Safety and Efficacy of ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematologic Malignancies.

Fate Therapeutics

Status and phase: Completed; Phase 1

Conditions: Allogeneic Stem Cell Transplantation; Hematologic Malignancies

Treatments: Drug: Tacrolimus; Drug: Melphalan; Drug: Antithymocyte Globulin; Drug: Fludarabine; Drug: Sirolimus

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00890500

FT1050-01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 12 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Factorial Assignment

Masking: None (Open label)

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12 participants in 2 patient groups

Group 1

Active Comparator group

Description:

2 umbilical cord units: Second cord blood unit modulated with ProHema

Treatment:

Drug: Tacrolimus

Drug: Fludarabine

Drug: Sirolimus

Drug: Melphalan

Drug: Antithymocyte Globulin

Group 2

Active Comparator group

Description:

2 umbilical cord units: First cord blood unit modulated with ProHema

Treatment:

Drug: Tacrolimus

Drug: Fludarabine

Drug: Sirolimus

Drug: Melphalan

Drug: Antithymocyte Globulin