Paroxysmal Nocturnal HemoglobinuriaNCT01374360
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
Trial summary
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- How many people
- 6,061 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Oct 2004to 14 Apr 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: PNH Patients
Who may be able to join
- 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with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.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.
Who cannot join
- 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 locations and contacts
1 site in 1 country.
United States1 site
Contact the PNH Registry at Alexion Pharmaceuticals, Inc. for worldwide locations.
Boston, Massachusetts, 02210
About this study
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Read the detailed description
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
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.
Measured: Ongoing (up to 13 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2011 (estimated)
- Study start29 Oct 2004
- Primary completion14 Apr 2025
- Study completion14 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 6,061 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Phillipe Gustovic, Study director, Alexion Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01374360
- Sponsor's protocol ID
- M07-001
- Status last verified
- Jan 2026
- First submitted
- 15 Apr 2011
- Last updated
- 20 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
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 May;101(5):939-948. doi: 10.1002/ajh.70268. Epub 2026 Mar 7.
PubMed 41795103Dingli 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.
PubMed 37199789Hill 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.
PubMed 36108594Chou 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.
PubMed 27766064Kelly 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.
PubMed 26352814
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
- ClinicalTrials.gov ID
- NCT01374360
- Acronym / sponsor ID
- M07-001
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH, Paroxysmal Nocturnal hemoglobinuria, Soliris, Eculizumab, Ravulizumab, Ultomiris
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 6,061 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Oct 2004 (actual)
- Primary completion
- 14 Apr 2025 (actual)
- Study completion
- 14 Apr 2025 (actual)
- First posted
- 16 Jun 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- PNH Patients
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Evaluate safety data specific to the use of Soliris and Ultomiris
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 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.
1 secondary outcome
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
Time frame: Ongoing (up to 13 years)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2026.
- Derived from this study: PubMed 41795103Roth 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 May;101(5):939-948. doi: 10.1002/ajh.70268. Epub 2026 Mar 7.
- Derived from this study: PubMed 37199789Dingli 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.
- Derived from this study: PubMed 36108594Hill 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.
- Derived from this study: PubMed 27766064Chou 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.
- Derived from this study: PubMed 26352814Kelly 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.
- Registry Summary OverviewLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Phillipe Gustovic study director
Alexion Pharmaceuticals, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate safety data specific to the use of Soliris and Ultomiris (time frame: Ongoing (up to 13 years)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01374360, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2026.