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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02222545




Registration number
NCT02222545
Ethics application status
Date submitted
18/08/2014
Date registered
21/08/2014
Date last updated
11/01/2019

Titles & IDs
Public title
Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies
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
Secondary ID [1] 0 0
2014-001032-11
Secondary ID [2] 0 0
OMS721-TMA-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Thrombotic Microangiopathies 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Other interventions - OMS721

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


Other interventions: OMS721


Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
4 to 24 weeks
Primary outcome [2] 0 0
Evaluate the clinical activity of multiple-dose administration of OMS721 in subjects with TMA - Clinical activity as assessed by platelet count
Timepoint [2] 0 0
4 to 24 weeks
Secondary outcome [1] 0 0
Pharmacokinetics (PK) of multiple-dose administration of OMS721 in subjects with TMA - PK parameters including maximum concentration, time to maximum concentration, elimination half-life, area under time-concentration curve, clearance, and volume of distribution
Timepoint [1] 0 0
Pre-dose and up to 204 days post-dose
Secondary outcome [2] 0 0
Pharmacodynamics (PD) of multiple-dose administration of OMS721 in subjects with TMA - PD measure of inhibition of ex vivo lectin pathway activation
Timepoint [2] 0 0
Pre-dose and up to 204 days post-dose
Secondary outcome [3] 0 0
Immunogenicity of multiple-dose administration of OMS721 in subjects with TMA - Presence of ADA response
Timepoint [3] 0 0
Pre-dose and up to 204 days post-dose

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
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
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)

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Minnesota
Country [3] 0 0
United States of America
State/province [3] 0 0
Missouri
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
North Carolina
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Wisconsin
Country [9] 0 0
Belgium
State/province [9] 0 0
Brussels
Country [10] 0 0
Belgium
State/province [10] 0 0
Leuven
Country [11] 0 0
Belgium
State/province [11] 0 0
Liege
Country [12] 0 0
Hong Kong
State/province [12] 0 0
Pok Fu Lam
Country [13] 0 0
Hong Kong
State/province [13] 0 0
Sha Tin
Country [14] 0 0
Italy
State/province [14] 0 0
Bergamo
Country [15] 0 0
Lithuania
State/province [15] 0 0
Vilnius
Country [16] 0 0
Malaysia
State/province [16] 0 0
Selangor
Country [17] 0 0
New Zealand
State/province [17] 0 0
Christchurch
Country [18] 0 0
New Zealand
State/province [18] 0 0
Hamilton
Country [19] 0 0
Poland
State/province [19] 0 0
Bialystok
Country [20] 0 0
Poland
State/province [20] 0 0
Katowice
Country [21] 0 0
Poland
State/province [21] 0 0
Krakow
Country [22] 0 0
Poland
State/province [22] 0 0
Warsaw
Country [23] 0 0
Poland
State/province [23] 0 0
Lódz
Country [24] 0 0
Thailand
State/province [24] 0 0
Bangkok
Country [25] 0 0
Thailand
State/province [25] 0 0
Chiang Mai
Country [26] 0 0
Thailand
State/province [26] 0 0
Pathum Thani
Country [27] 0 0
Thailand
State/province [27] 0 0
Pathumwan
Country [28] 0 0
Thailand
State/province [28] 0 0
Songkhla

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Omeros Corporation
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to assess the safety, tolerability, and clinical activity of
OMS721 in subjects with thrombotic microangiopathies (TMA), which include atypical hemolytic
uremic syndrome (aHUS), thrombotic thrombocytopenia (TTP), and hematopoietic stem cell
transplant -associated TMA (HSCT-associated TMA). The study will also evaluate clinical
activity, pharmacokinetics (PK), pharmacodynamics (PD), and anti-drug antibody response
(ADA).
Trial website
https://clinicaltrials.gov/show/NCT02222545
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Soyoung Han
Address 0 0
Country 0 0
Phone 0 0
206-676-0819
Fax 0 0
Email 0 0
shan@omeros.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02222545