MedDataX Logo

Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

NCT ID: NCT03805581

Last Updated: 2023-12-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-01-12

Study Completion Date

2023-11-07

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Sickle Cell Disease Acute Chest Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Interventional

Defibrotide 6.25 mg/kg IV q6h

Group Type EXPERIMENTAL

Defibrotide

Intervention Type DRUG

Defibrotide 6.25 mg/kg IV q6h up to 7 days

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Defibrotide

Defibrotide 6.25 mg/kg IV q6h up to 7 days

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Defitelio®

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

2 Years

Maximum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Johns Hopkins University

OTHER

Sponsor Role collaborator

New York Medical College

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Mitchell S Cairo, MD

Role: PRINCIPAL_INVESTIGATOR

New York Medical College

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

New York Medical College

Valhalla, New York, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

NYMC-192

Identifier Type: -

Identifier Source: org_study_id