Actively Recruiting
A Phase II Stratified Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With Sickle Cell Disease Without a Matched Sibling Donor
Led by University of Regensburg · Updated on 2024-05-13
212
Participants Needed
9
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating whether a haploidentical alpha/beta and CD19+ T-cell depleted hematopoietic stem cell transplantation (HSCT) can be a safe and effective alternative to matched sibling donor (MSD) transplantation for patients with sickle cell disease (SCD) who lack a suitable sibling donor. This Phase II trial focuses on improving disease-free survival while minimizing adverse events, graft rejection, and graft-versus-host disease (GvHD). The study aims to offer a curative option for a wider group of patients beyond those with MSDs, addressing current challenges in non-malignant disease treatment. The trial compares two approaches: the experimental arm involves haploidentical 5+/10 HSCT from a relative with alpha/beta T-cell depletion, and the control arm uses 10/10 matched sibling donor transplantation. It evaluates the safety and efficacy of the less aggressive conditioning regimen combined with TCR alpha/beta and CD19+ depleted grafts to reduce severe GvHD and graft failure. Both groups undergo transplantation procedures aligned with their donor type, with the experimental approach aiming to speed immune system recovery and reduce transplantation-related mortality. Participants are children and young adults aged 2 to 35 years with severe or moderate SCD complications. The study tracks event-free survival up to 180 days post-transplant and follows participants for up to two years to assess overall survival, disease-free survival, graft failure, and quality of life using standardized questionnaires. Researchers monitor immune recovery, fertility, and adverse events throughout, ensuring comprehensive safety and efficacy evaluations during and after transplantation.
CONDITIONS
Brief Title
A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 2 to 35 years
- Diagnosis of homozygous hemoglobin S disease or heterozygous hemoglobin SC or S 0/+
- Signed study-specific consent
- Presence of moderate to severe sickle cell disease complications such as stroke, silent crisis, neurocognitive deficit, abnormal brain MRI, high transcranial Doppler velocity, frequent vaso-occlusive crises, acute chest syndrome episodes, chronic transfusion needs, transfusion-refractory allo-immunization, multiple hospitalizations, early pulmonary hypertension, osteonecrosis in multiple sites, nephropathy, or recurrent priapism
You will not qualify if you...
- Karnofsky or Lansky Performance Score below 70%
- Presence of donor-specific antibodies against the stem cell donor detected by immune assays
- Major AB0 blood group incompatibility with donor
- Poor cardiac function including low ejection fraction or shortening fraction, or high-grade hypertension
- Reduced kidney function below specified thresholds for age
- Pulmonary function below set limits or low oxygen saturation
- Elevated liver enzymes or chronic active viral hepatitis
- Pregnancy or breastfeeding, or unwillingness to use effective birth control
- History of uncontrolled autoimmune disease or active treatment
- Prior hematopoietic stem cell transplant
- Vaccination with live virus vaccine during trial
- HIV infection
- Psychiatric illness affecting study understanding
- Severe uncontrolled medical diseases that may compromise participation
- Inability or unwillingness to comply with the study protocol or give informed consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Day 0 to Day 180
Participants receive either a haploidentical T-depleted stem cell transplantation or a matched sibling donor transplantation as a treatment for sickle cell disease.
Regular visits during transplantation and recovery period
Duration - Up to 2 years after transplantation
Participants are monitored for overall survival, disease-free survival, graft failure, and quality of life up to 2 years after transplantation.
Periodic follow-up visits over 2 years
Trial Site Locations
Total: 9 locations
1
St. Anna Kinderspital
Vienna, Austria
Not Yet Recruiting
2
University Hospital Aachen, Children's Hospital
Aachen, Germany
Not Yet Recruiting
3
Charité University medicine, Clinic for Hematology, Oncology
Berlin, Germany
Not Yet Recruiting
4
University Hospital Duesseldorf, Clinic for Pediatric Oncology, - Hemtaology and Clinical Immunology
Düsseldorf, Germany, 40225
Not Yet Recruiting
5
University Hospital of Frankfurt, Clinic for Paediatrics and Adolescent Medicine
Frankfurt, Germany
Not Yet Recruiting
6
University Hospital Heidelberg, Department of Pediatric Hematology, Oncology and Immunology
Heidelberg, Germany, 69120
Actively Recruiting
7
University Hospital Regensburg, Dept. of Ped. Hematology, Oncology and Stem Cell Transplantation
Regensburg, Germany, 93053
Actively Recruiting
8
University Children's Hospital Tübingen
Tübingen, Germany, 72076
Actively Recruiting
9
University Children's Hospital Würzburg
Würzburg, Germany, 97080 Würzburg
Actively Recruiting
Research Team
S
Selim Corbacioglu, MD
K
Katharina Kleinschmidt, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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