A Phase 1 Study of ProMetic Plasminogen (Human) Intravenous in Adults and Children With Plasminogen Deficiency

This is an interventional treatment trial for Type I Plasminogen Deficiency Read More

Inclusion Criteria:

1. Subject has provided informed consent/assent, or the subject's guardian has given consent if the subject is under 18 years
2. Subject is male or female between 12 and 80 years of age, inclusive
3. Subject has a diagnosis of hypoplasminogenemia evidenced by an abnormal, decreased plasminogen activity level, regardless of plasminogen antigen level
4. Subject has a plasminogen activity level ≤ 40%
5. Subject has documented immunity to hepatitis A virus (HAV) and hepatitis B virus (HBV) or has received the first dose of HAV and HBV vaccine prior to IMP administration and is scheduled to receive the second vaccine dose
6. Subject (male or female of reproductive age) agrees to use contraceptive methods from screening through 14 days after administration of IMP unless documented as biologically (e.g., post-menopausal, not begun menstruating) or surgically (vasectomized) sterile

Exclusion Criteria:

1. Subject has a history of severe reactions (e.g., anaphylaxis) to blood or blood products that may requiring resuscitation or otherwise prevent study participation in the opinion of the investigator
2. Subject has evidence of uncontrolled hypertension
3. Subject has clinical or laboratory evidence of an intercurrent infection as evidenced by symptoms including fever, tachycardia, or other specific signs and symptoms*
4. Subject is pregnant and/or lactating
5. Subject has or has had a malignancy, except for basal and squamous cell skin cancer, within 3 years of Baseline
6. Subject is a previous organ transplant recipient
7. Subject is receiving exogenous plasminogen (ocular or IV), including laboratory grade plasminogen and fresh lyophilized plasma within two weeks of Baseline
8. Subject has any psychiatric disorder, other mental disorder, or any other medical disorder that impairs the subject's ability to give informed consent or to comply with the requirements of the study protocol
9. Subject has experienced a severe adverse event in a prior cohort of this study.
10. Subject has evidence of renal dysfunction, defined as serum creatinine of > 2 times the upper limit of normal
11. Subject has evidence of hepatic dysfunction, defined as 3 times the upper limit of normal in ALT, and/or AST, and/or alkaline phosphatase (ALP)
12. Subject has participated in another IRB-approved interventional clinical trial of a drug, biologic, or device within 30 days of Baseline
13. Subject has a chronic or acute, clinically significant, inter-current illness (eg, cardiac, hepatic, renal, endocrine, neurologic, hematologic, neoplastic, immunological, and skeletal) that the investigator determines could interfere with the safety or pharmacokinetic assessments for this study
14. Subject is unable to adhere to the scheduling requirements of the protocol *Note: Subjects with an intercurrent infection cannot participate; however, subjects meeting this exclusion criterion at the Baseline Visit may return for a repeat Baseline Visit once the infection is considered resolved according to the investigator and can be included in the cohort that is actively recruiting at that time if all inclusion/exclusion criteria are met