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Stem Cell Transplantation

Stem Cell Transplant for Sickle Cell Disease

N/A
Recruiting
Led By Michele Nassin, MD
Research Sponsored by University of Chicago
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Between the ages of 2 and 25 years (Stage 1: 10-25 years; Stage II: 2-25 years)
Partially-matched family member with hemoglobin AA (normal) or hemoglobin AS (sickle trait) phenotype
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year post transplant
Awards & highlights

Study Summary

This trial will test whether a stem cell transplant can be a safe and effective treatment for sickle cell disease.

Who is the study for?
This trial is for children and young adults aged 2-25 with severe forms of sickle cell disease who lack a fully matched stem cell donor. Eligible participants may have had strokes, frequent pain episodes, or acute chest syndrome despite treatment. They must not be pregnant, lactating, or have certain organ dysfunctions like severe heart or kidney problems.Check my eligibility
What is being tested?
The study tests a new way to perform stem cell transplants in patients with severe sickle cell disease using αβ+ T-cell depletion from partially-matched family donors. It aims to establish if this method can safely cure the condition without requiring a perfect donor match.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include typical transplant-related complications such as immune reactions against the host's body (graft-versus-host disease), infections due to weakened immunity post-transplant, and other organ-specific issues.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 2 and 25 years old.
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I have a family member who is a partial match and has normal or sickle trait blood.
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I have a specific type of sickle cell disease.
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I have severe sickle cell disease with complications like stroke, acute chest syndrome, or organ damage.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year post transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year 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
Safety, as measured by incidence of graft failure, grade III/IV irreversible end organ toxicity, grade III/IV aGvHD, or death within 100 days post-Hap-HSCT
Secondary outcome measures
Estimate 1-year overall and event-free survival after Hap-HSCT
Observe incidence of severe acute GvHD as defined by grades III through IV
Observe incidence of severe chronic GvHD as defined by grades III and IV
+2 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Stage IIExperimental Treatment1 Intervention
Stage II will include eligible subjects between the ages of 2-25 years.
Group II: Stage IExperimental Treatment1 Intervention
Stage I will include eligible subjects between the ages of 10-25 years.

Find a Location

Who is running the clinical trial?

University of ChicagoLead Sponsor
997 Previous Clinical Trials
817,660 Total Patients Enrolled
Michele Nassin, MDPrincipal InvestigatorUniversity of Chicago
John Cunningham, MDPrincipal InvestigatorUniversity of Chicago
1 Previous Clinical Trials
126 Total Patients Enrolled

Media Library

Haploidentical Hematopoietic Stem Cell Transplantation (Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT04207320 — N/A
Sickle Cell Disease Research Study Groups: Stage I, Stage II
Sickle Cell Disease Clinical Trial 2023: Haploidentical Hematopoietic Stem Cell Transplantation Highlights & Side Effects. Trial Name: NCT04207320 — N/A
Haploidentical Hematopoietic Stem Cell Transplantation (Stem Cell Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04207320 — N/A

Frequently Asked Questions

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

How many participants have been enrolled in this clinical research?

"Absolutely correct. The clinicaltrials.gov website confirms that recruitment for this study is still ongoing, having been first posted on October 1st 2022 and recently updated on May 13th 2022. 38 participants are needed from a single trial site."

Answered by AI

What qualifications must one fulfill to participate in this research effort?

"This study is seeking 38 participants who are between two and 25 years old and have anemia, sickle cell. Eligible patients must meet the following criteria: Hemoglobin SS, SC, S-β0 Thalassemia or SO-Arab Sickle Cell Disease; Age within Stage 1 (10 to 25) or Stage II (2 to 25); Lack of a fully matched family donor in National Marrow Donor Program register; Partially-matched family member with hemoglobin AA/AS phenotype; Severe Phenotype as evidenced by Neurologic manifestations of sickle disease including CVA/TIA or abnormal MRI findings suggestive of"

Answered by AI

Does this research project accept participants aged 18 and above?

"Following the rules for participation, participants must range in age from 2 to 25 years old."

Answered by AI

Are there vacancies in this medical trial for volunteers?

"Per the records found on clinicaltrials.gov, this study is actively looking to recruit participants. The trial was inaugurated October 1st 2022 and has since been amended May 13th of the same year."

Answered by AI
~18 spots leftby Nov 2027