Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Alexion Pharmaceuticals

Status: Completed

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Study type: Observational

Funder types: Industry

Identifiers

NCT01374360

M07-001

Details and patient eligibility

About

This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Full description

Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Enrollment: 6,061 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients of any age, including minors, with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment. Patients who are minors must have parent/legal guardian consent and must be willing and able to give assent, if applicable as determined by the Ethics Committees/Institutional Review Boards. Upon attaining adulthood, these patients must be re-consented.
• Ability to comprehend and sign consent to have data entered in the PNH Registry.

Exclusion criteria

• Inability or unwillingness to sign informed consent.
• Patients currently enrolled in an interventional clinical trial for treatment of PNH cannot be enrolled in the Registry at the same time.

Trial design

6,061 participants in 2 patient groups

Receiving Soliris or Ultomiris

Description:

PNH patients of any age, including minors, that are receiving Soliris or Ultomiris

Not receiving Soliris or Ultomiris

Description:

PNH patients of any age, including minors, that are not receiving Soliris or Ultomiris