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Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome (aHUS)

This study is currently recruiting participants.
Verified June 2017 by Omeros Corporation
Sponsor:
ClinicalTrials.gov Identifier:
NCT03205995
First Posted: July 2, 2017
Last Update Posted: July 28, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Omeros Corporation
  Purpose
The purpose of this study is to evaluate the platelet count change from baseline and safety of OMS721 in adults and adolescents with atypical hemolytic uremic syndrome (aHUS). The study will also evaluate pharmacokinetics (PK), pharmacodynamics (PD), and anti-drug antibody response (ADA).

Condition Intervention Phase
Thrombotic Microangiopathies Atypical Hemolytic Uremic Syndrome Biological: OMS721 Phase 3

Access to an investigational treatment associated with this study is available outside the clinical trial.   More info ...

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3 Study to Evaluate the Safety and Efficacy of OMS721 for the Treatment of Atypical Hemolytic Uremic Syndrome (aHUS) in Adults and Adolescents

Resource links provided by NLM:


Further study details as provided by Omeros Corporation:

Primary Outcome Measures:
  • The effect of OMS721 as measured by platelet count change from baseline [ Time Frame: 26 weeks ]
    The effect of OMS721 will be evaluated in subjects with aHUS by changes in platelet count from baseline


Secondary Outcome Measures:
  • Safety as measured by incidences of Adverse Events, vital signs, ECG, and clinical laboratory tests [ Time Frame: Pre-dose and up to 771 days post-dose ]
    Assessment of safety of OMS721 in subjects with aHUS by incidence of Adverse Events, clinically significant vital sign abnormalities, ECG abnormalities, and clinical laboratory test abnormalities

  • TMA Response [ Time Frame: 26 weeks ]
    Complete TMA response defined as normalization of platelet count, normalization of serum LDH, and > 25% decrease in serum creatinine by at least 2 consecutive measures over at least 4 consecutive weeks, within the initial 26-week period

  • TMA event-free status [ Time Frame: 26 weeks ]
    No decrease in platelet count of > 25% from baseline, no plasma exchange or plasma infusion, and no initiation of new dialysis over at least 12 consecutive weeks, within the initial 26-week period

  • Increase in eGFR [ Time Frame: 26 weeks ]
    Increase of greater than 15 ml/min/1.73 m2 in eGFR calculated by the MDRD Equation

  • Hematological Normalization [ Time Frame: 26 weeks ]
    Normalization of platelet count and normalization of serum LDH by 2 consecutive measurements over at least 4 consecutive weeks, within the initial 26-week period

  • TMA Remission [ Time Frame: 26 weeks ]
    Platelet count greater than or equal to 150,000/μL over at least 2 consecutive weeks, within the initial 26-week period

  • Incidence of antidrug antibodies (ADA) [ Time Frame: 771 days post-dose ]
    Incidences of ADA in subjects with aHUS, administered OMS721

  • Change from baseline in serum creatinine [ Time Frame: 26 weeks ]
    Assessment of subject's change from baseline in serum creatinine

  • Change from baseline in serum LDH [ Time Frame: 26 weeks ]
    Assessment of subject's change from baseline in serum LDH

  • Change from baseline in haptoglobin [ Time Frame: 26 weeks ]
    Assessment of subject's change from baseline in haptoglobin

  • Pharmacokinetics (PK): Trough plasma concentration, lower limit of quantification (LLOQ) [ Time Frame: Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771 ]
  • Pharmacokinetics (PK): Maximum plasma concentrations (Cmax) [ Time Frame: Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771 ]
  • Pharmacokinetics (PK): Area under time-concentration curve (AUC) [ Time Frame: Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771 ]
  • Pharmacodynamics (PD): Inhibition of C3 activity [ Time Frame: Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771 ]
  • Pharmacodynamics (PD): Inhibition of C4 activity [ Time Frame: Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771 ]

