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| ID | Type | Description | Link |
|---|---|---|---|
| SU-04062009-2138 | Other Identifier | Stanford University | |
| 16213 | Other Identifier | Stanford IRB | |
| BMT206 | Other Identifier | OnCore |
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Non-myeloablative approach for allogeneic transplant is a reasonable option, especially given that the median age at diagnosis is 55-60 years and frequently present compromised skin in these patients, which increases the risk of infection. Therefore, we propose a clinical study with allogeneic hematopoietic stem cell transplantation (HSCT) using a unique non-myeloablative preparative regimen, TLI/ATG, to treat advanced mycosis fungoides/Sezary syndrome (MF/SS).
Primary Objectives
-To evaluate the graft versus lymphoma effect by monitoring rate of clinical response, event-free and overall survival.
Secondary Objectives
-To evaluate the incidence and extent of acute and chronic graft-versus-host disease (GVHD) and time to engraftment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Total lymphoid irradiation & anti-thymocyte immunoglobulin | Experimental | TLI is administered from a 6 MeV linear accelerator in 80c- 120c Gy fractions. Anti-thymocyte-Globulin (ATG) is administered intravenously for a total dose of 7.5 mg/kg. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| anti-thymocyte globulin | Drug | ATG will be administered five times intravenously at 1.5 mg/kg/day from day -11 through day -7 for a total dose of 7.5 mg/kg |
|
| Measure | Description | Time Frame |
|---|---|---|
| Progression-Free Survival (PFS) at 180 Days | Progression-Free Survival (PFS; time to disease progression or death from any cause) assessed at 180 days (Kaplan-Meier estimate). Disease progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions | 180 days |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Acute Graft-versus-host Disease (GVHD) | Cumulative incidence at 6 months. GvHD was assessed using the 2015 NIH consensus criteria. | 6 months |
| Number of Participants With Chronic Graft-versus-host Disease (GVHD) |
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Inclusion Criteria:
Donor Inclusion Criteria
Exclusion Criteria:
Donor Exclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| Wen-Kai Weng | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University School of Medicine | Stanford | California | 94305 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25529383 | Background | Jagasia MH, Greinix HT, Arora M, Williams KM, Wolff D, Cowen EW, Palmer J, Weisdorf D, Treister NS, Cheng GS, Kerr H, Stratton P, Duarte RF, McDonald GB, Inamoto Y, Vigorito A, Arai S, Datiles MB, Jacobsohn D, Heller T, Kitko CL, Mitchell SA, Martin PJ, Shulman H, Wu RS, Cutler CS, Vogelsang GB, Lee SJ, Pavletic SZ, Flowers ME. National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: I. The 2014 Diagnosis and Staging Working Group report. Biol Blood Marrow Transplant. 2015 Mar;21(3):389-401.e1. doi: 10.1016/j.bbmt.2014.12.001. Epub 2014 Dec 18. | |
| 7581076 |
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| ID | Title | Description |
|---|---|---|
| FG000 | Total Lymphoid Irradiation & Anti-thymocyte Immunoglobulin | Total lymphoid irradiation (TLI) is administered from a 6 MeV linear accelerator in 80c- 120c Gy fractions. Anti-thymocyte-Globulin (ATG) is administered intravenously for a total dose of 7.5 mg/kg. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jan 7, 2019 |
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|
| cyclosporine | Drug | 5 mg/kg PO or IV |
|
|
| Lymphoid radiation | Radiation | TLI is administered ten times in 80c- 120c Gy fractions on day -11 through day -7 and day -4 through day -1 |
|
|
Cumulative incidence at 6 months (any grade). GvHD was assessed using the 2015 NIH consensus criteria.
