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← refractory Childhood Acute Lymphoblastic Leukemia

NCT01251575 · STUDY IN CONTEXT

Sirolimus, Cyclosporine, and Mycophenolate Mofetil in Preventing Graft-versus-Host Disease in Treating Patients With Blood Cancer Undergoing Donor Peripheral Blood Stem Cell Transp

Draft scope assessment: unclear. Attached because the study's own record describes refractory disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full. Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

Number of patients with grades II-IV acute GVHD aGVHD Stages Skin: 1. a maculopapular eruption involving \< 25% BSA 2. a maculopapular eruption involving 25 - 50% BSA 3. generalized erythroderma 4. generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. bilirubin 2.0 - 3.0 mg/100 mL 2. bilirubin 3 - 5.9 mg/100 mL 3. bilirubin 6 - 14.9 mg/100 mL 4. bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death

Source null

→

secondary · POSTED

Number of patients expired without disease progression/relapse.

One subject aborted transplant during conditioning and was counted towards accrual but not evaluated with respect to outcome measures.

→

secondary · POSTED

Number of patients with grades III-IV acute GVHD aGVHD Stages Skin: 1. a maculopapular eruption involving \< 25% BSA 2. a maculopapular eruption involving 25 - 50% BSA 3. generalized erythroderma 4. generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. bilirubin 2.0 - 3.0 mg/100 mL 2. bilirubin 3 - 5.9 mg/100 mL 3. bilirubin 6 - 14.9 mg/100 mL 4. bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death

One subject aborted transplant during conditioning and was counted towards accrual but not evaluated with respect to outcome measures.

→

Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

No linked quality-of-life measure is recorded for this setting and study.

Full draft applicability and source-link record
trial
release id
clinical-indications-draft-1
disease state id
ds-acute-lymphoblastic-leukemia-pediatric-refractory-0acc9454
nct id
NCT01251575
trial label
Sirolimus, Cyclosporine, and Mycophenolate Mofetil in Preventing Graft-versus-Host Disease in Treating Patients With Blood Cancer Undergoing Donor Peripheral Blood Stem Cell Transp
applicability
unclear
applicability note
Attached because the study's own record describes refractory disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
provenance class
registry-condition-search
discovery note
Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.
outcome Count
3
harms Count
1
qol Count
0
outcomes
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-acute-lymphoblastic-leukemia-pediatric-refractory-0acc9454
    nct id
    NCT01251575
    outcome key
    om-0000
    source section id
    NCT01251575-om-0000
    source outcome index
    0
    endpoint
    Number of Patients With Grade II-IV Acute Graft Versus Host Disease (GVHD)
    endpoint definition
    Number of patients with grades II-IV acute GVHD aGVHD Stages Skin: 1. a maculopapular eruption involving \< 25% BSA 2. a maculopapular eruption involving 25 - 50% BSA 3. generalized erythroderma 4. generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. bilirubin 2.0 - 3.0 mg/100 mL 2. bilirubin 3 - 5.9 mg/100 mL 3. bilirubin 6 - 14.9 mg/100 mL 4. bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death
    time origin
    100 days post-transplant
    analysis role
    primary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-acute-lymphoblastic-leukemia-pediatric-refractory-0acc9454
    nct id
    NCT01251575
    outcome key
    om-0001
    source section id
    NCT01251575-om-0001
    source outcome index
    1
    endpoint
    Number of Non-Relapse Mortalities
    endpoint definition
    Number of patients expired without disease progression/relapse.
    time origin
    100 days post-transplant
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    One subject aborted transplant during conditioning and was counted towards accrual but not evaluated with respect to outcome measures.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-acute-lymphoblastic-leukemia-pediatric-refractory-0acc9454
    nct id
    NCT01251575
    outcome key
    om-0002
    source section id
    NCT01251575-om-0002
    source outcome index
    2
    endpoint
    Number of Patients With Grade III-IV Acute GVHD
    endpoint definition
    Number of patients with grades III-IV acute GVHD aGVHD Stages Skin: 1. a maculopapular eruption involving \< 25% BSA 2. a maculopapular eruption involving 25 - 50% BSA 3. generalized erythroderma 4. generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. bilirubin 2.0 - 3.0 mg/100 mL 2. bilirubin 3 - 5.9 mg/100 mL 3. bilirubin 6 - 14.9 mg/100 mL 4. bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death
    time origin
    100 days post-transplant
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    One subject aborted transplant during conditioning and was counted towards accrual but not evaluated with respect to outcome measures.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
arms
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-acute-lymphoblastic-leukemia-pediatric-refractory-0acc9454
    nct id
    NCT01251575
    outcome key
    om-0000
    arm key
    OG000
    arm label
    Class I Mismatch
    regimen
    Patients 1) mismatched at antigen level for any single class I locus (HLA-A, -B, -C) +/- an additional class mismatch at the allele level, or 2) mismatched at the allele level for any 2 class I loci.
    analyzed count
    51
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01251575-om-0000#groups/OG000
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-acute-lymphoblastic-leukemia-pediatric-refractory-0acc9454
    nct id
    NCT01251575
    outcome key
    om-0000
    arm key
    OG001
    arm label
    Class II Mismatch
    regimen
    Patients mismatched at the antigen or allele level for class II loci HLA-DRB1 and/or DQB1. Must be matched for at least one DRB1 allele and one DQB1 allele.
    analyzed count
    25
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01251575-om-0000#groups/OG001
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-acute-lymphoblastic-leukemia-pediatric-refractory-0acc9454
    nct id
    NCT01251575
    outcome key
    om-0001
    arm key
    OG000
    arm label
    Treatment (Fludarabine, Transplant, Immunosuppression)
    regimen
    CONDITIONING: Patients receive fludarabine phosphate IV over 30 minutes on days -4 to -2. Patients also undergo total-body irradiation on day 0. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplantation. IMMUNOSUPPRESSION: Patients receive sirolimus PO QD on days -3 to 180 with taper to day 365; cyclosporine PO BID on days -3 to 150 with taper to day 180; and mycophenolate mofetil PO TID on days 0-30 and then BID to day 100 with taper to day 150. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant Cyclosporine: Given PO or IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant Sirolimus: Given PO Total-Body Irradiation: Undergo total-body irradiation
    analyzed count
    76
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01251575-om-0001#groups/OG000
  4. release id
    clinical-indications-draft-1
    disease state id
    ds-acute-lymphoblastic-leukemia-pediatric-refractory-0acc9454
    nct id
    NCT01251575
    outcome key
    om-0002
    arm key
    OG000
    arm label
    Treatment (Fludarabine, Transplant, Immunosuppression)
    regimen
    CONDITIONING: Patients receive fludarabine phosphate IV over 30 minutes on days -4 to -2. Patients also undergo total-body irradiation on day 0. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplantation. IMMUNOSUPPRESSION: Patients receive sirolimus PO QD on days -3 to 180 with taper to day 365; cyclosporine PO BID on days -3 to 150 with taper to day 180; and mycophenolate mofetil PO TID on days 0-30 and then BID to day 100 with taper to day 150. Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant Cyclosporine: Given PO or IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo allogeneic peripheral blood stem cell transplant Sirolimus: Given PO Total-Body Irradiation: Undergo total-body irradiation
    analyzed count
    76
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01251575-om-0002#groups/OG000
harms
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-acute-lymphoblastic-leukemia-pediatric-refractory-0acc9454
    nct id
    NCT01251575
    harm key
    EG000
    source section id
    NCT01251575-ae
    event group key
    EG000
    event group label
    Treatment (Fludarabine, Transplant, Immunosuppression)
    serious affected
    0
    serious at risk
    76
    other affected
    25
    other at risk
    76
    frequency threshold
    0
    collection period
    AEs: From the start of conditioning to 100 Days post-transplant SAEs: From the start of conditioning to 200 Days post-transplant All-Cause Mortality: Conditioning through 1 Year
    event definition
    Source null
    undenominated note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
quality of life
    setting source links

      Preserved source evidence · Not medical advice