Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Treatment of Sickle Cell Anemia With Stem Cell Transplant

This study has been terminated.
(Poor accrual)
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Thomas Jefferson University ( Sidney Kimmel Cancer Center at Thomas Jefferson University )
ClinicalTrials.gov Identifier:
NCT01350232
First received: May 4, 2011
Last updated: October 19, 2016
Last verified: October 2016
Results First Received: November 13, 2014  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Sickle Cell Anemia
Sickle Cell-hemoglobin C Disease
Sickle Cell-β0-thalassemia
Interventions: Drug: Fludarabine
Drug: Cytarabine
Device: Cellular Infusions
Radiation: Total Body Irradiation
Drug: Cyclophosphamide
Drug: Bortezomib
Drug: Rituximab
Procedure: Plasmapheresis

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
HSCT

Subjects receive the preparative regimen in 2 steps. The "first step" will be with fludarabine and cytarabine and a low dose of total body irradiation. This will be followed by the "first step" of the transplant graft - the donor lymphocytes. The "second step" of the chemotherapy will be two doses of cyclophosphamide. This will then be followed by the "second step" of the transplant graft - the stem cells.

Only subjects with prior alloimmunization against donor will receive desensitization. Subjects who demonstrate alloimmunization against the HLA of the donor will receive bortezomib and rituximab in combination with plasmapheresis prior to the admission for transplant.

Fludarabine: Subjects will receive fludarabine at a dose of 30 mg/m2 daily for 4 days as part of the preparative regimen

Cytarabine: Subjects will receive cytarabine at a dose of 2 g/m2 daily for 4 days, approximately 4 hours after the fludarabine

Cellular Infusions: Subjects will receive the cellular


Participant Flow:   Overall Study
    HSCT
STARTED   2 
COMPLETED   2 
NOT COMPLETED   0 



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
HSCT

Subjects receive the preparative regimen in 2 steps. The "first step" will be with fludarabine and cytarabine and a low dose of total body irradiation. This will be followed by the "first step" of the transplant graft - the donor lymphocytes. The "second step" of the chemotherapy will be two doses of cyclophosphamide. This will then be followed by the "second step" of the transplant graft - the stem cells.

Only subjects with prior alloimmunization against donor will receive desensitization. Subjects who demonstrate alloimmunization against the HLA of the donor will receive bortezomib and rituximab in combination with plasmapheresis prior to the admission for transplant.

Fludarabine: Subjects will receive fludarabine at a dose of 30 mg/m2 daily for 4 days as part of the preparative regimen

Cytarabine: Subjects will receive cytarabine at a dose of 2 g/m2 daily for 4 days, approximately 4 hours after the fludarabine

Cellular Infusions: Subjects will receive the cellular


Baseline Measures
   HSCT 
Overall Participants Analyzed 
[Units: Participants]
 2 
Age 
[Units: Years]
Mean (Standard Deviation)
 32.3  (3.5) 
Age 
[Units: Participants]
 
<=18 years   0 
Between 18 and 65 years   2 
>=65 years   0 
Gender 
[Units: Participants]
 
Female   2 
Male   0 
Race (NIH/OMB) 
[Units: Participants]
 
American Indian or Alaska Native   0 
Asian   0 
Native Hawaiian or Other Pacific Islander   0 
Black or African American   1 
White   1 
More than one race   0 
Unknown or Not Reported   0 
Ethnicity (NIH/OMB) 
[Units: Participants]
 
Hispanic or Latino   0 
Not Hispanic or Latino   1 
Unknown or Not Reported   1 
Region of Enrollment 
[Units: Participants]
 
United States   2 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Stable Engraftment   [ Time Frame: 180 days post-infusion ]

2.  Secondary:   Organ Toxicity   [ Time Frame: 30 days post infusion ]

3.  Secondary:   Overall Survival   [ Time Frame: 6 months post infusion ]

4.  Secondary:   Acute Graft Versus Host Disease   [ Time Frame: 100 days post infusion ]

5.  Secondary:   Correction of Hemoglobinopathy   [ Time Frame: 100 days post infusion through 5 years post infusion ]

6.  Secondary:   Immune Recovery   [ Time Frame: 100 days post infusion through 5 years post infusion ]

7.  Secondary:   Quality of Life   [ Time Frame: Through 5 years post infusion ]

8.  Secondary:   Cytokine Profile   [ Time Frame: Through 5 years after infusion ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Study was terminated due to poor accrual. No reportable data has been collected.


  More Information
  Hide More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Joanne Filicko-O'Hara, MD
Organization: Thomas Jefferson University
phone: 215-955-8874
e-mail: jef001@jefferson.edu



Responsible Party: Thomas Jefferson University ( Sidney Kimmel Cancer Center at Thomas Jefferson University )
ClinicalTrials.gov Identifier: NCT01350232     History of Changes
Other Study ID Numbers: 09F.327
1-RC2HL101496-0 ( Other Grant/Funding Number: National Heart, Lung, and Blood Institute )
Study First Received: May 4, 2011
Results First Received: November 13, 2014
Last Updated: October 19, 2016
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board