International registry for patients with Paroxysmal Nocturnal Hemoglobinuria
The International PNH Interest Group PNH Registry
This study is collecting health information from people with Paroxysmal Nocturnal Hemoglobinuria to see how they are doing over time and how well their treatments are working.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 2000 (estimated) |
| Sex | All |
| Sponsor | International PNH Interest Group Academic / other |
| Locations | 1 site (Altamonte Springs, Florida) |
| Trial ID | NCT06524726 on ClinicalTrials.gov |
What this trial studies
The International PNH Interest Group PNH Registry is an observational database designed to collect real-world health information from patients diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH). This registry aims to gather data on clinical outcomes, patient-reported outcomes, and health resource utilization over time, while also ensuring long-term safety data for patients receiving anti-complement therapies. The registry is managed by a network of international PNH experts and is supported by pharmaceutical partners to fulfill post-approval regulatory commitments. Data collection occurs at enrollment and every six months thereafter.
Who should consider this trial
Good fit: Ideal candidates for this registry are patients with a confirmed diagnosis of PNH through flow cytometry.
Not a fit: Patients currently participating in an interventional PNH clinical trial may not benefit from this registry as their data collection will be paused.
Why it matters
Potential benefit: If successful, this registry could enhance understanding of PNH and improve treatment strategies for patients.
How similar studies have performed: Other similar registries have shown success in collecting valuable data that informs treatment and management strategies for rare diseases.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients with PNH confirmed by flow cytometry. * Patient and/or parent/legally authorized representative provide written informed consent/assent to participate in the registry in a manner approved by the Institutional Review Board/Independent Ethics Committee and local regulations. Exclusion Criteria: * Participating in an interventional PNH clinical trial. Note: A patient included in the registry, who enrolls in an interventional PNH clinical trial during the course of the registry, will be kept in the registry but data collection will be paused in the registry during their involvement in the clinical trial/extension study. Data collection in the registry will continue after patient involvement in the clinical trial/extension study has ended or trial protocol mandated data collection ceases.
Where this trial is running
Altamonte Springs, Florida
- International PNH Interest Group — Altamonte Springs, Florida, United States (Recruiting)
Study contacts
- Principal investigator: Richard Kelly, MBChB PhD — International PNH Interest Group
- Study coordinator: IPIG Registry Coordinator
- Email: registry@pnhinterestgroup.org
- Phone: Please email
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.