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

Phase 2

Withdrawn

• Conditions: MarchiafavaMicheli syndrome; stem cell disorder; 10038158

• Registration Number: NL-OMON48163
• Lead Sponsor: Regeneron Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 2

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.
- PNH granulocytes (denoted as polymorphonuclear [PMN]) >10% 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)

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>- The proportion of patients achieving adequate control of their intravascular<br /><br>hemolysis, defined as LDH * 1.5 x ULN at every scheduled time point between<br /><br>week 4 and week 26, inclusive<br /><br>- The proportion of patients achieving transfusion avoidance defined as no post<br /><br>baseline transfusion of RBCs per protocol through week 26</p><br>