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 (APPEX)

April 24, 2023 updated by: Assistance Publique - Hôpitaux de Paris
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

92

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France
        • AP-HP Hôpital Saint Louis

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

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.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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
Anti-C5 antibody treatment (i.e., eculizumab and ravulizumab)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
between Day 100 and Day 200

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
between Day 100 and Day 200
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).
Occurrence between Day 15 and Day 200

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Collaborators

Leeds Cancer Centre at St. James's University Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 18, 2023

Primary Completion (Actual)

March 1, 2023

Study Completion (Actual)

March 1, 2023

Study Registration Dates

First Submitted

April 24, 2023

First Submitted That Met QC Criteria

April 24, 2023

First Posted (Estimate)

May 4, 2023

Study Record Updates

Last Update Posted (Estimate)

May 4, 2023

Last Update Submitted That Met QC Criteria

April 24, 2023

Last Verified

March 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLNP023N12002R

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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