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Completed

NCT Number: NCT03805581

Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

This study evaluates the safety of defibrotide in subjects with sickle cell disease (SCD)-associated acute chest syndrome (ACS).

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Key information

Conditions

Sickle Cell Disease Acute Chest Syndrome Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Lung Diseases Respiration Disorders Respiratory Tract Diseases

Age range

2 year–40 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

New York Medical College

Valhalla, New York, 10595, United States

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • SCD-associated ACS with the presence of any two or more of the following signs not explained by other etiologies: Fever, Chest pain, Cough, Dyspnea, Tachypnea for age, Pulmonary infiltrate on CXR and/or Chest CT scan, Decreased O2 saturation with or without oxygen supplement;
  • Age 2 to 40 years of age;
  • Homozygous Hemoglobin S Disease, Hemoglobin SC Disease or Hemoglobin S 0/+ thalassemia;
  • Informed consent/assent;
  • Consent of patient/parent within ≤72 hours after inpatient admission for SCD-associated ACS.
  • Females of childbearing age will have a negative pregnancy test.

Exclusion criteria

  • Current Grade III or IV hemorrhage;
  • Previous hypersensitivity reaction to defibrotide;
  • Current systemic anti-coagulant therapy and/or fibrinolytic therapy;
  • Consent of patient/parent greater than 72 hours of inpatient admission for SCD-associated ACS;
  • No signed informed consent

Treatment and study plan

Defibrotide

Drug

Defibrotide 6.25 mg/kg IV q6h up to 7 days

Other names: Defitelio®

Primary outcomes

  1. Number of patients with grade III/IV allergic reaction to defibrotide

    Time frame: 30 days

    All patients will be monitored for allergic reaction probably or definitely related to defibrotide administration.

  2. Number of patients with grade III/IV hemorrhage

    Time frame: 30 days

    All patients will be monitored for hemorrhage probably or definitely related to defibrotide.

Secondary outcomes

  1. Number of patients with improvement in clinical signs of Acute Chest Syndrome

    Time frame: 30 days

    Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS

Sponsors and collaborators

Lead sponsor

New York Medical College

Other

Collaborators

  • Johns Hopkins University

Registry information

Official study title

A Phase II Study to Determine the Safety of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

Important dates

Study start
2018
Primary completion
2022
Study completion
2023
First posted
Jan 15, 2019
Registry last updated
Dec 8, 2023

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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