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Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies

Primary Purpose

Thrombotic Microangiopathies

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
OMS721
Sponsored by
Omeros Corporation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Thrombotic Microangiopathies focused on measuring TMA, aHUS, HSCT-associated TMA, TTP

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site
  • Omeros Investigational Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

OMS721 low dose

OMS721 medium dose

OMS721 high dose

Arm Description

Administration of OMS721 at a low dose

Administration of OMS721 at a medium dose

Administration of OMS721 at a high dose

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
August 18, 2014
Last Updated
July 1, 2021
Sponsor
Omeros Corporation
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1. Study Identification

Unique Protocol Identification Number
NCT02222545
Brief Title
Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2021
Overall Recruitment Status
Completed
Study Start Date
November 2, 2014 (Actual)
Primary Completion Date
January 30, 2020 (Actual)
Study Completion Date
August 11, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Omeros Corporation

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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).
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thrombotic Microangiopathies
Keywords
TMA, aHUS, HSCT-associated TMA, TTP

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
58 (Actual)

8. Arms, Groups, and Interventions

Arm Title
OMS721 low dose
Arm Type
Experimental
Arm Description
Administration of OMS721 at a low dose
Arm Title
OMS721 medium dose
Arm Type
Experimental
Arm Description
Administration of OMS721 at a medium dose
Arm Title
OMS721 high dose
Arm Type
Experimental
Arm Description
Administration of OMS721 at a high dose
Intervention Type
Biological
Intervention Name(s)
OMS721
Primary Outcome Measure Information:
Title
Assess the safety and tolerability of multiple-dose administration of OMS721 in subjects with TMA
Description
Incidence of Adverse Events, vital signs, ECG, and clinical laboratory tests
Time Frame
4 to 24 weeks
Title
Evaluate the response rate to OMS721 in patients with HSCT-TMA
Description
Improvement in TMA laboratory markers of platelet count and lactate dehydrogenase (LDH) and improvement in clinical status
Time Frame
4 to 24 weeks
Secondary Outcome Measure Information:
Title
Evaluate the following in patients with HSCT-TMA treated with OMS721: 100-day survival
Description
Vital status
Time Frame
Study Day 1 to 100 days later
Title
Evaluate the following in patients with HSCT-TMA treated with OMS721: Overall survival
Description
Vital status
Time Frame
Study Day 1 to up to 2 years following first dose of OMS721
Title
Evaluate the following in patients with HSCT-TMA treated with OMS721: Duration of response
Description
Number of days from the first response date to the first relapse date
Time Frame
Study Day 1 to up to 2 years following first dose of OMS721
Title
Evaluate the following in patients with HSCT-TMA treated with OMS721: Freedom from platelet transfusion
Description
Absence of platelet transfusions
Time Frame
Study Day -14 to 4 weeks following the last platelet transfusion
Title
Evaluate the following in patients with HSCT-TMA treated with OMS721: Freedom from red blood cell (RBC) transfusion
Description
Absence of RBC transfusions
Time Frame
Study Day -14 to 4 weeks following the last RBC transfusion
Title
Evaluate the following in patients with HSCT-TMA treated with OMS721: Change from baseline in platelet count, LDH, haptoglobin, hemoglobin (Hgb), creatinine
Description
Platelet count, LDH, haptoglobin, Hgb, creatinine
Time Frame
Study Day 1 to up to 2 years following the first dose of OMS721
Title
Evaluate the following in patients with HSCT-TMA, aHUS, and TTP: Pharmacokinetics (PK) of multiple-dose administration of OMS721
Description
PK parameters including maximum concentration, time to maximum concentration, elimination half-life, area under time-concentration curve, clearance, and volume of distribution
Time Frame
Pre-dose and up to 204 days post-dose
Title
Evaluate the following in patients with HSCT-TMA, aHUS, and TTP: Pharmacodynamics (PD) of multiple-dose administration of OMS721 in subjects with TMA
Description
PD measure in inhibition of ex vivo lectin pathway activation
Time Frame
Pre-dose and up to 204 days post-dose
Title
Evaluate the following in patients with HSCT-TMA, aHUS, and TTP: Immunogenicity of multiple-dose administration of OMS721 in subjects with TMA
Description
Presence of ADA response
Time Frame
Pre-dose and up to 204 days post-dose

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Are at least age 18 at screening (Visit 1) Have a diagnosis of primary aHUS, persistent HSCT-associated TMA or TTP No clinically apparent alternative explanation for thrombocytopenia and anemia Exclusion Criteria: Had eculizumab therapy within three months prior to screening Have STEC-HUS Have a positive direct Coombs test Have an active systemic bacterial or fungal infection requiring antimicrobial therapy (prophylactic antimicrobial therapy administered as standard of care is allowed)
Facility Information:
Facility Name
Omeros Investigational Site
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Facility Name
Omeros Investigational Site
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Omeros Investigational Site
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Omeros Investigational Site
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Omeros Investigational Site
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
Omeros Investigational Site
City
Brussels
Country
Belgium
Facility Name
Omeros Investigational Site
City
Leuven
Country
Belgium
Facility Name
Omeros Investigational Site
City
Liege
Country
Belgium
Facility Name
Omeros Investigational Site
City
Sofia
Country
Bulgaria
Facility Name
Omeros Investigational Site
City
PokFuLam
Country
Hong Kong
Facility Name
Omeros Investigational Site
City
Sha Tin
Country
Hong Kong
Facility Name
Omeros Investigational Site
City
Bergamo
Country
Italy
Facility Name
Omeros Investigational Site
City
Vilnius
Country
Lithuania
Facility Name
Omeros Investigational Site
City
Selangor
Country
Malaysia
Facility Name
Omeros Investigational Site
City
Christchurch
Country
New Zealand
Facility Name
Omeros Investigational Site
City
Katowice
Country
Poland
Facility Name
Omeros Investigational Site
City
Krakow
Country
Poland
Facility Name
Omeros Investigational Site
City
Warsaw
Country
Poland
Facility Name
Omeros Investigational Site
City
Łódź
Country
Poland
Facility Name
Omeros Investigational Site
City
Singapore
Country
Singapore
Facility Name
Omeros Investigational Site
City
Taichung
Country
Taiwan
Facility Name
Omeros Investigational Site
City
Taipei
Country
Taiwan
Facility Name
Omeros Investigational Site
City
Bangkok
Country
Thailand
Facility Name
Omeros Investigational Site
City
Pathum Thani
Country
Thailand
Facility Name
Omeros Investigational Site
City
Pathumwan
Country
Thailand

12. IPD Sharing Statement

Citations:
PubMed Identifier
35439028
Citation
Khaled SK, Claes K, Goh YT, Kwong YL, Leung N, Mendrek W, Nakamura R, Sathar J, Ng E, Nangia N, Whitaker S, Rambaldi A; OMS721-TMA-001 Study Group Members. Narsoplimab, a Mannan-Binding Lectin-Associated Serine Protease-2 Inhibitor, for the Treatment of Adult Hematopoietic Stem-Cell Transplantation-Associated Thrombotic Microangiopathy. J Clin Oncol. 2022 Aug 1;40(22):2447-2457. doi: 10.1200/JCO.21.02389. Epub 2022 Apr 19.
Results Reference
derived
PubMed Identifier
33783815
Citation
Pugh D, O'Sullivan ED, Duthie FA, Masson P, Kavanagh D. Interventions for atypical haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Mar 23;3(3):CD012862. doi: 10.1002/14651858.CD012862.pub2.
Results Reference
derived

Learn more about this trial

Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies

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