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Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

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: NCT01319851
Recruitment Status : Terminated (drug company is no longer making the drug)
First Posted : March 22, 2011
Results First Posted : March 9, 2015
Last Update Posted : July 27, 2017
Sponsor:
Collaborator:
Children's Healthcare of Atlanta
Information provided by (Responsible Party):
John Horan, Emory University

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Thalassemia
Sickle Cell Disease
Glanzmann Thrombasthenia
Wiskott-Aldrich Syndrome
Chronic-granulomatous Disease
Severe Congenital Neutropenia
Leukocyte Adhesion Deficiency
Schwachman-Diamond Syndrome
Diamond-Blackfan Anemia
Fanconi Anemia
Dyskeratosis-congenita
Chediak-Higashi Syndrome
Severe Aplastic Anemia
Intervention Drug: Alefacept
Enrollment 3
Recruitment Details Patients were enrolled at the Aflac Cancer and Blood Disorders Center within Children's Healthcare of Atlanta (CHOA) from November 2010 to November 2011.
Pre-assignment Details One subject with Fanconi Anemia (FA) received fludarabine (Flu) 25 mg/m2 on days -10 to -5 and cyclophosphamide (Cy) 10 mg/kg on days -5 to -2. The other two subjects received Flu 25 mg/m2 on days -6 to -1, Cy 50 mg/kg on day -2, and low-dose total body irradiation(TBI; 200 cGy) on day -1.
Arm/Group Title Alefacept
Hide Arm/Group Description Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
Period Title: Overall Study
Started 3
Completed 3
Not Completed 0
Arm/Group Title Alefacept
Hide Arm/Group Description Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
Overall Number of Baseline Participants 3
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
<=18 years
3
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants
7  (3.54)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
Female
2
  66.7%
Male
1
  33.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 3 participants
3
1.Primary Outcome
Title Feasibility of Alefacept Pre-conditioning, Measured by Number of Subjects With Full Donor Engraftment
Hide Description All subjects received alefacept prior to hematopoietic stem cell transplantation and were followed up to at least two years after transplantation to ensure successful engraftment.
Time Frame Two years post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Alefacept
Hide Arm/Group Description:
Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
3
2.Secondary Outcome
Title Number of Participants That Expressed Grade 2 or 3 Regimen-Related Toxicity
Hide Description Regimen-related toxicity was measured using the Bearman criteria. The Bearman criteria grades toxicity levels at Grade 1, Grade 2, Grade 3, and Grade 4. In this system, grade I toxicity is reversible without treatment and grade 2 is not life threatening, but requires treatment. Grade 3 requires life-support intervention and grade 4 is fatal. All regimen-related toxicities were determined to be unlikely attributable to the study drug.
Time Frame Day 42 post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Alefacept
Hide Arm/Group Description:
Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
2
3.Secondary Outcome
Title Number of Participants That Expressed Successful Neutrophil Engraftment
Hide Description Neutrophil engraftment was assessed with absolute neutrophils >500*10^8/kg by 100 days post transplant. Neutrophils were counted by performing a complete blood cell count (CBC).
Time Frame Day 100 post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Alefacept
Hide Arm/Group Description:
Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
3
4.Secondary Outcome
Title Incidence of Greater Than or Equal to 85% CD3 Donor Chimerism
Hide Description CD3 chimerism was measured from peripheral blood lymphocytes 30 days post transplant. DNA chimerism analysis was performed by amplified fragment length polymorphism.
Time Frame Day 30 post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Alefacept
Hide Arm/Group Description:
Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
3
5.Secondary Outcome
Title Incidence of 100% CD33 Donor Chimerism
Hide Description CD33 chimerism was measured from peripheral blood lymphocytes 30 days post transplant. DNA chimerism analysis was performed by amplified fragment length polymorphism.
Time Frame Day 30 post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Alefacept
Hide Arm/Group Description:
Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
3
6.Secondary Outcome
Title Number of Participants Who Experienced Acute Graft-versus-host Disease (aGVHD), Measured by NIH Consensus Criteria (NCC) Score: Grade II-IV
Hide Description Cumulative Incidence of Grade II-IV aGVHD Score at 30 Days. The NIH Consensus grading and severity criteria includes physical assessments of skin, oral cavity, eyes, gynecological and laboratory data and patient reports. Each domain is scored from Grade 0 (no involvement) to Grade IV (severe involvement).
Time Frame Day 30 post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Alefacept
Hide Arm/Group Description:
Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
Grade II 2
Grade III 1
Grade IV 0
7.Secondary Outcome
Title Number of Participants Who Experienced Chronic Graft-versus-host Disease (cGVHD), Measured by the NIH Criteria Consensus (NCC)
Hide Description The severity criteria of chronic graft-versus-host disease (cGVHD) recommended by the NIH Criteria Consensus (NCC) was employed. The number of organs involved and the severity of the disease in these organs dictated the global summary score used to define the disease as mild, moderate, or severe. Mild disease indicates one or two organs involved each with a maximal score of 1. Moderate disease indicates three or more organs involved with a score of 2 in any individual organ, or lung involvement with a score of 1. Severe global GVHD is defined by a score of 3 in any organ, or a lung score of 2.
Time Frame Day 100 post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Alefacept
Hide Arm/Group Description:
Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
Mild cGVHD 0
Moderate cGVHD 1
Severe cGVHD 1
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Alefacept
Hide Arm/Group Description Pediatric subjects with non-malignant diseases (NMD) received pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses).
All-Cause Mortality
Alefacept
Affected / at Risk (%)
Total   --/--    
Hide Serious Adverse Events
Alefacept
Affected / at Risk (%) # Events
Total   2/3 (66.67%)    
Gastrointestinal disorders   
Stomatitis *  2/3 (66.67%)  2
Immune system disorders   
Chronic graft versus host disease *  2/3 (66.67%)  3
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Alefacept
Affected / at Risk (%) # Events
Total   2/3 (66.67%)    
Blood and lymphatic system disorders   
Bacteremia *  1/3 (33.33%)  1
Infections and infestations   
Pulmonary aspergillosis *  1/3 (33.33%)  1
Cytomegalovirus (CMV) Viremia *  1/3 (33.33%)  1
Respiratory, thoracic and mediastinal disorders   
Sinusitis *  1/3 (33.33%)  1
*
Indicates events were collected by non-systematic assessment
Production of alefacept was halted in 2011.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. John Horan
Organization: Emory University
Phone: 404-785-1272
EMail: jthoran@emory.edu
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: John Horan, Emory University
ClinicalTrials.gov Identifier: NCT01319851    
Other Study ID Numbers: IRB00039680
BMT Alefacept ( Other Identifier: Other )
First Submitted: September 15, 2010
First Posted: March 22, 2011
Results First Submitted: February 25, 2015
Results First Posted: March 9, 2015
Last Update Posted: July 27, 2017