Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03805581

Enrollment

Registered

2019-01-15

Start date

2018-01-12

Completion date

2023-11-07

Last updated

2023-12-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Acute Chest Syndrome, Sickle Cell Disease

Brief summary

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

Interventions

DRUGDefibrotide

Defibrotide 6.25 mg/kg IV q6h up to 7 days

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to 40 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Number of patients with grade III/IV allergic reaction to defibrotide	30 days	All patients will be monitored for allergic reaction probably or definitely related to defibrotide administration.
Number of patients with grade III/IV hemorrhage	30 days	All patients will be monitored for hemorrhage probably or definitely related to defibrotide.

Secondary

Measure	Time frame	Description
Number of patients with improvement in clinical signs of Acute Chest Syndrome	30 days	Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 17, 2026