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)
Recruiting
Study Details
Medical Condition
- Unmapped
- Hemophilia A
- Hemophilia B
Study Drug
- Drug: SR604
- Drug: Placebo
Date
May 2024 - Apr 2026
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 18 - 60 Years years
Requirements Information
Inclusion and Exclusion Criteria
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 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:
- Participants must have one of the following bleeding disorders: Severe congenital Hemophilia A and Severe and/or moderately severe congenital Hemophilia B.
- Participants whose bleeding is not well controlled on their current treatment regimen.
- Medical records documenting a minimum of 2 years of bleeding event history.
- Willing to undergo a weaning period from prior Hemophilia A or Hemophilia B treatment or prophylaxis.
- 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.
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:
- 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.
- 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:
- FV Leiden mutation.
- APC resistant.
- PC or PS deficiency.
- Prothrombin 20210 mutation.
- ATIII deficiency.
- Impaired cardiac function or clinically significant cardiac disease, including any of the following:
- 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.
- QTcF \> 450 ms ECG or congenital Long QT Syndrome at the Screening Visit.
- 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:
- 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 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. 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. Participants with active infection will be excluded from the study.; b) 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.
Protocol Summary
This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.
Trial Locations
Location
Status
Location
California Clinical Trials Medical Group (CCTMG)
Glendale, California, United States, 91206
Status
Completed
Location
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Status
Not yet recruiting
Location
Rush University Medical Center
Chicago, Illinois, United States, 60612
Status
Not yet recruiting
Location
LA Center for Bleeding and Clotting Disorders - Metairie
Metairie, Louisiana, United States, 70001
Status
Recruiting
Location
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, United States, 48109
Status
Recruiting
Location
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Status
Recruiting
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