A trial with REGN3918 in patients Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not been treated previously with a Complement Inhibitor or Have Not Recently Received Complement Inhibitor Therapy

An Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-002734-20-GB

Enrollment

Registered

2019-03-22

Start date

2019-07-24

Completion date

Unknown

Last updated

2020-07-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857

Interventions

Product Name: REGN3918 Product Code: REGN3918 Pharmaceutical Form: Lyophilisate for solution for injection INN or Proposed INN: REGN3918 Current Sponsor code: REGN3918 Other descriptive name: REGN3918

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • Male or female = 18 years of age or legal age of majority at screening, whichever is greater •Diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) confirmed by high sensitivity flow cytometry •Active disease, as defined by the presence of 1 or more PNH related signs or symptoms or history of red blood cell (RBC) transfusion due to PNH within 3 months of screening. •Lactate dehydrogenase (LDH) level = 2 × upper limit of normal (ULN) at screening visit. •Type III PNH granulocytes (denoted as polymorphonuclear [PMN]) >10% at screening visit. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 30 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 12

Exclusion criteria

Exclusion criteria: •Prior treatment with a complement inhibitor either within 6 months prior to screening visit or at any time where the patient was refractory to complement inhibitor therapy, in the opinion of the investigator (with the exception of eculizumab refractory patients due to the C5 variant R885H/C)

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patients with active PNH who are treatment naive to complement inhibitor therapy or have not recently received complement inhibitor therapy;Secondary Objective: The secondary objectives of the study are: •To evaluate the safety and tolerability of REGN3918. •To evaluate the effect of REGN3918 on parameters of intravascular hemolysis •To assess the concentrations of total REGN3918 in serum. •To evaluate the incidence of treatment emergent anti drug antibodies to REGN3918. •To evaluate the effect of REGN3918 on patient reported outcomes (PROs) measuring fatigue and health related quality of life ;Primary end point(s): The co primary endpoints are: •The proportion of patients achieving adequate control of their intravascular hemolysis, defined as LDH = 1.5 x ULN at every scheduled time point between week 4 and week 26, inclusive •The proportion of patients achieving transfusion avoidance defined as no post baseline transfusion of RBCs per protocol over 26 weeks;Timepoint(s) of evaluation of this end point: 26 weeks	—

Measure

Time frame

Main Objective: The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patients with active PNH who are treatment naive to complement inhibitor therapy or have not recently received complement inhibitor therapy;Secondary Objective: The secondary objectives of the study are: •To evaluate the safety and tolerability of REGN3918. •To evaluate the effect of REGN3918 on parameters of intravascular hemolysis •To assess the concentrations of total REGN3918 in serum. •To evaluate the incidence of treatment emergent anti drug antibodies to REGN3918. •To evaluate the effect of REGN3918 on patient reported outcomes (PROs) measuring fatigue and health related quality of life ;Primary end point(s): The co primary endpoints are: •The proportion of patients achieving adequate control of their intravascular hemolysis, defined as LDH = 1.5 x ULN at every scheduled time point between week 4 and week 26, inclusive •The proportion of patients achieving transfusion avoidance defined as no post baseline transfusion of RBCs per protocol over 26 weeks;Timepoint(s) of evaluation of this end point: 26 weeks

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Secondary

Measure	Time frame
Secondary end point(s): The secondary endpoints include: •The rate of breakthrough hemolysis over 26 weeks, defined as the measurement of LDH = 2 x ULN concomitant with associated signs or symptoms at any time subsequent to an initial achievement of disease control (ie, LDH = 1.5 x ULN) •The proportion of patients achieving normalization of their intravascular hemolysis, defined as LDH = 1.0 x ULN at every scheduled time point between week 4 through week 26, inclusive •Time to first LDH = 1.5 x ULN •Percentage of days with LDH = 1.5 x ULN between week 4 and week 26, inclusive. •Change and percent change in LDH levels from baseline to week 26;Timepoint(s) of evaluation of this end point: 26 weeks	—

Measure

Time frame

Secondary end point(s): The secondary endpoints include: •The rate of breakthrough hemolysis over 26 weeks, defined as the measurement of LDH = 2 x ULN concomitant with associated signs or symptoms at any time subsequent to an initial achievement of disease control (ie, LDH = 1.5 x ULN) •The proportion of patients achieving normalization of their intravascular hemolysis, defined as LDH = 1.0 x ULN at every scheduled time point between week 4 through week 26, inclusive •Time to first LDH = 1.5 x ULN •Percentage of days with LDH = 1.5 x ULN between week 4 and week 26, inclusive. •Change and percent change in LDH levels from baseline to week 26;Timepoint(s) of evaluation of this end point: 26 weeks

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Countries

Canada, Germany, Hong Kong, Hungary, Italy, Korea, Democratic People's Republic of, Malaysia, Netherlands, Poland, Romania, Singapore, South Africa, Taiwan, United Kingdom, United States

Contacts

Public ContactClinical Trial Information

Regeneron Pharmaceuticals, Inc.

clinicaltrials@regeneron.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026