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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years post-transplant

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years post-transplant

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Donor engraftment

Secondary outcome measures

Effect of BMT on cardiac function

Effect of BMT on hepatic function

Effect of BMT on neurologic function

+9 more

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

0 / 17Eligibility criteria met

Find a Location

Who is running the clinical trial?

Washington University School of MedicineLead Sponsor

1,933 Previous Clinical Trials

2,299,696 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

Non-Malignant Disorders Research Study Groups: RIC Prep Regimen & GVHD Prophylaxis

Non-Malignant 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there available openings for those wishing to participate in this research initiative?

"Clinicaltrials.gov has reported that this experiment is in need of new participants as its last update was on February 4th of 2022, after it had been posted to the site back in March 20th 2017."

Answered by AI

What prerequisites must be met for individuals to qualify as participants in this investigation?

"This clinical research is enrolling 29 patients, ranging from the day of birth to 21 years old, who have hemoglobinopathies. Additional qualifying criteria includes: sustained elevated transcranial doppler velocities in spite of transfusion therapy; recurrent bouts of acute chest syndrome with respiatory issues; performance score greater than 50; sickle nephropathy or other nonmalignant disorders necessitating bone marrow transplantation such as metabolic diseases, immunologic illnesses and haemoglobonopathy ; red cell alloimmunization requiring persistent blood transfusions; repeated osteonecrosis or multiple joint incapacitation due to av"

Answered by AI

What is the sample size for this clinical investigation?

"Affirmative. The official clinicaltrials.gov page states that recruitment for the trial, which was initially posted on March 20th 2017, is still ongoing. As of this time, 29 individuals are being sought out from two different medical facilities."

Answered by AI

Is this experiment open to participants of a certain age or higher?

"Qualified applicants for this trial are aged between 1 Day and 21. 413 clinical trials have been established to treat those under 18, while 788 exist for individuals over 65+."

Answered by AI

Recent research and studies

formation/NLM/NIH, NCBI. “Gprobe”. 2017. Web. <https://github.com/ncbi/gprobe>..Journal

Gprobe

Information/NLM/NIH, NCBI. 2017. “Gprobe”. GitHub repository. https://github.com/ncbi/gprobe.

clinicaltrials.govStudy

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

2017. "Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03128996.

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