Estimated Enrollment: 80
Actual Study Start Date: February 23, 2017
Estimated Study Completion Date: February 2020
Estimated Primary Completion Date: February 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: OMS721
Administration of OMS721
Biological: OMS721
Intravenous loading dose followed by daily subcutaneous injections

Detailed Description:
This is a Phase 3, multicenter study of OMS721 in adults and adolescents with atypical hemolytic uremic syndrome (aHUS). The uncontrolled, open-label study will evaluate the effect of OMS721 in subjects with plasma therapy-resistant aHUS and plasma therapy-responsive aHUS. This study has four periods: Screening, Treatment Induction, Treatment Maintenance, and Follow-up. Approximate enrollment is 80 subjects. An interim analysis will be performed after 40 subjects have completed 26 weeks of treatment for potential registration.
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Competent to provide informed consent, or if a minor, have at least one parent or legal guardian to provide informed consent with written assent from the subject.
  • Are at least 12 years old at screening (Visit 1).
  • Have a clinically diagnosis of primary atypical hemolytic uremic syndrome (aHUS), with ADAMTS13 activity greater than 5% in plasma.
  • Plasma therapy-resistant aHUS patients must have a screening platelet count less than 150,000/uL, evidence of microangiopathic hemolysis, and serum creatinine greater than upper limit of normal.
  • Plasma therapy-responsive aHUS patients must have documented history of requiring plasma therapy to prevent aHUS exacerbation and received plasma therapy at least once every 2 weeks at an unchanged frequency for at least 8 weeks before first dose of OMS721.

Exclusion Criteria:

  • Have STEC-HUS, a direct positive Coombs test, history of hematopoietic stem cell transplant, and/or HUS from an identified drug.
  • History of vitamin B12 deficiency-related HUS, systemic lupus erythematosus, and/or antiphospholipid syndrome.
  • Active cancer or history of cancer (except non-melanoma skin cancers) within 5 years of screening.
  • Have been on hemodialysis or peritoneal dialysis for greater than or equal to 12 weeks.
  • Have an active systemic bacterial or fungal infection requiring systemic antimicrobial therapy (prophylactic antimicrobial therapy administered as standard of care is allowed).
  • Baseline resting heart rate less than 45 beats per minute or greater than 115 beats per minute.
  • Baseline QTcF greater than 470 milliseconds.
  • Have malignant hypertension (diastolic blood pressure [BP] greater than 120 mm Hg with bilateral hemorrhages or "cotton-wool" exudates on funduscopic examination).
  • Have a poor prognosis with a life expectancy of less than three months in the opinion of the Investigator.
  • Are pregnant or lactating.
  • Have received treatment with an investigational drug or device within four weeks prior to screening.
  • Have abnormal liver function tests defined as ALT or AST > five times ULN.
  • Have HIV infection.
  • History of cirrhosis of the liver.
  • Have previously completed treatment in an OMS721study.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03205995


Contacts
Contact: Zeny Gonda 206-676-0885 zgonda@omeros.com
Contact: Danielle Barnhart 206-676-5051 dbarnhart@omeros.com

Locations
United States, California
Omeros Investigational Site Not yet recruiting
Los Angeles, California, United States, 90025
United States, Illinois
Omeros Investigational Site Recruiting
Chicago, Illinois, United States, 60643
Sponsors and Collaborators
Omeros Corporation
Investigators
Study Director: Steve Whitaker, M.D. Omeros Corporation
  More Information

Responsible Party: Omeros Corporation
ClinicalTrials.gov Identifier: NCT03205995     History of Changes
Other Study ID Numbers: OMS721-HUS-002
First Submitted: April 17, 2017
First Posted: July 2, 2017
Last Update Posted: July 28, 2017
Last Verified: June 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Omeros Corporation:
TMA
aHUS

Additional relevant MeSH terms:
Syndrome
Hemolysis
Azotemia
Hemolytic-Uremic Syndrome
Atypical Hemolytic Uremic Syndrome
Thrombotic Microangiopathies
Disease
Pathologic Processes
Uremia
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombocytopenia
Blood Platelet Disorders