Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

NCT01374360 | Completed

• 1 other identifier: M07-001
• Study Type: observational
• Target: 6,061
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

PNH; Paroxysmal Nocturnal hemoglobinuria; Soliris; Eculizumab; Ravulizumab; Ultomiris

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• 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

  Ongoing (up to 13 years)

Study Arms (2)

Receiving Soliris or Ultomiris

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

Not receiving Soliris or Ultomiris

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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

PNH Patients

You may qualify if:

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with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (1)

Contact the PNH Registry at Alexion Pharmaceuticals, Inc. for worldwide locations.

Boston, Massachusetts, 02210, United States

Location

Related Publications (5)

• Roth A, Patriquin CJ, Szer J, Terriou L, Patel AS, Metzger J, Gustovic P, Nishimura JI, Brodsky RA. Real-World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International PNH Registry. Am J Hematol. 2026 Mar 7. doi: 10.1002/ajh.70268. Online ahead of print.

  PMID: 41795103 DERIVED

• Dingli D, Maciejewski JP, Larratt L, Go RS, Hochsmann B, Zu K, Gustovic P, Kulagin AD. Relationship of paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size to disease burden and risk of major vascular events in untreated patients: results from the International PNH Registry. Ann Hematol. 2023 Jul;102(7):1637-1644. doi: 10.1007/s00277-023-05269-4. Epub 2023 May 18.

  PMID: 37199789 DERIVED

• Hill A, de Latour RP, Kulasekararaj AG, Griffin M, Brodsky RA, Maciejewski JP, Marantz JL, Gustovic P, Schrezenmeier H. Concomitant Immunosuppressive Therapy and Eculizumab Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Analysis. Acta Haematol. 2023;146(1):1-13. doi: 10.1159/000526979. Epub 2022 Sep 15.

  PMID: 36108594 DERIVED

• Chou WC, Huang WH, Wang MC, Chang CS, Yeh SP, Chiou TJ, Chen YC, Lin TH, Shen MC; Taiwan PNH study group. Characteristics of Taiwanese patients of PNH in the international PNH registry. Thromb J. 2016 Oct 4;14(Suppl 1):39. doi: 10.1186/s12959-016-0094-0. eCollection 2016.

  PMID: 27766064 DERIVED

• Kelly RJ, Hochsmann B, Szer J, Kulasekararaj A, de Guibert S, Roth A, Weitz IC, Armstrong E, Risitano AM, Patriquin CJ, Terriou L, Muus P, Hill A, Turner MP, Schrezenmeier H, Peffault de Latour R. Eculizumab in Pregnant Patients with Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2015 Sep 10;373(11):1032-9. doi: 10.1056/NEJMoa1502950.

  PMID: 26352814 DERIVED

Related Links

• Registry Summary Overview

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• Phillipe Gustovic

  Alexion Pharmaceuticals, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 15, 2011
• First Posted: June 16, 2011
• Study Start: October 29, 2004
• Primary Completion: April 14, 2025
• Study Completion: April 14, 2025
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)