Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...

Alemtuzumab, Fludarabine Phosphate, and Total-Body Irradiation Followed by a Donor Stem Cell Transplant in Treating Patients With Immunodeficiency or Other Nonmalignant Inherited Disorders

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
National Cancer Institute (NCI)
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Lauri Burroughs, Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier:
NCT00553098
First received: November 2, 2007
Last updated: April 13, 2017
Last verified: April 2017
Results First Received: April 13, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: No masking;   Primary Purpose: Treatment
Conditions: Immunodeficiency Syndrome
Non-Cancer Diagnosis
Interventions: Biological: Alemtuzumab
Procedure: Allogeneic Bone Marrow Transplantation
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Drug: Cyclosporine
Drug: Fludarabine Phosphate
Other: Laboratory Biomarker Analysis
Drug: Mycophenolate Mofetil
Radiation: Total-Body Irradiation

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Treatment (Chemotherapy, Low Dose Radiation)

CONDITIONING REGIMEN: *Patients with no life-threatening viral or fungal infections within 1 month before the planned HCT receive alemtuzumab IV over 6 hours on day -10 and fludarabine phosphate IV over 30 minutes on days -4 to -2. They also undergo low-dose TBI on day 0. Patients with HLH, IPEX syndrome, DiGeorge syndrome, or life-threatening viral or fungal infections within 1 month before the planned HCT receive fludarabine phosphate IV over 30 minutes on days -4 to -2 and undergo 2 low doses of TBI on day 0.

HEMATOPOIETIC CELL TRANSPLANTATION: Patients undergo HCT on day 0. IMMUNOSUPPRESSION: Patients receive cyclosporine IV or PO 2-3 times daily beginning on day -3 and continuing until day 100 followed by a taper until day 180. They also receive mycophenolate mofetil IV or PO 3 times daily beginning on day 0 and continuing until day 40 followed by a taper until day 96.

Alemtuzumab: Given IV Allogeneic Bone Marrow Transplantation: Undergo HCT Allogeneic Hematopoiet


Participant Flow:   Overall Study
    Treatment (Chemotherapy, Low Dose Radiation)
STARTED   29 
COMPLETED   28 
NOT COMPLETED   1 



  Baseline Characteristics
  Hide 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
Treatment (Chemotherapy, Low Dose Radiation)

CONDITIONING REGIMEN: *Patients with no life-threatening viral or fungal infections within 1 month before the planned HCT receive alemtuzumab IV over 6 hours on day -10 and fludarabine phosphate IV over 30 minutes on days -4 to -2. They also undergo low-dose TBI on day 0. Patients with HLH, IPEX syndrome, DiGeorge syndrome, or life-threatening viral or fungal infections within 1 month before the planned HCT receive fludarabine phosphate IV over 30 minutes on days -4 to -2 and undergo 2 low doses of TBI on day 0.

HEMATOPOIETIC CELL TRANSPLANTATION: Patients undergo HCT on day 0.

IMMUNOSUPPRESSION: Patients receive cyclosporine IV or PO 2-3 times daily beginning on day -3 and continuing until day 100 followed by a taper until day 180. They also receive mycophenolate mofetil IV or PO 3 times daily beginning on day 0 and continuing until day 40 followed by a taper until day 96.

Alemtuzumab: Given IV Allogeneic Bone Marrow Transplantation: Undergo HCT Allogeneic Hematopoiet


Baseline Measures
   Treatment (Chemotherapy, Low Dose Radiation) 
Overall Participants Analyzed 
[Units: Participants]
 28 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      24  85.7% 
Between 18 and 65 years      4  14.3% 
>=65 years      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      12  42.9% 
Male      16  57.1% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      1   3.6% 
Not Hispanic or Latino      27  96.4% 
Unknown or Not Reported      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      0   0.0% 
Native Hawaiian or Other Pacific Islander      1   3.6% 
Black or African American      1   3.6% 
White      22  78.6% 
More than one race      4  14.3% 
Unknown or Not Reported      0   0.0% 
Region of Enrollment 
[Units: Participants]
 
United States   28 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Patients Who Achieve Greater Than 50% Donor T-cell Chimerism   [ Time Frame: At 1 year post transplant ]

2.  Secondary:   Overall Survival   [ Time Frame: 1 year ]

3.  Secondary:   Immune Reconstitution by 1 Year Post Transplant   [ Time Frame: 1 year ]

4.  Secondary:   Disease Response by 1 Year Post Transplant   [ Time Frame: 1 year ]

5.  Secondary:   Greater Than 50% CD33+ Donor Chimerisms at 1 Year Post Transplant   [ Time Frame: 1 year ]

6.  Secondary:   Greater Than 50% CD19+ Donor Chimerisms at 1 Year Post Transplant   [ Time Frame: 1 year ]

7.  Secondary:   Clinical Significant Infection, Requiring Treatment, Within 100 Days Post Transplant   [ Time Frame: 100 days ]

8.  Secondary:   Number of Patients Diagnosed With Acute GVHD   [ Time Frame: Day 100 ]

9.  Secondary:   Number of Patients Diagnosed With Overall Grade 1 or Grade 2 Acute GVHD   [ Time Frame: Day 100 ]

10.  Secondary:   Number of Patients Diagnosed With Overall Grade III or Grade IV Acute GVHD   [ Time Frame: Day 100 ]

11.  Secondary:   Number of Patients Diagnosed With Chronic GVHD   [ Time Frame: 1 year ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide 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
No text entered.


  More Information
  Hide More Information

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:  
Name/Title: Dr. Lauri Burroughs
Organization: Fred Hutch Cancer Research
phone: 206-667-2396
e-mail: lburroug@fredhutch.org



Responsible Party: Lauri Burroughs, Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier: NCT00553098     History of Changes
Other Study ID Numbers: 2007.00
NCI-2009-01550 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2007
2007.00 ( Other Identifier: Fred Hutch/University of Washington Cancer Consortium )
P30CA015704 ( US NIH Grant/Contract Award Number )
Study First Received: November 2, 2007
Results First Received: April 13, 2017
Last Updated: April 13, 2017