Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia
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|ClinicalTrials.gov Identifier: NCT02224872|
Recruitment Status : Recruiting
First Posted : August 25, 2014
Last Update Posted : December 20, 2018
|Condition or disease||Intervention/treatment||Phase|
|Severe Aplastic Anemia Bone Marrow Failure Syndromes||Procedure: Bone marrow transplant Drug: Thymoglobulin Drug: Fludarabine Drug: Cyclophosphamide Radiation: TBI Drug: Mesna Drug: Tacrolimus Drug: Mycophenolic acid mofetil||Phase 2|
This research is being done to find out if bone marrow transplantation (BMT) followed by chemotherapy will help people with aplastic anemia who have failed other treatments.
You have a severe, life threatening disease (severe aplastic anemia) in your bone marrow. Your disease has come back or not responded after receiving one or more immunosuppressive treatments. High dose chemotherapy followed by bone marrow transplantation (BMT) has been used to treat blood diseases like yours but complications from Graft vs. Host disease (GVHD) and graft failure have limited the survival for those people.
A small study done at Johns Hopkins has shown that in subjects with other diseases (blood cancers) some immunosuppressive drugs given after the BMT have decreased how often subjects had complications of GVHD and engraftment failure.
People with aplastic anemia who have refractory disease (not responding to standard treatment) may join.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes|
|Study Start Date :||August 2014|
|Estimated Primary Completion Date :||November 2023|
|Estimated Study Completion Date :||November 2023|
Experimental: Bone marrow transplant
Thymoglobulin on days -9 to -7 Fludarabine on days -6 to -2 Cyclophosphamide on days -6, -5, 3, 4 TBI on day -1 BMT on day 0 Mesna on days 3, 4 Tacrolimus on days 5-365 Mycophenolic acid mofetil on days 5-35
Procedure: Bone marrow transplant
0.5 mg/kg IV on Day -9 2 mg/kg IV on Days -8, -7
30 mg/M2 IV on days -6 to -2
14.5 mg/kg IV on days -6, -5, 3, 4
Other Name: CTX
200 cGy on day -1
40 mg/kg IV on days 3, 4
For patients 18 years or older, tacrolimus will be given per institutional standards; may be increased or later changed to a PO BID schedule. Treatment to continue until Day 365 or longer if GVHD present
Drug: Mycophenolic acid mofetil
15 mg/kg PO/IV TID beginning on day 5 through day 35
Other Name: MMF
- Is this type of transplantation for SAA feasible and safe? [ Time Frame: 5 years ]Feasibility will be met with the following conditions: the patient has the transplant, is assessed for the safety endpoint, and survives one year. The safety monitoring plan is included to monitor graft failure (day 60), grade 2-4 acute graft versus host disease (day100), 6 month mortality (day 180), and chronic graft versus host disease (day 180).
- Number of patients that have survived at one year [ Time Frame: 5 years ]
- Number of patients that have acheived full donor chimerism by day 60 after transplant [ Time Frame: 5 years ]Donor chimerism will be measured in the peripheral blood around day 30 and day 60. Patients with >5% donor chimerism around day 60 will be considered as having engrafted.
- Number of patients that expired due to non-relapsed-related mortality following transplant [ Time Frame: 5 years ]
- Major toxicities related to transplant [ Time Frame: 5 years ]
- Number of patients that expired due to transplant related mortality [ Time Frame: 5 years ]
- Number of patients with primary or secondary graft failure following transplant [ Time Frame: 5 years ]
Graft failure: < 5% donor chimerism in blood and/or bone marrow on ~Day 30 or after and on all subsequent measurements.
Primary graft failure: < 5% donor chimerism in blood and/or bone marrow by ~ Day 56 Secondary graft failure: achievement of > 5% donor chimerism, followed by sustained <5% donor chimerism in blood and/or bone marrow.
- Number of patients with grade II-IV or grade III-IV acute GVHD [ Time Frame: 5 years ]
- Number of patients with chronic GVHD [ Time Frame: 5 years ]
- Length of time required for patients to recover ANC and platelet counts after transplant [ Time Frame: 5 years ]CBC drawn daily with a WBC differential once the total WBC is greater than 100 until ANC > 500 for three days or two consecutive measurements over a three day period; then CBC drawn weekly with differential.
- Length of GVHD free, relapse free survival (GRFS) in patients [ Time Frame: 5 years ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02224872
|Contact: Amy DeZern, MDfirstname.lastname@example.org|
|United States, Maryland|
|The Sidney Kimmel Comprehensive Cancer Center||Recruiting|
|Baltimore, Maryland, United States, 21287|
|Contact: Amy DeZern, MD 410-502-7208 email@example.com|
|Contact: Clinical Trials Office- SKCCC 410-955-8804 firstname.lastname@example.org|
|Principal Investigator: Amy DeZern, MD|
|United States, Wisconsin|
|Medical College of Wisconsin/Children's Hospital of Wisconsin||Recruiting|
|Milwaukee, Wisconsin, United States, 53226|
|Contact: David Margolis, MD 414-337-7066 email@example.com|
|Contact: Monica Thakar, MD 414-955-7546 firstname.lastname@example.org|
|Principal Investigator:||Amy DeZern, MD||Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins|