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

Hematologic Malignancies Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00890500
• Other study ID #: FT1050-01
• Status: Completed
• Phase: Phase 1
• First received: April 28, 2009
• Last updated: October 7, 2013
• Start date: January 2011
• Est. completion date: October 2013

Study information

• Verified date: October 2013
• Source: Fate Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: October 2013
• Est. primary completion date: May 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Factorial Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Allogeneic Stem Cell Transplantation
• Hematologic Malignancies
• Neoplasms

Intervention

Drug:
• Fludarabine
30mg/m2/day IV x 6 days
• Melphalan
100 mg/m2/day IV x 1 day
• Antithymocyte Globulin
1mg/kg/day x 4 days
• Sirolimus
GVHD Prophylaxis: Target range 3-12 ng/ml
• Tacrolimus
GVHD Prophylaxis: Target range 5-10 ng/ml

Locations

Country	Name	City	State
United States	Dana-Farber Cancer Institute	Boston	Massachusetts
United States	Massachusetts General Hospital	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Fate Therapeutics	Dana-Farber Cancer Institute

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes
Secondary	Time to engraftment of umbilical cord blood units		2 years	No
Secondary	Fractional chimerism of transplanted cord blood units		2 years	No
Secondary	Rates of acute and chronic GVHD		2 years	Yes
Secondary	30- and 100-day treatment related mortality		2 years	Yes
Secondary	Measures of immune reconstitution and relapse-free and overall survival at 1 and 2 years after transplantation		2 years	No