Sickle Cell DiseasePhase 2NCT03805581
Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
A Phase 2 study testing Defibrotide in people with Sickle Cell Disease and Acute Chest Syndrome, run by New York Medical College at 1 site in United States.
Trial summary
This study evaluates the safety of defibrotide in subjects with sickle cell disease (SCD)-associated acute chest syndrome (ACS). Read more about this study
- Who is it for?
- Ages 2 to 40 years
- Conditions the study is for
- Sickle Cell Disease, Acute Chest Syndrome
- What is being tested?
- Defibrotide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Jan 2018to 7 Nov 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II Study to Determine the Safety of 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).
- Conditions
- Sickle Cell Disease, Acute Chest Syndrome
- Being tested
- Defibrotide
- Medical subject headings
- Anemia, Sickle Cell, Acute Chest Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DefibrotideDrug
Also called Defitelio®
Defibrotide 6.25 mg/kg IV q6h up to 7 days
| Study group | Role | What they receive |
|---|---|---|
| Interventional | Experimental | Defibrotide 6.25 mg/kg IV q6h Receives: Drug: Defibrotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 30 days
Number of patients with grade III/IV hemorrhage
All patients will be monitored for hemorrhage probably or definitely related to defibrotide.
Measured: 30 days
Secondary outcomes (1)
Number of patients with improvement in clinical signs of Acute Chest Syndrome
Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS
Measured: 30 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2019
- Study start12 Jan 2018
- Primary completion1 Jul 2022
- Study completion7 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Collaborators
- Johns Hopkins University
- Responsible party
- Sponsor
- Investigators
- Mitchell S Cairo, MD, Principal investigator, New York Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03805581
- Sponsor's protocol ID
- NYMC-192
- Status last verified
- Dec 2023
- First submitted
- 18 Dec 2018
- Last updated
- 8 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study to Determine the Safety of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
- ClinicalTrials.gov ID
- NCT03805581
- Acronym / sponsor ID
- NYMC-192
- Conditions
- Sickle Cell Disease, Acute Chest Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Collaborators
- Johns Hopkins University
- Study start
- 12 Jan 2018 (actual)
- Primary completion
- 1 Jul 2022 (actual)
- Study completion
- 7 Nov 2023 (actual)
- First posted
- 15 Jan 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study evaluates the safety of defibrotide in subjects with sickle cell disease (SCD)-associated acute chest syndrome (ACS).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Interventional | Experimental | Drug: DefibrotideDefibrotide 6.25 mg/kg IV q6h |
Who may qualify
- Ages
- 2 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DefibrotideAlso: Defitelio®Defibrotide 6.25 mg/kg IV q6h up to 7 days | Drug | Interventional |
Primary outcome
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.
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.
1 secondary outcome
Number of patients with improvement in clinical signs of Acute Chest Syndrome
Time frame: 30 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mitchell S Cairo, MD principal investigator
New York Medical College
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Defibrotide in people with Sickle Cell Disease and Acute Chest Syndrome, run by New York Medical College at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with grade III/IV allergic reaction to defibrotide (time frame: 30 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2023.