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

NCT00890500 | Completed Phase 1 DMC

• 1 other identifier: FT1050-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

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.

Conditions

Hematologic Malignancies; Allogeneic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• 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

Study Arms (2)

Group 1

ACTIVE COMPARATOR

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

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

Group 2

ACTIVE COMPARATOR

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

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

Interventions

Fludarabine DRUG

30mg/m2/day IV x 6 days

Group 1; Group 2

Melphalan DRUG

100 mg/m2/day IV x 1 day

Group 1; Group 2

Antithymocyte Globulin DRUG

1mg/kg/day x 4 days

Group 1; Group 2

Sirolimus DRUG

GVHD Prophylaxis: Target range 3-12 ng/ml

Group 1; Group 2

Tacrolimus DRUG

GVHD Prophylaxis: Target range 5-10 ng/ml

Group 1; Group 2

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• years of age
• ECOG Performance Status 0-2

You may not qualify if:

• 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

Sponsors & Collaborators

• Fate Therapeutics: lead
• Dana-Farber Cancer Institute: collaborator

Study Sites (2)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Related Publications (1)

• 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.

  PMID: 23996087 DERIVED

MeSH Terms

Conditions

Hematologic Neoplasms

Interventions

fludarabine; Melphalan; Antilymphocyte Serum; Sirolimus; Tacrolimus

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Macrolides; Lactones

Study Officials

• Corey Cutler, MD, MPH, FRCP(C)

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: FACTORIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 28, 2009
• First Posted: April 29, 2009
• Study Start: January 1, 2011
• Primary Completion: May 1, 2013
• Study Completion: October 1, 2013
• Last Updated: October 8, 2013

Record last verified: 2013-10

Locations

US (2)