NCT01279616

A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease

A Pilot Study of an Immunosuppressive and Myeloablative Preparative Regimen for Allogeneic Unrelated Donor Hematopoietic Stem Cell Transplantation (HSCT) for Severe Sickle Cell Disease

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Nationwide Children's Hospital · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

Summary

Majority of patients who are eligible for allogeneic HSCT for cure of severe sickle cell disease lack a matched family donor. This study aims for cure of sickle cell disease by performing unrelated donor (outside family) allogeneic HSCT. Donors or unrelated cord blood units will be selected from the NMDP database. It is designed to estimate the safety of a novel reduced toxicity, yet an immunosuppressive and myeloablative preparative regimen. This is meant for patients \<21 years old who have severe complications from sickle cell and do not have matched sibling donors in the family to undergo stem cell transplant. Patients will undergo transplant using unrelated donor stem cells after receiving the protocol therapy. They will be followed for 1 year to monitor for engraftment of donor cells and complications like graft versus host disease (GVHD), infections and death.

Detailed description

The primary goal of this pilot study is to determine the safety and feasibility of the preparative regimen for HSCT using a novel reduced toxicity regimen for stem cell transplant with unrelated donors. Analysis will be geared to confirm if the study regimen, followed by an appropriately HLA-matched unrelated donor (MUD)or unrelated cord blood HSCT, can lead to durable donor engraftment with reasonable toxicity, inhibiting sickle erythropoiesis and limiting disease related organ toxicity in patients who are at high risk for morbidity and mortality associated with sickle cell disease (SCD).

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Fludarabine monophosphate	180 mg/m2 over 6 days.
DRUG	Rituximab	375 mg/m2 on day -13 and day -3
DRUG	Busulfan	AUC 1000-1200 microM.mt
DRUG	ATG	2.5 mg/kg for 3 days
DRUG	Cyclophosphamide	50 mg/kg on day +3
DRUG	Mycophenolate mofetil	15 mg/kg q 8 hours
DRUG	Tacrolimus	0.03 mg/kg /d

Timeline

• Start date: 2010-09-01
• Primary completion: 2015-01-01
• Completion: 2015-01-01
• First posted: 2011-01-19
• Last updated: 2019-04-03
• Results posted: 2019-04-03

Locations

1 site across 1 country: United States