Hypoplasminogenemia MedDRA version: 18.1 Level: PT Classification code 10035493 Term: Plasminogen decreased System Organ Class: 10022891 - Investigations
Conditions
Interventions
Product Name: Plasminogen (Human) ProMetic
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: Human Plasminogen
Other descriptive name: PLASMINOGEN (HUMAN) INTRAVENOUS LYOPHIL
Sponsors
ProMetic BioTherapeutics Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject or legal guardian has provided informed consent (as well as assent by subjects with ages dictated by local Investigational Review Board (IRB) guidelines). 2. Subject is male or female between the ages of 2 and 80 years (inclusive). 3. Subject has a documented history of lesions and symptoms consistent with a diagnosis of hypoplasminogenemia. 4. Subject has plasminogen levels ==65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: 1. Subject has a history of anaphylactic reactions to blood or blood products that may interfere with participation in the study in the opinion of the investigator. 2. Subject has uncontrolled hypertension. 3. Subject has clinical or laboratory evidence of an intercurrent infection as evidenced by symptoms including fever, tachycardia, or other systemic signs and symptoms. (Note: Subjects with an intercurrent clinically significant infection cannot participate; however, once the infection has resolved according to the investigator, they can be re-screened if enrollment is still open.) 4. Subject is pregnant and/or lactating. 5. Subject has a malignancy, except for basal or squamous cell skin cancer, within 3 years before screening. 6. Subject is a previous organ transplant recipient. 7. Subject is in receipt of exogenous plasminogen (ocular or IV), such as laboratory grade plasminogen, fresh frozen plasma, or ProMetic Plasminogen (Human) within 2 weeks of the first dose of the IMP. 8. Subject has a psychiatric disorder, other mental disorder, or any other medical disorder that impairs the subject's ability to provide informed consent or to comply with the requirements of the study protocol. 9. Subject has evidence of renal dysfunction defined as of > 2 x the upper limit of normal (ULN) in serum creatinine. 10. Subject has evidence of hepatic dysfunction defined as > 3 x ULN in alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP). 11. Subject has participated in another IRB-approved interventional clinical trial of drug, biologic, or device within 30 days before the first dose of the IMP. 12. Subject has a chronic or acute clinically significant intercurrent illness (e.g., cardiac, hepatic, renal, endocrine, neurologic, hematologic, neoplastic, immunological, and skeletal) that the investigator determines could interfere with the assessments in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To achieve an increase of individual trough plasminogen activity by at least an absolute 10% (i.e., 10 U/dL) from baseline in adult and pediatric subjects with hypoplasminogenemia during the 12 weeks of plasminogen replacement therapy in Segment 2. ;Secondary Objective: To evaluate the safety and tolerability of plasminogen replacement therapy during the 12 weeks of dosing in Segment 2. To evaluate the efficacy of plasminogen replacement therapy on clinically evident or visible symptoms of hypoplasminogenemia during the 12 weeks of dosing in Segment 2.;Primary end point(s): The number and percentage of subjects who achieve the target plasminogen activity trough levels for at least 3 measurements by the 12 week time-point during Segment 2. The target trough level is defined as an increase in plasminogen activity level of at least an absolute 10% (10 U/dL) from the subject's individual baseline level. Baseline level is defined as the plasminogen activity level measured before Segment 1 dosing (or Segment 2, Dose #1, if Segment 1 is not required).;Timepoint(s) of evaluation of this end point: After 12 weeks treatment during Segment 2 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): For analysis of safety and tolerability, TEAEs and SAEs will be summarized descriptively. Clinical laboratory tests (hematology, biochemistry, urinalysis, fibrinolysis/coagulation) will be presented in summary and shift tables. The numbers of subjects who had changes from baseline in viral tests and immunogenicity tests will be presented in individual subject listings and summary tables. The secondary efficacy endpoints are: - Number and size of lesions assessed at each study site visit. - Evidence of improvement in affected organ functionality assessed at each study visit, if appropriate e.g. FEV1, FVC, FEV1/FVC ratio for subjects with bronchial involvement. - Patient assessments of general overall health status (Quality of Life - 10-point scale) at each study visit. - Physician global clinical impression at each study visit (7-point scale). Efficacy endpoints are considered achieved if 30% of subjects (i.e. 4 or more) demonstrate at least a 50% improvement in lesion number/size or functionality impact from baseline. ;Timepoint(s) of evaluation of this end point: Safety and tolerability: At each study visit during dosing in Segment 1 (Day -3) and Segment 2 (during 12 weeks) and at study discontinuation, which is 30 days after the final dose of IMP. Efficacy: At each study visit during 12 weeks treatment in Segment 2 and at study discontinuation. | — |
Countries
Norway, United States
Contacts
Public ContactVice President, Clinical Trial Serv
KLIFO A/S
Outcome results
None listed