Phase
Condition
Hemophilia
Treatment
Placebo
SR604
Clinical Study ID
Ages 18-60 Male Accepts Healthy Volunteers
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Part A:
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 physicalexamination, 12-lead electrocardiogram, and clinical laboratory tests.
Sexually active men must commit to use an effective method of birth control whiletaking the study intervention and for 90 days after the dose of study intervention.
Part B:
Participants must have one of the following bleeding disorders: Severe congenitalHemophilia A and Severe and/or moderately severe congenital Hemophilia B.
Participants whose bleeding is not well controlled on their current treatmentregimen.
Medical records documenting a minimum of 2 years of bleeding event history.
Willing to undergo a weaning period from prior Hemophilia A or Hemophilia Btreatment or prophylaxis.
Sexually active men must commit to use an effective method of birth control whiletaking the study intervention and for 90 days after the dose of study intervention.
Exclusion
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 perminute (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 beforeScreening, or is planned to undergo surgery during the study.
Participant has received vaccination within 14 days before the dose of studyintervention 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 liverdisease, 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 ofthe Investigator, participation would not be in the best interest of the participantor could prevent, limit or confound protocol-specified assessments.
Participants with a history of all types of thrombosis, including any arterialand/or venous thrombosis, superficial thrombophlebitis, or embolism. Additionally,participants with a history of thrombotic microangiopathy, stroke, and transientischemic attack (TIA), or abnormal findings in any prior laboratory thrombophiliaevaluation will be excluded.
Part B:
Participants with a history of all types of thrombosis, including any arterialand/or venous thrombosis, superficial thrombophlebitis, or embolism. Additionally,participants with a history of thrombotic microangiopathy, stroke, and TIA, orabnormal findings in any prior laboratory thrombophilia evaluation will be excluded.
History of one or more of the following in participants and/or family members:
FV Leiden mutation.
APC resistant.
PC or PS deficiency.
Prothrombin 20210 mutation.
ATIII deficiency.
- Impaired cardiac function or clinically significant cardiac disease, including anyof the following:
Clinically significant and/or uncontrolled heart disease such as congestiveheart failure requiring treatment (New York Heart Association Grade >=2), leftventricular ejection fraction < 50% as determined by multiple gated acquisitionor echocardiogram, or clinically significant arrhythmia.
QTcF > 450 ms ECG or congenital Long QT Syndrome at the Screening Visit.
Acute myocardial infarction or unstable angina pectoris < 3 months prior tostudy entry.
Uncontrolled hypertension (systolic BP > 150 mmHg and diastolic BP > 100 mmHg), ahistory of hypertension crisis, or a history of hypertensive encephalopathy.
Participant with the following laboratory abnormalities:
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 1.5 ×upper limit of normal (ULN);
Total bilirubin ˃3.0 × ULN and direct bilirubin ˃1.5 × ULN (unless due toGilbert's syndrome).
Calculated creatinine clearance ˂ 60 mL/min using the 2021 Chronic Kidney DiseaseEpidemiology Collaboration (CKD-EPI) formula at the Screening Visit.
Participant has positive test result for human immunodeficiency virus (HIV)antibody. a) 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. Participantswith active infection will be excluded from the study.; b) Participants who testpositive for hepatitis C virus antibody will be required to have a negative resultfor hepatitis C viral ribonucleic acid (RNA) PCR before enrollment. Individuals withpositive results for hepatitis C PCR will be excluded from the study.
Chronic liver disease (Child-Pugh class C hepatic impairment), or history of liveror kidney transplants.
Injury, trauma, and/or major surgery (mediastinoscopy, insertion of a central venousaccess device, and insertion of a feeding tube are not considered major surgery),major dental procedures (extractions, etc.) within 4 weeks of the first dose ofSR604 or planned surgery during the study.
Active infection requiring systemic antibiotic or antiviral therapy or in a sepsiscondition 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 thestudy outcome at undue risk.
Study Design
Study Description
Connect with a study center
McMaster University Medical Centre, Hamilton Health Sciences
Hamilton, Ontario L8N 3Z5
CanadaSite Not Available
California Clinical Trials Medical Group (CCTMG)
Glendale, California 91206
United StatesCompleted
Children's Hospital Los Angeles
Los Angeles, California 90027
United StatesSite Not Available
Rush University Medical Center
Chicago, Illinois 60612
United StatesSite Not Available
LA Center for Bleeding and Clotting Disorders - Metairie
Metairie, Louisiana 70001
United StatesActive - Recruiting
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan 48109
United StatesActive - Recruiting
Washington University School of Medicine
Saint Louis, Missouri 63110
United StatesActive - Recruiting
Brody School of Medicine at East Carolina University
Greenville, North Carolina 27834
United StatesSite Not Available
Penn State Milton S Hershey Medical Center Pediatrics
Hershey, Pennsylvania 17033
United StatesSite Not Available
Perelman Center for Advanced Medicine (PCAM)- Penn Blood Disorders Program
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
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