A Phase 1 interventional study of peripheral blood stem cell graft that are CD34+ selected in Sickle Cell-thalassemia Disease and Thalassemia, sponsored by Catherine Bollard. Completed at 1 site in United States. Open to participants aged Up to 22 Years. Per ClinicalTrials.gov, last updated 2024-12-19.
Sponsored by Catherine Bollard · Phase 1, Interventional, and Treatment
The study is designed as a Pilot/Phase 1 trial of reduced intensity Haploidentical HSCT in patients with sickle cell disease and thalassemia. The purpose of the study is to assess the safety and toxicity of reduced intensity conditioning haploidentical hematopoietic stem cell transplantation.
Research subjects will undergo reduced intensity conditioning (Hydroxyurea, ATG, Fludarabine, Thiotepa, Melphalan) followed by infusion of a peripheral blood stem cell graft collected from haploidentical family donors that are CD34+ positively selected using the CliniMACS device. Sirolimus will be used for GVHD prophylaxis and given for 9 months post-transplant and then tapered off by one year
The use of the CliniMACS device for CD34 selection will be performed at CNMC through cross-reference of the master file for CliniMACS CD34+ Reagent by Milteyni Biotech (BB-MF 8061).
CliniMACs is an electromechanical device intended to isolate certain cell subsets from mixed cell populations. When used in combination with the CliniMACs CD34 reagent, it is possible to prepare extremely pure populations of CD34+ cells with upwards of 5 logs depletion of contaminating T cells within a closed and sterile system.
We intend to use this system to select cells from HLA haploidentical related donors who have been mobilized with G-CSF prior to stem cell collection. Since previous investigations of this strategy in adult patients have not translated into enhanced long term survival, we intend to limit this protocol to patients under the age of 22 as they have more rapid immune reconstitution.
416 studies on the registry are indexed under Thalassemia; 67 are open to participants now.
This study's enrollment of 7 is below the median of 37 across 277 interventional studies indexed under Thalassemia.
Browse Thalassemia studies →Catherine Bollard is the lead sponsor of 14 studies on the registry; 1 is open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
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Exclusion Criteria:
peripheral blood stem cell graft that are CD34+ selected. All patients will undergo reduced intensity conditioning regimen which followed by infusion of a peripheral blood stem cell graft collected from haploidentical family donors that are CD34+ positively selected using the CliniMACS device and Sirolimus will be used for GVHD prophylaxis and given for 9 months post-transplant and then tapered off by one year (see intervention).
Drug: peripheral blood stem cell graft that are CD34+ selected
The preparatory regimen will consist of Hydroxyurea from Days -50 to -22, Alemtuzumab from days -21 to -19 (test dose Alemtuzumab on day -22), Fludarabine days -8 to -4, Thiotepa Day -4, Melphalan day -3 to -2 (Table 4a). In patients with intolerance to or have received alemtuzumab in the prior 6 months, alemtuzumab will be replaced with rabbit ATG on days -10 through -7, followed by infusion of a peripheral blood stem cell graft collected from haploidentical family donors that are CD34+ positively selected using the CliniMACS device. Sirolimus will be used for GVHD prophylaxis and given for 9 months post-transplant and then tapered off by one year.
Also known as: Reduced intensity conditioning, Sirolimus
Incidence of transplant related adverse outcomes
The primary endpoint of this trial is safety. Transplant related adverse outcomes and non-hematological toxicity will be measured through Day +60 on this objective to include: * Non-hematological severe (Grade IV and V) organ specific toxicity according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0) * Rates of non-engraftment * Severe acute (Grade III-IV) * Veno-occlusive disease of the liver * Idiopathic pneumonia syndrome * Seizures/Posterior reversible encephalopathy syndrome (PRES)
Time frame: 60 days
Overall survival
Overall survival upto 2 years
Time frame: 2 years
Graft failure
Graft failure upto 2 years
Time frame: 2 years
Grades II-IV and III-IV acute GVHD
Grades II-IV and III-IV acute GVHD at day +180
Time frame: 180 days
Chronic GVHD
Chronic GVHD by 1 yea
Time frame: 1 year
Transplant-related mortality
Transplant-related mortality at Day+ 100
Time frame: 100 days
Viral infection rates
Viral infection rates at 6 months: Reactivation of CMV, Adenovirus and EBV detected on peripheral blood monitoring or any visceral disease with documented molecular studies for these viruses within the first six months post transplantation will be recorded
Time frame: 6 months
Lymphocyte reconstitution
Lymphocyte reconstitution upto 1 year post transplant
Time frame: 1 year
This study is completed, as verified in Dec 2024. You cannot join it, but the record below documents what was studied.
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Catherine Bollard