chicago, Illinois, United States Recruiting -
LA Center for Bleeding and Clotting Disorders - Metairie
Metairie, Louisiana, United States Recruiting -
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, United States Recruiting -
Washington University School of Medicine
St Louis, Missouri, United States Recruiting -
Penn State Milton S Hershey Medical Center Pediatrics
Hershey, Pennsylvania, United States Recruiting
Show 4 more locations
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Perelman Center for Advanced Medicine (PCAM)- Penn Blood Disorders Program
Philadelphia, Pennsylvania, United States Recruiting -
McMaster University Medical Centre, Hamilton Health Sciences
Hamilton, Ontario, Canada Recruiting -
California Clinical Trials Medical Group (CCTMG)
Glendale, California, United States Completed -
Brody School of Medicine at East Carolina University
Greenville, North Carolina, United States Not yet recruiting
Study contacts
- Study coordinator: Clinical Trials
- Email: inf@equilibrabioscience.com
- Phone: 925-490-0278
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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SR604: a new treatment tested in adults with Hemophilia A/B or congenital Factor VII deficiency
This study tests a single and multiple doses of SR604 to see if it is safe and effective for adults with Hemophilia A or B, with or without inhibitors, and for those with congenital Factor VII deficiency.
-
ATHNdataset registry for people with bleeding and clotting disorders
This registry collects health, treatment, and patient-reported information from people with bleeding and clotting disorders seen at ATHN-affiliated centers to help improve care and outcomes.
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Is synovial proliferation in haemophilia joints active or inactive?
This project will test whether ultrasound, MRI and elastography can tell if subclinical synovial proliferation in people with severe haemophilia is currently active so clinicians can treat earlier.
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Observational study of blood clotting and bleeding disorders in Chinese patients
This study looks at blood clotting and bleeding problems in Chinese patients to see how common they are and what factors might affect them.
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Non-invasive prenatal diagnosis for single gene disorders
This study is testing a new way to safely diagnose single-gene disorders in unborn babies using a simple blood test from the mother, starting as early as 9 weeks into the pregnancy.
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Testing marstacimab for treating hemophilia in children
This study is testing a new medication called marstacimab to see if it can help children with severe hemophilia control their bleeding and improve their health.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.