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

NCT05842486 | Completed

• 1 other identifier: CLNP023N12002R
• Study Type: observational
• Target: 92
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this non-interventional secondary use of data study is to evaluate hematological response in patients with paroxysmal nocturnal hemoglobinuria and anemia in the 6-month period after initiation of anti-C5 antibody treatment using real-world data obtained from multiple datasets. The results will be used to contextualize results from the APPOINT-PNH (NCT04820530) trial with iptacopan.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Increase from baseline in hemoglobin

  Increase from baseline in hemoglobin of ≥ 2 g/dL in the absence of transfusions occurring after Day 14 post index date

  between Day 100 and Day 200

Secondary Outcomes (2)

• Hemoglobin Response

  between Day 100 and Day 200

• Transfusion avoidance

  Occurrence between Day 15 and Day 200

Study Arms (1)

Real-world cohort

The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy.

Other: Anti-C5 antibody treatment

Interventions

Anti-C5 antibody treatment OTHER

Anti-C5 antibody treatment (i.e., eculizumab and ravulizumab)

Real-world cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy. Patient data originate from the Hôpital Saint-Louis, France and St. James's University Hospital, Leeds, United Kingdom.

You may qualify if:

• Incident users of anti-C5 antibody treatment (i.e., first use of anti-C5 antibody with no evidence of use prior to that)
• Confirmed clinical diagnosis of PNH (including PNH clone size \>10% in RBC percent/white blood cell (WBC) percent)
• Adult patients ≥18 years of age
• Baseline Hb \<10 g/dL.

You may not qualify if:

• Bone marrow transplantation within one year prior to initiating anti-C5 treatment
• Hereditary complement deficiency
• Reticulocyte count \<60x10\^9/L.
• Pregnancy

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• Leeds Cancer Centre at St. James's University Hospital: collaborator

Study Sites (1)

AP-HP Hôpital Saint Louis

Paris, France

Location

Related Publications (1)

• 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.

  PMID: 40395624 DERIVED

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 24, 2023
• First Posted: May 6, 2023
• Study Start: January 18, 2023
• Primary Completion: March 1, 2023
• Study Completion: March 1, 2023
• Last Updated: May 6, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)