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Immunosuppressive Drug
Low-Dose Radiation + Immunosuppressants for Sickle Cell Disease
Phase 1 & 2
Waitlist Available
Led By John F Tisdale, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year and 2 year
Awards & highlights
No Placebo-Only Group
Summary
This trial is exploring whether a bone marrow transplant procedure that uses a low dose of radiation and two immunosuppressive drugs is safe for people who are typically excluded from the procedure because of their age.
Who is the study for?
This trial is for adults aged 18-65 with severe congenital anemias like sickle cell disease, who have a sibling as a stem-cell donor. Participants must meet specific health criteria such as normal organ function and not be pregnant or lactating. Those with uncontrolled infections or significant heart/lung problems are excluded.
What is being tested?
The study tests a bone marrow transplant (BMT) method using low-dose radiation and immunosuppressive drugs Alemtuzumab and Sirolimus, instead of chemotherapy. This nonmyeloablative BMT aims to be safer for patients normally too old for traditional BMT.
What are the potential side effects?
Potential side effects include immune system suppression leading to increased infection risk, possible infusion reactions from the drugs used, fatigue, blood count changes, and mild radiation-associated risks.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year and 2 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year and 2 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 That Experience Treatment Success Following Stem Cell Transplant
Secondary study objectives
Mean Myeloid Chimerism Level
Number of Participants Overall Survival
Number of Participants That Experienced a Transplant-related Mortality
+4 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell TransplantsExperimental Treatment3 Interventions
Nonmyeloablative transplant regiment, consisting of alemtuzumab (1 mg/kg in divided doses), total-body irradiation (300 cGy), sirolimus, and infusion of unmanipulated filgrastim mobilized peripheral blood stem cells from human leukocyte antigen-matched siblings.
Group II: Human Leukocyte Antigens (HLA) Matched Related Stem Cell DonorExperimental Treatment1 Intervention
Participants received Filgrastim to mobilize peripheral blood stem cells for apheresis collection. Collected stem cells of donor will then be infused to HLA matched sibling.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Alemtuzumab
2004
Completed Phase 4
~1880
Sirolimus
2013
Completed Phase 4
~2750
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Logistics
Participation is compensated
You will be compensated for participating in this trial.
Who is running the clinical trial?
National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,934 Previous Clinical Trials
47,792,166 Total Patients Enrolled
John F Tisdale, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
14 Previous Clinical Trials
1,197 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am over 18 and have high ferritin or bilirubin levels indicating liver issues.I need regular blood transfusions.I am mostly bedridden due to my health condition.I don't have any ongoing serious infections.I do not have any major illnesses that would make a stem cell transplant impossible.Not applicable.You must have one of the following diseases:I have sickle cell disease with severe complications not improved by medication.I have beta-thalassemia with significant iron overload.I have had a stroke confirmed by an MRI or an abnormal Doppler exam.I am between 5 and 10 years old with normal kidney function.I am eligible for a stem cell transplant and have been hospitalized more than once a year despite taking the highest dose of hydroxyurea I can tolerate.I have kidney problems due to sickle cell disease.I have had at least two long-lasting erections involving all parts of the penis.I haven't followed my iron removal treatment as prescribed since my first blood transfusion.I am between 10 and 15 years old with normal kidney function.I am over 15 years old and my kidney function test is normal.I am taking the highest dose of hydroxyurea I can tolerate.My heart valve's flow speed is high and I'm over 18, measured after a recent health crisis.I have had episodes of severe pain due to blocked blood flow.I have had acute chest syndrome.I have had 2 acute chest syndromes after age 3 and have received proper asthma treatment.I am eligible for a stem cell transplant and have been treated with hydroxyurea.I have bone damage in two or more of my joints.My daily activities are significantly limited, but I can still care for myself.My liver extends more than 2 cm below my rib cage.My hemoglobin hasn't increased more than 1 g/dL on hydroxyurea.I am under 16 and have chosen not to undergo a bone marrow transplant.I am 4 years old or older.I have a family donor who is a perfect match for me.I am 5 years old or younger with normal kidney function.I've been admitted to the hospital 3 times in the last year and can take hydroxyurea.I am eligible for a stem cell transplant and my hemoglobin levels have not significantly increased with hydroxyurea treatment.I have at least one of the listed complications.
Research Study Groups:
This trial has the following groups:- Group 1: Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants
- Group 2: Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor
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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