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Chemotherapy

RIC + BMT for Non-Malignant Disorders

Phase 1 & 2
Recruiting
Led By Shalini Shenoy, MD
Research Sponsored by Washington University School of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Serum creatinine </= 1.5x upper limit of normal for age and/or GFR > 70 mL/min/1.73m2
Recurrent acute chest syndrome with significant respiratory compromise each time
Must not have
Patients with active autoimmune disease (e.g. sarcoidosis, lupus, scleroderma)
Patients who have an HLA-identical sibling who is able and willing to donate bone marrow
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years post-transplant
Awards & highlights
No Placebo-Only Group

Summary

This trial will study whether it is safe and effective to give bone marrow transplants to patients with certain non-cancerous diseases who are less than 21 and might benefit from the transplant.

Who is the study for?
This trial is for young people (up to age 20.99) with non-malignant disorders like sickle cell disease, bone marrow failure, or metabolic diseases. They must have specific health criteria met such as normal kidney function and no severe liver issues. Participants need a performance score of at least 50 and agree to contraception methods post-transplant.
What is being tested?
The study tests the effectiveness and safety of familial HLA mismatched bone marrow transplants in patients under 21 with non-cancerous diseases. It includes a reduced intensity conditioning (RIC) regimen before transplant and graft-versus-host disease (GVHD) prevention afterwards.
What are the potential side effects?
Potential side effects may include reactions from the immune system due to mismatched donor cells, complications from conditioning treatments prior to transplant, infection risks, organ toxicity, and graft-versus-host disease despite prophylaxis.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My kidney function is within the normal range.
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I have had multiple severe episodes of acute chest syndrome.
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I frequently visit the hospital for pain crises, use opioids often, and miss a lot of school.
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I need regular blood transfusions due to an immune reaction.
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I have had bone tissue death more than once or in several joints.
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I have severe symptoms of sickle cell disease.
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I need a bone marrow transplant for a non-cancerous condition.
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I have kidney problems due to sickle cell disease.
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My lung function is good, with oxygen levels above 90% without assistance.
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My liver does not have cirrhosis, bridging fibrosis, or active hepatitis, confirmed by a biopsy.
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I agree to use two forms of birth control or practice abstinence for 12 months after my transplant.
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I am 20 years old or younger.
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I have had a stroke or high brain blood flow speeds despite getting blood transfusions.
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My bilirubin levels are less than twice the normal limit.
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My hemoglobin S level is below 30% as required for a treatment I'm starting.
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My liver enzymes are within a safe range.
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I am mostly independent and can do most of my daily activities.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have an active autoimmune disease like lupus.
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I have a sibling who matches my bone marrow and is willing to donate.
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I haven't used any experimental drugs or devices in the last 3 months.
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I am not pregnant or breastfeeding.
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I have severe liver scarring or active hepatitis.
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I have had a stem cell transplant before.
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I have not had a severe infection in the last 6 weeks.

Timeline

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

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Donor engraftment
Secondary study objectives
Effect of BMT on cardiac function
Effect of BMT on hepatic function
Effect of BMT on neurologic function
+9 more

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: RIC Prep Regimen & GVHD ProphylaxisExperimental Treatment2 Interventions
Single arm study. All patients receive the same Reduced Intensity Conditioning (RIC) regimen and GVHD prophylaxis regimen

Find a Location

Who is running the clinical trial?

Washington University School of MedicineLead Sponsor
1,991 Previous Clinical Trials
2,295,831 Total Patients Enrolled
Shalini Shenoy, MDPrincipal InvestigatorWashington University School of Medicine
2 Previous Clinical Trials
241 Total Patients Enrolled

Media Library

RIC regimen (Chemotherapy) Clinical Trial Eligibility Overview. Trial Name: NCT03128996 — Phase 1 & 2
Hemoglobin Disorders Research Study Groups: RIC Prep Regimen & GVHD Prophylaxis
Hemoglobin Disorders Clinical Trial 2023: RIC regimen Highlights & Side Effects. Trial Name: NCT03128996 — Phase 1 & 2
RIC regimen (Chemotherapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03128996 — Phase 1 & 2
~4 spots leftby Apr 2026
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