Clinical trial NCT01220297: Sirolimus & Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative, Matched Related Donor HCT
Official title: Sirolimus and Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative, Matched Related Donor Hematopoietic Cell Transplantation
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Hematologic Diseases
Acute-graft-versus-host Disease
Leukemia
Non-Hodgkin Lymphoma (NHL)
Hodgkin Lymphoma
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2010-10-13
Enrollment: 3
Sex: ALL
Minimum age (years): 2
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A continuation study of sirolimus and mycophenolate mofetil (MMF) for graft-vs-host disease (GvHD) prophylaxis for patients undergoing matched related allogeneic hematopoietic stem cell transplantation (HSCT) for acute and chronic leukemia, myelodysplastic syndrome (MDS), high risk non-Hodgkin lymphoma (NHL), or Hodgkin lymphoma (HL)

Detailed description: To explore the novel combination of sirolimus and mycophenolate mofetil (MMF) as graft-vs-host disease (GvHD) prevention in human leukocyte antigen (HLA)-matched related donor peripheral blood stem cell (PBSC) or marrow transplantation (BMT), collectively hematopoietic stem cell transplantation (HSCT). This study will report the toxicities associated with this drug combination.

For all treatments and procedures, Study Day is based on the day of HSCT as Day 0.

Eligibility criteria: INCLUSION CRITERIA

* Acute myelogenous leukemia (AML), beyond 2nd remission or relapsed/refractory disease, age 2 to 60 years
* AML, in first or subsequent remission or relapsed/refractory disease, age 51 to 60 years of age
* AML with multilineage dysplasia
* Acute lymphoblastic leukemia (ALL), beyond 2nd remission or relapsed/refractory disease, age 2 to 60 years
* ALL, age 51 - 60 years in first or subsequent remission or relapsed/refractory disease
* Chronic myeloid leukemia (CML), beyond 2nd chronic phase or in blast crisis
* Myelodysplastic syndrome (MDS), including World Health Organization (WHO)classifications of refractory anemia with excess blasts-1 (RAEB-1), RAEB-2 and therapy-related MDS
* MDS with poor long-term survival including myeloid metaplasia and myelofibrosis
* Myeloproliferative disorders
* High-risk non-Hodgkin lymphoma (NHL) in 1st emission
* Relapsed or refractory NHL
* Hodgkin lymphoma (HL) beyond first remission
* Males and females of any ethnic background, 2 to 60 years of age
* Karnofsky Performance Status (KPS) ≥ 70% or Lansky performance status \> 70% for patients \< 16 years of age.
* Related, matched-donor identified \[6/6 human leukocyte antigen (HLA)-A, B and DRB1\]
* Willingness to take oral medications during the transplantation period
* Ability to understand and the willingness to sign a written informed consent document

EXCLUSION CRITERIA

* Prior myeloablative allogeneic or autologous hematopoietic stem cell transplant (HSCT)
* HIV infection
* Pregnant
* Lactating
* Evidence of uncontrolled active infection
* Serum creatinine \> 1.5 mg/dL or 24-hour creatinine clearance \< 50 mL/min
* Direct bilirubin, Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> 2 x upper limit of normal (ULN)
* Carbon monoxide diffusing capacity (DlCO) \< 60% predicted (adults) OR and in-room air oxygen saturation \< 92% (children)
* Left ventricular ejection fraction \< 45% (adults) OR shortening fraction \< 26%(children)
* Fasting cholesterol \> 300 mg/dL or Triglycerides \> 300 mg/dL while on lipid-lowering agents.
* Receiving investigational drugs unless cleared by the Principal Investigator (PI).
* Prior malignancies except basal cell carcinoma or treated carcinoma in-situ.
* Cancer treated with curative intent ≤ 5 years (EXCEPTION BY PI DISCRETION) (Cancer treated with curative intent \> 5 years will be allowed).

Primary outcomes:
measure: Acute Graft-vs-Host Disease (GvHD) (Grade 2 to 4)
timeFrame: 100 days post-transplant
Secondary outcomes:
measure: Acute GvHD (Grade 3 to 4)
timeFrame: 100 days post-transplant
measure: Disease-free Survival (DFS)
timeFrame: 2 years
measure: Overall Survival
timeFrame: 2 years
measure: Veno-occlusive Disease (VoD)
timeFrame: 100 days post-transplant
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University School of Medicine
state: California
Study officials:
affiliation: Stanford University
name: Laura Johnston
role: PRINCIPAL_INVESTIGATOR
References: