Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

Official title:

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01374360
• Other study ID #: M07-001
• Status: Recruiting
• Start date: October 29, 2004
• Est. completion date: December 31, 2024

Study information

• Verified date: February 2024
• Source: Alexion Pharmaceuticals, Inc.
• Contact: Alexion Pharmaceuticals, Inc. (Sponsor)
• Phone: 1-855-752-2356
• Email: clinicaltrials[@]alexion.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

Description:

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5950
• Est. completion date: December 31, 2024
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.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.

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Locations

Country	Name	City	State
United States	Contact the PNH Registry at Alexion Pharmaceuticals, Inc. for worldwide locations.	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluate safety data specific to the use of Soliris and Ultomiris	Primary analyses will assess safety endpoints, including occurrence and time to first event for the following: meningococcal infections, infections with serious outcomes, formation of human anti-drug antibodies (ADA) to Soliris and Ultomiris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, impaired hepatic function, hemolysis, pregnancies, lactation, infusion reactions, bone marrow transplant, serious adverse events, and mortality.	Ongoing (up to 13 years)
Secondary	Collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris or Ultomiris and non-Soliris or non- Ultomiris treated patients	Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.	Ongoing (up to 13 years)