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Trial record 51 of 833 for:    Texas Children's Hospital | ( Map: United States )

Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease (MUNCHR)

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ClinicalTrials.gov Identifier: NCT00578643
Recruitment Status : Completed
First Posted : December 21, 2007
Results First Posted : November 9, 2018
Last Update Posted : November 9, 2018
Sponsor:
Collaborator:
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
Condition Chronic Granulomatous Disease
Interventions Drug: Busulfan
Biological: Alemtuzumab
Drug: Cyclophosphamide
Drug: Fludarabine
Drug: Cyclosporine
Procedure: Stem Cell Infusion
Enrollment 15
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Allogeneic Unrelated Transplant
Hide Arm/Group Description

Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion.

Busulfan: Days -9 through -6

1 mg/kg initially (based on weight)

Alemtuzumab: Day -5 through Day -2

Dose is based on weight:

Less than 15 kg: 3 mg

More than 15 kg to 30 kg: 5 mg

More than 30 kg: 15 mg

Cyclophosphamide: Days -5 through -2

50 mg/kg

Fludarabine: Day -5 through Day -2

30 mg/m^2

Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours.

Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10^8/kg recipient weight; for cord blood ≥ 3 X 10^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10^/kg CD34+ cells.

Period Title: Overall Study
Started 15
Completed 13
Not Completed 2
Reason Not Completed
Death             1
Lost to Follow-up             1
Arm/Group Title Allogeneic Unrelated Transplant
Hide Arm/Group Description

Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion.

Busulfan: Days -9 through -6

1 mg/kg initially (based on weight)

Alemtuzumab: Day -5 through Day -2

Dose is based on weight:

Less than 15 kg: 3 mg

More than 15 kg to 30 kg: 5 mg

More than 30 kg: 15 mg

Cyclophosphamide: Days -5 through -2

50 mg/kg

Fludarabine: Day -5 through Day -2

30 mg/m^2

Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours.

Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10^8/kg recipient weight; for cord blood ≥ 3 X 10^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10^/kg CD34+ cells.

Overall Number of Baseline Participants 15
Hide Baseline Analysis Population Description
Analysis included 15 patients who underwent HLA matched unrelated or non-genotype identical related donor transplantation for chronic granulomatous disease(CGD).
Age, Continuous  
Median (Inter-Quartile Range)
Unit of measure:  Years
Number Analyzed 15 participants
6
(2 to 10)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants
Female
1
   6.7%
Male
14
  93.3%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Race/Ethnicity Number Analyzed 15 participants
White
7
  46.7%
Black or African American
1
   6.7%
Asian
1
   6.7%
Hispanic or Latino
5
  33.3%
Unknown
1
   6.7%
1.Primary Outcome
Title Percentage of Participants With Engraftment
Hide Description To estimate the engraftment rate for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors.
Time Frame 28 days post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Allogeneic Unrelated Transplant
Hide Arm/Group Description:

Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion.

Busulfan: Days -9 through -6

1 mg/kg initially (based on weight)

Alemtuzumab: Day -5 through Day -2

Dose is based on weight:

Less than 15 kg: 3 mg

More than 15 kg to 30 kg: 5 mg

More than 30 kg: 15 mg

Cyclophosphamide: Days -5 through -2

50 mg/kg

Fludarabine: Day -5 through Day -2

30 mg/m^2

Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours.

Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10^8/kg recipient weight; for cord blood ≥ 3 X 10^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10^/kg CD34+ cells.

Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: percentage of participants
100
2.Secondary Outcome
Title Number of Patients That Have Complete Donor Chimerism After Transplant.
Hide Description To estimate the likelihood of complete donor chimerism for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors.
Time Frame 120 days post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
One patient died on day 62 post transplant and was not included in the analysis.
Arm/Group Title Allogeneic Unrelated Transplant
Hide Arm/Group Description:

Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion.

Busulfan: Days -9 through -6

1 mg/kg initially (based on weight)

Alemtuzumab: Day -5 through Day -2

Dose is based on weight:

Less than 15 kg: 3 mg

More than 15 kg to 30 kg: 5 mg

More than 30 kg: 15 mg

Cyclophosphamide: Days -5 through -2

50 mg/kg

Fludarabine: Day -5 through Day -2

30 mg/m^2

Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours.

Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10^8/kg recipient weight; for cord blood ≥ 3 X 10^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10^/kg CD34+ cells.

Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: participants
13
3.Secondary Outcome
Title Number of Patients That Have Acute GVHD and Regimen Related Morbidity/Mortality Post Transplant.
Hide Description To estimate the risk for acute GVHD and regimen related morbidity/mortality for patients with CGD following stem cell transplant from 5/6 or 6/6 HLA matched unrelated or 5/6 or 6/6 HLA phenotype matched related donors.
Time Frame Assessed between day 0 and day 100 post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
One patient who died on day 62 post transplant without developing acute GVHD was not assessed for this Outcome Measure.
Arm/Group Title Allogeneic Unrelated Transplant
Hide Arm/Group Description:

Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion.

Busulfan: Days -9 through -6

1 mg/kg initially (based on weight)

Alemtuzumab: Day -5 through Day -2

Dose is based on weight:

Less than 15 kg: 3 mg

More than 15 kg to 30 kg: 5 mg

More than 30 kg: 15 mg

Cyclophosphamide: Days -5 through -2

50 mg/kg

Fludarabine: Day -5 through Day -2

30 mg/m^2

Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours.

Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10^8/kg recipient weight; for cord blood ≥ 3 X 10^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10^/kg CD34+ cells.

Overall Number of Participants Analyzed 14
Measure Type: Count of Participants
Unit of Measure: Participants
None
8
  57.1%
Grade I
6
  42.9%
Grade II
0
   0.0%
Grade III-IV
0
   0.0%
4.Secondary Outcome
Title Number of Patients That Have Chronic GVHD and Regimen Related Morbidity/Mortality Post Transplant.
Hide Description To estimate the risk for chronic GVHD and regimen related morbidity/mortality for patients with CGD following stem cell transplant from 5/6 or 6/6 HLA matched unrelated or 5/6 or 6/6 HLA phenotype matched related donors.
Time Frame Assessed between day 100 and day 365 post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
One patient died on day 62 post transplant, and one patient was lost to F/U on day 163 post transplant. They were not assessed for this Outcome Measure.
Arm/Group Title Allogeneic Unrelated Transplant
Hide Arm/Group Description:

Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion.

Busulfan: Days -9 through -6

1 mg/kg initially (based on weight)

Alemtuzumab: Day -5 through Day -2

Dose is based on weight:

Less than 15 kg: 3 mg

More than 15 kg to 30 kg: 5 mg

More than 30 kg: 15 mg

Cyclophosphamide: Days -5 through -2

50 mg/kg

Fludarabine: Day -5 through Day -2

30 mg/m^2

Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours.

Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10^8/kg recipient weight; for cord blood ≥ 3 X 10^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10^/kg CD34+ cells.

Overall Number of Participants Analyzed 13
Measure Type: Count of Participants
Unit of Measure: Participants
Yes
0
   0.0%
No
13
 100.0%
Time Frame Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant.
Adverse Event Reporting Description An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
 
Arm/Group Title Allogeneic Unrelated Transplant
Hide Arm/Group Description

Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion.

Busulfan: Days -9 through -6

1 mg/kg initially (based on weight)

Alemtuzumab: Day -5 through Day -2

Dose is based on weight:

Less than 15 kg: 3 mg

More than 15 kg to 30 kg: 5 mg

More than 30 kg: 15 mg

Cyclophosphamide: Days -5 through -2

50 mg/kg

Fludarabine: Day -5 through Day -2

30 mg/m^2

Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours.

Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10^8/kg recipient weight; for cord blood ≥ 3 X 10^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10^/kg CD34+ cells.

