Clinical trial NCT00299767: Phase I Study of Sequential Cord Blood Transplants
Official title: Phase I Study of Sequential Cord Blood Transplants
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Lymphoma
Leukemia
Multiple Myeloma
Myelodysplastic Syndrome
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Dana-Farber Cancer Institute
First posted: 2006-03-07
Enrollment: 21
Sex: ALL
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the safety of sequential cord blood transplantation (2 cord blood units) for patients who have diseases that are capable of being cured by allogeneic stem cell transplant but do not have a matched family or volunteer unrelated donor.

Detailed description: Eligible patients will receive conditioning therapy with fludarabine 30 mg/m2/day x 6 days, melphalan 100 mg/m2/day x 1 day, rabbit antithymocyte globulin 1.5 mg/kg/day x 4 days. GVHD prophylaxis will consist of cyclosporine starting on day -1 and mycophenolate mofetil starting on day 0. Sequential cord blood units will be infused on Day 0.

Eligibility criteria: Inclusion Criteria:

* Disease Status: NHL, HD, or MM refractory to chemotherapy or relapsed; CLL Rai Stage III/IV, or lymphocyte doubling time of 6 months, or Stage I/II resistant to 2 chemotherapy regimens; AML or ALL in second or subsequent remission or in first remission with adverse cytogenetics or antecedent hematologic disorder
* Estimated disease-free survival of less than one year
* ECOG performance status of 0, 1, 2
* Lack of 6/6 or 5/6 HLA matched related or 6/6 matched unrelated donor, or a donor is not available within the time frame necessary to perform a potentially curative stem cell transplant
* 4/6 or greater HLA A, B, CRB1 cord blood units with a combined nucleated cell dose from of \> 3.7 x 10(7) NC/kg

Exclusion Criteria:

* Cardiac disease: symptomatic congestive hearth failure or RVG or echocardiogram determined left ventricular ejection fraction of \< 45%, active angina pectoris, or uncontrolled hypertension
* Pulmonary Disease: severe chronic obstructive lung disease, or symptomatic restrictive lung disease, or corrected DLCO of \< 50%
* Renal disease: serum creatinine \> 2.0 mg/dl
* Hepatic disease: serum bilirubin \> 2.0 mg/dl (except in the case of Gilbert's syndrome), SGPT or SGOT \> 3 x normal
* Neurologic disease: symptomatic leukoencephalopathy, active DNS malignancy or other neuropsychiatric abnormalities believed to preclude transplantation
* HIV antibody or Hepatitis B surface antigen positivity
* Uncontrolled infection
* Pregnancy or breast-feeding mother

Primary outcomes:
measure: To evaluate the 100-day transplant-related (non-relapse) mortality, including relapse-related mortality associated with Grade 4 toxicity.
timeFrame: 3 years
Secondary outcomes:
measure: To evaluate the 6 month transplant related (non-relapse) mortality.
timeFrame: 3 years
measure: To evaluate the days to neutrophil engraftment (ANC > 500).
timeFrame: 3 years
measure: To evaluate the days of platelet engraftment (platelet count > 20K unsupported).
timeFrame: 3 years
measure: To evaluate the risk of acute and chronic graft versus host disease.
timeFrame: 3 years
measure: To evaluate percent donor chimerism - contribution of each cord unit.
timeFrame: 3 years
measure: To evaluate relapse rate.
measure: To evaluate overall survival.
measure: To evaluate transfusion support needed to cord blood transplant recipients.
timeFrame: 3 years
Other outcomes:
Locations:
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital, Harvard University
name: Karen Ballen, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Dana Farber Cancer Institute, Harvard Univeristy
name: Joseph Antin, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Beth Israel Deaconess Medical Center
name: David Avigan, M.D.
role: PRINCIPAL_INVESTIGATOR
References: