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Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China (Soliris)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05886244
Recruitment Status : Recruiting
First Posted : June 2, 2023
Last Update Posted : September 15, 2023
Sponsor:
Collaborator:
AstraZeneca
Information provided by (Responsible Party):
Alexion

Study Description
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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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria Drug: Eculizumab Phase 3

Detailed Description:
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 PNH in China who previously have not been treated with complement inhibitors. Approximately 25 eligible participants in China will be enrolled.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: open label
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-Label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
Actual Study Start Date : July 5, 2023
Estimated Primary Completion Date : April 30, 2025
Estimated Study Completion Date : April 30, 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Eculizumab

Arms and Interventions
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Arm Intervention/treatment
Experimental: Eculizumab
Eculizumab will be administered by IV infusion.
 Drug: Eculizumab
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.


Outcome Measures
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Primary Outcome Measures :
  1. Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12 [ Time Frame: Baseline, Week 12 ]
    To assess efficacy of eculizumab in participants with PNH


Secondary Outcome Measures :
  1. Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline through Week 64 ]
    To assess the safety and tolerability of eculizumab in participants with PNH

  2. Pharmacokinetics (PK): Serum Eculizumab Concentration [ Time Frame: Baseline through Week 64 (predose and postdose) ]
    To characterize the pharmacokinetics of eculizumab in participants with PNH

  3. Change from baseline in Serum Free and total Complement 5 (C5) Concentration [ Time Frame: Baseline through week 64 (predose and postdose) ]
    To characterize the pharmacodynamics of eculizumab in participants with PNH

  4. Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab [ Time Frame: Baseline through Week 64 ]
    To characterize the immunogenicity of eculizumab in participants with PNH

  5. Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64 [ Time Frame: Baseline through Week 64 ]
    To assess the efficacy of eculizumab in participants with PNH

  6. Number(%) of participants with Breakthrough Hemolysis [ Time Frame: Baseline through Week 64 ]
    To assess the efficacy of eculizumab in participants with PNH

  7. Number(%) of participants achieving LDH Normalization [ Time Frame: Baseline through Week 64 ]
    To assess the efficacy of eculizumab in participants with PNH

  8. Number(%) of participants needing Blood Transfusion [ Time Frame: Baseline through Week 64 ]
    To assess the efficacy of eculizumab in participants with PNH

  9. Number(%) of participants changes from baseline in vital signs and laboratory parameters at all scheduled visits. [ Time Frame: Baseline through Week 64 ]
    To characterize the safety profile of eculizumab in participants with PNH


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • Meningitidis infection or unresolved meningococcal disease
  • Significant bone marrow failure
  • Other significant systemic diseases that might have impact on efficacy and safety assessment
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05886244


Contacts
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Contact: AstraZeneca Clinical Study Information Center 1-877-240-9479 information.center@astrazeneca.com

Locations
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China
Research Site Recruiting
Beijing, China, CN-100730
Research Site Recruiting
Hangzhou, China, 310003
Research Site Recruiting
Nantong, China, 226001
Research Site Recruiting
Shanghai, China, 200040
Research Site Recruiting
Tianjian, China, 300020
Research Site Recruiting
Tianjin, China, 300052
Research Site Recruiting
Wuhan, China, 430022
Sponsors and Collaborators
Alexion
AstraZeneca
More Information
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Responsible Party: Alexion
ClinicalTrials.gov Identifier: NCT05886244    
Other Study ID Numbers: D7414C00001
First Posted: June 2, 2023    Key Record Dates
Last Update Posted: September 15, 2023
Last Verified: September 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

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.
Supporting Materials: Study Protocol
Statistical Analysis Plan (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.
URL: https://astrazenecagroup-dt.pharmacm.com/DT/Home

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion:
paroxysmal nocturnal hemoglobinuria
PNH
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
Male Urogenital Diseases
Urological Manifestations
 Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases
Eculizumab
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs