A Treatment Protocol for Expanded Access Administration of Prometic Plasminogen Due to Closure of Clinical Trial

A Treatment Protocol for Extended Administration of Prometic Plasminogen (Human) by Intravenous Infusion in Subjects With Hypoplasminogenemia Requiring Plasminogen Replacement Therapy

The clinical trial is no longer enrolling and is currently closing and Prometic will continue to provide Plasminogen (Human) under a treatment protocol to subjects in the United States (US) with hypoplasminogenemia requiring plasminogen replacement therapy who completed the End of Study visit in the following Prometic-sponsored clinical trials: 2002C011G, 2002C013G, 2002C016G, 2002C017G and 2002C019G.

Study Overview

• Status: No longer available
• Conditions: Hypoplasminogenemia
• Intervention / Treatment: Biological: Ryplazim

Detailed Description

US adult and pediatric subjects with hypoplasminogenemia requiring plasminogen replacement therapy who completed the End of Study visit in the following Prometic-sponsored clinical studies: 2002C011G, 2002C013G, 2002C016G, 2002C017G and 2002C019G will continue to receive their repeat-dose study of Plasminogen (Human) under a treatment protocol. Approximately, 15 subjects will be enrolled in the treatment protocol under an expanded access to continue to receive Plasminogen (Human) at a dose of 6.6 mg/kg as a 10 to 30-minute intravenous (IV) infusion at site visits or at home by either the subject or caregiver at the same frequency as in their prior Prometic-sponsored clinical study. Subjects or caregivers may be trained for study drug administration as deemed appropriate by the Investigator. Dosing frequency will be adjusted by the Investigator based on clinical response and plasminogen activity trough levels.

Subjects who completed the End of Study visit in studies 2002C011G, 2002C013G, 2002C016G, 2002C017G and 2002C019G and signed informed consent (and assent, if applicable) for the present study will be enrolled in this study; this visit will be designated as Day 1 of the present study. Subjects will return to their study site every 26 weeks, or more often as determined by the Investigator, for safety assessments and measurements of plasminogen activity trough levels at the Investigator's discretion.

Treatment with Plasminogen (Human) will continue until the product has been approved and is commercially available or discontinued at any time by the Sponsor or at the discretion of the Investigator.

• Study Type: Expanded Access
• Expanded Access Type: Individual Patients; Intermediate-size Population; Treatment IND/Protocol

Contacts and Locations

Study Locations

• United States
  • California
    • Beverly Hills, California, United States, 90211
      • Tower Hematology and Oncology
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Indiana Hemophilia and Thrombosis Center
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Subjects must meet all the criteria below to participate in this study:

  1. The subject or the subject's caregiver has provided informed consent (as well as assent by subjects with ages dictated by local Investigational Review Board [IRB] guidelines).
  2. Subject has a diagnosis of hypoplasminogenemia requiring replacement therapy with Plasminogen (Human).
  3. Subject has completed the End of Study visit at a United States site in the following Prometic-sponsored clinical studies: 2002C011G, 2002C013G, 2002C016G, 2002C017G and 2002C019G.
  4. Female and male subject agrees to use contraceptive methods from Day 1 through 14 days after last dose of study treatment (unless documented as biologically or surgically sterile [e.g., postmenopausal, vasectomized]), or has not reached reproductive age.

Exclusion Criteria:

• Subjects who are pregnant and/or lactating are excluded from participating in the study.

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Prometic Biotherapeutics, Inc.
• Investigators: Principal Investigator: Jeremy Lorber, MD, Tower Hematology, Oncology; Principal Investigator: Heather McDaniel, MD, Vanderbilt Childrens Hospital

Study record dates

Study Major Dates

• Study Start: December 7, 2022
• Primary Completion: December 7, 2022
• Study Completion: December 7, 2022

Study Registration Dates

• First Submitted: August 10, 2018
• First Submitted That Met QC Criteria: August 20, 2018
• First Posted (Actual): August 22, 2018

Study Record Updates

• Last Update Posted (Estimate): December 9, 2022
• Last Update Submitted That Met QC Criteria: December 8, 2022
• Last Verified: December 1, 2022

More Information

Terms related to this study

• Other Study ID Numbers: 2002C018G

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No