Thrombotic MicroangiopathiesPhase 2NCT02222545

Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies

A Phase 2 study testing OMS721 in people with Thrombotic Microangiopathies, run by Omeros Corporation at 24 sites in 12 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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). Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Thrombotic Microangiopathies
What is being tested?: OMS721
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 58 participants
Where: 24 sitesin 12 countries
Study dates: 2 Nov 2014to 11 Aug 2020
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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)

Trial locations and contacts

24 sites in 12 countries.

Country

City, postcode or hospital

24 sites in 12 countries

United States5 sites

Omeros Investigational Site
Duarte, California, 91010
Omeros Investigational Site
Durham, North Carolina, 27710
Omeros Investigational Site
Madison, Wisconsin, 53792
Omeros Investigational Site
New York, New York, 10065
Omeros Investigational Site
Rochester, Minnesota, 55905

Other trials in United States

Poland4 sites

Omeros Investigational Site
Katowice
Omeros Investigational Site
Krakow
Omeros Investigational Site
Lodz
Omeros Investigational Site
Warsaw

Other trials in Poland

Belgium3 sites

Omeros Investigational Site
Brussels
Omeros Investigational Site
Leuven
Omeros Investigational Site
Liège

Other trials in Belgium

Thailand3 sites

Omeros Investigational Site
Ban Pathumwan
Omeros Investigational Site
Bangkok
Omeros Investigational Site
Pathum Thani

Other trials in Thailand

Taiwan2 sites

Omeros Investigational Site
Taichung
Omeros Investigational Site
Taipei

Other trials in Taiwan

Bulgaria1 site

Omeros Investigational Site
Sofia

Other trials in Bulgaria

Hong Kong1 site

Omeros Investigational Site
Shatin

Other trials in Hong Kong

Italy1 site

Omeros Investigational Site
Bergamo

Other trials in Italy

Lithuania1 site

Omeros Investigational Site
Vilnius

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Malaysia1 site

Omeros Investigational Site
Selangan

Other trials in Malaysia

New Zealand1 site

Omeros Investigational Site
Christchurch

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Singapore1 site

Omeros Investigational Site
Singapore

Other trials in Singapore

About this study

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

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).

Read the 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.

Conditions: Thrombotic Microangiopathies
Being tested: OMS721
Keywords: TMA, aHUS, HSCT-associated TMA, TTP
Medical subject headings: Thrombotic Microangiopathies, Atypical Hemolytic Uremic Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

OMS721Biological

Study groups in NCT02222545
Study group	Role	What they receive
OMS721 low dose	Experimental	Administration of OMS721 at a low dose Receives: Biological: OMS721
OMS721 medium dose	Experimental	Administration of OMS721 at a medium dose Receives: Biological: OMS721
OMS721 high dose	Experimental	Administration of OMS721 at a high dose Receives: Biological: OMS721

What is being measured

The primary outcome is the main question the study is designed to answer.

Assess the Safety and Tolerability of Multiple-dose Administration of OMS721 in Participants With TMA
Incidence of treatment-emergent adverse events (AEs): clinically significant changes in vital signs, ECG, and laboratory tests were reported as AEs.
Measured: Day 1 to 37 days after end of treatment, approximately up to 31 weeks.
Number of Participants With HSCT-TMA Who Respond to OMS721
Response defined as: Improvement in TMA laboratory markers of platelet count and lactate dehydrogenase (LDH) and improvement in clinical status
Measured: Day 1 to up to 2 years following the first dose of OMS721

Secondary outcomes (15)

Participants With HSCT-TMA Treated With OMS721: 100-day Survival
Number of participants alive from the date of TMA diagnosis
Measured: Study Day of HSCT-TMA diagnosis to 100 days later
Participants With HSCT-TMA Treated With OMS721: Overall Survival
Survival days from the day of TMA diagnosis
Measured: Study Day of HSCT-TMA diagnosis to up to 2 years following first dose of OMS721
Participants With HSCT-TMA Treated With OMS721: Duration of Response
Number of days from the first response date to the first relapse date
Measured: Study Day 1 to up to 2 years following first dose of OMS721
Participants With HSCT-TMA Treated With OMS721: Freedom From Platelet Transfusion
Number of participants with absence of platelet transfusions
Measured: Study Day -14 to 4 weeks following the last platelet transfusion
Participants With HSCT-TMA Treated With OMS721: Freedom From Red Blood Cell (RBC) Transfusion
Number of participants with absence of RBC transfusions
Measured: Study Day -14 to 4 weeks following the last RBC transfusion
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Platelet Count
Changes from baseline in Platelet count
Measured: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA: Pharmacokinetics (PK) of Multiple-dose Administration of OMS721
PK parameters including clearance rate
Measured: Pre-dose and up to 204 days post-dose
Participants With HSCT-TMA (ADA)
Presence of ADA response. Immunogenicity of multiple-dose administration of OMS721 in subjects with TMA
Measured: Pre-dose and up to 204 days post-dose
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in LDH
Changes from baseline in LDH
Measured: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Creatine
Changes from baseline in Creatine
Measured: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Haptoglobin
Changes from baseline in Haptoglobin
Measured: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Hemoglobin
Changes from baseline in Hemoglobin
Measured: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA: Pharmacokinetics (PK) of Multiple-dose Administration of OMS721
PK parameters Apparent volume of the central compartment (V1)
Measured: Pre-dose and up to 204 days post-dose
Participants With HSCT-TMA: Pharmacokinetics (PK) of Multiple-dose Administration of OMS721
PK parameters Concentration of OMS721 that achieves half maximum elimination rate (KM) (ug/mL)
Measured: Pre-dose and up to 204 days post-dose
Participants With HSCT-TMA: Pharmacodynamics (PD)
PD measure is expressed as percentage inhibition of C4d to assess ex-vivo lectin pathway activation
Measured: Pre-dose and up to 204 days post-dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

2 Nov 201411 Aug 2020

First posted21 Aug 2014 (estimated)
Study start2 Nov 2014
Primary completion30 Jan 2020
Study completion11 Aug 2020

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Thrombotic MicroangiopathiesStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 58 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02222545
Sponsor's protocol ID: OMS721-TMA-001
Other IDs: 2014-001032-11, 2014
Status last verified: Jan 2024
First submitted: 18 Aug 2014
Last updated: 28 Aug 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Expanded access: Available

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.
PubMed 35439028
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.
PubMed 33783815

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 28 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT02222545
Acronym / sponsor ID: OMS721-TMA-001
Conditions: Thrombotic Microangiopathies
Keywords: TMA, aHUS, HSCT-associated TMA, TTP
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 58 participants (actual)
Lead sponsor: Omeros CorporationIndustry (pharma, biotech, devices)
Study start: 2 Nov 2014 (actual)
Primary completion: 30 Jan 2020 (actual)
Study completion: 11 Aug 2020 (actual)
First posted: 21 Aug 2014 (estimated)
Results: Posted 28 Aug 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 3

Arm	Type	Receives
OMS721 low dose	Experimental	Biological: OMS721Administration of OMS721 at a low dose
OMS721 medium dose	Experimental	Biological: OMS721Administration of OMS721 at a medium dose
OMS721 high dose	Experimental	Biological: OMS721Administration of OMS721 at a high dose

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

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

Key exclusion criteria (4 listed)

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)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
OMS721	Biological	OMS721 high dose, OMS721 low dose, OMS721 medium dose

Primary outcome

Assess the Safety and Tolerability of Multiple-dose Administration of OMS721 in Participants With TMA
Time frame: Day 1 to 37 days after end of treatment, approximately up to 31 weeks.
Incidence of treatment-emergent adverse events (AEs): clinically significant changes in vital signs, ECG, and laboratory tests were reported as AEs.
Number of Participants With HSCT-TMA Who Respond to OMS721
Time frame: Day 1 to up to 2 years following the first dose of OMS721
Response defined as: Improvement in TMA laboratory markers of platelet count and lactate dehydrogenase (LDH) and improvement in clinical status

15 secondary outcomes

Participants With HSCT-TMA Treated With OMS721: 100-day Survival
Time frame: Study Day of HSCT-TMA diagnosis to 100 days later
Participants With HSCT-TMA Treated With OMS721: Overall Survival
Time frame: Study Day of HSCT-TMA diagnosis to up to 2 years following first dose of OMS721
Participants With HSCT-TMA Treated With OMS721: Duration of Response
Time frame: Study Day 1 to up to 2 years following first dose of OMS721
Participants With HSCT-TMA Treated With OMS721: Freedom From Platelet Transfusion
Time frame: Study Day -14 to 4 weeks following the last platelet transfusion
Participants 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
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Platelet Count
Time frame: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA: Pharmacokinetics (PK) of Multiple-dose Administration of OMS721
Time frame: Pre-dose and up to 204 days post-dose
Participants With HSCT-TMA (ADA)
Time frame: Pre-dose and up to 204 days post-dose
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in LDH
Time frame: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Creatine
Time frame: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Haptoglobin
Time frame: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA Treated With OMS721: Change From Baseline in Hemoglobin
Time frame: Study Day 1 to Day 97, approximately 13 weeks
Participants With HSCT-TMA: Pharmacokinetics (PK) of Multiple-dose Administration of OMS721
Time frame: Pre-dose and up to 204 days post-dose
Participants With HSCT-TMA: Pharmacokinetics (PK) of Multiple-dose Administration of OMS721
Time frame: Pre-dose and up to 204 days post-dose
Participants With HSCT-TMA: Pharmacodynamics (PD)
Time frame: Pre-dose and up to 204 days post-dose

Results and safety

Results posted to the registry on 28 Aug 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
OMS721 Low Dose	3	2
OMS721 Medium Dose	3	3
OMS721 High Dose	52	27

Primary outcome: Assess the Safety and Tolerability of Multiple-dose Administration of OMS721 in Participants With TMA

Time frame: Day 1 to 37 days after end of treatment, approximately up to 31 weeks.. Count of participants. Unit: Participants

Measure	OMS721 Low Dose n = 0	OMS721 Medium Dose n = 0	OMS721 High Dose n = 28
Value	0	0	27

Population analysed: 28 participants entered the study with a diagnosis of HSCT-TMA; all participants were in the High Dose Arm

Primary outcome: Number of Participants With HSCT-TMA Who Respond to OMS721

Time frame: Day 1 to up to 2 years following the first dose of OMS721. Count of participants. Unit: Participants

Measure	OMS721 Low Dose n = 0	OMS721 Medium Dose n = 0	OMS721 High Dose n = 28
Value	0	0	17

Population analysed: Only subjects with HSCT-TMA were analyzed; 28 subjects with HSCT were in the Full Analysis Set (FAS)

15 secondary outcomes are reported in the full results.

Adverse events

Time frame: The Adverse Event period of time was from the date of Consent to 2 years after the first dose of study drug.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
HSCT-TMA-001 High Dose (N=28)	17 of 28 (60.7%)	27 of 28 (96.4%)	16 of 28
aHUS OMS721-TMA-001 Low Dose	2 of 3 (66.7%)	2 of 3 (66.7%)	0 of 3
aHUS OMS721-TMA-001 Medium Dose	0 of 2 (0%)	1 of 2 (50%)	0 of 2
aHUS OMS721-TMA-001 High Dose	12 of 20 (60%)	19 of 20 (95%)	3 of 20
TTP OMS721-TMA-001 Medium Dose	0 of 1 (0%)	0 of 1 (0%)	0 of 1
TTP OMS721-TMA-001 High Dose	2 of 4 (50%)	3 of 4 (75%)	0 of 4

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 35439028Khaled 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.
Derived from this study: PubMed 33783815Pugh 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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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

24 sites in 12 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing OMS721 in people with Thrombotic Microangiopathies, run by Omeros Corporation at 24 sites in 12 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 24 sites in United States, Poland, Belgium, Thailand, Taiwan and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 28 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Assess the Safety and Tolerability of Multiple-dose Administration of OMS721 in Participants With TMA (time frame: Day 1 to 37 days after end of treatment, approximately up to 31 weeks.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02222545, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Aug 2024.