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New treatment option for Severe Sickle Cell Disease
Official title Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
ClinicalTrials.gov ID: NCT03128996
What this study is testing
What is RIC regimen?
RIC regimen is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for severe sickle cell disease.
Also referred to as Transplant Preparative Regimen, Transplant Conditioning Regimen.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 21
You may be able to join if
- Nonmalignant disorder requiring bone marrow transplant including bone marrow failure syndromes, metabolic disorders, immunologic disorders, or...
- For patients with sickle cell disease, must have one of the following severe manifestations:
- Overt or silent stroke or persistently elevated transcranial doppler velocities despite transfusion therapy
- Recurrent acute chest syndrome with significant respiratory compromise each time
- Sickle nephropathy
You likely can't join if
- Patients who have an HLA-identical sibling who is able and willing to donate bone marrow
- Patients with cirrhosis or established bridging fibrosis of the liver or active hepatitis
- Uncontrolled bacterial, viral, or fungal infection within 6 weeks prior to enrollment
- Evidence of HIV infection or known HIV positive serology
- Patients who have received a previous stem cell transplant
- Patients who have received an investigational drug or device or off-label use of a drug or device within 3 months of enrollment
See the full eligibility criteria
- Nonmalignant disorder requiring bone marrow transplant including bone marrow failure syndromes, metabolic disorders, immunologic disorders, or hemoglobinopathy
- For patients with sickle cell disease, must have one of the following severe manifestations:
- Overt or silent stroke or persistently elevated transcranial doppler velocities despite transfusion therapy
- Recurrent acute chest syndrome with significant respiratory compromise each time
- Sickle nephropathy
- Recurrent admissions for vaso-occlusive episodes resulting in prolonged opioid use and poor quality of life with interrupted school attendance activity
- Red cell alloimmunization with the need for chronic transfusions
- Recurrent osteonecrosis or multiple joint involvement from avascular necrosis
- Patients with sickle cell disease must have hemoglobin S \< 30% within 30 days prior to beginning alemtuzumab
- Age \</= 20.99 years at the time of enrollment
- Performance score \>/= 50
- Left ventricular ejection fraction \> 40% or left ventricular shortening fraction \> 26% by echocardiogram
- DLCO \> 40% (corrected for hemoglobin) or pulse oximetry with a baseline O2 saturation of \>/= 90% on room air if too young to perform PFTs
- Serum creatinine \ 70 mL/min/1.73m2
- Direct bilirubin \< 2x upper limit of normal for age
- ALT and AST \< 5x upper limit of normal for age
- Participants who have or are receiving \>/= 8 packed red blood cell transfusions for \>/= 1 year or \>/= 20 packed red blood cell transfusions (lifetime cumulative) will undergo liver MRI for estimation of hepatic iron...
- Female people of childbearing potential, must agree to practice 2 methods of contraception at the same time from the time of signing of informed consent through 12 months post transplant. Male people must agree to...
- Written informed consent must be obtained from all recipients in accordance with the guidelines of the institution's Human Studies Committee.
- Patients who have an HLA-identical sibling who is able and willing to donate bone marrow
- Patients with cirrhosis or established bridging fibrosis of the liver or active hepatitis
- Uncontrolled bacterial, viral, or fungal infection within 6 weeks prior to enrollment
- Evidence of HIV infection or known HIV positive serology
- Patients who have received a previous stem cell transplant
- Patients who have received an investigational drug or device or off-label use of a drug or device within 3 months of enrollment
- Females who are pregnant or breast feeding
- Patients with active autoimmune disease (e.g. sarcoidosis, lupus, scleroderma)
The study team makes the final eligibility decision.
Where it's taking place
- New Haven, Connecticut, United States
- Wilmington, Delaware, United States
- Grand Rapids, Michigan, United States
- St Louis, Missouri, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 day to 21 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include New Haven, Connecticut, United States; Wilmington, Delaware, United States; Grand Rapids, Michigan, United States; St Louis, Missouri, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.