All-Cause Mortality
Allogeneic Unrelated Transplant
Affected / at Risk (%)
Total   1/15 (6.67%)    
Show Serious Adverse Events Hide Serious Adverse Events
Allogeneic Unrelated Transplant
Affected / at Risk (%) # Events
Total   9/15 (60.00%)    
Cardiac disorders   
Hypertension  1  1/15 (6.67%)  1
Gastrointestinal disorders   
Diarrhea patients without colostomy  1  1/15 (6.67%)  1
Gastrointestinal-Other: Eosinofilic enteritis  1  1/15 (6.67%)  1
Pancreatitis  1  1/15 (6.67%)  1
General disorders   
Constitutional Symptoms - Other: Multiorgan failure  1  1/15 (6.67%)  1
Immune system disorders   
Allergic reaction/hypersensitivity (including drug fever)  1  1/15 (6.67%)  1
Infections and infestations   
Hematuria (in the absence of vaginal bleeding)  1  1/15 (6.67%)  1
Infection (documented clinically or microbiologically) with grade 3 or 4 neutropenia (ANC <1.0 x 10e  1  1/15 (6.67%)  2
Infection without neutropenia  1  1/15 (6.67%)  1
Infection - Other: Adenovirus viremia, BK viruria, CMV viremia  1  1/15 (6.67%)  1
Infection - Other: Hemorrhagic cystitis with BK virus, adenovirus, and CMV  1  1/15 (6.67%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils - Blood  1  1/15 (6.67%)  1
Metabolism and nutrition disorders   
SGOT (AST) (serum glutamic oxaloacetic transaminase)  1  1/15 (6.67%)  1
Nervous system disorders   
Seizure  1  1/15 (6.67%)  1
Renal and urinary disorders   
Renal failure  1  1/15 (6.67%)  1
Respiratory, thoracic and mediastinal disorders   
Pleural effusion (non-malignant) 1  1  1/15 (6.67%)  1
Pneumonitis/pulmonary infiltrates  1  1/15 (6.67%)  1
Pulmonary/Upper Respiratory - Other: Respiratory distress  1  1/15 (6.67%)  1
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Allogeneic Unrelated Transplant
Affected / at Risk (%) # Events
Total   14/15 (93.33%)    
Cardiac disorders   
Hypertension  1  3/15 (20.00%)  3
Gastrointestinal disorders   
Dehydration  1  1/15 (6.67%)  1
Diarrhea  1  1/15 (6.67%)  1
Diarrhea patients without colostomy  1  1/15 (6.67%)  3
Stomatitis/pharyngitis (oral/pharyngeal mucositis)  1  1/15 (6.67%)  1
Hepatobiliary disorders   
GGT (Gamma-Glutamyl transpeptidase)  1  2/15 (13.33%)  3
SGOT (AST) (serum glutamic oxaloacetic transaminase)  1  2/15 (13.33%)  2
SGPT (ALT) (serum glutamic pyruvic transaminase)  1  1/15 (6.67%)  1
Infections and infestations   
Hematuria (in the absence of vaginal bleeding)  1  1/15 (6.67%)  1
Catheter-related infection  1  1/15 (6.67%)  1
Infection (documented clinically or microbiologically) with grade 3 or 4 neutropenia(ANC<1.0x10e9/L)  1  3/15 (20.00%)  3
Infection without neutropenia  1  2/15 (13.33%)  5
Infection with normal ANC or Grade 1 or 2 neutrophils - Bladder (urinary)  1  1/15 (6.67%)  1
Metabolism and nutrition disorders   
ALT, SGPT (serum glutamic pyruvic transaminase)  1  1/15 (6.67%)  1
AST, SGOT(serum glutamic oxaloacetic transaminase)  1  5/15 (33.33%)  6
Acidosis (metabolic or respiratory)  1  1/15 (6.67%)  2
Amylase  1  1/15 (6.67%)  2
Calcium, serum-low (hypocalcemia)  1  1/15 (6.67%)  1
GGT (gamma-Glutamyl transpeptidase)  1  1/15 (6.67%)  1
Hypocalcemia  1  2/15 (13.33%)  2
Hyponatremia  1  2/15 (13.33%)  2
Lipase  1  1/15 (6.67%)  1
Nervous system disorders   
Seizure(s)  1  1/15 (6.67%)  1
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Robert Krance, MD
Organization: Baylor College of Medicine
Phone: (832) 824-4661
Responsible Party: Robert Krance, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00578643     History of Changes
Other Study ID Numbers: 14771-MUNCHR
First Submitted: December 19, 2007
First Posted: December 21, 2007
Results First Submitted: August 21, 2018
Results First Posted: November 9, 2018
Last Update Posted: November 9, 2018