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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.
Must not have
Poor pulmonary function: FEV1 and FVC<40% predicted.
Prior transfusions from donor or recipient if caused alloimmunization vs. donor cells.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights
No Placebo-Only Group

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.
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.
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 below
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I am fully active or able to carry out light work.
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I have a diagnosed form of sickle cell disease or another significant hemoglobin disorder.
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I still experience severe pain from my condition despite treatment with hydroxyurea or blood transfusions.
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I have a long-term lung condition that is getting worse over time.
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I am between 1 and 70 years old.
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I have had a stroke or brain damage confirmed by MRI.
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I have had repeated severe chest pain needing hospital stays.
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I have moderate to severe kidney disease.
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I need regular blood transfusions for my thalassemia.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My lung function tests are very low.
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I have had a reaction to a previous blood transfusion.
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I am not pregnant and use effective birth control methods.
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I have difficulty performing daily activities without help.
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My heart's pumping ability is significantly reduced.
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My direct bilirubin level is above 3.1 mg/dl.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Transplant-related Mortality (TRM)
Secondary study objectives
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 • NCT00801632
100%
Engraftment syndrome
80%
Proteinuria
60%
Thrombocytopenia
60%
Urinary tract infection
40%
Anaemia
40%
Weight increased
40%
Transplant rejection
40%
Clostridium difficile colitis
40%
Alanine aminotransferase increased
40%
Hypophosphataemia
20%
Pyrexia
20%
Atrial fibrillation
20%
Stomatitis
20%
Neutropenia
20%
Vomiting
20%
Limb injury
20%
Thrombotic microangiopathy
20%
Gastroenteritis
20%
Gastritis
20%
Viral infection
20%
Catheter related complication
20%
Post procedural haemorrhage
20%
Diarrhoea
20%
Blood creatinine increased
20%
Nausea
20%
Pulmonary embolism
20%
Haematoma
20%
Thrombosis
20%
Drug toxicity
20%
Hyponatraemia
20%
Catheter site infection
20%
Hyperkalaemia
20%
Renal failure acute
20%
Hyperglycaemia
100%
80%
60%
40%
20%
0%
Study treatment Arm
MEDI-507

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: 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
~2210
Fludarabine
2012
Completed Phase 4
~1860
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
217 Previous Clinical Trials
63,530 Total Patients Enrolled
Adetola A Kassim, MDPrincipal InvestigatorVanderbilt-Ingram Cancer Center
1 Previous Clinical Trials
100 Total Patients Enrolled

Media Library

Bone marrow transplantation Clinical Trial Eligibility Overview. Trial Name: NCT01850108 — N/A
Sickle Cell Disease Research Study Groups: Non-Myeloablative Conditioning and Bone Marrow Transplantation
Sickle Cell Disease Clinical Trial 2023: Bone marrow transplantation Highlights & Side Effects. Trial Name: NCT01850108 — N/A
Bone marrow transplantation 2023 Treatment Timeline for Medical Study. Trial Name: NCT01850108 — N/A
~2 spots leftby Nov 2025
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