Last reviewed · How we verify

NCT01251575

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

Completed Phase 2 Results posted Last updated 9 December 2019
What this trial tests

Phase 2 trial testing Allogeneic Hematopoietic Stem Cell Transplantation in Adult Acute Lymphoblastic Leukemia in 77 participants. Completed in 11 February 2019.

Timeline
1 December 2010
Primary endpoint
23 November 2018
11 February 2019

Quick facts

Lead sponsorFred Hutchinson Cancer Center
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment77
Start date1 December 2010
Primary completion23 November 2018
Estimated completion11 February 2019
Sites5 locations across Denmark, United States

Drugs / interventions tested

Conditions studied

Sponsor

Fred Hutchinson Cancer Center — full company profile →

Who can join

Eligibility, any sex, with Adult Acute Lymphoblastic Leukemia or Adult Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Patients With Grade II-IV Acute Graft Versus Host Disease (GVHD) Primary · 100 days post-transplant

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 invo

GroupValue95% CI
Class I Mismatch15
Class II Mismatch12
Number of Non-Relapse Mortalities Secondary · 100 days post-transplant

Number of patients expired without disease progression/relapse.

GroupValue95% CI
Treatment (Fludarabine, Transplant, Immunosuppression)3
Number of Patients With Grade III-IV Acute GVHD Secondary · 100 days post-transplant

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 inv

GroupValue95% CI
Treatment (Fludarabine, Transplant, Immunosuppression)2

Adverse events — posted to ClinicalTrials.gov

Time frame: 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. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treatment (Fludarabine, Transplant, Immunosuppression)
Serious: 0/76 (0%)
Deaths: 21/76
Other adverse events (34 terms — click to expand)

ReactionSystemTreatment (Fludarabine, Tr…
Acute kidney injuryRenal and urinary disorders
Febrile neutropeniaBlood and lymphatic system disorders
HypoxiaRespiratory, thoracic and mediastinal disorders
Blood bilirubin increasedInvestigations
Creatinine increasedInvestigations
DiarrheaGastrointestinal disorders
SepsisInfections and infestations
Bronchopulmonary hemorrhageRespiratory, thoracic and mediastinal disorders
Chronic kidney diseaseRenal and urinary disorders
HypotensionVascular disorders
Left ventricular systolic dysfunctionCardiac disorders
Lung infectionInfections and infestations
Thromboembolic eventVascular disorders
AscitesGastrointestinal disorders
Atrial fibrillationCardiac disorders
Atrial flutterCardiac disorders
Ejection fraction decreasedInvestigations
Encephalitis infectionInfections and infestations
Gastric hemorrhageGastrointestinal disorders
GastritisGastrointestinal disorders
Heart failureCardiac disorders
HemolysisBlood and lymphatic system disorders
Hemolytic uremic syndromeBlood and lymphatic system disorders
Mucositis oralGastrointestinal disorders
Multi-organ failureGeneral disorders
MyalgiaMusculoskeletal and connective tissue disorders
Paroxysmal atrial tachycardiaCardiac disorders
Pleural effusionRespiratory, thoracic and mediastinal disorders
Respiratory failureRespiratory, thoracic and mediastinal disorders
Small intestine infectionInfections and infestations
SyncopeNervous system disorders
Treatment related secondary malignancyNeoplasms benign, malignant and unspecified (incl cysts and polyps)
TyphlitisGastrointestinal disorders
FeverGeneral disorders

Data from ClinicalTrials.gov NCT01251575 adverse events section.

Sponsor's own description

This phase II trial studies how well sirolimus, cyclosporine and mycophenolate mofetil works in preventing graft-vs-host disease (GVHD) in patients with blood cancer undergoing donor peripheral blood stem cell (PBSC) transplant. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving total-body irradiation together with sirolimus, cyclosporine, and mycophenolate mofetil before and after transplant may stop this from happening.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Acute graft-versus-host disease: a bench-to-bedside update.
    Holtan SG, Pasquini M, Weisdorf DJ. · · 2014 · cited 152× · PMID 24914140 · DOI 10.1182/blood-2014-01-514786
  2. Sirolimus with CSP and MMF as GVHD prophylaxis for allogeneic transplantation with HLA antigen-mismatched donors.
    Kornblit B, Storer BE, Andersen NS, Maris MB, et al · · 2020 · cited 22× · PMID 32603426 · DOI 10.1182/blood.2020005338
  3. Total body irradiation dose escalation decreases risk of progression and graft rejection after hematopoietic cell transplantation for myelodysplastic syndromes or myeloproliferative neoplasms.
    Monaco F, Scott BL, Chauncey TR, Petersen FB, et al · · 2019 · cited 17× · PMID 30630975 · DOI 10.3324/haematol.2018.199398
  4. Advances in the regulatory mechanisms of mTOR in necroptosis.
    Xie Y, Zhao G, Lei X, Cui N, et al · · 2023 · cited 12× · PMID 38164133 · DOI 10.3389/fimmu.2023.1297408

Verify or expand the search:

Other trials of Allogeneic Hematopoietic Stem Cell Transplantation

Trials testing the same drug.

Other recruiting trials for Adult Acute Lymphoblastic Leukemia

Currently open trials in the same condition.

Other Fred Hutchinson Cancer Center trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01251575.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing