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Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Clinical Trials | Clareo Health

COMPLETEDPHASE2

Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

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

NCT03946748•Regeneron Pharmaceuticals

View on ClinicalTrials.gov

Summary

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. 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 over time * To evaluate the effect of REGN3918 on patient-reported outcomes (PROs) measuring fatigue and health-related quality of life

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) confirmed by high-sensitivity flow cytometry
•PNH granulocytes \> 10% at screening visit
•Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms (eg, fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 g/dL\], history of a MAVE \[including thrombosis\], dysphagia, or erectile dysfunction) 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.

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)
•History of bone marrow transplantation
•Body weight \< 40 kilograms at screening visit
•Peripheral blood absolute neutrophil count (ANC) \<500/μL \[\<0.5 x 109/L\] or peripheral blood platelet count \<50,000/μL
•Documented history of systemic fungal disease or unresolved tuberculosis, or evidence of active or latent tuberculosis infection (LTBI) during screening period
•Any contraindication for receiving Neisseria meningitidis vaccination and antibiotic prophylaxis therapy as recommended in the study
•Any active, ongoing infection within 2 weeks of screening or during the screening period
•Any clinically significant abnormality identified at the time of screening that in the judgment of the Investigator or any sub-Investigator would preclude safe completion of the study or constrain endpoints assessment such as major systemic diseases, or patients with short life expectancy
•Women who are pregnant, breastfeeding, or who have a positive pregnancy test at screening visit or day 1

Locations (12)

Regeneron Study Site

Shatin, Hong Kong

Regeneron Study Site

Budapest, Hungary

Regeneron Study Site

Kaposvár, Hungary

Regeneron Study Site

Kota Bharu, Kelantan, Malaysia

Regeneron Study Site

Sibu, Sarawak, Malaysia

Regeneron Study Site

Kuala Terengganu, Terengganu, Malaysia

Regeneron Study Site

Seoul, South Korea

Regeneron Study Site

Seoul, South Korea

Regeneron Study Site

Seoul, South Korea

Regeneron Study Site

Seoul, South Korea

Key Dates

Start Date

May 16, 2019

Primary Completion Date

June 9, 2021

Completion Date

June 10, 2021

Last Updated

June 26, 2023

Enrollment

ACTUAL participants

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

REGN3918

DRUG

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

NCT03520647

Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)+1 more

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RECRUITINGPHASE1

Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment

NCT07152288

Paroxysmal Nocturnal Hemoglobinuria (PNH)

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 26, 2023Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Inclusion Criteria

Exclusion Criteria

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment

Hematological Disorders in EHPVO Patients

ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy

Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy