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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

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

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

Primary outcome measures

dichotomous positive/negative outcome where a positive response is defined by absence of graft rejection

Secondary outcome measures

Incidence of graft rejection defined as HbS >10% when donors have HbAA and HbS >50% when donors have sickle cell trait

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

0 / 2Eligibility criteria met

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

3,836 Previous Clinical Trials

47,851,016 Total Patients Enrolled

Courtney F Joseph, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)

3 Previous Clinical Trials

327 Total Patients Enrolled

Courtney D Fitzhugh, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)

5 Previous Clinical Trials

349 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

Frequently Asked Questions

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

How many total participants are anticipated for this research project?

"That is correct. The information available on clinicaltrials.gov suggests that the trial is still looking for patients. The posting date was October 24th, 2019 and the last update was July 20th, 2020. They are hoping to find 30 individuals willing to participate at a single location."

Answered by AI

Do the elderly qualify for participation in this experiment?

"This clinical trial is currently recruiting patients that fall in the age range of 2 to 80 years old. Out of the 218 total studies, 133 are for individuals below 18 and 85 are for those over 65."

Answered by AI

Could you tell me if this clinical trial is still enrolling participants?

"This medical trial is still open to new participants, according to the latest information on clinicaltrials.gov. The study was first posted on October 24th, 2019, and was last updated on July 20th, 2022."

Answered by AI

Would I be able to join this research project if I wanted to?

"This study is looking for 30 myeloid chimerism participants that meet the following requirements: they must be between 2 and 80 years old, have a related donor willing to go through clinical donation evaluations, age 2 years <80 years old ,and have a DLCO greater than or equal to 35%."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

2019. "Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04008368.

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