RecruitingPhase 2NCT04201210

A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD

A Phase II Stratified Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With Sickle Cell Disease With no Available Sibling Donor


Sponsor

University of Regensburg

Enrollment

212 participants

Start Date

Jun 30, 2021

Study Type

INTERVENTIONAL

Conditions

Summary

HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß/CD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection.


Eligibility

Min Age: 2 YearsMax Age: 35 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a type of stem cell transplant for patients with sickle cell disease (SCD). Sickle cell disease is a genetic blood disorder that causes abnormally shaped red blood cells, leading to pain crises, organ damage, and other serious complications. The transplant uses stem cells from a half-matched (haploidentical) donor and involves removing certain immune cells (T-cells) to reduce the risk of rejection. Stem cell transplantation is currently the only known cure for sickle cell disease, but it requires a well-matched donor. This approach aims to expand donor options for patients who don't have a perfect match, potentially making a cure available to more people. You may be eligible if: - You are between 2 and 35 years old - You have sickle cell disease (SS, SC, or S0/S+ type) - You have had serious SCD-related complications such as stroke, frequent pain crises, chest syndrome episodes, or organ damage - A suitable half-matched donor is available You may NOT be eligible if: - You have poor performance status (Karnofsky/Lansky score below 70%) - You have donor-specific antibodies against your potential donor - You have major ABO blood type incompatibility with your donor - You have significant heart, kidney, lung, or liver problems - You are pregnant or not willing to use contraception - You have HIV infection or active uncontrolled hepatitis - You have had a prior stem cell transplant Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

OTHERTCRα/β+ and CD19+ depleted haploidentical stem cell transplantation

Haploidentical 5+/10 HSCT from a relative, α/β T-depleted

OTHERMatched sibling donor transplantation

10/10 HSCT - matched family donor


Locations(9)

St. Anna Kinderspital

Vienna, Austria

University Hospital Aachen, Children's Hospital

Aachen, Germany

Charité University medicine, Clinic for Hematology, Oncology

Berlin, Germany

University Hospital Duesseldorf, Clinic for Pediatric Oncology, - Hemtaology and Clinical Immunology

Düsseldorf, Germany

University Hospital of Frankfurt, Clinic for Paediatrics and Adolescent Medicine

Frankfurt, Germany

University Hospital Heidelberg, Department of Pediatric Hematology, Oncology and Immunology

Heidelberg, Germany

University Hospital Regensburg, Dept. of Ped. Hematology, Oncology and Stem Cell Transplantation

Regensburg, Germany

University Children's Hospital Tübingen

Tübingen, Germany

University Children's Hospital Würzburg

Würzburg, Germany

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NCT04201210


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