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Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

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ClinicalTrials.gov Identifier: NCT00301834
Recruitment Status : Completed
First Posted : March 13, 2006
Results First Posted : May 16, 2013
Last Update Posted : September 28, 2017
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
University of California, San Francisco

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Congenital Amegakaryocytic Thrombocytopenia
Diamond-blackfan Anemia
Leukemia
Myelodysplastic Syndromes
Severe Congenital Neutropenia
Interventions: Biological: alemtuzumab
Drug: busulfan
Drug: cyclosporine
Drug: fludarabine phosphate
Drug: methotrexate
Drug: methylprednisolone
Procedure: allogeneic bone marrow transplantation
Procedure: allogeneic hematopoietic stem cell transplantation
Procedure: peripheral blood stem cell transplantation
Procedure: umbilical cord blood transplantation

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Subjects recruited from 9/2005 through 9/2010. Patients identified by the Pediatric BMT (Bone Marrow Transplant) Program and consented by study investigators as outpatients in the BMT clinic.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Patients were ineligible if a cord blood was being used for the transplant or if they met any of the other ineligibility criteria in the protocol or did not meet eligibility criteria such as having Fanconi's Anemia.

Reporting Groups
  Description
Single Arm - Transplant Pre-conditioning Per Study Protocol All eligible patients received pre-transplant conditioning with Alemtuzumab, fludarabine and targeted busulfan followed by allogeneic transplant. The initial dose of busulfan was determined by performing PK (pharmacokinetics) analysis on a test dose of busulfan on the day prior to initiating conditioning. Based on the PK results a starting dose of busulfan was determined. A second PK study was done after the starting dose to ensure that the targeted dose was achieved. If it wasn't achieved, then a dose modification was made and a third PK study done.

Participant Flow:   Overall Study
    Single Arm - Transplant Pre-conditioning Per Study Protocol
STARTED   35 
COMPLETED   34 
NOT COMPLETED   1 
Death                1 



  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.
Thirty-five children undergoing allogeneic hemotopoietic stem cell transplants (HSCTs) for malignant and nonmalignant diseases; 12 with HLA-matched related donors (MRDs), 16 with 10 of 10 HLA allele-matched unrelated donors (MUDs), 7 with 9 of 10 allele MUDs.

Reporting Groups
  Description
Single Arm - Transplant Pre-conditioning Per Study Protocol All eligible patients received pre-transplant conditioning with Alemtuzumab, fludarabine and targeted busulfan followed by allogeneic transplant. The initial dose of busulfan was determined by performing PK (pharmacokinetics) analysis on a test dose of busulfan on the day prior to initiating conditioning. Based on the PK results a starting dose of busulfan was determined. A second PK study was done after the starting dose to ensure that the targeted dose was achieved. If it wasn't achieved, then a dose modification was made and a third PK study done.

Baseline Measures
   Single Arm - Transplant Pre-conditioning Per Study Protocol 
Overall Participants Analyzed 
[Units: Participants]
 35 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      34  97.1% 
Between 18 and 65 years      1   2.9% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 7.58  (5.25) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      13  37.1% 
Male      22  62.9% 
Region of Enrollment 
[Units: Participants]
 
United States   35 


  Outcome Measures

1.  Primary:   Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation   [ Time Frame: 6 weeks post-transplant ]

2.  Secondary:   Treatment-related Mortality at 100 Days and 1 Year Post Transplantation   [ Time Frame: 100 days and 1 year ]

3.  Secondary:   Toxicity Grade ≥ 3 From Start of Conditioning Through the First Year Post Transplantation   [ Time Frame: 1 year post-transplantation ]

4.  Secondary:   Cytomegalovirus (CMV) Viral Infection and Disease Symptoms   [ Time Frame: Up to one year post-transplant ]

5.  Secondary:   Disease-free Survival With Correction of Disease at One Year Post Transplantation   [ Time Frame: 1 year post-transplantation ]


  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
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  More Information

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


Results Point of Contact:  
Name/Title: Dr. Morton Cowan
Organization: University of California San Francisco
phone: 4154762188
e-mail: mcowan@peds.ucsf.edu



Responsible Party: University of California, San Francisco
ClinicalTrials.gov Identifier: NCT00301834     History of Changes
Other Study ID Numbers: CDR0000462406
UCSF-04152 ( Other Identifier: UCSF Helen Diller Family Comprehensive Cancer Center )
UCSF-00452 ( Other Identifier: UCSF Helen Diller Family Comprehensive Cancer Center )
UCSF-H411-25738-02 ( Other Identifier: UCSF Committee on Human Research (CHR) )
First Submitted: March 9, 2006
First Posted: March 13, 2006
Results First Submitted: January 31, 2013
Results First Posted: May 16, 2013
Last Update Posted: September 28, 2017