NCT05886244

Brief Summary

This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal hemoglobinuria (PNH) in China.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jul 2023

Geographic Reach
1 country

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 24, 2023

Completed
9 days until next milestone

First Posted

Study publicly available on registry

June 2, 2023

Completed
1 month until next milestone

Study Start

First participant enrolled

July 5, 2023

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 14, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 14, 2025

Completed
Last Updated

April 16, 2025

Status Verified

April 1, 2025

Enrollment Period

1.8 years

First QC Date

May 24, 2023

Last Update Submit

April 15, 2025

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

paroxysmal nocturnal hemoglobinuriaPNH

Outcome Measures

Primary Outcomes (1)

  • Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12

    To assess efficacy of eculizumab in participants with PNH

    Baseline, Week 12

Secondary Outcomes (9)

  • Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Baseline through Week 64

  • Pharmacokinetics (PK): Serum Eculizumab Concentration

    Baseline through Week 64 (predose and postdose)

  • Change from baseline in Serum Free and total Complement 5 (C5) Concentration

    Baseline through week 64 (predose and postdose)

  • Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab

    Baseline through Week 64

  • Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64

    Baseline through Week 64

  • +4 more secondary outcomes

Study Arms (1)

Eculizumab

EXPERIMENTAL

Eculizumab will be administered by IV infusion.

Drug: Eculizumab

Interventions

EculizumabDRUG

Participants will receive 600 milligrams (mg) once a week on Day 1, 8, 15, and 22 followed by 900 mg every 2 weeks from Day 29 to Day 435.

Eculizumab

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Adult C5 inhibitor naïve PNH patients (age\>=18), which is confirmed by flow cytometry evaluation
  • Must be vaccinated against N meningitidis

You may not qualify if:

  • Meningitidis infection or unresolved meningococcal disease
  • Significant bone marrow failure
  • Other significant systemic diseases that might have impact on efficacy and safety assessment

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Research Site

Beijing, CN-100730, China

Location

Research Site

Hangzhou, 310003, China

Location

Research Site

Nantong, 226001, China

Location

Research Site

Shanghai, 200040, China

Location

Research Site

Tianjin, 300020, China

Location

Research Site

Tianjin, 300050, China

Location

Research Site

Wuhan, 430022, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: open label
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 24, 2023

First Posted

June 2, 2023

Study Start

July 5, 2023

Primary Completion

April 14, 2025

Study Completion

April 14, 2025

Last Updated

April 16, 2025

Record last verified: 2025-04

Data Sharing

IPD Sharing
Will share

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Access Criteria
When a request has been approved AstraZeneca will provide access to the deidentified individual patient-level data in an approved sponsored tool . Signed Data Sharing Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information. Additionally, all users will need to accept the terms and conditions of the SAS MSE to gain access. For additional details, please review the Disclosure Statements at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
More information

Locations

CN(7)