Hematological Response in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Anti-C5 Antibody: an External Control Arm Study for Iptacopan Use in Anti-C5 naïve Patients
NCT ID: NCT05842486
Last Updated: 2023-05-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
92 participants
OBSERVATIONAL
2023-01-18
2023-03-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
RETROSPECTIVE
Study Groups
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Real-world cohort
The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy.
Anti-C5 antibody treatment
Anti-C5 antibody treatment (i.e., eculizumab and ravulizumab)
Interventions
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Anti-C5 antibody treatment
Anti-C5 antibody treatment (i.e., eculizumab and ravulizumab)
Eligibility Criteria
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Inclusion Criteria
2. Confirmed clinical diagnosis of PNH (including PNH clone size \>10% in RBC percent/white blood cell (WBC) percent)
3. Adult patients ≥18 years of age
4. Baseline Hb \<10 g/dL.
Exclusion Criteria
1. Bone marrow transplantation within one year prior to initiating anti-C5 treatment
2. Hereditary complement deficiency
3. Reticulocyte count \<60x10\^9/L.
4. Pregnancy
18 Years
ALL
No
Sponsors
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Leeds Cancer Centre at St. James's University Hospital
OTHER
Assistance Publique - Hôpitaux de Paris
OTHER
Responsible Party
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Locations
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AP-HP Hôpital Saint Louis
Paris, , France
Countries
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References
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Holt M, Kelly RJ, Fermont JM, Ansari S, Dahlke M, Brindel I, Maafa L, de Fontbrune FS, de Latour RP. Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor-Naive Patients With Paroxysmal Nocturnal Haemoglobinuria. EJHaem. 2025 May 20;6(3):e270055. doi: 10.1002/jha2.70055. eCollection 2025 Jun.
Other Identifiers
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CLNP023N12002R
Identifier Type: -
Identifier Source: org_study_id
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