RECRUITING

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)

Conditions

Healthy Participants Hemophilia A Hemophilia B Factor VII Deficiency Single ascending dose Multiple ascending dose Factor IX Factor VIII Factor VII

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

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)

Quick Facts

Study Start:2024-05-10

Study Completion:2026-04-27

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06349473

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 60 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:Yes

Standard Ages:ADULT

Inclusion Criteria	Exclusion Criteria
* 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. * 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.	* 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. * 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.

Inclusion Criteria

Exclusion Criteria

* 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.
* 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.

* 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.
* 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.

Contacts and Locations

Study Contact

Clinical Trials

CONTACT

925-490-0278

inf@equilibrabioscience.com

Study Locations (Sites)

California Clinical Trials Medical Group (CCTMG)

Glendale, California, 91206

United States

Children's Hospital Los Angeles

Los Angeles, California, 90027

United States

Rush University Medical Center

Chicago, Illinois, 60612

United States

LA Center for Bleeding and Clotting Disorders - Metairie

Metairie, Louisiana, 70001

United States

University of Michigan Hospitals - Michigan Medicine

Ann Arbor, Michigan, 48109

United States

Washington University School of Medicine

St Louis, Missouri, 63110

United States

Brody School of Medicine at East Carolina University

Greenville, North Carolina, 27834

United States

Penn State Milton S Hershey Medical Center Pediatrics

Hershey, Pennsylvania, 17033

United States

Perelman Center for Advanced Medicine (PCAM)- Penn Blood Disorders Program

Philadelphia, Pennsylvania, 19104

United States

Collaborators and Investigators

Sponsor: Equilibra Bioscience LLC

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-10

Study Completion Date2026-04-27

Study Record Updates

Study Start Date2024-05-10

Study Completion Date2026-04-27

Terms related to this study

Keywords Provided by Researchers

Single ascending dose
Multiple ascending dose
Factor IX
Factor VIII
Factor VII

Additional Relevant MeSH Terms

Healthy Participants
Hemophilia A
Hemophilia B
Factor VII Deficiency