Paroxysmal Nocturnal Hemoglobinuria, Natural History StudyNCT05125341
Natural History Study of Adult Patients With Paroxysmal Nocturnal Hemoglobinuria of High-risk Hemolysis in China
An observational study of Paroxysmal Nocturnal Hemoglobinuria, Natural History Study, run by CARE Pharma Shanghai Ltd. at 1 site in China.
Trial summary
This is a single-center observational study conducted in adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis. This observational study consists of two parts, one part is retrospective study which aims to collect medical chart data to calculate the mean change or mean incidence rates of LDH… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria, Natural History Study
- How many people
- 67 participants
- Where
- 1 siteChina
- Study dates
- 25 Nov 2021to 30 Apr 2022
- 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 18 years and older
- All sexes
- Adult, Older adult
Study population: Adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis in China
Who may be able to join
- Meet the diagnostic criteria for paroxysmal nocturnal hemoglobinuria;
- Age ≥ 18 years old;
- PNH clone size of granulocytes or monocytes (CD59 or Flaer) ≥ 10%;
- LDH≥1.5 ULN;
- Accompanied by at least one PNH-related symptoms: fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia symptoms, major adverse vascular events (including thrombosis), dysphagia, erectile dysfunction;
- Transfusion-dependent PNH patients;
- There are at least 6 months consecutive data of diagnosis and treatment after the data collection time point;
- Patients agreed to participate in the study by signing informed consent or giving oral informed consent.
Who cannot join
- Patients are under the treatment of the C1/C3/C5 complement inhibitors currently or in the past;
- Patients have received bone marrow transplantation;
- platelet count <30*10 9/L or absolute neutrophil count <0.5*10 9/L;
- Patients have clinically significant heart, liver, or lung diseases, or have related medical history.
- Patients have comorbidities of rheumatoid disease, HIV infection, active HBV infection, HCV infection and other diseases that affect the body's immune function;
- Severe missing data in the records of diagnosis and treatment after the data collection time point;
- Other patients who are not suitable to participate in the study.
Trial locations and contacts
1 site in 1 country.
About this study
This is a single-center observational study conducted in adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis.
This observational study consists of two parts, one part is retrospective study which aims to collect medical chart data to calculate the mean change or mean incidence rates of LDH, hemoglobin, PNH-related symptoms and PNH-related events over 6 months.
The other part is cross-sectional study to detect the total C5 level in PUMCH at the latest follow-up visit in eligible PNH patients with high-risk hemolysis, to show the difference between eligible PNH patients and healthy people and to explore the related clinical factor influencing high-level total C5 using logistic regression model.
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What is being measured
The primary outcome is the main question the study is designed to answer.
the mean change of LDH
the mean change of LDH over 6 months
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2021
- Study start25 Nov 2021
- Primary completion16 Mar 2022
- Study completion30 Apr 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria, Natural History StudyStudies of the same condition, open and under way.
- 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
- Other
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- CARE Pharma Shanghai Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bing Han, MD, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05125341
- Sponsor's protocol ID
- CAN106-01001
- Status last verified
- Nov 2021
- First submitted
- 8 Nov 2021
- Last updated
- 5 Jul 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History Study of Adult Patients With Paroxysmal Nocturnal Hemoglobinuria of High-risk Hemolysis in China
- ClinicalTrials.gov ID
- NCT05125341
- Acronym / sponsor ID
- CAN106-01001
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 67 participants (actual)
- Lead sponsor
- CARE Pharma Shanghai Ltd.Industry (pharma, biotech, devices)
- Study start
- 25 Nov 2021 (actual)
- Primary completion
- 16 Mar 2022 (actual)
- Study completion
- 30 Apr 2022 (actual)
- First posted
- 18 Nov 2021 (actual)
- 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 is a single-center observational study conducted in adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis.
This observational study consists of two parts, one part is retrospective study which aims to collect medical chart data to calculate the mean change or mean incidence rates of LDH, hemoglobin, PNH-related symptoms and PNH-related events over 6 months.
The other part is cross-sectional study to detect the total C5 level in PUMCH at the latest follow-up visit in eligible PNH patients with high-risk hemolysis, to show the difference between eligible PNH patients and healthy people and to explore the related clinical factor influencing high-level total C5 using logistic regression model.
Study design
- Observational model
- Other
- Time perspective
- Other
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis in China
Key inclusion criteria (8 listed)
- Meet the diagnostic criteria for paroxysmal nocturnal hemoglobinuria;
- Age ≥ 18 years old;
- PNH clone size of granulocytes or monocytes (CD59 or Flaer) ≥ 10%;
- LDH≥1.5 ULN;
- Accompanied by at least one PNH-related symptoms: fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia symptoms, major adverse vascular events (including thrombosis), dysphagia, erectile dysfunction;
Key exclusion criteria (7 listed)
- Patients are under the treatment of the C1/C3/C5 complement inhibitors currently or in the past;
- Patients have received bone marrow transplantation;
- platelet count <30*10 9/L or absolute neutrophil count <0.5*10 9/L;
- Patients have clinically significant heart, liver, or lung diseases, or have related medical history.
- Patients have comorbidities of rheumatoid disease, HIV infection, active HBV infection, HCV infection and other diseases that affect the body's immune function;
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
the mean change of LDH
Time frame: 6 months
the mean change of LDH over 6 months
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 5 Jul 2022.
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.
Bing Han, MD principal investigator
Peking Union Medical College Hospital
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, Natural History Study, run by CARE Pharma Shanghai Ltd. at 1 site in China.
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 8 inclusion and 7 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 5 Jul 2022. 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 China. 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?
the mean change of LDH (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Jul 2022; AzTrials last refreshed its copy on 8 Nov 2026.