NCT03128996 Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
| NCT ID | NCT03128996 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Washington University School of Medicine |
| Condition | Severe Sickle Cell Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 29 participants |
| Start Date | 2017-03-20 |
| Primary Completion | 2028-04 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 29 participants in total. It began in 2017-03-20 with a primary completion date of 2028-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion 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: 1. Overt or silent stroke or persistently elevated transcranial doppler velocities despite transfusion therapy 2. Recurrent acute chest syndrome with significant respiratory compromise each time 3. Sickle nephropathy 4. Recurrent admissions for vaso-occlusive episodes resulting in prolonged opioid use and poor quality of life with interrupted school attendance activity 5. Red cell alloimmunization with the need for chronic transfusions 6. 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 \</= 1.5x upper limit of normal for age and/or GFR \> 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 content. 1\. Liver biopsy is indicated for hepatic iron content \>/= 7mg Fe/mg liver dry weight by liver MRI. Histologic examination of the liver must document for the absence of cirrhosis, bridging fibrosis, and active hepatitis * Female subjects 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 subjects must agree to practice effective barrier contraception or practice true abstinence from the time of signing 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. Exclusion Criteria: * 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)
Contact & Investigator
Shalini Shenoy, MD
PRINCIPAL INVESTIGATOR
Washington University School of Medicine
Frequently Asked Questions
Who can join the NCT03128996 clinical trial?
This trial is open to participants of all sexes, aged 1 Day or older, up to 21 Years, studying Severe Sickle Cell Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT03128996 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT03128996 currently recruiting?
Yes, NCT03128996 is actively recruiting participants. Contact the research team at shalinishenoy@wustl.edu for enrollment information.
Where is the NCT03128996 trial being conducted?
This trial is being conducted at New Haven, United States, Wilmington, United States, Grand Rapids, United States, St Louis, United States.
Who is sponsoring the NCT03128996 clinical trial?
NCT03128996 is sponsored by Washington University School of Medicine. The principal investigator is Shalini Shenoy, MD at Washington University School of Medicine. The trial plans to enroll 29 participants.
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