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

Completed

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Other: Anti-C5 antibody treatment

• Registration Number: NCT05842486
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-01-18
• Status Verified: 2023-03
• Primary Completion: 2023-03-01
• Study Completion: 2023-03-01
• Study First Submitted: 2023-04-24
• Study First Submitted QC: 2023-04-24
• Last Update Submitted: 2023-04-24
• Study First Posted: 2023-05-06
• Last Update Posted: 2023-05-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 92

Inclusion Criteria

1. Incident users of anti-C5 antibody treatment (i.e., first use of anti-C5 antibody with no evidence of use prior to that)
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.

Read More

Exclusion Criteria

Patients who met any of the following criteria were not eligible for inclusion.

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

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Real-world cohort	Anti-C5 antibody treatment	The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy.

Primary Outcome Measures

Name	Time	Method
Increase from baseline in hemoglobin	between Day 100 and Day 200	Increase from baseline in hemoglobin of ≥ 2 g/dL in the absence of transfusions occurring after Day 14 post index date

Secondary Outcome Measures

Name	Time	Method
Hemoglobin Response	between Day 100 and Day 200	Achieving hemoglobin of ≥ 12 g/dL in the absence of blood transfusions occurring after Day 14 post index date
Transfusion avoidance	Occurrence between Day 15 and Day 200	Transfusion avoidance (dichotomous endpoint) defined proportion of subjects without transfusions during the initial 6-month treatment period starting from 2 Weeks after index date (i.e., date of first anti-C5 antibody treatment for PNH).

Trial Locations

Locations (1)

AP-HP Hôpital Saint Louis; 🇫🇷 Paris, France