POZELIMAB for Paroxysmal nocturnal hemoglobinuria

Inclusion criteria

• {"criterion_text":"- Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing.\n- Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months.\n- LDH persistently > 1.5 × Upper Limit of Normal (ULN) in the previous 6 months that the Principal Investigator (PI) attributes is due to intravascular hemolysis.\n- At least 2 screening LDH values from different visits as described in the protocol.\n- Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol and agreement to continue to remain up to date with these vaccinations during the study.\n- Note: Other protocol-defined Inclusion Criteria apply."}

Exclusion criteria

• {"criterion_text":"- Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants.\n- Body weight <40 kilograms at screening visit.\n- Patients with a known or suspected C5 mutation that is refractory to their current C5i treatment as described in the protocol.\n- Any active or ongoing infection within 2 weeks of screening or during the screening period or any recent infection as described in the protocol.\n- Known hereditary complement deficiency.\n- Note: Other protocol-defined Exclusion Criteria apply."}

Primary endpoints

• {"endpoint_text":"- Percent change in Lactate Dehydrogenase (LDH) during treatment period (TP) from baseline to week 28.","definition_or_measurement_approach":"Percent change in LDH from baseline to week 28 during the treatment period (measurement of LDH at baseline and at week 28 to calculate percent change)."}

Secondary endpoints

• {"endpoint_text":"- Normalization of LDH through week 52.","definition_or_measurement_approach":"Normalization of LDH measured through week 52 (LDH values assessed up to week 52)."}
• {"endpoint_text":"- Adequate control of hemolysis (LDH ≤1.5 × ULN) through week 52.","definition_or_measurement_approach":"Proportion/time with LDH ≤1.5 × ULN assessed through week 52."}
• {"endpoint_text":"- Transfusion avoidance through week 52.","definition_or_measurement_approach":"Assessment of transfusion avoidance (no transfusions) through week 52."}
• {"endpoint_text":"- Hemoglobin stabilization through week 52.","definition_or_measurement_approach":"Assessment of hemoglobin stabilization through week 52 (as defined in protocol)."}
• {"endpoint_text":"- Change in hemoglobin through week 52.","definition_or_measurement_approach":"Change from baseline in hemoglobin measured through week 52."}
• {"endpoint_text":"- Change in fatigue through week 52, as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale.","definition_or_measurement_approach":"Change from baseline in FACIT-Fatigue Scale scores through week 52."}
• {"endpoint_text":"- Occurrence of all Adverse Events (AEs) through week 52.","definition_or_measurement_approach":"Recording and tabulation of all AEs through week 52."}
• {"endpoint_text":"- Severity of all AEs through week 52.","definition_or_measurement_approach":"Assessment and grading of AE severity through week 52."}
• {"endpoint_text":"- Occurrence of all Treatment-Emergent Adverse Events (TEAEs) through week 52.","definition_or_measurement_approach":"Recording and tabulation of TEAEs through week 52."}
• {"endpoint_text":"- Severity of all TEAEs through week 52.","definition_or_measurement_approach":"Assessment and grading of TEAE severity through week 52."}
• {"endpoint_text":"- Change from baseline in Total Complement Hemolytic Activity Assay (CH50) through week 52.","definition_or_measurement_approach":"Change from baseline in CH50 assay values through week 52."}
• {"endpoint_text":"- Concentrations of total pozelimab through week 52.","definition_or_measurement_approach":"Measurement of total pozelimab serum concentrations through week 52."}
• {"endpoint_text":"- Concentrations of cemdisiran through week 52.","definition_or_measurement_approach":"Measurement of cemdisiran plasma concentrations through week 52."}
• {"endpoint_text":"- Concentrations of total C5 through week 52.","definition_or_measurement_approach":"Measurement of total C5 concentrations through week 52."}
• {"endpoint_text":"- Incidence of Anti-Drug Antibody (ADA) to pozelimab through week 52.","definition_or_measurement_approach":"Incidence of ADA to pozelimab assessed through week 52."}
• {"endpoint_text":"- Magnitude of ADA to pozelimab though week 52.","definition_or_measurement_approach":"Assessment of ADA titers/magnitude to pozelimab through week 52."}
• {"endpoint_text":"- Incidence of ADA to cemdisiran through week 52.","definition_or_measurement_approach":"Incidence of ADA to cemdisiran assessed through week 52."}
• {"endpoint_text":"- Magnitude of ADA to cemdisiran through week 52.","definition_or_measurement_approach":"Assessment of ADA titers/magnitude to cemdisiran through week 52."}
• {"endpoint_text":"- Percent change in LDH during extension treatment period (EP) from baseline to week 24 and week 52.","definition_or_measurement_approach":"Percent change in LDH from baseline to week 24 and week 52 during extension treatment period."}

Methods

• Recruitment at participating hospital/clinic trial sites (site-based recruitment as per listed trial sites in Italy, Spain, Poland).
• Patient recruitment/retention services provided by Clariness GmbH (as listed under sponsor third parties). Recruitment materials and procedures documented (K1/K2 recruitment documents listed).

Poland

Earliest CTIS Part Ii Submission Date

25-09-2025

Latest Decision Or Authorization Date

23-03-2026

Processing Time Days

179

Number Of Sites

1

Number Of Participants

2

Italy

Earliest CTIS Part Ii Submission Date

26-09-2025

Latest Decision Or Authorization Date

24-03-2026

Processing Time Days

179

Number Of Sites

3

Number Of Participants

3

Spain

Earliest CTIS Part Ii Submission Date

21-07-2025

Latest Decision Or Authorization Date

25-03-2026

Processing Time Days

247

Number Of Sites

5

Number Of Participants

3

Primary sponsor

Full Name

Regeneron Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Parexel International (IRL) Limited

Responsibilities

Contract Research Organisation (CRO)

Third parties

• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Contract Research Organisation (CRO)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Yprime LLC","duties_or_roles":"Electronic Clinical Outcome Assessment (eCOA)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Germany","full_name":"Clariness GmbH","duties_or_roles":"Patient Recruitment/Retention","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Iqvia Rds Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Slovakia","full_name":"SanaClis s.r.o.","duties_or_roles":"IP distribution","organisation_type":"Pharmaceutical company"}