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NCT Number: NCT05125341

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

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.

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria, Natural History Study Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Peking Union Medical College Hospital

Beijing, China

Who can participate

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

Primary outcomes

  1. the mean change of LDH

    Time frame: 6 months

    the mean change of LDH over 6 months

Sponsors and collaborators

Lead sponsor

CARE Pharma Shanghai Ltd.

Industry

Registry information

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Nov 18, 2021
Registry last updated
Jul 5, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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