The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria

NCT04079257 | Completed

• 1 other identifier: PREPARE
• Study Type: observational
• Target: 27
• Locations: 1 country
• Sites: 1

Brief Summary

Because of the inter and intra individual variability in pharmacokinetics and pharmacodynamics of eculizumab in PNH patients, a tailored treatment approach for the individual is probably preferable. The starting point of a robust tailored dosing approach for eculizumab is the development of a population pharmacokinetic-pharmacodynamic model. In this cross-sectional observational pharmacokinetic and pharmacodynamic study, trough and peak concentrations of eculizumab are measured to describe the pharmacokinetics and complement activation markers to describe the pharmacodynamics.

Conditions

Paroxysmal Nocturnal Hemoglobinuria; Eculizumab; PK-PD

Outcome Measures

Primary Outcomes (2)

• Clearance

  Peak and trough concentrations will be measured in order to determine clearance

  9 months

• Volume of distribution

  Peak and trough concentrations will be measured in order to determine volume of distribution

  9 months

Study Arms (1)

Patients with paroxysmal nocturnal hemoglobinuria

Patients are already treated with eculizumab. The only intervention is the collection of extra blood samples to measure peak concentrations of eculizumab

Other: Blood collection for measurement of eculizumab peak concentrations

Interventions

Blood collection for measurement of eculizumab peak concentrations OTHER

collection of blood

Also known as: blood collection

Patients with paroxysmal nocturnal hemoglobinuria

Eligibility Criteria

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

Study Population

PNH patients treated with eculizumab

You may qualify if:

• Diagnosis of PNH
• Treated with eculizumab
• Willing to give informed consent

You may not qualify if:

• Not applicable

Sponsors & Collaborators

• Radboud University Medical Center: lead

Study Sites (1)

Radboudumc

Nijmegen, Netherlands

Location

Related Publications (1)

• Ter Avest M, Langemeijer SMC, van den Heuvel LPWJ, Baas LM, van de Kar NCAJ, Ter Heine R. Model-Informed Precision Dosing of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria. Clin Pharmacokinet. 2025 Aug;64(8):1139-1147. doi: 10.1007/s40262-025-01536-x. Epub 2025 Jun 25.

  PMID: 40560515 DERIVED

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Design

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

Study Record Dates

• First Submitted: July 11, 2019
• First Posted: September 6, 2019
• Study Start: February 23, 2021
• Primary Completion: July 1, 2022
• Study Completion: July 1, 2022
• Last Updated: August 24, 2022

Record last verified: 2022-08

Locations

NL (1)