| 2 years |
| Overall Survival (OS) | Overall survival (OS) is the time measurement between the day of allogeneic transplant and death from any cause (Kaplan-Meier estimate). | 2 years |
| Overall Survival (OS) | Overall survival (OS) is the time measurement between the day of allogeneic transplant and death from any cause (Kaplan-Meier estimate). | 5 years |
| Mortality | Total count of non-relapsed mortality and mortality from relapsed disease. | Up to 5 years |
| Treatment Related Mortality | Up to 5 years |
| Event Free Survival (EFS) | Event-free survival (EFS) is the time measurement between the day of allogeneic transplant and the first documented recurrence or death from any cause (Kaplan-Meier estimate). | 2 years |
| Event Free Survival (EFS) | Event-free survival (EFS) is the time measurement between the day of allogeneic transplant and the first documented recurrence or death from any cause (Kaplan-Meier estimate). | 5 years |
| Background |
| Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, Thomas ED. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant. 1995 Jun;15(6):825-8. |
| 32941647 | Result | Weng WK, Arai S, Rezvani A, Johnston L, Lowsky R, Miklos D, Shizuru J, Muffly L, Meyer E, Negrin RS, Wang E, Almazan T, Million L, Khodadoust M, Li S, Hoppe RT, Kim YH. Nonmyeloablative allogeneic transplantation achieves clinical and molecular remission in cutaneous T-cell lymphoma. Blood Adv. 2020 Sep 22;4(18):4474-4482. doi: 10.1182/bloodadvances.2020001627. |
| 24307695 | Derived | Weng WK, Armstrong R, Arai S, Desmarais C, Hoppe R, Kim YH. Minimal residual disease monitoring with high-throughput sequencing of T cell receptors in cutaneous T cell lymphoma. Sci Transl Med. 2013 Dec 4;5(214):214ra171. doi: 10.1126/scitranslmed.3007420. |
| Completed Assigned Treatment |
|
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Total Lymphoid Irradiation & Anti-thymocyte Immunoglobulin | TLI is administered from a 6 MeV linear accelerator in 80c- 120c Gy fractions. Anti-thymocyte-Globulin (ATG) is administered intravenously for a total dose of 7.5 mg/kg. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
| ||||||||||||||||||
| Diagnosis | Count of Participants | Participants |
| ||||||||||||||||||
| Retro-CLIPI | Risk stratification using the Retrospective Cutaneous Lymphoma International Prognostic Index (Retro-CLIPI). | Participants who completed assigned treatment | Count of Participants | Participants |
| ||||||||||||||||
| Time from diagnosis to allogeneic HCT | HCT: hematopoietic cell transplantation | Participants who completed assigned treatment | Median | Full Range | months |
| |||||||||||||||
| Active disease at the time of conditioning | Participants may have had more than one active disease | Participants who completed assigned treatment | Count of Participants | Participants |
| ||||||||||||||||
| Donor | Participants who completed assigned treatment | Count of Participants | Participants |
| |||||||||||||||||
| Donor-recipient cytomegalovirus (CMV) status | Participants who completed assigned treatment | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Progression-Free Survival (PFS) at 180 Days | Progression-Free Survival (PFS; time to disease progression or death from any cause) assessed at 180 days (Kaplan-Meier estimate). Disease progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions | Participants who completed assigned treatment | Posted | Number | 95% Confidence Interval | percentage of participants | 180 days |
|
|
| |||||||||||||||||||||||||
| Secondary | Number of Participants With Acute Graft-versus-host Disease (GVHD) | Cumulative incidence at 6 months. GvHD was assessed using the 2015 NIH consensus criteria. | Participants who completed assigned treatment | Posted | Count of Participants | Participants | 6 months |
|
| |||||||||||||||||||||||||||
| Secondary | Number of Participants With Chronic Graft-versus-host Disease (GVHD) | Cumulative incidence at 6 months (any grade). GvHD was assessed using the 2015 NIH consensus criteria. | Participants who completed assigned treatment | Posted | Count of Participants | Participants | 2 years |
|
| |||||||||||||||||||||||||||
| Secondary | Overall Survival (OS) | Overall survival (OS) is the time measurement between the day of allogeneic transplant and death from any cause (Kaplan-Meier estimate). | Participants who completed assigned treatment | Posted | Number | 95% Confidence Interval | percentage of participants | 2 years |
|
| ||||||||||||||||||||||||||
| Secondary | Overall Survival (OS) | Overall survival (OS) is the time measurement between the day of allogeneic transplant and death from any cause (Kaplan-Meier estimate). | Participants who completed assigned treatment | Posted | Number | 95% Confidence Interval | percentage of participants | 5 years |
|
| ||||||||||||||||||||||||||
| Secondary | Mortality | Total count of non-relapsed mortality and mortality from relapsed disease. | Participants who completed assigned treatment | Posted | Count of Participants | Participants | Up to 5 years |
|
| |||||||||||||||||||||||||||
| Secondary | Treatment Related Mortality | Participants who completed assigned treatment | Posted | Count of Participants | Participants | Up to 5 years |
|
| ||||||||||||||||||||||||||||
| Secondary | Event Free Survival (EFS) | Event-free survival (EFS) is the time measurement between the day of allogeneic transplant and the first documented recurrence or death from any cause (Kaplan-Meier estimate). | Participants who completed assigned treatment | Posted | Number | 95% Confidence Interval | percentage of participants | 2 years |
