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

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

— APPEX  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05842486
• Other study ID #: CLNP023N12002R
• Status: Completed
• Start date: January 18, 2023
• Est. completion date: March 1, 2023

Study information

• Verified date: March 2023
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 92
• Est. completion date: March 1, 2023
• Est. primary completion date: March 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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. 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

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

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

Locations

Country	Name	City	State
France	AP-HP Hôpital Saint Louis	Paris

Sponsors (2)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	Leeds Cancer Centre at St. James's University Hospital

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Hemoglobin Response	Achieving hemoglobin of = 12 g/dL in the absence of blood transfusions occurring after Day 14 post index date	between Day 100 and Day 200
Secondary	Transfusion avoidance	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).	Occurrence between Day 15 and Day 200