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Trial record 9 of 17 for:    "severe congenital neutropenia"

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

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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: 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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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.

Reporting Groups
  Description
Alefacept 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).

Participant Flow:   Overall Study
    Alefacept
STARTED   3 
COMPLETED   3 
NOT COMPLETED   0 



  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
Alefacept 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).

Baseline Measures
   Alefacept 
Overall Participants Analyzed 
[Units: Participants]
 3 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      3 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 7  (3.54) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      2  66.7% 
Male      1  33.3% 
Region of Enrollment 
[Units: Participants]
 
United States   3 


  Outcome Measures

1.  Primary:   Feasibility of Alefacept Pre-conditioning, Measured by Number of Subjects With Full Donor Engraftment   [ Time Frame: Two years post-transplant ]

2.  Secondary:   Number of Participants That Expressed Grade 2 or 3 Regimen-Related Toxicity   [ Time Frame: Day 42 post-transplant ]

3.  Secondary:   Number of Participants That Expressed Successful Neutrophil Engraftment   [ Time Frame: Day 100 post-transplant ]

4.  Secondary:   Incidence of Greater Than or Equal to 85% CD3 Donor Chimerism   [ Time Frame: Day 30 post-transplant ]

5.  Secondary:   Incidence of 100% CD33 Donor Chimerism   [ Time Frame: Day 30 post-transplant ]

6.  Secondary:   Number of Participants Who Experienced Acute Graft-versus-host Disease (aGVHD), Measured by NIH Consensus Criteria (NCC) Score: Grade II-IV   [ Time Frame: Day 30 post-transplant ]

7.  Secondary:   Number of Participants Who Experienced Chronic Graft-versus-host Disease (cGVHD), Measured by the NIH Criteria Consensus (NCC)   [ Time Frame: Day 100 post-transplant ]


  Serious Adverse Events


  Other Adverse Events


  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
Production of alefacept was halted in 2011.


  More Information

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


Results Point of Contact:  
Name/Title: Dr. John Horan
Organization: Emory University
phone: 404-785-1272
e-mail: jthoran@emory.edu


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: John Horan, Emory University
ClinicalTrials.gov Identifier: NCT01319851     History of Changes
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