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Alkylating agents
Immune Suppression Treatment for Sickle Cell Disease
Phase 2
Recruiting
Led By Maria Cancio, MD
Research Sponsored by Memorial Sloan Kettering Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
No Placebo-Only Group
Summary
This trial is studying a new way of doing an HCT: using drugs to lower the activity of the body's immune system before transplanting healthy stem cells to replace diseased or damaged cells.
Who is the study for?
This trial is for people aged 12-35 with sickle cell disease or β-thalassemia who need a stem cell transplant. They must have had certain complications like stroke, organ damage, or frequent pain episodes and agree to use contraception. Excluded are those over age 50, with recent strokes, uncontrolled infections, liver issues, HIV/hepatitis B/C, pregnancy, obesity (BMI >40), prior transplants or other cancers.
What is being tested?
The study tests if immune suppression drugs like fludarabine and dexamethasone before standard therapy can prevent graft failure and GvHD in patients receiving stem cell transplants. Other drugs may be added based on blood test results to improve immune suppression.
What are the potential side effects?
Possible side effects include weakened immunity leading to infections; reactions related to infusion; potential damage to organs such as the liver and kidneys; blood disorders; nausea; hair loss from chemotherapy agents used.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Number of participants with treatment related mortality/TRM or primary graft failure
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: Participants with Sickle Cell Disease or β-ThalassemiaExperimental Treatment8 Interventions
Participants will have severe sickle cell disease or transfusion-dependent β-thalassemia.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fludarabine
2012
Completed Phase 4
~1860
Cyclophosphamide
2010
Completed Phase 4
~2310
Tacrolimus
2019
Completed Phase 4
~5510
Mycophenolate Mofetil
1997
Completed Phase 4
~2380
Rabbit ATG
2001
Completed Phase 2
~60
Dexamethasone
2007
Completed Phase 4
~2650
Bortezomib
2005
Completed Phase 3
~1410
Rituximab
1999
Completed Phase 4
~2990
Find a Location
Who is running the clinical trial?
Memorial Sloan Kettering Cancer CenterLead Sponsor
1,973 Previous Clinical Trials
597,714 Total Patients Enrolled
2 Trials studying Thalassemia
15 Patients Enrolled for Thalassemia
Maria Cancio, MDPrincipal InvestigatorMemorial Sloan Kettering Cancer Center
2 Previous Clinical Trials
32 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had 2 or more episodes of prolonged erections in the last 2 years.I have had a bone marrow transplant from a donor.I have not had a stroke or brain surgery in the last 6 months.I am mostly active and can care for myself.I do not have any uncontrolled infections.I have thalassemia of any genetic type.I have mild liver fibrosis, not severe cirrhosis.My kidney function is good, as per the Schwartz formula.My lung function test shows I can breathe well enough, or if I'm under 7, my oxygen level is above 92% without help.I have had at least 2 episodes of acute chest syndrome in the last 2 years.My major organs are functioning well.I have SCD with complications.My direct bilirubin levels are within normal range.I am between 13 and 34 years old, or 2-12/35-50 with specific trial conditions met.I am not pregnant and can bear children.I've had 3 or more pain crises in the last 2 years.I have had osteomyelitis or osteonecrosis.I have received more than 225 mL/kg of red blood cells in a year or over 15 transfusions in my lifetime.I have an enlarged liver and spleen.I, or my legal guardian, can sign the consent form. If I'm 7-17, I can agree to participate.I am over 18 and my kidney function is good.I have had a neurological event that caused lasting symptoms for more than a day.I have active hepatitis B or C.I have been cancer-free for at least 2 years, except for certain skin or cervical conditions.I needed blood transfusions regularly since I was a baby.
Research Study Groups:
This trial has the following groups:- Group 1: Participants with Sickle Cell Disease or β-Thalassemia
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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