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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT01350232
Other study ID # 09F.327
Secondary ID 1-RC2HL101496-0
Status Terminated
Phase N/A
First received May 4, 2011
Last updated November 13, 2014
Start date September 2009
Est. completion date August 2011

Study information

Verified date November 2014
Source Thomas Jefferson University
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug AdministrationUnited States: Institutional Review Board
Study type Interventional

Clinical Trial Summary

This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor. It is believed this regimen is sufficiently efficacious and sufficiently gentle to apply to patients with sickle cell anemia and related disorders. It is proposed to characterize the efficacy and toxicity of this regimen in high risk patients with sickle cell anemia using criteria for patient selection that have been accepted in prior BMT trials in patients with sickle cell disease, specifically only the subset of patients whose prior clinical behavior indicates that they are at high risk for serious morbidity and early mortality. In addition, it is proposed to characterize the pathophysiology of a consistent febrile response seen in the haploidentical BMT regimen the investigators have developed at Thomas Jefferson University (TJU).

The primary goal of this study is to determine the response rate to a reduced intensity conditioning regimen which consists of fludarabine, cytarabine, low dose total body irradiation and cyclophosphamide in patients with severe sickle cell anemia.


Description:

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Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Intervention

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Locations

Country Name City State
United States Thomas Jefferson University Philadelphia Pennsylvania

Sponsors (2)

Lead Sponsor Collaborator
Thomas Jefferson University National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Stable Engraftment To determine if the reduced intensity preparative regimen of fludarabine, cytarabine, cyclophosphamide and low-dose total body irradiation will generate stable engraftment with donor hematopoietic stem cells in at least 80% of patients with severe sickle cell anemia. 180 days post-infusion No
Secondary Organ Toxicity To assess organ toxicity related to fludarabine, cytarabine, cyclophosphamide and low-dose total body irradiation in a population with severe sickle cell anemia. 30 days post infusion Yes
Secondary Overall Survival To determine the overall survival at 6 months post-transplant in patients receiving a matched or partially-matched related donor transplant after reduced-intensity conditioning. 6 months post infusion Yes
Secondary Acute Graft Versus Host Disease To describe the incidence and severity of acute and chronic GVHD following this reduced intensity transplant from partially matched related donors using a combination of cyclophosphamide, tacrolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis. 100 days post infusion Yes
Secondary Correction of Hemoglobinopathy To evaluate the extent of correction of hemoglobinopathy following this reduced intensity transplant. 100 days post infusion through 5 years post infusion No
Secondary Immune Recovery To assess the pace of lymphoid recovery and associated risk for opportunistic infections and relapse (return to recipient erythropoiesis) in this patient population. 100 days post infusion through 5 years post infusion Yes
Secondary Quality of Life To describe the quality of life and functional status following transplantation. Through 5 years post infusion No
Secondary Cytokine Profile To characterize the profiles of cytokines released following administration of the lymphoid portion of the transplant (donor lymphocyte infusion [DLI]). Through 5 years after infusion No
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