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Clinical Trials List

Protocol NumberR3918-PNH-2050
NCT Number(ClinicalTrials.gov Identfier)NCT06028594
Active

2023-03-01 - 2028-10-16

Phase III

Not yet recruiting8

Recruiting1

ICD-10D59.5

Paroxysmal nocturnal hemoglobinuria [Marchiafava-Micheli]

ICD-10D59.6

Hemoglobinuria due to hemolysis from other external causes

ICD-10D59.8

Other acquired hemolytic anemias

ICD-9283.2

Hemoglobinuria due to hemolysis from external causes

An Expanded Access Program of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

  • Sponsor

    PPD, Inc. Taiwan Branch

  • Trial scale

    Multi-Regional Multi-Center

  • Update

    2026/02/01

Investigators and Locations

Principal Investigator 陳宇欽 Division of Hematology & Oncology
Tri-Service General Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Su-Peng Yeh Division of Hematology & Oncology
China Medical University Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator 黃威翰 Division of Hematology & Oncology
Hualien Tzu Chi Hospital

Co-Principal Investigator

  • 黃聖娟 Division of Hematology & Oncology

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator 林炫聿 Division of Hematology & Oncology
CHANGHUA CHRISTIAN HOSPITAL

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator HSIN-AN HOU Division of General Internal Medicine
National Taiwan University Hospital

Co-Principal Investigator

  • Chien-Chin Lin Division of General Internal Medicine
  • MING YAO Division of General Internal Medicine
  • Wen-Chien Chou Division of General Internal Medicine
  • - - Division of General Internal Medicine

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Tsai-Yun Chen Division of Hematology & Oncology
National Taiwan University Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Hung Chang Division of Hematology & Oncology
Linkou Chang Gung Medical Foundation

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator Hui-Hua Hsiao
Kaohsiung Medical Univeristy Chung-Ho Memorial Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator HSIN-CHEN LIN Division of Hematology & Oncology
Taichung Veterans General Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Condition/Disease

Paroxysmal Nocturnal Hemoglobinuria

Objectives

The primary objective of this trial is to describe the long-term safety, tolerability, and efficacy of pozelimab + cemdisiran combination therapy in subjects with paroxysmal nocturnal hemoglobinuria (PNH). Secondary objectives of this trial are to characterize the long-term effects of pozelimab + cemdisiran on: - Measurement of vascular hemolysis - Transfusion parameters - Heme concentration - Fatigue as assessed by Patient-Reported Outcomes (PROs) - Physical function as assessed by PROs - Changes in global health status (GHS) as assessed by PROs - Complement activation - To assess total pozelimab concentration in serum and cemdisiran concentration in plasma relative to total complement factor 5 (C5) protein concentration - Immunogenicity of pozelimab and cemdisiran

Test Drug

PozelimabCemdisiran

Active Ingredient

Pozelimab
Cemdisiran

Dosage Form

SC
SC

Dosage

2 mL/vial
200 mg/mL

Endpoints

The primary safety endpoints were the incidence and severity of treatment-emergent serious adverse events (SAEs), adverse events of special interest (AESIs), and adverse events (AEs) leading to permanent discontinuation of treatment at 108 weeks of OLEP.
The primary efficacy endpoint was the percent change in LDH from baseline to week 36.

Inclution Criteria

Key Inclusion Criteria:

Patients who have completed either the end of treatment visit of the open-label treatment period or open-label extension period in one of the following parent studies:

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (R3918-PNH-2050 [NCT05744921])
A Randomized, Open-Label, Two-Arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy (R3918-PNH-2092 [NCT04811716])
A Single Arm, Open-Label Study to Assess the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switch from Eculizumab Therapy (R3918-PNH-20105 [NCT04888507]). Note: In some countries, patients that would normally enter into the R3918-PNH-2050 (NCT05744921) parent study before entering this expanded access program, may be given the opportunity to skip R3918-PNH-2050 (NCT05744921) and go straight into this expanded access program, with Regeneron's permission
Patients who have been enrolled in R3918-PNH-2022 (NCT05131204), with Regeneron's permission
Willing and able to comply with clinic visits and related standard-of-care procedures
With Regeneron's permission, patients who have been enrolled in other PNH studies as appropriate

Exclusion Criteria

Key Exclusion Criteria:

Significant history or concerns of non-compliance that could impact the patient's safety per the treating physician
Any new condition or worsening of an existing condition which, in the opinion of the treating physician, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the program

The Estimated Number of Participants

  • Taiwan

    26 participants

  • Global

    202 participants

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