Actively Recruiting
A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD
Led by University of Regensburg · Updated on 2024-05-13
212
Participants Needed
9
Research Sites
456 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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 years to 35 years
- Diagnosis of homozygous hemoglobin S disease or heterozygous hemoglobin SC or S 0/+
- Signed study-specific consent
- Presence of severe or moderate sickle cell disease complications such as:
- Clinically significant neurological event (stroke) or deficit
- Silent crisis or neurocognitive deficit
- Pathological angio-MRI with TOF Sequence
- Transcranial Doppler velocity over 200 cm/s on two occasions more than 1 month apart
- More than 5 vaso-occlusive crises in the past year or over 20 in lifetime
- Two or more episodes of acute chest syndrome in lifetime or one episode in past 24 months
- Chronic transfusion requirement or more than 8 transfusions or one exchange transfusion in lifetime
- Transfusion-refractory allo-immunization
- More than five sickle cell-related hospitalizations in lifetime
- Beginning pulmonary hypertension
- Osteonecrosis at more than 2 sites
- Beginning sickle cell nephropathy
- Recurrent priapism (more than 2 episodes)
You will not qualify if you...
- Karnofsky or Lansky performance score less than 70%
- Presence of donor-specific antibodies against the potential stem cell donor detected by cell-based crossmatch, flow cytometry crossmatch, or solid-phase immunoassays including modified tests
- Major ABO blood group incompatibility between donor and recipient
- Cardiac function with ejection fraction less than 45% or shortening fraction less than 27% by echocardiogram or radionuclide scan
- Grade II or higher hypertension by Common Toxicity Criteria
- Renal function below defined thresholds for age, including creatinine clearance or GFR below specified limits
- Pulmonary function with DLCO less than 50%, FVC or FEV1 less than 50%, or oxygen saturation under 92% on room air (for children unable to perform tests)
- Liver function with total bilirubin over twice the upper limit of normal (unless Gilbert's Syndrome), ALT or AST over 2.5 times upper limit, or chronic active viral hepatitis
- Pregnancy or breastfeeding; women of childbearing potential must have negative pregnancy test and agree to effective contraception during treatment and for 12 months after
- History of uncontrolled autoimmune disease or ongoing treatment
- Inability to comply with treatment protocol
- Prior hematopoietic stem cell transplant
- Receipt of live virus vaccine during the trial
- HIV infection
- History of psychiatric illness or conditions affecting understanding of study requirements
- Unwillingness or inability to give informed consent
- Concurrent severe or uncontrolled medical conditions such as uncontrolled diabetes, congestive heart failure, recent myocardial infarction, unstable hypertension, chronic renal disease, or active uncontrolled infection
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
K
Katharina Kleinschmidt, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here