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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02222545
Registration number
NCT02222545
Ethics application status
Date submitted
18/08/2014
Date registered
21/08/2014
Date last updated
28/08/2024
Titles & IDs
Public title
Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies
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Scientific 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
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Secondary ID [1]
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2014-001032-11
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Secondary ID [2]
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OMS721-TMA-001
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Thrombotic Microangiopathies
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Condition category
Condition code
Cardiovascular
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Diseases of the vasculature and circulation including the lymphatic system
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Blood
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Other blood disorders
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Intervention/exposure
Study type
Interventional(has expanded access)
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Description of intervention(s) / exposure
Experimental: OMS721 low dose - Administration of OMS721 at a low dose
Experimental: OMS721 medium dose - Administration of OMS721 at a medium dose
Experimental: OMS721 high dose - Administration of OMS721 at a high dose
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Assess the Safety and Tolerability of Multiple-dose Administration of OMS721 in Participants With TMA
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Assessment method [1]
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Incidence of treatment-emergent adverse events (AEs): clinically significant changes in vital signs, ECG, and laboratory tests were reported as AEs.
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Timepoint [1]
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Day 1 to 37 days after end of treatment, approximately up to 31 weeks.
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Primary outcome [2]
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Number of Participants With HSCT-TMA Who Respond to OMS721
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Assessment method [2]
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Response defined as: Improvement in TMA laboratory markers of platelet count and lactate dehydrogenase (LDH) and improvement in clinical status
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Timepoint [2]
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Day 1 to up to 2 years following the first dose of OMS721
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Secondary outcome [1]
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Participants With HSCT-TMA Treated With OMS721: 100-day Survival
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Assessment method [1]
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Number of participants alive from the date of TMA diagnosis
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Timepoint [1]
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Study Day of HSCT-TMA diagnosis to 100 days later
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Secondary outcome [2]
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Participants With HSCT-TMA Treated With OMS721: Overall Survival
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Assessment method [2]
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Survival days from the day of TMA diagnosis
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Timepoint [2]
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Study Day of HSCT-TMA diagnosis to up to 2 years following first dose of OMS721
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Secondary outcome [3]
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Participants With HSCT-TMA Treated With OMS721: Duration of Response
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Assessment method [3]
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Number of days from the first response date to the first relapse date
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Timepoint [3]
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Study Day 1 to up to 2 years following first dose of OMS721
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Secondary outcome [4]
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Participants With HSCT-TMA Treated With OMS721: Freedom From Platelet Transfusion
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Assessment method [4]
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Number of participants with absence of platelet transfusions
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Timepoint [4]
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Study Day -14 to 4 weeks following the last platelet transfusion
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Secondary outcome [5]
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Participants With HSCT-TMA Treated With OMS721: Freedom From Red Blood Cell (RBC) Transfusion
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Assessment method [5]
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Number of participants with absence of RBC transfusions
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Timepoint [5]
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Study Day -14 to 4 weeks following the last RBC transfusion
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Secondary outcome [6]
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Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Platelet Count
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Assessment method [6]
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Changes from baseline in Platelet count
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Timepoint [6]
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Study Day 1 to Day 97, approximately 13 weeks
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Secondary outcome [7]
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Participants With HSCT-TMA: Pharmacokinetics (PK) of Multiple-dose Administration of OMS721
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Assessment method [7]
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PK parameters including clearance rate
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Timepoint [7]
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Pre-dose and up to 204 days post-dose
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Secondary outcome [8]
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Participants With HSCT-TMA (ADA)
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Assessment method [8]
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Presence of ADA response. Immunogenicity of multiple-dose administration of OMS721 in subjects with TMA
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Timepoint [8]
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Pre-dose and up to 204 days post-dose
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Secondary outcome [9]
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Participants With HSCT-TMA Treated With OMS721: Change From Baseline in LDH
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Assessment method [9]
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Changes from baseline in LDH
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Timepoint [9]
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Study Day 1 to Day 97, approximately 13 weeks
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Secondary outcome [10]
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Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Creatine
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Assessment method [10]
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Changes from baseline in Creatine
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Timepoint [10]
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Study Day 1 to Day 97, approximately 13 weeks
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Secondary outcome [11]
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Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Haptoglobin
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Assessment method [11]
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Changes from baseline in Haptoglobin
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Timepoint [11]
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Study Day 1 to Day 97, approximately 13 weeks
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Secondary outcome [12]
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Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Hemoglobin
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Assessment method [12]
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Changes from baseline in Hemoglobin
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Timepoint [12]
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Study Day 1 to Day 97, approximately 13 weeks
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Secondary outcome [13]
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Participants With HSCT-TMA: Pharmacokinetics (PK) of Multiple-dose Administration of OMS721
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Assessment method [13]
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PK parameters Apparent volume of the central compartment (V1)
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Timepoint [13]
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Pre-dose and up to 204 days post-dose
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Secondary outcome [14]
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Participants With HSCT-TMA: Pharmacokinetics (PK) of Multiple-dose Administration of OMS721
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Assessment method [14]
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PK parameters Concentration of OMS721 that achieves half maximum elimination rate (KM) (ug/mL)
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Timepoint [14]
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Pre-dose and up to 204 days post-dose
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Secondary outcome [15]
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Participants With HSCT-TMA: Pharmacodynamics (PD)
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Assessment method [15]
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PD measure is expressed as percentage inhibition of C4d to assess ex-vivo lectin pathway activation
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Timepoint [15]
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Pre-dose and up to 204 days post-dose
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Eligibility
Key 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
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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)
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
2/11/2014
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
11/08/2020
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Sample size
Target
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Accrual to date
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Final
58
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
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United States of America
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California
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United States of America
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Minnesota
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United States of America
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New York
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United States of America
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North Carolina
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United States of America
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Wisconsin
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Country [6]
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Belgium
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State/province [6]
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Brussels
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Belgium
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Leuven
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Belgium
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Liege
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Bulgaria
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Sofia
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Hong Kong
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Sha Tin
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Italy
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Bergamo
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Lithuania
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Vilnius
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Malaysia
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Selangan
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New Zealand
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Christchurch
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Poland
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Katowice
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Poland
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Krakow
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Poland
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Warsaw
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Poland
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Lódz
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Singapore
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Singapore
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Taiwan
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Taichung
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Taiwan
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Taipei
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Thailand
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Ban Pathumwan
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Thailand
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Bangkok
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Thailand
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Pathum Thani
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Omeros Corporation
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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).
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Trial website
https://clinicaltrials.gov/study/NCT02222545
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Trial related presentations / publications
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. 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.
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Public notes
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Contacts
Principal investigator
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Contact person for public queries
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/45/NCT02222545/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/45/NCT02222545/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT02222545
Download to PDF