Paediatric Diseases Working Party (PDWP)
Type d'étude:
Numéro de l'étude:
Eudra-CT 2018-002652-33
Type de traitement à base de cellules souches:
Allogeneic - Haploidentical
Maladies:
Other non-malignant disorders
Titre court:
T-Haplo-HSCT for SCD
Objectif principal:
Primary efficacy endpoint: Composite Endpoint: Event free survival (EFS). Event is defined as incidence of acute GvHD (Grade II – IV), chronic GvHD (moderate/severe), graft failure (GF), or death (from any reason).
Key secondary endpoint(s): (i) Overall survival (OS); (ii) Disease-free survival (DFS); (iii) Graft failure (GF); (iv) immune-reconstitution; (v) Quality of life (QOL); (vi) Fertility
Key secondary endpoint(s): (i) Overall survival (OS); (ii) Disease-free survival (DFS); (iii) Graft failure (GF); (iv) immune-reconstitution; (v) Quality of life (QOL); (vi) Fertility
Principaux critères d'inclusion:
• Age 1yr to 35yrs
• Homozygous hemoglobin S disease or heterozygous hemoglobin SC or S 0/+
• Preexisting severe or moderate SCD related complications:
o Clinically significant neurological event (stroke) or deficit >24h o Silent crisis,
neurocognitive deficit, pathological Angio-MRI with TOF Sequence
o A TCD velocity >200cm/s at 2 occasions >1 month apart
o More than five vaso-occlusive crises (VOC) in the past 1 year
o Two or more episodes of acute chest syndrome (ACS) in their lifetime or one episode of
ACS in the past 24 months
o Receiving chronic, monthly blood transfusions
o Transfusion-refractory allo-immunization
o More than five VOC in a lifetime
o More than five SCD-related hospitalizations in a lifetime
o More than eight transfusions or one exchange transfusion
o Beginning pulmonary hypertension
o Osteonecrosis at more than 2 sites
o Beginning SCD Nephropathy
• Homozygous hemoglobin S disease or heterozygous hemoglobin SC or S 0/+
• Preexisting severe or moderate SCD related complications:
o Clinically significant neurological event (stroke) or deficit >24h o Silent crisis,
neurocognitive deficit, pathological Angio-MRI with TOF Sequence
o A TCD velocity >200cm/s at 2 occasions >1 month apart
o More than five vaso-occlusive crises (VOC) in the past 1 year
o Two or more episodes of acute chest syndrome (ACS) in their lifetime or one episode of
ACS in the past 24 months
o Receiving chronic, monthly blood transfusions
o Transfusion-refractory allo-immunization
o More than five VOC in a lifetime
o More than five SCD-related hospitalizations in a lifetime
o More than eight transfusions or one exchange transfusion
o Beginning pulmonary hypertension
o Osteonecrosis at more than 2 sites
o Beginning SCD Nephropathy
Pays:
Allemagne
Investigateur principal:
Selim Corbacioglu
Coordinateur EBMT de l'étude:
Arnaud Dalissier
E-mail du coordinateur de l'étude:
arnaud.dalissier@upmc.fr