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Stem Cell Transplantation

Stem Cell Transplant for Sickle Cell Anemia

Phase 1 & 2
Recruiting
Led By Courtney F Joseph, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patient with history of SCD or beta thalassemia who previously underwent allogeneic hematopoietic stem cell transplantation (HSCT)
Age greater than or equal to 4 years
Must not have
History of secondary malignancies (other than localized skin cancer)
Major anticipated illness or organ failure incompatible with survival from PBSC transplant
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a second treatment for people with sickle cell disease who had a transplant but the disease returned.

Who is the study for?
This trial is for people aged 4 and older with sickle cell disease who had a previous stem cell transplant but the disease came back. They need some remaining donor cells in their body, good heart and lung function, and can't be pregnant or breastfeeding. Donors must be relatives, at least 2 years old, without serious health issues.
What is being tested?
The study tests a second peripheral blood stem cell transplantation using CliniMACS CD34 Reagent to treat sickle cell disease when it returns after an initial transplant. Participants will undergo extensive screening, receive medications to accept new donor cells, and have follow-up visits for up to five years.
What are the potential side effects?
Potential side effects may include reactions from the long IV line placement, complications from drugs that help the body accept donor cells (like immune system suppression), risks associated with bone marrow expansion procedures, and general risks of transfusions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have had a stem cell transplant for SCD or beta thalassemia.
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I am at least 4 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had cancer before, but it wasn't just skin cancer.
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I do not have any major illnesses or organ failures that would make a stem cell transplant impossible.
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I am mostly bedridden or am a child with significantly reduced activity.
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My body has strong immune responses against a transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
dichotomous positive/negative outcome where a positive response is defined by absence of graft rejection
Secondary study objectives
Incidence of graft rejection defined as HbS >10% when donors have HbAA and HbS >50% when donors have sickle cell trait

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: 2Experimental Treatment1 Intervention
patients with haploidentical donors
Group II: 1Experimental Treatment1 Intervention
patients with HLA-matched sibling donors

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,944 Previous Clinical Trials
47,799,800 Total Patients Enrolled
Courtney F Joseph, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
3 Previous Clinical Trials
329 Total Patients Enrolled
Courtney D Fitzhugh, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
5 Previous Clinical Trials
351 Total Patients Enrolled
Emily M Limerick, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
1 Previous Clinical Trials
70 Total Patients Enrolled

Media Library

Repeat Peripheral Blood Stem Cell Transplantation (Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT04008368 — Phase 1 & 2
Myeloid Chimerism Research Study Groups: 2, 1
Myeloid Chimerism Clinical Trial 2023: Repeat Peripheral Blood Stem Cell Transplantation Highlights & Side Effects. Trial Name: NCT04008368 — Phase 1 & 2
Repeat Peripheral Blood Stem Cell Transplantation (Stem Cell Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04008368 — Phase 1 & 2
~16 spots leftby Jan 2030
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