Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies

July 1, 2021 updated by: Omeros Corporation

A Phase 2, Uncontrolled, Three-Stage, Dose-Escalation Cohort Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Clinical Activity of OMS721 in Adults With Thrombotic Microangiopathies

The purpose of this study is to assess the safety, efficacy, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of OMS721 in patients with thrombotic microangiopathies (TMA).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This is a Phase 2, uncontrolled, 3-stage, ascending-dose-escalation study in patients with 1 of 3 forms of TMA: atypical hemolytic uremic syndrome (aHUS), thrombotic thrombocytopenia (TTP), and hematopoietic stem cell transplant - associated TMA (HSCT-associated TMA). In Stage 1 of the study, OMS721 was administered to 3 cohorts, with dose escalation by cohort to identify the optimal dosing regimen. In Stage 2, the dose selected in the first stage was administered to expanded cohorts of patients with distinct etiologies (aHUS alone in 1 cohort and TTP or HSCT-TMA in the other cohort). Patients completing Stage 2 were eligible for continued treatment in Stage 3 if they tolerated OMS721 treatment and derived clinical benefit. Enrollment in the study has been completed.

Study Type

Interventional

Enrollment (Actual)

58

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Brussels, Belgium
        • Omeros Investigational Site
      • Leuven, Belgium
        • Omeros Investigational Site
      • Liege, Belgium
        • Omeros Investigational Site
  • Bulgaria
      • Sofia, Bulgaria
        • Omeros Investigational Site
  • Hong Kong
      • PokFuLam, Hong Kong
        • Omeros Investigational Site
      • Sha Tin, Hong Kong
        • Omeros Investigational Site
  • Italy
      • Bergamo, Italy
        • Omeros Investigational Site
  • Lithuania
      • Vilnius, Lithuania
        • Omeros Investigational Site
  • Malaysia
      • Selangor, Malaysia
        • Omeros Investigational Site
  • New Zealand
      • Christchurch, New Zealand
        • Omeros Investigational Site
  • Poland
      • Katowice, Poland
        • Omeros Investigational Site
      • Krakow, Poland
        • Omeros Investigational Site
      • Warsaw, Poland
        • Omeros Investigational Site
      • Łódź, Poland
        • Omeros Investigational Site
  • Singapore
      • Singapore, Singapore
        • Omeros Investigational Site
  • Taiwan
      • Taichung, Taiwan
        • Omeros Investigational Site
      • Taipei, Taiwan
        • Omeros Investigational Site
  • Thailand
      • Bangkok, Thailand
        • Omeros Investigational Site
      • Pathum Thani, Thailand
        • Omeros Investigational Site
      • Pathumwan, Thailand
        • Omeros Investigational Site
  • United States
    • California
      • Duarte, California, United States, 91010
        • Omeros Investigational Site
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Omeros Investigational Site
    • New York
      • New York, New York, United States, 10065
        • Omeros Investigational Site
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Omeros Investigational Site
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Omeros Investigational Site

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.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Are at least age 18 at screening (Visit 1)
  2. Have a diagnosis of primary aHUS, persistent HSCT-associated TMA or TTP
  3. No clinically apparent alternative explanation for thrombocytopenia and anemia

Exclusion Criteria:

  1. Had eculizumab therapy within three months prior to screening
  2. Have STEC-HUS
  3. Have a positive direct Coombs test
  4. Have an active systemic bacterial or fungal infection requiring antimicrobial therapy (prophylactic antimicrobial therapy administered as standard of care is allowed)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: OMS721 low dose
Administration of OMS721 at a low dose
Biological: OMS721
Experimental: OMS721 medium dose
Administration of OMS721 at a medium dose
Biological: OMS721
Experimental: OMS721 high dose
Administration of OMS721 at a high dose
Biological: OMS721

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assess the safety and tolerability of multiple-dose administration of OMS721 in subjects with TMA
Time Frame: 4 to 24 weeks
Incidence of Adverse Events, vital signs, ECG, and clinical laboratory tests
4 to 24 weeks
Evaluate the response rate to OMS721 in patients with HSCT-TMA
Time Frame: 4 to 24 weeks
Improvement in TMA laboratory markers of platelet count and lactate dehydrogenase (LDH) and improvement in clinical status
4 to 24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluate the following in patients with HSCT-TMA treated with OMS721: 100-day survival
Time Frame: Study Day 1 to 100 days later
Vital status
Study Day 1 to 100 days later
Evaluate the following in patients with HSCT-TMA treated with OMS721: Overall survival
Time Frame: Study Day 1 to up to 2 years following first dose of OMS721
Vital status
Study Day 1 to up to 2 years following first dose of OMS721
Evaluate the following in patients with HSCT-TMA treated with OMS721: Duration of response
Time Frame: Study Day 1 to up to 2 years following first dose of OMS721
Number of days from the first response date to the first relapse date
Study Day 1 to up to 2 years following first dose of OMS721
Evaluate the following in patients with HSCT-TMA treated with OMS721: Freedom from platelet transfusion
Time Frame: Study Day -14 to 4 weeks following the last platelet transfusion
Absence of platelet transfusions
Study Day -14 to 4 weeks following the last platelet transfusion
Evaluate the following in patients with HSCT-TMA treated with OMS721: Freedom from red blood cell (RBC) transfusion
Time Frame: Study Day -14 to 4 weeks following the last RBC transfusion
Absence of RBC transfusions
Study Day -14 to 4 weeks following the last RBC transfusion
Evaluate the following in patients with HSCT-TMA treated with OMS721: Change from baseline in platelet count, LDH, haptoglobin, hemoglobin (Hgb), creatinine
Time Frame: Study Day 1 to up to 2 years following the first dose of OMS721
Platelet count, LDH, haptoglobin, Hgb, creatinine
Study Day 1 to up to 2 years following the first dose of OMS721
Evaluate the following in patients with HSCT-TMA, aHUS, and TTP: Pharmacokinetics (PK) of multiple-dose administration of OMS721
Time Frame: Pre-dose and up to 204 days post-dose
PK parameters including maximum concentration, time to maximum concentration, elimination half-life, area under time-concentration curve, clearance, and volume of distribution
Pre-dose and up to 204 days post-dose
Evaluate the following in patients with HSCT-TMA, aHUS, and TTP: Pharmacodynamics (PD) of multiple-dose administration of OMS721 in subjects with TMA
Time Frame: Pre-dose and up to 204 days post-dose
PD measure in inhibition of ex vivo lectin pathway activation
Pre-dose and up to 204 days post-dose
Evaluate the following in patients with HSCT-TMA, aHUS, and TTP: Immunogenicity of multiple-dose administration of OMS721 in subjects with TMA
Time Frame: Pre-dose and up to 204 days post-dose
Presence of ADA response
Pre-dose and up to 204 days post-dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Omeros Corporation

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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 Major Dates

Study Start (Actual)

November 2, 2014

Primary Completion (Actual)

January 30, 2020

Study Completion (Actual)

August 11, 2020

Study Registration Dates

First Submitted

August 18, 2014

First Submitted That Met QC Criteria

August 20, 2014

First Posted (Estimate)

August 21, 2014

Study Record Updates

Last Update Posted (Actual)

July 6, 2021

Last Update Submitted That Met QC Criteria

July 1, 2021

Last Verified

July 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • OMS721-TMA-001
  • 2014-001032-11 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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