Observational study on the outcomes of ravulizumab in patients with PNH
Real-life Treatment Outcomes of Ravulizumab in Polish Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). Prospective and Retrospective, Multicenter, Non-interventional Study.
This study looks at how well the medication ravulizumab works for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) in real-life situations.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 40 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | AstraZeneca Industry-sponsored |
| Drugs / interventions | ravulizumab |
| Locations | 13 sites (Bialystok and 12 other locations) |
| Trial ID | NCT06633536 on ClinicalTrials.gov |
What this trial studies
The PNH-RECORD study is a multicenter observational study conducted in Poland that collects data on the characteristics and clinical outcomes of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are treated with ravulizumab. This non-interventional study involves retrospective data collection with prospective follow-up to assess real-life treatment outcomes. The aim is to understand how ravulizumab performs in routine clinical practice among adult patients diagnosed with PNH.
Who should consider this trial
Good fit: Ideal candidates for this study are adult patients aged 18 and older with a confirmed diagnosis of PNH who are currently receiving ravulizumab treatment.
Not a fit: Patients who have previously participated in clinical trials involving ravulizumab or those with cognitive incapacity or language barriers may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the effectiveness and safety of ravulizumab for patients with PNH in real-world settings.
How similar studies have performed: Other studies have shown success in evaluating treatment outcomes for PNH, but this specific observational approach focusing on real-life data is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Bialystok and 12 other locations
- Research Site — Bialystok, Poland (Not_yet_recruiting)
- Research Site — Brzozow, Poland (Recruiting)
- Research Site — Bydgoszcz, Poland (Recruiting)
- Research Site — Gdansk, Poland (Recruiting)
- Research Site — Katowice, Poland (Not_yet_recruiting)
- Research Site — Krakow, Poland (Not_yet_recruiting)
- Research Site — Lodz, Poland (Recruiting)
- Research Site — Lublin, Poland (Not_yet_recruiting)
- Research Site — Opole, Poland (Recruiting)
- Research Site — Szczecin, Poland (Not_yet_recruiting)
- Research Site — Walbrzych, Poland (Recruiting)
- Research Site — Warsaw, Poland (Not_yet_recruiting)
- Research Site — Wroclaw, Poland (Recruiting)
Study contacts
- Study coordinator: AstraZeneca Clinical Study Information Center
- Email: information.center@astrazeneca.com
- Phone: 1-877-240-9479
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.