Your session is about to expire
← Back to Search
Non-Myeloablative Bone Marrow Transplant for Sickle Cell Disease
N/A
Waitlist Available
Led By Adetola A Kassim, MD
Research Sponsored by Vanderbilt-Ingram Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Good performance status (ECOG 0 or 1; Karnofsky and Lansky 70-100)
Eligible diagnoses: Patients with sickle cell anemia such as sickle cell anemia (Hb SS), Hb Sβ° thalassemia, Hb Sβ+thalassemia, Hb SC disease, Hb SE disease, Hb SD disease, Hemoglobin SO- Arab disease HbS with hereditary persistence of fetal hemoglobin. Other significant hemoglobinopathies.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights
Study Summary
This trial is testing a new way to do an allogeneic blood or marrow transplantation (alloBMT) that may be less toxic and more effective than the current standard method.
Who is the study for?
This trial is for patients with sickle cell anemia or related hemoglobin disorders, aged 1-70 years, who have a compatible first-degree relative donor willing to donate bone marrow. Participants must not be pregnant, HIV-positive, or have severe organ dysfunction. They should be able to consent and commit to all treatment stages.Check my eligibility
What is being tested?
The study tests a non-myeloablative conditioning regimen before bone marrow transplantation (alloBMT) in patients with sickle cell disease and thalassemia. It aims to reduce toxicity by using immunosuppressive drugs like Thymoglobulin and Sirolimus instead of high-dose chemotherapy or radiation.See study design
What are the potential side effects?
Potential side effects include immune system suppression leading to increased infection risk, reactions from the infusion of drugs, liver and kidney function changes, gastrointestinal issues such as nausea or diarrhea, mouth sores from Fludarabine and Cyclophosphamide.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am fully active or able to carry out light work.
Select...
I have a diagnosed form of sickle cell disease or another significant hemoglobin disorder.
Select...
I still experience severe pain from my condition despite treatment with hydroxyurea or blood transfusions.
Select...
I have a long-term lung condition that is getting worse over time.
Select...
I am between 1 and 70 years old.
Select...
I have had a stroke or brain damage confirmed by MRI.
Select...
I have had repeated severe chest pain needing hospital stays.
Select...
I have moderate to severe kidney disease.
Select...
I need regular blood transfusions for my thalassemia.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 2 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Transplant-related Mortality (TRM)
Secondary outcome measures
Number of Participants With Hematologic and Non-hematologic Toxicities Following minihaploBMT
Number of Patients Who Developed Grade I-IV Acute Graft-vs.-Host Disease
Number of Patients With Donor Hematopoietic Chimerism in Peripheral Blood <95% at 6 Months After Mini-haploBMT
Side effects data
From 2014 Phase 2 trial • 5 Patients • NCT00801632100%
Engraftment syndrome
80%
Proteinuria
60%
Thrombocytopenia
60%
Urinary tract infection
40%
Transplant rejection
40%
Alanine aminotransferase increased
40%
Weight increased
40%
Hypophosphataemia
40%
Anaemia
40%
Clostridium difficile colitis
20%
Stomatitis
20%
Gastroenteritis
20%
Viral infection
20%
Post procedural haemorrhage
20%
Blood creatinine increased
20%
Pulmonary embolism
20%
Haematoma
20%
Thrombosis
20%
Neutropenia
20%
Diarrhoea
20%
Gastritis
20%
Nausea
20%
Drug toxicity
20%
Limb injury
20%
Hyperglycaemia
20%
Hyperkalaemia
20%
Hyponatraemia
20%
Atrial fibrillation
20%
Renal failure acute
20%
Vomiting
20%
Catheter related complication
20%
Pyrexia
20%
Catheter site infection
20%
Thrombotic microangiopathy
100%
80%
60%
40%
20%
0%
Study treatment Arm
MEDI-507
Trial Design
1Treatment groups
Experimental Treatment
Group I: Non-Myeloablative Conditioning and Bone Marrow TransplantationExperimental Treatment8 Interventions
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Total body irradiation
2008
Completed Phase 3
~1080
Thymoglobulin
2005
Completed Phase 4
~1500
Fludarabine
2012
Completed Phase 3
~1100
Cyclophosphamide (CTX)
2002
Completed Phase 3
~1600
Mesna
2003
Completed Phase 2
~1380
Sirolimus
2013
Completed Phase 4
~2750
Mycophenolate mofetil (MMF)
2008
Completed Phase 3
~2250
Bone marrow transplantation
2008
Completed Phase 3
~590
Find a Location
Who is running the clinical trial?
Vanderbilt-Ingram Cancer CenterLead Sponsor
213 Previous Clinical Trials
60,907 Total Patients Enrolled
Adetola A Kassim, MDPrincipal InvestigatorVanderbilt-Ingram Cancer Center
1 Previous Clinical Trials
100 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My lung function tests are very low.My donor's blood type is slightly different but compatible.I have had a reaction to a previous blood transfusion.I am not pregnant and use effective birth control methods.I am fully active or able to carry out light work.I have a diagnosed form of sickle cell disease or another significant hemoglobin disorder.I have difficulty performing daily activities without help.My heart's pumping ability is significantly reduced.I still experience severe pain from my condition despite treatment with hydroxyurea or blood transfusions.I have a long-term lung condition that is getting worse over time.My direct bilirubin level is above 3.1 mg/dl.I can make medical decisions for myself or have someone who can.I don't have a fully matched sibling donor, so I can have a half-matched bone marrow transplant.I am between 1 and 70 years old.I have had a stroke or brain damage confirmed by MRI.I have had repeated severe chest pain needing hospital stays.I have moderate to severe kidney disease.I need regular blood transfusions for my thalassemia.
Research Study Groups:
This trial has the following groups:- Group 1: Non-Myeloablative Conditioning and Bone Marrow Transplantation
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 conditions typically necessitate Bone marrow transplantation?
"Bone marrow transplantation is a widely used medical intervention for organ transplants. It also has been demonstrated to be beneficial in cases of immunosuppressive treatment, multiple sclerosis, leukemia, myelocytic and acute disorders."
Answered by AI
Does this clinical experiment include participants over the age of eighty?
"According to the prerequisites, participants must be between 1 Year and 70 years old. There are 513 clinical trials specifically for minors and 872 studies for elderly individuals."
Answered by AI
To what extent has Bone marrow transplantation been explored in clinical research?
"Currently, there are 1,043 clinical studies in progress for Bone marrow transplantation; of these trials, 186 have advanced to Phase 3. These research efforts are largely situated within the confines of Philadelphia, Pennsylvania with a total of 29,950 locations running related experiments."
Answered by AI
Is it possible to become a participant in this clinical research study?
"This trial seeks patients afflicted with a hemoglobinopathy between the ages of 1 and 70. Approximately 21 individuals will be recruited to partake in this study."
Answered by AI
How many subjects are enrolled in this research project?
"Currently, no further enrollment is being considered for this trial. It was first published on May 1st 2013 and last updated on December 20th 2021. However, those looking to join a clinical study might consider one of the 212 trials related to hemoglobinopathies or 1043 Bone marrow transplantation studies presently enrolling patients."
Answered by AI
Is there still an opportunity to enroll in this clinical experiment?
"Unfortunately, this investigation is not enrolling at the moment. It was initially listed on May 1st 2013 and last edited December 20th 2021. If you are hoping to join a study, there currently 212 trials for hemoglobinopathies that are taking partakers and 1043 studies involving bone marrow transplants have open slots."
Answered by AI
Recent research and studies
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger