Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease
ConditionSickle Cell Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 22
SponsorSt. Jude Children's Research Hospital
About this trial
The purpose of this study it to evaluate a reduced toxicity conditioning regimen for haploidentical donor HCT followed by a GVHD prophylaxis regimen comprising of post-transplant cyclophosphamide, sirolimus and abatacept with the goal to improve the GVHD-free rejection-free survival (GRFS) to greater than 90% after haploidentical donor HCT in children and young adults with SCD.
Primary Objective:
\- To assess the GVHD-free and rejection free survival (GRFS) after haploidentical donor HCT in children and young adults with SCD.
Secondary Objectives:
* Assess the overall survival (OS) and disease-free survival (DFS) after haploidentical donor HCT for SCD. * Estimate incidence and severity of acute and chronic GVHD after haploidentical donor HCT for SCD. * Assess the neutrophil and platelet engraftment kinetics after haploidentical donor HCT for SCD.
Eligibility criteria
Qualifiers
Age less than or equal to 22 years.
Patients without a suitable HLA-matched sibling donor but with a suitable single haplotype matched (≥ 3 of 6) family member donor. Potential donors do not need to undergo eligibility determination prior to the recipients enrolling on the study. As long as a potential donor is identified and willing to donate hematopoietic progenitor cells, recipients can enroll on the study.
History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique (or ≥185 cm/sec by the imaging technique) measured at a minimum of two separate occasions.
History of cerebral infarction on brain MRI (overt stroke, or silent cerebral infarct).
Disqualifiers
Karnofsky or Lansky performance score <60.
Pregnant, as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment (if female).
Breast feeding.
Uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms) within 1 month prior to conditioning. Patients with febrile illness or suspected minor infection should await clinical resolution prior to starting conditioning. Patients with confirmed seropositivity or positive NAAT for HIV are excluded.
Trial design
Treatments tested in this trial
- Cyclophosphamide 50mg
- Abatacept
- Alemtuzumab
- Hydroxyurea
- Thiotepa
- Filgrastim
- Sirolimus
- Azathioprine
- TBI
- Hematopoietic Progenitor Cell Infusion
Treatment groups
45 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov