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Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders (MASCI)

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ClinicalTrials.gov Identifier: NCT00579137
Recruitment Status : Terminated (slow accrual)
First Posted : December 21, 2007
Results First Posted : July 2, 2013
Last Update Posted : July 2, 2013
Sponsor:
Collaborators:
Center for Cell and Gene Therapy, Baylor College of Medicine
Texas Children's Hospital
Information provided by (Responsible Party):
Robert Krance, Baylor College of Medicine

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Severe Combined Immunodeficiency Disease
Severe Primary Immunodeficiency Disorder
Undefined T Cell Deficiency Disorder
Wiskott-Aldrick Syndrome
Interventions Biological: Campath -1H
Drug: Fludarabine
Biological: Anti-CD45
Procedure: Stem cell infusion
Enrollment 3
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Participants With SCID or Primary Immunodeficiency Disorder
Hide Arm/Group Description

Participants received an allogeneic stem cell transplant with the following conditioning:

Day 8 Campath 1H as per CAGT SOP, Fludarabine 10 kg or less: 1 mg/kg; > 10 kg: 30 mg/m2

D7 Campath 1H as per CAGT SOP, Fludarabine 10 kg or less: 1 mg/kg; > 10 kg: 30 mg/m2

D6 Campath 1H as per CAGT SOP, Fludarabine 10 kg or less: 1 mg/kg; > 10 kg: 30 mg/m2

D5 Anti-CD45 MAb 400ug/kg over 6 hr, Fludarabine 10 kg or less: 1 mg/kg; > 10 kg: 30 mg/m2

D4 Anti-CD45 MAb 400ug/kg over 6 hr, Fludarabine 10 kg or less: 1 mg/kg; > 10 kg: 30 mg/m2

D3 Anti-CD45 MAb 400ug/kg over 6 hr

D2 Anti-CD45 MAb 400ug/kg over 6 hr

D1 rest

D0 Stem Cell Infusion

Campath dose is weight based: for patients less than 15 kg administer Campath 3 mg; for patients >15 kg to 30 kg administer Campath 5 mg; for patients > 30 kg administer Campath 10 mg. Campath will be dosed and administered as per CAGT SOP.

Anti-CD45 infusion will be administered according to CAGT SOPs.

Period Title: Overall Study
Started 3
Completed 3
Not Completed 0
Arm/Group Title Single Group
Hide Arm/Group Description only one group
Overall Number of Baseline Participants 3
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
<=1 years Number Analyzed 3 participants
3
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
Female
0
   0.0%
Male
3
 100.0%
1.Primary Outcome
Title Number of Patients With Donor Engraftment
Hide Description [Not Specified]
Time Frame 100 Days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Single Group
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only one group
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
1
2.Secondary Outcome
Title Patients Alive at 1 Year
Hide Description [Not Specified]
Time Frame 1 Year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Single Group
Hide Arm/Group Description:
only one group
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
3
3.Secondary Outcome
Title Number of Patients With Grade III or IV Toxicity
Hide Description [Not Specified]
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Single Group
Hide Arm/Group Description:
only one group
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
0
4.Secondary Outcome
Title Number of Patients With Grade III to IV Acute GVHD
Hide Description [Not Specified]
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Single Group
Hide Arm/Group Description:
only one group
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
0
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Single Group
Hide Arm/Group Description only one group
All-Cause Mortality
Single Group
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Single Group
Affected / at Risk (%) # Events
Total   0/3 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Single Group
Affected / at Risk (%) # Events
Total   3/3 (100.00%)    
Blood and lymphatic system disorders   
Hemoglobin   1/3 (33.33%)  5
Leukocytes (total WBC)   1/3 (33.33%)  17
Lymphopenia   1/3 (33.33%)  23
Neutrophils/granulocytes (ANC/AGC)   1/3 (33.33%)  10
PTT (Partial Thromboplastin Time)   1/3 (33.33%)  1
Platelets   1/3 (33.33%)  1
Gastrointestinal disorders   
Constipation   1/3 (33.33%)  3
Diarrhea   1/3 (33.33%)  5
Mucositis/stomatitis (clinical exam) - Oral cavity   1/3 (33.33%)  1
Nausea   1/3 (33.33%)  3
Vomiting   1/3 (33.33%)  5
General disorders   
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)   1/3 (33.33%)  1
Pain - Abdomen NOS   1/3 (33.33%)  1
Hepatobiliary disorders   
Hepatobiliary/Pancreas - Other: Liver GvHD   1/3 (33.33%)  1
Immune system disorders   
Allergic reaction/hypersensitivity (including drug fever)   1/3 (33.33%)  1
Infections and infestations   
Infection (documented clinically or microbiologically) with Grade 3 or 4 neutrophils (ANC <1.0 x 10e   1/3 (33.33%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils - Catheter-related   1/3 (33.33%)  1
Metabolism and nutrition disorders   
AST, SGOT(serum glutamic oxaloacetic transaminase)   1/3 (33.33%)  5
Albumin, serum-low (hypoalbuminemia)   1/3 (33.33%)  5
Calcium, serum-low (hypocalcemia)   1/3 (33.33%)  2
Glucose, serum-low (hypoglycemia)   1/3 (33.33%)  1
Phosphate, serum-low (hypophosphatemia)   1/3 (33.33%)  4
Potassium, serum-high (hyperkalemia)   1/3 (33.33%)  7
Sodium, serum-low (hyponatremia)   2/3 (66.67%)  2
Triglyceride, serum-high (hypertriglyceridemia)   1/3 (33.33%)  1
ALT, SGPT (serum glutamic pyruvic transaminase)   1/3 (33.33%)  5
Respiratory, thoracic and mediastinal disorders   
Bronchospasm, wheezing   1/3 (33.33%)  1
Cough   1/3 (33.33%)  3
Dyspnea (shortness of breath)   1/3 (33.33%)  2
Pulmonary/Upper Respiratory - Other: Nasal congestion   1/3 (33.33%)  1
Skin and subcutaneous tissue disorders   
Dermatology/Skin - Other: Livedo reticularis   1/3 (33.33%)  2
Dry skin   1/3 (33.33%)  1
Rash/desquamation   1/3 (33.33%)  3
Indicates events were collected by systematic assessment
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: Robert Krance, MD
Organization: Baylor
Phone: 832-824-4661
EMail: rkrance@bcm.edu
Layout table for additonal information
Responsible Party: Robert Krance, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00579137     History of Changes
Obsolete Identifiers: NCT00609258
Other Study ID Numbers: 21123-MASCI
First Submitted: December 19, 2007
First Posted: December 21, 2007
Results First Submitted: March 30, 2013
Results First Posted: July 2, 2013
Last Update Posted: July 2, 2013