Protocol summary
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Study aim
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The purpose of this study is to test with optimal approaches as post transplant cyclophosphamide in combination with Antithymocyte Globulin (ATG) whether we will achieve acceptable results in haploidentical transplantation in patients with Severe Aplastic Anemia.
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Design
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Phase 1-2 prospective single-arm interventional pilot study with 10 patients.
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Settings and conduct
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Severe Aplastic Anemia patients candidates for allogeneic transplantation are admitted at the hematopoietic stem cell transplant department of Shariati Hospital and will receive the conditioning regimen and will be transplanted.
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Participants/Inclusion and exclusion criteria
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Inclusion criteria: Patients from 18 to 60 years old, with Severe Aplastic Anemia and unresponsive to prior immune suppressive treatments, no full matched (sibling or unrelated) donor available.
Exclusion criteria: Prior allogeneic stem cell transplantation
Symptomatic coronary artery disease, Karnofsky Performance Score < 60, patients with uncontrolled bacterial, viral or fungal infection, a known life-threatening reaction (anaphylaxis) to Thymoglobin, females who are pregnant or breastfeeding, patients seropositive for human immunodeficiency virus or active hepatitis B or C with detectable viral load, received Antithymocyte globulin drug within 2 weeks before enrolling in the study, previous malignancies.
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Intervention groups
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Intervention group: Thymoglobulin (Genzyme) 0.5 mg/kg/D, Day -9 and Thymoglobulin 2.5 mg/kg/D, Day -8 and -7, Cyclophosphamide (Baxter) 14.5 mg/kg, Day -6 and -5, Fludarabin (ACTOVERCO) 30 mg/m2, Day -6 to -2, Busulfan (Nanoalvand) 3.2 mg/kg/D, Day -3 and -2. Cyclophosphamide (Baxter) 50 mg/kg, Day +3 and Day +4 after transplantation.
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Main outcome variables
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- One year overall survival
- Incidence of acute and chronic graft versus host disease
- Transplant related mortality
General information
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Reason for update
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Acronym
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IRCT registration information
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IRCT registration number:
IRCT20140818018842N29
Registration date:
2023-01-31, 1401/11/11
Registration timing:
prospective
Last update:
2024-04-12, 1403/01/24
Update count:
1
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Registration date
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2023-01-31, 1401/11/11
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Registrant information
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Recruitment status
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Recruitment complete
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Funding source
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Expected recruitment start date
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2023-02-20, 1401/12/01
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Expected recruitment end date
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2024-03-20, 1403/01/01
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Actual recruitment start date
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empty
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Actual recruitment end date
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empty
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Trial completion date
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empty
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Scientific title
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Evaluation of the safety and effectiveness of Haploidentical hematopoietic stem cell transplantation for the treatment of severe aplastic anemia patients who lack an Human leukocyte antigen (HLA)-matched (sibling or unrelated) donor.
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Public title
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Haploidentical hematopoietic stem cell transplantation for the treatment of severe aplastic anemia patients.
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Purpose
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Treatment
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Inclusion/Exclusion criteria
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Inclusion criteria:
Patients from 18 to 60 years old
Patients with Severe Aplastic Anemia
Patients unresponsive to prior immune suppressive treatments.
No full matched (sibling or unrelated) donor available.
Exclusion criteria:
Prior allogeneic stem cell transplantation
Symptomatic coronary artery disease
Karnofsky Performance Score < 60
Patients with uncontrolled bacterial, viral or fungal infection
A known life-threatening reaction (anaphylaxis) to Thymoglobin.
Females who are pregnant or breastfeeding.
Patients seropositive for human immunodeficiency virus or active hepatitis B or C with detectable viral load.
Received Antithymocyte globulin drug within 2 weeks before enrolling in the study.
Previous malignancies other than resected basal cell carcinoma or cervical cancer treated in situ.
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Age
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From 18 years old to 60 years old
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Gender
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Both
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Phase
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2
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Groups that have been masked
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No information
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Sample size
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Target sample size:
10
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Randomization (investigator's opinion)
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N/A
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Randomization description
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Blinding (investigator's opinion)
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Not blinded
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Blinding description
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Placebo
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Not used
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Assignment
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Single
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Other design features
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Ethics committees
1
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Ethics committee
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Approval date
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2023-01-25, 1401/11/05
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Ethics committee reference number
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IR.TUMS.HORCSCT.REC.1401.025
Health conditions studied
1
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Description of health condition studied
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Aplastic Anemia
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ICD-10 code
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D61.9
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ICD-10 code description
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Aplastic anemia, unspecified
Primary outcomes
1
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Description
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overall survival
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Timepoint
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Monthly for 12 months after transplantation
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Method of measurement
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Visiting the patient and performing monthly lab tests in outpatient clinique
Secondary outcomes
1
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Description
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Incidence of acute graft versus host disease
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Timepoint
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Monthly for 4 months after transplantation
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Method of measurement
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Visiting the patient in person and performing monthly lab tests in outpatient clinique
2
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Description
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Incidence of chronic graft versus host disease
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Timepoint
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Monthly for 12 months after transplantation
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Method of measurement
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Visiting the patient in person and performing monthly lab tests in outpatient clinique
3
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Description
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Incidence of infectious complications post-transplant
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Timepoint
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Monthly for 12 months after transplantation
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Method of measurement
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Visiting the patient in person and performing monthly lab tests in outpatient clinique
4
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Description
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Transplant Related Mortality
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Timepoint
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Monthly for 12 months after transplantation
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Method of measurement
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Visiting the patient in person and performing monthly lab tests in outpatient clinique
Intervention groups
1
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Description
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Intervention group: patients with Severe Aplastic Anemia who are candidates for allogeneic Hematopoietic Cell Transplantation and are between 18-60 years old and are unresponsive to prior immune suppressive treatments and no have no full matched (sibling or unrelated) donor. They will be transplanted according to the reduced-intensity conditioning regimen. The conditioning regimen consists of: Thymoglobulin (Genzyme) 0.5 mg/kg/D, Day -9 and Thymoglobulin 2.5 mg/kg/D, Day -8 and -7, Cyclophosphamide (Baxter) 14.5 mg/kg, Day -6 and -5, Fludarabin (ACTOVERCO) 30 mg/m2, Day -6 to -2, Busulfan (Nanoalvand) 3.2 mg/kg/D, Day -3 and -2. Cyclophosphamide (Baxter) 50 mg/kg, Day +3 and Day +4 after transplantation.
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Category
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Treatment - Other
1
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Sponsor
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Grant name
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Grant code / Reference number
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Is the source of funding the same sponsor organization/entity?
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Yes
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Title of funding source
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Tehran University of Medical Sciences
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Proportion provided by this source
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100
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Public or private sector
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Public
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Domestic or foreign origin
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Domestic
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Category of foreign source of funding
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empty
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Country of origin
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Type of organization providing the funding
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Academic
Sharing plan
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Deidentified Individual Participant Data Set (IPD)
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No - There is not a plan to make this available
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Justification/reason for indecision/not sharing IPD
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There is no further information
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Study Protocol
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No - There is not a plan to make this available
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Statistical Analysis Plan
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No - There is not a plan to make this available
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Informed Consent Form
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No - There is not a plan to make this available
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Clinical Study Report
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No - There is not a plan to make this available
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Analytic Code
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No - There is not a plan to make this available
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Data Dictionary
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No - There is not a plan to make this available