Natural History Study of Adult Patients With Paroxysmal Nocturnal Hemoglobinuria of High-risk Hemolysis in China

June 30, 2022 updated by: CARE Pharma Shanghai Ltd.

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 Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

67

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Beijing, China
        • Peking Union Medical College Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis in China

Description

Inclusion Criteria:

  1. Meet the diagnostic criteria for paroxysmal nocturnal hemoglobinuria;
  2. Age ≥ 18 years old;
  3. PNH clone size of granulocytes or monocytes (CD59 or Flaer) ≥ 10%;
  4. LDH≥1.5 ULN;
  5. Accompanied by at least one PNH-related symptoms: fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia symptoms, major adverse vascular events (including thrombosis), dysphagia, erectile dysfunction;
  6. Transfusion-dependent PNH patients;
  7. There are at least 6 months consecutive data of diagnosis and treatment after the data collection time point;
  8. Patients agreed to participate in the study by signing informed consent or giving oral informed consent.

Exclusion Criteria:

  1. Patients are under the treatment of the C1/C3/C5 complement inhibitors currently or in the past;
  2. Patients have received bone marrow transplantation;
  3. platelet count <30*10 9/L or absolute neutrophil count <0.5*10 9/L;
  4. Patients have clinically significant heart, liver, or lung diseases, or have related medical history.
  5. Patients have comorbidities of rheumatoid disease, HIV infection, active HBV infection, HCV infection and other diseases that affect the body's immune function;
  6. Severe missing data in the records of diagnosis and treatment after the data collection time point;
  7. Other patients who are not suitable to participate in the study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
the mean change of LDH
Time Frame: 6 months
the mean change of LDH over 6 months
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CARE Pharma Shanghai Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 25, 2021

Primary Completion (Actual)

March 16, 2022

Study Completion (Actual)

April 30, 2022

Study Registration Dates

First Submitted

November 8, 2021

First Submitted That Met QC Criteria

November 8, 2021

First Posted (Actual)

November 18, 2021

Study Record Updates

Last Update Posted (Actual)

July 5, 2022

Last Update Submitted That Met QC Criteria

June 30, 2022

Last Verified

November 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAN106-01001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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