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

Phase 1

Completed

• Conditions: Hematologic Malignancies; Allogeneic Stem Cell Transplantation
• Interventions: Drug: Fludarabine; Drug: Melphalan; Drug: Antithymocyte Globulin; Drug: Sirolimus; Drug: Tacrolimus

• Registration Number: NCT00890500
• Lead Sponsor: Fate Therapeutics

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.

Study Timeline

• Study First Submitted: 2009-04-28
• Study First Submitted QC: 2009-04-28
• Study First Posted: 2009-04-29
• Study Start: 2011-01
• Primary Completion: 2013-05
• Status Verified: 2013-10
• Study Completion: 2013-10
• Last Update Submitted: 2013-10-07
• Last Update Posted: 2013-10-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: FACTORIAL

Arm && Interventions

Group	Intervention	Description
Group 1	Antithymocyte Globulin	2 umbilical cord units: Second cord blood unit modulated with ProHema
Group 2	Antithymocyte Globulin	2 umbilical cord units: First cord blood unit modulated with ProHema
Group 1	Fludarabine	2 umbilical cord units: Second cord blood unit modulated with ProHema
Group 1	Melphalan	2 umbilical cord units: Second cord blood unit modulated with ProHema
Group 1	Sirolimus	2 umbilical cord units: Second cord blood unit modulated with ProHema
Group 1	Tacrolimus	2 umbilical cord units: Second cord blood unit modulated with ProHema
Group 2	Fludarabine	2 umbilical cord units: First cord blood unit modulated with ProHema
Group 2	Melphalan	2 umbilical cord units: First cord blood unit modulated with ProHema
Group 2	Tacrolimus	2 umbilical cord units: First cord blood unit modulated with ProHema
Group 2	Sirolimus	2 umbilical cord units: First cord blood unit modulated with ProHema

Primary Outcome Measures

Name	Time	Method
Determine the safety of ProHema modulated umbilical cord blood units when used for transplantation in a reduced intensity, sequential umbilical cord blood transplantation model.	2 years

Secondary Outcome Measures

Name	Time	Method
Time to engraftment of umbilical cord blood units	2 years
Fractional chimerism of transplanted cord blood units	2 years
Rates of acute and chronic GVHD	2 years
30- and 100-day treatment related mortality	2 years
Measures of immune reconstitution and relapse-free and overall survival at 1 and 2 years after transplantation	2 years

Trial Locations

Locations (2)

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Dana-Farber Cancer Institute; 🇺🇸 Boston, Massachusetts, United States