|
| ||||||||||||||||||||||||||
| Secondary | Event Free Survival (EFS) | Event-free survival (EFS) is the time measurement between the day of allogeneic transplant and the first documented recurrence or death from any cause (Kaplan-Meier estimate). | Participants who completed assigned treatment | Posted | Number | 95% Confidence Interval | percentage of participants | 5 years |
|
|
Up to 5 years
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Total Lymphoid Irradiation & Anti-thymocyte Immunoglobulin | TLI is administered from a 6 MeV linear accelerator in 80c- 120c Gy fractions. Anti-thymocyte-Globulin (ATG) is administered intravenously for a total dose of 7.5 mg/kg. | 21 | 38 | 7 | 38 | 20 | 38 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Secondary Malignancy | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment | Acute lymphoblastic leukemia |
| |
| Secondary Malignancy | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment | Metastatic cutaneous squamous cell carcinoma |
| |
| Acute Viral Hepatitis | Hepatobiliary disorders | Systematic Assessment | Hepatitis B infection |
| |
| Liver Failure | Hepatobiliary disorders | Systematic Assessment | Liver failure due to disease progression |
| |
| Sepsis | Infections and infestations | Systematic Assessment |
| ||
| GI Bleeding | Gastrointestinal disorders | Systematic Assessment | Acute GI GVHD |
| |
| Hyperbilirubinemia | Hepatobiliary disorders | Systematic Assessment | Acute Liver GVHD |
| |
| Multi-organ failure | General disorders | Systematic Assessment | Secondary to acute GVHD |
| |
| Acute appendicitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Hemorrhage | General disorders | Systematic Assessment | Internal bleeding from anti-coagulation |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Serum sickness | Injury, poisoning and procedural complications | Systematic Assessment | Serum sickness from ATG infusion |
| |
| Disease Relapse | General disorders | Systematic Assessment | Relapse of lymphoma |
| |
| Secondary Malignancy | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment | Localized cutaneous squamous cell carcinoma |
| |
| Anemia | Blood and lymphatic system disorders | Systematic Assessment | Anemia needing transfusion |
| |
| Neutropenia | Blood and lymphatic system disorders | Systematic Assessment | Neutrophil <0.5 |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | Systematic Assessment | Platelet count <10,000 |
| |
| Neutropenic fever | Infections and infestations | Systematic Assessment |
| ||
| Infection, non-specified | Infections and infestations | Systematic Assessment | Central-line related infection |
| |
| Vaginal bleeding | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Post transplant lymphoproliferative disorder (PTLD) | Infections and infestations | Systematic Assessment | Post-transplant lymphoproliferative disorder (PTLD) secondary to Epstein Barr virus (EBV) reactivation |
| |
| CMV viremia | Infections and infestations | Systematic Assessment |
| ||
| EBV viremia | Infections and infestations | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Wen-Kai Weng, Associate Professor of Medicine | Stanford University Medical Center | 650-723-7689 | wkweng@stanford.edu |
| Mar 27, 2023 |
| Prot_SAP_001.pdf |
| ICF | No | No | Yes | Informed Consent Form | Mar 13, 2019 | Feb 18, 2020 | ICF_000.pdf |
| ID | Term |
|---|---|
| D009181 | Mycoses |
| D012751 | Sezary Syndrome |
| D016410 | Lymphoma, T-Cell, Cutaneous |
| D008228 | Lymphoma, Non-Hodgkin |
| ID | Term |
|---|---|
| D001423 | Bacterial Infections and Mycoses |
| D007239 | Infections |
| D016399 | Lymphoma, T-Cell |
| D008223 | Lymphoma |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D000961 | Antilymphocyte Serum |
| D016572 | Cyclosporine |
| D015182 | Lymphatic Irradiation |
| ID | Term |
|---|---|
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D003524 | Cyclosporins |
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
| D011878 | Radiotherapy |
| D013812 | Therapeutics |
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|
| Unknown or Not Reported |
|
|
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| Asian |
|
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| Unknown/not reported |
|
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| Intermediate risk |
|
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| Low risk |
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| Blood |
|
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| Lymph node |
|
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| Visceral (site: bone marrow) |
|
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| Visceral (site: tonsil) |
|
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| Unrelated - matched |
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| Unrelated - unmatched |
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| Donor and recipient seronegative |
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|
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Non-relaped, treatment related |
| |||||
| Non-relaped, not treatment related |
| |||||
| Disease relapse |
|
| Title |
|---|
| Denominators |
|---|
| Categories |
|---|
| Acute GVHD |
| |||||
| Chronic GVHD |
| |||||
| Secondary malignancy |
| |||||
| Hepatitis B |
| |||||
| Hemorrhage secondary to anticoagulation |
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