The International PNH Interest Group PNH Registry

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

2000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-05-10

Study Completion Date

2029-05-10

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH covering clinical outcomes, patient reported outcomes (PROs), and health-resource utilization (HRU) on all enrolled patients, as well as long term safety data.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

NCT01374360

Paroxysmal Nocturnal Hemoglobinuria

COMPLETED

Renal HYPODYSPLASIA : Genetic and Familial Assessment

NCT00925379

Renal HYPODYSPLASIA, Nonsyndromic, 1

COMPLETED

The Molecular Biology of Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT00721864

Hemoglobinuria, Paroxysmal

COMPLETED

Hereditary Paraganglioma: Evaluation of Screening Methods to Detect Tumors in SDH Positive Carriers

NCT00188019

Paraganglioma Pheochromocytoma

COMPLETED NA

STOP-HSP.Net: a Registry for Hereditary Spastic Paraplegia as an Integration Tool for Future Therapeutic Strategies

NCT06572046

Hereditary Spastic Paraplegia

RECRUITING

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

The International PNH Interest Group (IPIG) PNH Registry is an international, observational database collecting real-world health information about PNH patients over time. The registry is owned and managed by IPIG, a not-for-profit network of international PNH experts dedicated to improving care and treatment for PNH patients. Several pharmaceutical partners are collaborating with IPIG to fund the registry, which will provide data to support their post-approval regulatory commitments.

The IPIG PNH Registry aims to collect data about all PNH patients including clinical outcomes, patient reported outcomes and health resource usage, as well as long term safety data for patients treated with anti-complement therapies.

The IPIG PNH Registry is comprised of a 'core' PNH disease registry collecting a defined set of core variables on all patients with PNH. In addition, there are several product-specific 'silos' initiated by marketing authorization holders including patients treated with PNH-specific therapies. The silos collect both the core dataset and additional variables to address specific objectives or requests from regulatory authorities (e.g. post-authorization safety data).

Data is collected at enrollment and at 6 monthly intervals during follow-up.

This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44).

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Paroxysmal Nocturnal Hemoglobinuria

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

PNH patients not receiving anti-complement treatment

PNH patients of any age who are not receiving any anti-complement treatment

No interventions assigned to this group

PNH patients receiving anti-complement treatment

PNH patients of any age who are receiving an approved anti-complement treatment

No interventions assigned to this group

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Eligible Sex

ALL

Accepts Healthy Volunteers

No