Severe Sickle Cell Disease study: RIC regimen vs GVHD prophylaxis regimen
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?
This is an investigational combination of medicines and a bone marrow transplant being studied for severe sickle cell disease. It is designed to use drugs to lower the body's immune system and clear space in the bone marrow so that new, healthy stem cells from a donor can grow and take over.
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 testing whether a bone marrow transplant from a family member works well. It also checks how safe the treatment is and what side effects it causes.
- Who it's for
- You may be able to join if you are under 21 and have a serious blood or immune disease.
- What's involved
- You would get a bone marrow transplant from a family member. You would also get a lower amount of medicine to prepare your body.
- Time commitment
- The study involves the transplant and later check-ins to see how you are doing.
- 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...
- 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...
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
- 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.
- 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 is enrolling
- New Haven, Connecticut, United States
- Wilmington, Delaware, United States
- Grand Rapids, Michigan, United States
- St Louis, Missouri, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
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, but 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?
This study is enrolling now at 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 enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.