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Completed

NCT Number: NCT05842486

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

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.

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

AP-HP Hôpital Saint Louis

Paris, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

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

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

Treatment and study plan

Anti-C5 antibody treatment

Other

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

Primary outcomes

  1. Increase from baseline in hemoglobin

    Time frame: 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 outcomes

  1. Hemoglobin Response

    Time frame: 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

  2. Transfusion avoidance

    Time frame: 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).

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Collaborators

  • Leeds Cancer Centre at St. James's University Hospital

Registry information

Acronym: APPEX

Important dates

Study start
2023
Primary completion
2023
Study completion
2023
First posted
May 6, 2023
Registry last updated
May 6, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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