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Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02222545
Recruitment Status : Completed
First Posted : August 21, 2014
Last Update Posted : July 6, 2021
Sponsor:
Information provided by (Responsible Party):
Omeros Corporation

Tracking Information
First Submitted Date  ICMJE August 18, 2014
First Posted Date  ICMJE August 21, 2014
Last Update Posted Date July 6, 2021
Actual Study Start Date  ICMJE November 2, 2014
Actual Primary Completion Date January 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 16, 2020)
  • 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
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: August 20, 2014)
  • Assess the safety and tolerability of multiple-dose administration of OMS721 in subjects with TMA [ Time Frame: 4 weeks ]
    Incidence of Adverse Events
  • Evaluate the clinical activity of multiple-dose administration of OMS721 in subjects with TMA [ Time Frame: 4 weeks ]
    • Platelet count
    • Serum LDH
    • Serum haptoglobin
    • Hemoglobin
    • Serum creatinine
    • TMA-related symptoms
    • Need for plasma therapy (plasma exchange or plasma infusion)
    • Need for dialysis
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 16, 2020)
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: August 20, 2014)
  • Pharmacokinetics (PK) of multiple-dose administration of OMS721 in subjects with TMA [ Time Frame: Pre-dose and up to 56 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
  • Pharmacodynamics (PD) of multiple-dose administration of OMS721 in subjects with TMA [ Time Frame: Pre-dose and up to 56 days post-dose ]
    PD measure inhibition of pathway activation
  • Immunogenicity of multiple-dose administration of OMS721 in subjects with TMA [ Time Frame: Pre-dose and up to 56 days post-dose ]
    Presence of ADA response
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies
Official Title  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Thrombotic Microangiopathies
Intervention  ICMJE Biological: OMS721
Study Arms  ICMJE
  • Experimental: OMS721 low dose
    Administration of OMS721 at a low dose
    Intervention: Biological: OMS721
  • Experimental: OMS721 medium dose
    Administration of OMS721 at a medium dose
    Intervention: Biological: OMS721
  • Experimental: OMS721 high dose
    Administration of OMS721 at a high dose
    Intervention: Biological: OMS721
Publications * 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:CD012862. doi: 10.1002/14651858.CD012862.pub2.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 16, 2020)
58
Original Estimated Enrollment  ICMJE
 (submitted: August 20, 2014)
29
Actual Study Completion Date  ICMJE August 11, 2020
Actual Primary Completion Date January 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Bulgaria,   Hong Kong,   Italy,   Lithuania,   Malaysia,   New Zealand,   Poland,   Singapore,   Taiwan,   Thailand,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02222545
Other Study ID Numbers  ICMJE OMS721-TMA-001
2014-001032-11 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Omeros Corporation
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Omeros Corporation
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Omeros Corporation
Verification Date July 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP