Real-life Treatment Outcomes of Ravulizumab in PNH (PNH-RECORD)

Real-life Treatment Outcomes of Ravulizumab in Polish Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). Prospective and Retrospective, Multicenter, Non-interventional Study.

The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.

Study Overview

• Status: Active, not recruiting
• Conditions: Paroxysmal Nocturnal Haemoglobinuria
• Intervention / Treatment: Drug: Ravulizumab

Detailed Description

PNH-RECORD study aims to aims to collect data on the patients' characteristics and clinical outcomes of ravulizumab administered in the scope of routine clinical practice in PNH.

• Study Type: Observational
• Enrollment (Actual): 64

Contacts and Locations

Study Locations

• Poland
  • Bialystok, Poland
    • Research Site
  • Brzozów, Poland
    • Research Site
  • Bydgoszcz, Poland
    • Research Site
  • Gdansk, Poland
    • Research Site
  • Krakow, Poland
    • Research Site
  • Lodz, Poland
    • Research Site
  • Lublin, Poland
    • Research Site
  • Opole, Poland
    • Research Site
  • Szczecin, Poland
    • Research Site
  • Warsaw, Poland
    • Research Site
  • Wałbrzych, Poland
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult (aged ≥18) patients with PNH

Description

Inclusion Criteria:

1. Adult (aged ≥18) patients with PNH receiving ravulizumab treatment in the frames of NDP in Poland.
2. Patients willing to participate in the study and signed ICF.

Exclusion Criteria:

1. Those who participated in ravulizumab clinical trial in the past, and/or those who participated/plans to participate in clinical trial on/after the date of first ravulizumab infusion through NDP.
2. Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Ravulizumab group; Open-label	Drug: Ravulizumab; open-label arm with ravulizumab administrated within the scope of routine clinical practice

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in LDH levels from index date (first dose of ravulizumab) at every 6 month	up to 36 months
Proportion of patients achieving or remaining at LDH<1.5 ULN at every 6 month	up to 36 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of transfusion-free patients from index date at every 6 month		up to 36 months
Number of blood units used per patient-year		up to 36 months
To present patients characteristics	Mean age (years) at PNH diagnosis	Baseline
To present patients characteristics	Mean age (years) at index date	Baseline
To present patients characteristics	Proportion (%) of men and women	Baseline
To present patients characteristics	Mean body mass index (kg/m²)	Baseline
To present clinical characteristics	Mean and median (years) duration of PNH	Baseline
To present clinical characteristics	Mean and median (months) time from diagnosis to complement inhibitors treatment initiation	Baseline
To present clinical characteristics	Proportion (%) of patients with complication of haemolysis, thrombosis, serious vascular event andaplastic anemia	Baseline
To present clinical characteristics	Proportion (%) of patients naïve to complement inhibitors	Baseline

Collaborators and Investigators

• Sponsor: AstraZeneca

Study record dates

Study Major Dates

• Study Start (Actual): February 6, 2025
• Primary Completion (Estimated): August 1, 2028
• Study Completion (Estimated): August 1, 2028

Study Registration Dates

• First Submitted: August 9, 2024
• First Submitted That Met QC Criteria: October 8, 2024
• First Posted (Actual): October 9, 2024

Study Record Updates

• Last Update Posted (Actual): April 1, 2026
• Last Update Submitted That Met QC Criteria: March 31, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: PNH; ravulizumab; Paroxysmal nocturnal haemoglobinuria
• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Hemic and Lymphatic Diseases; Hemoglobinuria, Paroxysmal; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Complement Inactivating Agents; ravulizumab
• Other Study ID Numbers: D9289R00001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

• IPD Sharing Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• IPD Sharing Access Criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No