Defibrotide for Anemia, Sickle Cell

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Anemia, Sickle Cell+1 More
Defibrotide - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

This trial will assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation.

Eligible Conditions
  • Anemia, Sickle Cell

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Anemia, Sickle Cell

Study Objectives

2 Primary · 0 Secondary · Reporting Duration: 1 year

1 year
All patients will be monitored for the development of SOS.
100 days
All patients will be monitored for known and unknown side effects of defibrotide with daily physical exams while in the hospital and then as needed in addition to daily laboratory values including chemistries, hematology labs as needed

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Anemia, Sickle Cell

Side Effects for

Defibrotide Plus LPS
81%Headache
38%Flu-like Symptoms
19%Chills
13%Nausea
13%Arthralgia
6%Precollapse
6%Vertigo
6%Dizziness
This histogram enumerates side effects from a completed 2018 Phase 4 trial (NCT02876601) in the Defibrotide Plus LPS ARM group. Side effects include: Headache with 81%, Flu-like Symptoms with 38%, Chills with 19%, Nausea with 13%, Arthralgia with 13%.

Trial Design

1 Treatment Group

Defibrotide prophylaxis
1 of 1
Experimental Treatment

40 Total Participants · 1 Treatment Group

Primary Treatment: Defibrotide · No Placebo Group · Phase 2

Defibrotide prophylaxis
Drug
Experimental Group · 1 Intervention: Defibrotide · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Defibrotide
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year

Who is running the clinical trial?

Dana-Farber Cancer InstituteOTHER
1,012 Previous Clinical Trials
393,319 Total Patients Enrolled
7 Trials studying Anemia, Sickle Cell
2,262 Patients Enrolled for Anemia, Sickle Cell
New York Medical CollegeLead Sponsor
65 Previous Clinical Trials
5,644 Total Patients Enrolled
4 Trials studying Anemia, Sickle Cell
81 Patients Enrolled for Anemia, Sickle Cell
Medical College of WisconsinOTHER
554 Previous Clinical Trials
1,152,132 Total Patients Enrolled
14 Trials studying Anemia, Sickle Cell
12,811 Patients Enrolled for Anemia, Sickle Cell
Children's Hospital Los AngelesOTHER
203 Previous Clinical Trials
5,071,676 Total Patients Enrolled
7 Trials studying Anemia, Sickle Cell
459 Patients Enrolled for Anemia, Sickle Cell
Baylor College of MedicineOTHER
914 Previous Clinical Trials
5,981,999 Total Patients Enrolled
10 Trials studying Anemia, Sickle Cell
12,138 Patients Enrolled for Anemia, Sickle Cell
Tufts Medical CenterOTHER
235 Previous Clinical Trials
244,496 Total Patients Enrolled
1 Trials studying Anemia, Sickle Cell
21 Patients Enrolled for Anemia, Sickle Cell
University of California, Los AngelesOTHER
1,404 Previous Clinical Trials
6,942,407 Total Patients Enrolled
5 Trials studying Anemia, Sickle Cell
107 Patients Enrolled for Anemia, Sickle Cell
Johns Hopkins UniversityOTHER
2,055 Previous Clinical Trials
30,974,941 Total Patients Enrolled
12 Trials studying Anemia, Sickle Cell
1,612 Patients Enrolled for Anemia, Sickle Cell
Mitchell Cairo, MDPrincipal Investigator - New York Medical College
Fort Washington Medical Center, Holy Cross Germantown Hospital, Holy Cross Hospital of Silver Spring, Inova Alexandria Hospital, Inova Fairfax Hospital, MedStar Southern Maryland, Said M Ali Mdpc, Washington Adventist Hospital
University Of Cairo (Medical School)
Aultman Hospital (Residency)
12 Previous Clinical Trials
314 Total Patients Enrolled

Eligibility Criteria

Age < 65 · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have a stroke or any neurologic deficit lasting >24 hours that is accompanied by an infarct on cerebral MRI.
You have sickle cell nephropathy.
You have had at least 3 painful events in the 2 years prior to enrollment.
At least one silent infarct lesion on a MRI scan of the head.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 23rd, 2021

Last Reviewed: October 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.