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Trial record 6 of 17 for:    "severe congenital neutropenia"

Stem Cell Transplant for Hemoglobinopathy

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ClinicalTrials.gov Identifier: NCT00176852
Recruitment Status : Active, not recruiting
First Posted : September 15, 2005
Results First Posted : May 9, 2017
Last Update Posted : December 5, 2017
Sponsor:
Collaborator:
National Marrow Donor Program
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Sickle Cell Disease
Thalassemia
Severe Congenital Neutropenia
Diamond-Blackfan Anemia
Shwachman-Diamond Syndrome
Interventions: Drug: Busulfan, Fludarabine, ATG, TLI
Drug: Busulfan, Cyclophosphamide, ATG, GCSF
Drug: Campath, Fludarabine, Cyclophosphamide
Radiation: Total Body Irradiation
Procedure: Stem cell infusion

  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
Full Conditioning (Discontinued / A)

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy

Busulfan Conditioning (B)

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC >2500 x 2 days.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Campath and TBI Conditioning (A2)

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0


Participant Flow:   Overall Study
    Full Conditioning (Discontinued / A)   Busulfan Conditioning (B)   Campath and TBI Conditioning (A2)
STARTED   3   5   14 
COMPLETED   3   5   14 
NOT COMPLETED   0   0   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
Full Conditioning (Discontinued / A)

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

Busulfan, Fludarabine, ATG, TLI: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy

Busulfan Conditioning (B)

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

Busulfan, Cyclophosphamide, ATG, GCSF: Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC >2500 x 2 days.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Campath and TBI Conditioning (A2)

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

Campath, Fludarabine, Cyclophosphamide: Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Total Total of all reporting groups

Baseline Measures
   Full Conditioning (Discontinued / A)   Busulfan Conditioning (B)   Campath and TBI Conditioning (A2)   Total 
Overall Participants Analyzed 
[Units: Participants]
 3   5   14   22 
Age 
[Units: Participants]
Count of Participants
       
<=18 years      3 100.0%      5 100.0%      12  85.7%      20  90.9% 
Between 18 and 65 years      0   0.0%      0   0.0%      2  14.3%      2   9.1% 
>=65 years      0   0.0%      0   0.0%      0   0.0%      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
       
Female      2  66.7%      1  20.0%      8  57.1%      11  50.0% 
Male      1  33.3%      4  80.0%      6  42.9%      11  50.0% 


  Outcome Measures

1.  Primary:   Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity   [ Time Frame: 1 year ]

2.  Secondary:   The Incidence of Chimerism at 100 Days   [ Time Frame: 100 days ]

3.  Secondary:   The Incidence of Chimerism at 6 Months   [ Time Frame: 6 months ]

4.  Secondary:   The Incidence of Chimerism at 1 Year   [ Time Frame: 1 year ]

5.  Secondary:   The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD)   [ Time Frame: 100 days ]

6.  Secondary:   The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD)   [ Time Frame: 100 days ]

7.  Secondary:   The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)   [ Time Frame: 6 months ]

8.  Secondary:   The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)   [ Time Frame: 1 year ]

9.  Secondary:   Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment   [ Time Frame: pre-transplant ]

10.  Secondary:   Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment   [ Time Frame: 1 year ]

11.  Secondary:   Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment   [ Time Frame: 2 years ]

12.  Secondary:   Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment   [ Time Frame: 5 years ]

13.  Secondary:   Determine Physical Characteristics and Biologic Effects of Mixed Populations of Donor and Host Red Blood Cells   [ Time Frame: During study ]

14.  Secondary:   Determine the Concentration of Campath in the Serum   [ Time Frame: Day 0 ]

15.  Secondary:   Overall Survival   [ Time Frame: 100 days ]

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

17.  Secondary:   Disease Free Survival   [ Time Frame: 100 days ]

18.  Secondary:   Disease Free Survival   [ Time Frame: 1 year ]


  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
No text entered.


  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. Angela Smith
Organization: Masonic Cancer Center, University of Minnesota
phone: 612-626-2778
e-mail: smith719@umn.edu



Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00176852     History of Changes
Obsolete Identifiers: NCT00005897
Other Study ID Numbers: MT2002-07
0206M26241 ( Other Identifier: IRB, University of Minnesota )
First Submitted: September 12, 2005
First Posted: September 15, 2005
Results First Submitted: March 28, 2017
Results First Posted: May 9, 2017
Last Update Posted: December 5, 2017