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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
Study 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.Check my eligibility
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.See study design
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
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of Participants That Experience Treatment Success Following Stem Cell Transplant
Secondary outcome measures
Mean Myeloid Chimerism Level
Number of Participants Overall Survival
Number of Participants That Experienced a Transplant-related Mortality
+4 moreTrial 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
~1890
Sirolimus
2013
Completed Phase 4
~2750
Find a Location
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
3,837 Previous Clinical Trials
47,851,497 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.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What medical indications is Alemtuzumab typically prescribed for?
"Alemtuzumab has demonstrated efficacy in treating immune thrombocytopenic purpura, as well as autoimmune hemolytic anemia, organ transplantation and multiple sclerosis."
Answered by AI
Who is eligible to partake in this trial?
"Recruitment to this study necessitates that the participant is diagnosed with congenital hemolytic anemia and between 2 and 65 years of age; as many as 150 individuals will be allowed into the trial."
Answered by AI
What other research has been undertaken for the purpose of understanding Alemtuzumab?
"Presently, the therapeutic efficacy of Alemtuzumab is being examined in 147 active trials with 16 Phase 3 studies. While Philadelphia has several medical centres running clinical tests on this medication, there are 1251 locations across the globe conducting research into it."
Answered by AI
Does the study include participants aged 40 or older?
"The eligibility criteria stipulates that the minimum age for participation is 2 and no participant should exceed 65 years of age."
Answered by AI
Are there vacancies available for participants in this research experiment?
"Per clinicaltrials.gov, this investigation is still actively seeking participants with the original posting date being 16th July 2004 and last modified on 15th November 2022."
Answered by AI
What is the aggregate of participants engaged in this clinical experiment?
"Affirmative. Clinicaltrials.gov details this research project, which was initially posted on July 16th 2004 and most recently amended November 15th 2022, is currently searching for participants to fill its quota of 150 individuals at 2 sites."
Answered by AI
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