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Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

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ClinicalTrials.gov Identifier: NCT00004143
Recruitment Status : Completed
First Posted : January 27, 2003
Results First Posted : November 11, 2013
Last Update Posted : December 5, 2014
Sponsor:
Information provided by (Responsible Party):
David Rizzieri, MD, Duke University

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Sickle Cell Anemia
Severe Aplastic Anemia
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Pure Red Cell Aplasia
Intervention Drug: Campath, Chemo and/or TBI Allo SCT
Enrollment 2
Recruitment Details The protocol was reopened in 2003 for non-malignant conditions only. Enrollment closed in 2008 due to lack of enrollment.
Pre-assignment Details  
Arm/Group Title All Patients
Hide Arm/Group Description [Not Specified]
Period Title: Overall Study
Started 2
Completed 2
Not Completed 0
Arm/Group Title All Patients
Hide Arm/Group Description [Not Specified]
Overall Number of Baseline Participants 2
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants
<=18 years
0
   0.0%
Between 18 and 65 years
2
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants
Female
0
   0.0%
Male
2
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 2 participants
2
1.Primary Outcome
Title Number of Patients With Neutrophil Engraftment
Hide Description Number of patients with neutrophil engraftment: Absolute Neutrophil Count (ANC) > 500/μL and hemoglobin level remaining above 10 g/dL without transfusion support, with tests showing at least 2.5% donor cells present. Primary graft failure is defined as absence of establishment of adequate donor hematopoiesis by day 42 with bone marrow cellularity < 5%, peripheral White Blood Count (WBC) < 500/μL, peripheral ANC < 100/μL, and/or platelets < 10,000/μL by day 120 with absence of megakaryocytes in the bone marrow (in the absence of disease relapse).
Time Frame 1 year post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title All Patients
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: participants
2
2.Primary Outcome
Title Number of Patients With Platelet Engraftment
Hide Description Number of patients with platelet engraftment - Platelets > 20,000/μL and hemoglobin level remaining above 10 g/dL without transfusion support, with tests showing at least 2.5% donor cells present. Primary graft failure is defined as absence of establishment of adequate donor hematopoiesis by day 42 with bone marrow cellularity < 5%, peripheral White Blood Count (WBC) < 500/μL, peripheral ANC < 100/μL, and/or platelets < 10,000/μL by day 120 with absence of megakaryocytes in the bone marrow (in the absence of disease relapse).
Time Frame 1 year post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title All Patients
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: participants
2
3.Primary Outcome
Title Number of Patients With Grade 3-4 Acute Graft Versus Host Disease (GVHD)
Hide Description Number of patients with Grade 3-4 acute Graft Versus Host Disease (GVHD). GVHD will be monitored at least two times per week through day 45, then weekly through day 60 and graded by 2 persons at each institution, to ensure internal consistency in grading.
Time Frame 60 days post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title All Patients
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: participants
2
4.Primary Outcome
Title Number of Participants With Grade 3-4 Unexpected Adverse Events
Hide Description An unexpected adverse event is one that differs in the nature, severity, or frequency from (a) the research procedures that are described in the protocol-related documents, (such as the IRB-approved research protocol and informed consent document) as expected, and/or (b) the characteristics of the subject population being studied.
Time Frame 45 days post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title All Patients
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: participants
0
5.Primary Outcome
Title Number of Participants With Transplant-related Mortality
Hide Description Number of patients who died due to transplant-related complications
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title All Patients
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: participants
0
6.Secondary Outcome
Title Overall Survival
Hide Description Number of patients alive 2 years after transplant
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title All Patients
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: participants
2
Time Frame 45 days post transplant
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title All Patients
Hide Arm/Group Description [Not Specified]
All-Cause Mortality
All Patients
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
All Patients
Affected / at Risk (%) # Events
Total   0/2 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
All Patients
Affected / at Risk (%) # Events
Total   2/2 (100.00%)    
Cardiac disorders   
Hypertension * 1  1/2 (50.00%)  1
Cardiac ischemia / infarction * 1  1/2 (50.00%)  1
Infections and infestations   
Parainfluenza * 1  1/2 (50.00%)  1
Nervous system disorders   
Headache * 1  1/2 (50.00%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (2.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Mitchell Horwitz, MD
Organization: Duke University Medical Center
Phone: 919-668-1045
EMail: Mitchell.Horwitz@duke.edu
Layout table for additonal information
Responsible Party: David Rizzieri, MD, Duke University
ClinicalTrials.gov Identifier: NCT00004143     History of Changes
Other Study ID Numbers: Pro00008771
DUMC-1340-99-7 ( Other Identifier: Duke University Medical Center Institutional Review Board )
NCI-G99-1617 ( Other Grant/Funding Number: National Cancer Institute )
CDR0000067374
First Submitted: December 10, 1999
First Posted: January 27, 2003
Results First Submitted: September 6, 2013
Results First Posted: November 11, 2013
Last Update Posted: December 5, 2014