Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

NCT03805581 | Completed Phase 2 FDA Drug

• 1 other identifier: NYMC-192
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Sickle Cell Disease; Acute Chest Syndrome

Outcome Measures

Primary Outcomes (2)

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

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

  30 days

• Number of patients with grade III/IV hemorrhage

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

  30 days

Secondary Outcomes (1)

• Number of patients with improvement in clinical signs of Acute Chest Syndrome

  30 days

Study Arms (1)

Interventional

EXPERIMENTAL

Defibrotide 6.25 mg/kg IV q6h

Drug: Defibrotide

Interventions

Defibrotide DRUG

Defibrotide 6.25 mg/kg IV q6h up to 7 days

Also known as: Defitelio®

Interventional

Eligibility Criteria

• Age: 2 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• New York Medical College: lead
• Johns Hopkins University: collaborator

Study Sites (1)

New York Medical College

Valhalla, New York, 10595, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Acute Chest Syndrome

Interventions

defibrotide

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lung Diseases; Respiratory Tract Diseases; Respiration Disorders

Study Officials

• Mitchell S Cairo, MD

  New York Medical College

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2018
• First Posted: January 15, 2019
• Study Start: January 12, 2018
• Primary Completion: July 1, 2022
• Study Completion: November 7, 2023
• Last Updated: December 8, 2023

Record last verified: 2023-12

Locations

US (1)