Umbilical Cord Transplantation for the Elderly Population

NCT01484470 | Completed Phase 2 DMC

• 1 other identifier: 202041
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

While cord blood transplants have been performed safely in elderly patients, many still relapse. The investigators propose to intensify the preparative regimen for this patient group in an attempt to decrease relapses, and combine this with an ex vivo expanded Umbilical Cord Blood (UCB) unit.

Conditions

Hematologic Malignancies

Keywords

Stem cell transplantation; Stem Cell expansion; Elderly

Outcome Measures

Primary Outcomes (1)

• Efficacy of StemEx

  The primary endpoint is to demonstrate the efficacy of StemEx® vs. unmanipulated UCB transplantation in the elderly population (\>55years of age) following RIC regimen by demonstrating engraftment with full donor chimerism (\>98%) by Day 100 of more than 60% of the patients who received transplants expanded by the StemEx method.

  100 days

Secondary Outcomes (1)

• Time to engraftment

  42 days

Study Arms (2)

Unmanipulated arm

NO INTERVENTION

Participants that do not meet criteria for StemEx®, will be registered into the unmanipulated UCB arm and receive the standard conditioning regimen.

Stemx Arm

EXPERIMENTAL

StemEx is a stem/progenitor cell-based product of ex-vivo expanded allogeneic UCB, which is administered to the subject in combination with the non-manipulated portion of the same cord blood unit (CBU). The CBU must be cryopreserved in two portions of which the larger (or equal) CBU portion contains at least 1.5 x 107 total nucleated cells (TNC)/Kg. This portion remains unmanipulated and is transplanted on Day 0. StemEx is derived from the smaller (or equal) CBU portion, which is expanded ex vivo for 21 days starting pre-transplant in the presence of cytokines TPO, IL-6, Flt-3L and SCF at a concentration of 50ng/ml and 5μM tetraethylenepentamine (TEPA)

Biological: StemEx

Interventions

StemEx BIOLOGICAL

For patients allocated to StemEx® arm: * Day -20: Start small (or equal) portion expansion at processing site. (II) Conditioning Phase * Day -6 to -1: Subject receives a RIC regimen containing Fludarabine, Cyclophosphamide and Total Body Irradiation (TBI) (III) Transplantation and Follow-up Phase * Day 0: CBU unmanipulated portion transplantation (for StemEx® arm) or unmanipulated CBU transplantation. * Day 1: StemEx® transplantation. * Day 2 to 3 years: Post transplant follow-up.

Stemx Arm

Eligibility Criteria

• Age: 55 Years - 73 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Ages 55-73
• Patients will have one of the following malignancies:
• Acute myelogenous leukemia (AML) deNovo in first CR with adverse cytogenetic abnormalities, M0, M6, M7 subtypes, extramedullary disease in remission or high CD34+ disease (\> 50%)
• AML in early relapse (5-10% blasts on bone marrow aspirate or biopsy), or beyond CR-1 with no circulating blasts
• AML at any time if resulting from a previous myelodysplasia
• Acute lymphocytic leukemia or lymphoblastic lymphoma (ALL) in first CR with adverse prognostic features: t (9; 22), extra medullary disease, or mature B cell phenotype
• Acute lymphoid leukemia or lymphoblastic lymphoma in early relapse (5- 10% blasts on aspirate), or beyond CR-1
• Acute Undifferentiated Leukemia or biphenotypic leukemia in CR1 or CR2
• Transfusion dependent myelodysplastic syndrome (MDS) or refractory anemia with excess blasts (RAEB) or RAEB-in transition, CMMOL, or any myelodysplasia with 7q-, 5q-, 7-, 5- or resulting from prior anti cancer therapy.
• Relapsed Non-Hodgkin's Lymphoma (NHL), including those that have relapsed after an autologous marrow/blood stem cell transplant
• Chronic lymphocytic leukemia (CLL) patient who has had fludarabine and either failed or relapsed. Prior autologous transplant patients are eligible.
• Patients with adequate organ function and performance status criteria
• Subject must have at least one or the following back-up stem cell sources in case of engraftment failure:
• Subject is willing to undergo BM harvest or peripheral blood progenitor cells (PBPC) collection for use in case of engraftment failure (when clinically applicable).
• Subject has a second CBU as a possible back up.

You may not qualify if:

• Patient with suitable related donor as defined per institutional guidelines
• Chemotherapy resistant or active AML, ALL, AUL, biphenotypic leukemia
• AML evolved from myelofibrosis
• MDS with 20% or greater bone marrow blasts at pre-transplant workup. Patients may receive therapy and if in remission, are eligible
• Prior allogeneic hematopoeitic stem cell transplant at any time
• Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except for hydroxyurea)
• Uncontrolled bacterial, fungal or viral infection at the time of study enrollment
• Seropositive or NAT positive for HIV, HTLV-1 and Hepatitis C
• Subjects with signs and symptoms of active central nervous system (CNS) disease
• Females who are pregnant or breastfeeding
• Allergy to bovine proteins or to aminoglycoside antibiotics (e.g. gentamicin) or to any product, which may interfere with the treatment.
• Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up and research tests.
• Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by the primary investigator.

Sponsors & Collaborators

• Loyola University: lead
• Gamida Cell ltd: collaborator

Study Sites (1)

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms

Condition Hierarchy (Ancestors)

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

Study Officials

• Patrick Stiff, MD

  Loyola Universtiy Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: November 30, 2011
• First Posted: December 2, 2011
• Study Start: January 1, 2010
• Primary Completion: November 21, 2018
• Study Completion: November 21, 2018
• Last Updated: August 21, 2019

Record last verified: 2019-08

Locations

US (1)