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NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02833805
Recruitment Status : Completed
First Posted : July 14, 2016
Results First Posted : July 12, 2022
Last Update Posted : July 12, 2022
Sponsor:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Severe Aplastic Anemia
Aplastic Anemia
Bone Marrow Failure
Immunosuppression
Interventions Drug: Thymoglobulin
Drug: Fludarabine
Drug: Cyclophosphamide
Radiation: Total body irradiation
Drug: Tacrolimus
Drug: Mycophenolate mofetil
Enrollment 21
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as graft-versus-host disease (GVHD) prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Period Title: Overall Study
Started 21
Completed 21
Not Completed 0
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Baseline Participants 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
<=18 years
7
  33.3%
Between 18 and 65 years
14
  66.7%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 21 participants
25
(3 to 69)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
Female
8
  38.1%
Male
13
  61.9%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   4.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
4
  19.0%
White
14
  66.7%
More than one race
0
   0.0%
Unknown or Not Reported
2
   9.5%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
African American
4
  19.0%
Caucasian
14
  66.7%
Other
3
  14.3%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 21 participants
21
 100.0%
1.Primary Outcome
Title Overall Survival and Engraftment at One Year
Hide Description Number of enrolled participants who receive BMT, achieve engraftment, and are alive at one year post bone marrow transplant (BMT).
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
19
  90.5%
2.Secondary Outcome
Title Overall Survival at One Year
Hide Description Number of participants alive at one year after BMT.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
19
  90.5%
3.Secondary Outcome
Title Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts
Hide Description Probability of neutrophil recovery will be assessed by the number of participants who have recovered neutrophil counts at 1 year (>500 ANC).
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
21
 100.0%
4.Secondary Outcome
Title Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts
Hide Description Probability of platelet recovery will be assessed by the number of participants who have recovered platelet counts at 1 year.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
20
  95.2%
5.Secondary Outcome
Title Number of Participants Who Experience Primary Graft Failure
Hide Description Number of participants who experience primary graft failure by one year after BMT.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
21
 100.0%
6.Secondary Outcome
Title Number of Participants Who Experience Secondary Graft Failure
Hide Description Number of participants who experience secondary graft failure by one year after BMT.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
19
  90.5%
7.Secondary Outcome
Title Number of Participants Who Experience Grades II-IV Acute GVHD
Hide Description Number of participants who experience grade II, III, or IV acute GVHD by Day 100.
Time Frame Day 100
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
4
  19.0%
8.Secondary Outcome
Title Number of Participants Who Experience Grades III-IV Acute GVHD
Hide Description Number of participants who experience grade III or IV acute GVHD by Day 100.
Time Frame Day 100
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
2
   9.5%
9.Secondary Outcome
Title Number of Participants Who Experience Chronic GVHD
Hide Description Number of participants who experience chronic GVHD by two years after BMT.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
1
   4.8%
10.Secondary Outcome
Title Number of Participants With Full Donor Chimerism
Hide Description Number of participants with full donor chimerism at Day 60.
Time Frame Day 60
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
21
 100.0%
11.Secondary Outcome
Title GVHD-free Relapse-free Survival (GRFS)
Hide Description Number of participants alive, without relapse, and without GVHD at 1 year.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
19
  90.5%
12.Secondary Outcome
Title Transplant-related Mortality
Hide Description Number of participants deceased for reasons related to BMT at 1 year.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description:

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
2
   9.5%
Time Frame Adverse events were monitored and collected from time of consent up to one year.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Bone Marrow Transplant
Hide Arm/Group Description

Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.

Thymoglobulin: Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily

Fludarabine: Days -6 through -2: 30 mg/m^2 IV daily

Cyclophosphamide: Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily

Total body irradiation: Day -1: 200 centigray (cGy) in a single fraction

Tacrolimus: Start on Day 5 through Day 365

Mycophenolate mofetil: Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)

All-Cause Mortality
Bone Marrow Transplant
Affected / at Risk (%)
Total   2/21 (9.52%) 
Hide Serious Adverse Events
Bone Marrow Transplant
Affected / at Risk (%)
Total   2/21 (9.52%) 
Infections and infestations   
Gram negative rods Bacteremia *  2/21 (9.52%) 
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Bone Marrow Transplant
Affected / at Risk (%)
Total   0/21 (0.00%) 
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
Layout table for Results Point of Contact information
Name/Title: Dr. Amy DeZern
Organization: Johns Hopkins University
Phone: 410-502-7208
EMail: adezerb1@jhmi.edu
Layout table for additonal information
Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier: NCT02833805    
Other Study ID Numbers: J1688
IRB00107139 ( Other Identifier: JHMIRB )
First Submitted: July 12, 2016
First Posted: July 14, 2016
Results First Submitted: May 5, 2022
Results First Posted: July 12, 2022
Last Update Posted: July 12, 2022