Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

• ClinicalTrials.gov Identifier: NCT01374360
• Recruitment Status: Recruiting
• First Posted: June 16, 2011
• Last Update Posted: April 12, 2019
• Sponsor: Alexion Pharmaceuticals
• Information provided by (Responsible Party): Alexion Pharmaceuticals

Tracking Information

• First Submitted Date: April 15, 2011
• First Posted Date: June 16, 2011
• Last Update Posted Date: April 12, 2019
• Actual Study Start Date: January 2007
• Estimated Primary Completion Date: December 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 22, 2018)

Evaluate safety data specific to the use of Soliris [ Time Frame: Ongoing (up to 13 years) ]

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, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, impaired hepatic function, hemolysis, pregnancies, lactation, infusion reactions, bone marrow transplant, serious adverse events, and mortality.

Original Primary Outcome Measures (submitted: June 15, 2011)

The PNH Registry will collect data to evaluate safety data specific to the use of Soliris. [ Time Frame: Ongoing (up to 13 years) ]

Primary analyses will assess safety endpoints, including occurrence and time to first event for the following: meningococcal infections, infections with serious outcomes, formation of HAHA to Soliris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, serious hemolysis, pregnancies, infusion reactions, targeted adverse events, bone marrow transplant, and mortality.

• Change History: Complete list of historical versions of study NCT01374360 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures (submitted: February 22, 2018)

Collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris and non-Soliris treated patients [ Time Frame: Ongoing (up to 13 years) ]

Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.

Original Secondary Outcome Measures (submitted: June 15, 2011)

PNH Registry will collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris and non-Soliris treated patients. [ Time Frame: Ongoing (up to 13 years) ]

Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
• Official Title: Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
• Brief Summary: This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
• Detailed Description: Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
• Study Type: Observational
• Study Design: Observational Model: Other; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Probability Sample
• Study Population: PNH Patients
• Condition: Paroxysmal Nocturnal Hemoglobinuria
• Intervention: Not Provided

Study Groups/Cohorts

• Receiving Soliris
  PNH patients of any age, including minors, that are receiving Soliris
• Not receiving Soliris
  PNH patients of any age, including minors, that are not receiving Soliris

Publications *

• 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. eCollection 2016.
• Kelly RJ, Höchsmann B, Szer J, Kulasekararaj A, de Guibert S, Röth 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.

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: January 11, 2018): 5000
• Original Estimated Enrollment (submitted: June 15, 2011): 2000
• Estimated Study Completion Date: December 2025
• Estimated Primary Completion Date: December 2025 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Patients of any age, including minors, with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No

Contacts

Contact: Alexion Pharmaceuticals, Inc.; ClinicalTrials@alexion.com

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01374360
• Other Study ID Numbers: M07-001
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Alexion Pharmaceuticals
• Study Sponsor: Alexion Pharmaceuticals
• Collaborators: Not Provided

Investigators

• Study Director: Phillipe Gustovic; Alexion Pharmaceuticals

• PRS Account: Alexion Pharmaceuticals
• Verification Date: April 2019