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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01279616
Recruitment Status : Terminated (PI moving to a different institution.)
First Posted : January 19, 2011
Results First Posted : April 3, 2019
Last Update Posted : April 3, 2019
Sponsor:
Information provided by (Responsible Party):
Nationwide Children's Hospital

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Sickle Cell Disease
Interventions Drug: Fludarabine monophosphate
Drug: Rituximab
Drug: Busulfan
Drug: ATG
Drug: Cyclophosphamide
Drug: Mycophenolate mofetil
Drug: Tacrolimus
Enrollment 8
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description

Drug: Fludarabine monophosphate 180 mg/m2 over 6 days.

Drug: Rituximab 375 mg/m2 on day -13 and day -3 Other Names: •Rituxan

Drug: Busulfan AUC 1000-1200 microM.mt Other Names: •busulfex

Drug: ATG 2.5 mg/kg for 3 days Other Names: •Thymoglobulin

Drug: Cyclophosphamide 50 mg/kg on day +3 Other Names: •Cytoxan

Drug: Mycophenolate mofetil 15 mg/kg q 8 hours Other Names: •MMF, Cell-cept.

Drug: Tacrolimus 0.03 mg/kg /d Other Names: •FK-506

Period Title: Overall Study
Started 8
Completed 1
Not Completed 7
Reason Not Completed
donor could not be located             5
no signs of sickle cell disease             2
Arm/Group Title Hematopoietic Stem Cell Transplant
Hide Arm/Group Description

Stem cell infusion on Day 0.

Fludarabine monophosphate: 180 mg/m2 over 6 days.

Rituximab: 375 mg/m2 on day -13 and day -3

Busulfan: AUC 1000-1200 microM.mt

ATG: 2.5 mg/kg for 3 days

Cyclophosphamide: 50 mg/kg on day +3

Mycophenolate mofetil: 15 mg/kg q 8 hours

Tacrolimus: 0.03 mg/kg /d

Overall Number of Baseline Participants 0
Hide Baseline Analysis Population Description
No baseline data are available for this study as the study was terminated. The PI has left the institution and cannot be contacted.
Age, Categorical  
Number Analyzed 0 participants
<=18 years
Between 18 and 65 years
>=65 years
Age, Continuous  
Number Analyzed 0 participants
Sex: Female, Male  
Number Analyzed 0 participants
Female
Male
Race and Ethnicity Not Collected   [1] 
Number Analyzed 0 participants
[1]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Region of Enrollment  
Unit of measure:  Participants
United States Number Analyzed 0 participants
Study-Specific Measure  
Number Analyzed 0 participants
1.Primary Outcome
Title Event-free Survival
Hide Description Event-free survival
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
The study was terminated and the PI has left the institution. Efforts were made to contact the PI but unsuccessful. No outcome measure data is available for the study.
Arm/Group Title HSCT Transplant
Hide Arm/Group Description:
HSCT transplant
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame From enrollment of first participation to study termination.
Adverse Event Reporting Description The study was terminated and the PI has left the institution. The only available information is in respect to the serious adverse events.
 
Arm/Group Title No Arm Analyzed: N/A
Hide Arm/Group Description No outcome date are available for this study as the study was terminated. The PI has left the institution and cannot be contacted.
All-Cause Mortality
No Arm Analyzed: N/A
Affected / at Risk (%)
Total   0/0 
Hide Serious Adverse Events
No Arm Analyzed: N/A
Affected / at Risk (%)
Total   0/8 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
No Arm Analyzed: N/A
Affected / at Risk (%)
Total   0/0 
No outcome data are available for this study as the study was terminated. The PI has left the institution and cannot be contacted.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
No Outcome data are available for this study as the study was terminated. The PI has left the institution and cannot be contacted.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Kristy Ott
Organization: Nationwide Children's Hospital
Phone: 614-722-6313
EMail: kristy.ott@nationwidechildrens.org
Layout table for additonal information
Responsible Party: Nationwide Children's Hospital
ClinicalTrials.gov Identifier: NCT01279616    
Other Study ID Numbers: 09-00383
First Submitted: January 18, 2011
First Posted: January 19, 2011
Results First Submitted: February 21, 2019
Results First Posted: April 3, 2019
Last Update Posted: April 3, 2019