Clinical trial NCT00890500: Safety and Efficacy of ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematologic Malignancies.
Official title: A Phase I Study of Reduced Intensity, Sequential Double Umbilical Cord Blood Transplantation Using ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematological Malignancies.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hematologic Malignancies
Allogeneic Stem Cell Transplantation
Lead sponsor: Fate Therapeutics
Sponsor class: INDUSTRY
Collaborators: name: Dana-Farber Cancer Institute
First posted: 2009-04-29
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: FACTORIAL
Primary purpose: TREATMENT

Brief summary: The purpose of this research study is to determine the safety and efficacy of a reduced intensity conditioning regimen during a double umbilical cord blood unit transplant with one of the cord blood units modulated with ProHema.

Detailed description: The purpose of this research study is to determine the safety and efficacy of a reduced intensity conditioning regimen during a double umbilical cord blood unit transplant with one of the cord blood units modulated with ProHema.

-As part of this research study cord units will be modulated in the laboratory with ProHema before it is given to the participant. Two different treatment groups will be tested. Group 1: will have the second cord blood unit modulated with ProHema; Group 2: will have the first cord blood unit modulated with ProHema.

Eligibility criteria: Inclusion Criteria:

* Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate
* Patient must be ineligible for traditional myeloablative transplantation according to treating physician
* Lack of 6/6 or 5/6 HLA-matched related, 8/8 HLA-matched unrelated donor, or unrelated donor not available within a time frame necessary to perform a potentially curative stem cell transplant
* 18-65 years of age
* ECOG Performance Status 0-2

Exclusion Criteria:

* The following hematologic malignancies are excluded:

  * Myelofibrosis unless there has been exposure to cytotoxic chemotherapy for the treatment of progression to acute myeloid leukemia
  * Chronic Myelogenous Leukemia, unless there has been exposure to cytotoxic chemotherapy for the treatment of blast phase, 3) Aplastic anemia, in the absence of transformation to Myelodysplastic disorder
* Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular dysfunction as measured by gated radionucleotide ventriculogram or echocardiogram; active angina pectoris, or uncontrolled hypertension
* Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of \< 50% of predicted, corrected hemoglobin
* Renal disease: serum creatinine \> 2.0mg/dl
* Hepatic disease: serum bilirubin \> 2.0mg/dl (expect in the case of Gilbert's syndrome or ongoing hemolytic anemia), SGOT or SGPT \> 3 x upper limit of normal
* Neurologic disease: symptomatic leukoencephalopathy, active CNS malignancy or other neuropsychiatric abnormalities believed to preclude transplantation
* HIV antibody
* Uncontrolled infection
* Pregnancy or breast feeding mother
* Inability to comply with the requirements for care after allogeneic stem cell transplantation

Primary outcomes:
measure: Determine the safety of ProHema modulated umbilical cord blood units when used for transplantation in a reduced intensity, sequential umbilical cord blood transplantation model.
timeFrame: 2 years
Secondary outcomes:
measure: Time to engraftment of umbilical cord blood units
timeFrame: 2 years
measure: Fractional chimerism of transplanted cord blood units
timeFrame: 2 years
measure: Rates of acute and chronic GVHD
timeFrame: 2 years
measure: 30- and 100-day treatment related mortality
timeFrame: 2 years
measure: Measures of immune reconstitution and relapse-free and overall survival at 1 and 2 years after transplantation
timeFrame: 2 years
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
Study officials:
affiliation: Dana-Farber Cancer Institute
name: Corey Cutler, MD, MPH, FRCP(C)
role: PRINCIPAL_INVESTIGATOR
References:
citation: Cutler C, Multani P, Robbins D, Kim HT, Le T, Hoggatt J, Pelus LM, Desponts C, Chen YB, Rezner B, Armand P, Koreth J, Glotzbecker B, Ho VT, Alyea E, Isom M, Kao G, Armant M, Silberstein L, Hu P, Soiffer RJ, Scadden DT, Ritz J, Goessling W, North TE, Mendlein J, Ballen K, Zon LI, Antin JH, Shoemaker DD. Prostaglandin-modulated umbilical cord blood hematopoietic stem cell transplantation. Blood. 2013 Oct 24;122(17):3074-81. doi: 10.1182/blood-2013-05-503177. Epub 2013 Aug 30.