Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-99
SponsorOmeros Corporation

About this trial

The purpose of this study is to assess the long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV administration at 8-week intervals in patients with PNH.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Have completed the last dosing visit of the prior OMS906 PNH study.

Female patients of child bearing potential must have a negative result from a highly sensitive urine pregnancy test prior to each dose of OMS906.

Females must use highly effective birth control to prevent pregnancy during the clinical trial and for 20 weeks following their last dose of study drug.

Males must use highly effective birth control with a female partner to prevent pregnancy during the clinical trial and for 20 weeks after last dose of study drug.

Disqualifiers

Platelet count <30,000/µL or absolute neutrophil count <500 cells/µL at the start of the Evaluation Period.

Elevation of liver function tests, defined as total bilirubin > 2 x ULN, direct bilirubin > 1.5 x ULN, and elevated transaminases (alanine or aspartate aminotransferase), > 2 X ULN unless due to PNH-related hemolysis.

History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation.

Patients with unresolved serious infections caused by encapsulated bacteria including H. influenzae, S. pneumoniae and N. meningitidis.

Trial design

Design model

Single group

Treatments tested in this trial

  • OMS906 study drug

    Drug

    OMS906 study drug repeat-dose 5mg/kg IV administration at 8-week intervals

Treatment groups

25 Participants
are divided into 1 treatment group
Group A: OMS906 study drugExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

To assess overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients.

Treatment-emergent adverse events, including clinically significant clinical laboratory tests, 12-lead electrocardiograms, vital signs, and physical examinations recorded as an adverse event.

Time frame
104 weeks

Secondary outcomes

1

To assess efficacy measured by hemoglobin (Hgb).

Measured by patients achieving Hb ≥ 12.0 g/dL and by proportion of patients maintaining an increase in Hb ≥ 2 g/dL, achieved in the prior study, through the duration of the long-term extension.

Time frame
6 month intervals
2

To assess efficacy by transfusion requirements.

Measure proportion of patients who are transfusion free and mean change from baseline in transfusion frequency from the start of the long-term extension.

Time frame
Weeks 48 and 96
3

To assess efficacy by measurement of lactate dehydrogenase (LDH).

Measure mean LDH change from baseline.

Time frame
Weeks 48 and 96
4

To assess efficacy by measurement of reticulocyte count.

Measure mean change in reticulocyte count from baseline.

Time frame
Weeks 48 and 96

Other outcomes

Sponsors and contacts

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