Phase 3
N=9
A Study of Prometic Plasminogen IV Infusion in Subjects With Hypoplasminogenemia
Hypoplasminogenemia · Congenital Plasminogen Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT02690714 ↗Enrolled (actual)
9
Serious AEs
20.0%
Results posted
Apr 2021
Primary outcome: Primary: Overall Clinical Success in Number and Size of Lesions as Measured by Photographic or Other Imaging Modality Depending on the Organ System Affected or Change in Affected Organ Functionality at 48 Weeks. — 34; 10; 3 Lesions
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Plasminogen (Human) intravenous (Biological)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Prometic Biotherapeutics, Inc.
- Primary completion
- Dec 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Clinical Success in Number and Size of Lesions as Measured by Photographic or Other Imaging Modality Depending on the Organ System Affected or Change in Affected Organ Functionality at 48 Weeks. |
34; 10; 3 | — |
| PRIMARY Number and Percentage of Particpants Who Achieved the Target Plasminogen Activity Trough Levels for at Least 3 Measurements in 12 Weeks During Segment 2 |
15 | — |
| SECONDARY Overall Clinical Success in Number and Size of Lesions as Measured by Photographic or Other Imaging Modality Depending on the Organ System Affected or Change in Affected Organ Functionality at 12 Weeks |
26; 14; 2; 5 | — |
| SECONDARY Clinical Global Impression-Global Improvement (CGI-I) Scores at Week 12 |
0; 11; 4; 0; 0; 0 | — |
| SECONDARY Clinical Global Impression-Global Improvement (CGI-I) Scores at Week 48 |
0; 13; 2; 0; 0; 0 | — |
| SECONDARY Number of Participants With Improved Quality of Life (QOL) Score After 12 Weeks of Study Treatment |
0; 0; 0; 0; 0; 1 | — |
| SECONDARY Number of Participants With Improved Quality of Life (QOL) Score After 48 Weeks of Study Treatment |
0; 0; 0; 0; 0; 1 | — |
| SECONDARY Plasminogen Activity Trough Levels Between Week 2 and Week 120 |
45.1; 48.9; 52.4; 48.3; 50.2; 51.0 | — |
| SECONDARY Plasminogen Antigen Trough Levels Between Week 2 and Week 120 |
6.550; 7.800; 7.268; 6.607; 9.460; 7.340 | — |
| SECONDARY Half-life (t1/2) of Plasminogen Activity After First Dose and at Week 12 |
34; 39.2 | — |
| SECONDARY AUClast of Plasminogen Activity After the First Dose and at Week 12 |
3063.6; 4656.0 | — |
| SECONDARY Cmax of Plasminogen Activity After First Dose and at Week 12 |
95; 125 | — |
| SECONDARY Cl of Plasminogen Activity After First Dose and at Week 12 |
1.44; 0.92 | — |
| SECONDARY AUCinf of Plasminogen Activity After First Dose and at Week 12 |
3605.8; 5731.8 | — |
| SECONDARY MRTlast for Plasminogen Activity After First Dose and at Week 12 |
30.6; 33.5 | — |
| SECONDARY Vss for Plasminogen Activity After First Dose and at Week 12 |
63.3; 49.3 | — |
Summary
This is a Phase 2/3 pivotal study to evaluate pharmacokinetics (PK), efficacy, and safety of Prometic Plasminogen (Human) Intravenous Lyophilized Solution, the investigational medicinal product (IMP), in pediatric and adult subjects with hypoplasminogenemia.
Eligibility Criteria
Inclusion Criteria
- Subject is a male or female between the ages of 2 and 80 years (inclusive), is able to provide informed consent or assent, and agrees to use contraceptive methods during the study (unless documented as biologically or surgically sterile or has not reached reproductive age).
- Subject has documented history of hypoplasminogenemia and has plasminogen activity level ≤ 45%.
- Subject had a documented history of lesions and symptoms consistent with a diagnosis of congenital plasminogen deficiency.
- Subject has documented vaccination to hepatitis A virus (HAV) and hepatitis B virus (HBV), or has received the first dose of HAV and HBV vaccine prior to the first dose of IMP and is scheduled to receive the second vaccine dose.
Exclusion Criteria
- Subject has uncontrolled hypertension; clinical or laboratory evidence of an intercurrent infection; a malignancy within 3 years, except for basal or squamous cell skin cancer; a psychiatric disorder; chronic or acute clinically significant inter-current illness; or evidence of renal and hepatic dysfunction.
- Subject is pregnant or lactating
- Subject has a history of anaphylactic reactions to blood or blood products that may interfere with participation in study in the opinion of the investigator.
- Subject is a previous organ transplant recipient; has received exogenous plasminogen within 2 weeks of the screening; has a history of anaphylactic reactions to blood or blood products; or has received another IRB-approved interventional clinical trial of a drug, biologic, or device within 30 days before the first dose of the IMP.
Data sourced from ClinicalTrials.gov (NCT02690714). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.