An observational study in Paroxysmal Nocturnal Hemoglobinuria, sponsored by Aplastic Anemia and MDS International Foundation. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2025-02-20.
Sponsored by Aplastic Anemia and MDS International Foundation · Observational
Among 68 studies in Hemoglobinuria, Paroxysmal with a site in United States — 10 open to participants now, 16 under way, 4 of them phase 3. Hemoglobinuria, Paroxysmal · United States.
Among the 68 studies in Hemoglobinuria, Paroxysmal with a site in United States, this one is the 4th largest by planned enrollment of the 13 observational ones, is one of 5 that started in 2021, is one of 13 observational ones, reaches primary completion later than 0 of the 15 other live ones, and was the 59th registered.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
This study's planned enrollment of 500 is above the median of 90 across 37 observational studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →10 of the 68 studies in Hemoglobinuria, Paroxysmal with a site in United States are open now, in 12 countries. Each count lists the studies.
Of the 30 Hemoglobinuria, Paroxysmal studies recruiting now, 14 (47%) set no upper age limit and 12 (40%) stop at 75 or younger; 12 take children under 18; 4 take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →68 studies; the count open now beside each. This study's row is marked.
First in 1996; this study’s year is marked. 5 started in 2021.
Median enrollment 53 across the 54 interventional studies, 75 across the 13 observational. Of the 50 that completed or were terminated, 26 (52%) have results posted.
35 of 68 have an industry lead sponsor. The 10 most frequent:
This is the only study on the registry with Aplastic Anemia and MDS International Foundation as lead sponsor.
Aplastic Anemia and MDS International Foundation has 1 study filed under Hemoglobinuria, Paroxysmal — fewer than ten, so there is no comparison to draw.
3 of the studies in Hemoglobinuria, Paroxysmal with a site in United States that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04432584Phase 3Hoffmann-La RocheA Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors | 2027-04-18 |
| NCT04747613Phase 3Novartis PharmaceuticalsLong-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria | 2027-10-18 |
| NCT03520647National Heart, Lung, and Blood Institute (NHLBI)Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis | 2027-06-01 |
Among the 68 studies in Hemoglobinuria, Paroxysmal with a site in United States, from their registry updates:
The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the disease and its course and pace over time. Other registry objectives include the following:
The Global PNH Patient Registry is a prospective, longitudinal, web-based, observational natural history study. Participants with PNH will be followed throughout the course of their lives with either the participant or authorized respondents contributing data at varying intervals throughout the course of the study. Data will be collected at the start of the study (baseline), at least once per year or can be updated by the participant as needed. Data will be collected on demographics, quality of life, medical history, disease phenotypes, disease-related events, personal experience with PNH, general health status, medications, and diagnoses. The Global PNH Patient Registry provides a convenient online platform for participants; facilitates communication about PNH; gives researchers the ability to characterize the PNH population as a whole; assists the PNH community with the development of recommendations and standards of care and; and serves as a case-finding resource to be used for researchers to study the pathophysiology of PNH.
A Registry Advisory Board, that may include scientists, doctors, and patient advocates, will be assembled to oversee the conduct of the study. The Advisory Board will review aggregate registry data and the use of this registry, ensure proper evaluation of protocols requesting to use registry data and/or contact registry participants, and review any protocol or confidentiality deviations on a case- by by-case basis and ensure that any such deviations are reported to the IRB.
The registry will use a web-based interface to maximize accessibility to participants and clinicians world-wide. Following informed consent, participants will be invited to enter their data and information which will be stored indefinitely or until a participant revokes their consent to participate in the study. No experimental intervention is involved in participation in the Global PNH Patient registry study.
Annual maintenance will be funded by the Aplastic Anemia and MDS International Foundation (AAMDSIF) with support from industry partners. Registry participants will be automatically enrolled in NORD's Natural History Study Program (NHS), and their de-identified information aggregated with information from other rare diseases may be used for the purposes of cross disease analysis and cross-disease research to facilitate advocacy and further NORD's mission. De-identified information may be shared with other databases such as the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP). This will allow more researchers to use the information to do research.
Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Individuals must be willing to provide . Participants can be:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion.
Characterize and describe the Global PNH population
Understand disease prevalence and phenotype as well as the rate of progression of disease characteristics. Gather patient experiences including demographics, diagnostics, treatment, symptoms, and quality of life.
Time frame: 5-10 years
Develop a communications registry
* Be a case-finding resource to be used for researchers who seek to study the pathophysiology of PNH, retrospectively collate intervention outcomes, and design prospective trials of novel treatments. * Notify patients of research studies and clinical trials as appropriate.
Time frame: 5-10 years
Plan to share: Yes — It is anticipated that the Global PNH Patient Registry will be a valuable resource for current and future research. A Global PNH Patient Registry Advisory Board will ensure proper evaluation of protocols that request use of registry data and/or contact registry participants. To promote use of the repository, aggregate information about database contents will be updated quarterly or semi-annually and made available to the public. Such information may include number of registrants, prevalence of common diagnoses of registrants, demographic information, and percent willing to be contacted for future research. Investigators wanting to use the registry or contact participants will need to apply to the Advisory Board. The application will require information concerning: Principal Investigator, aims and hypotheses of the proposed research, where the research will be performed, and how the research will be funded.
Supporting information: Clinical study report
Time frame: Data will be available 2 years after study start and will be analyzed through study ending.
Access criteria: Sharing is subject to review by the Medical Advisory Committee
Started May 2021 (actual). Primary completion is estimated for May 2026, 60 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
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