Umbilical Cord Blood Transplantation From Unrelated Donors

• Register: ClinicalTrials.gov
• Last refreshed on: 2 August 2023
• Main ID: NCT03016806
• Date of registration: 26/12/2016
• Prospective Registration: No
• Primary sponsor: University of Rochester
• Public title: Umbilical Cord Blood Transplantation From Unrelated Donors
• Scientific title: Umbilical Cord Blood Transplantation From Unrelated Donors
• Date of first enrolment: June 2015
• Target sample size: 30
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03016806
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Jane L Liesveld, MD

• Name: Jane L Liesveld, MD
• Affiliation: Medical Director, Blood & Marrow Transplant Unit

• Name: Jane L Liesveld, MD

Key inclusion & exclusion criteria

Inclusion Criteria:

- Appropriate diagnosis: Patients must have a disease or syndrome amenable to therapy
with hematopoietic stem cell transplantation. Diagnoses include, but are not limited
to:

- Congenital and Other Non-malignant Disorders:

- Immunodeficiency disorders (e.g. Severe Combined Immunodeficiency, Wiskott-Aldrich
Syndrome)

- Congenital hematopoietic stem cell defects (e.g. Chediak-Higashi Syndrome, Congenital
Osteopetrosis, Osteogenesis Imperfecta)

- Metabolic disorders (e.g. Hurler's Syndrome)

- Severe aplastic anemia

- High-Risk Leukemia:

- Acute Myelogenous Leukemia

- Refractory to standard induction therapy (more than 1 cycle required to achieve
remission)

- Recurrent (in CR = 2)

- Treatment-related AML or MDS

- Evolved from myelodysplastic syndrome

- Presence of FLT3 abnormalities

- FAB M6 or M7

- Adverse cytogenetics

- Myelodysplastic Syndrome

- Acute Lymphoblastic Leukemia including T lymphoblastic leukemia:

- Refractory to standard induction therapy (time to CR >4 weeks)

- Recurrent (in CR = 2)

- WBC count >30,000/mcL at diagnosis

- Age >30 at diagnosis

- Adverse cytogenetics, such as t(9:22), t(1:19), t(4:11), and other MLL rearrangements.

- Chronic Myelogenous Leukemia in accelerated phase or blast crisis

- Biphenotypic or undifferentiated leukemia

- Burkitt's leukemia or lymphoma

- Lymphoma:

- Large cell, Mantle cell, Hodgkin lymphoma refractory or recurrent, chemo-sensitive,
and ineligible for an autologous stem cell transplant or previously treated with
autologous SCT

- Marginal zone or follicular lymphoma that is progressive after at least two prior
therapies

- Multiple Myeloma, recurrent following high-dose therapy and autologous SCT or
ineligible for an autologous HSCT

- Solid tumors, with efficacy of allogeneic HSCT demonstrated for the specific disease
and disease status

- Adequate organ function:

- Cardiac - LVEF >45%, or shortening fraction >25%, Absence of congestive heart failure
or conduction disturbances with high risk for sudden death

- Pulmonary - DLCO (corrected for hemoglobin), FEV1 and FVC = 50% predicted;

- Renal - serum Cr < 1.5 times the upper limit of normal for age or GFR = 50
ml/min/1.73m2

- Hepatic - total bilirubin level < 2 times the upper limit of normal (except for
patients with Gilbert's syndrome or hemolysis); if the primary disease process is
causal, this criterion will be reconsidered. ALT, AST, and Alkaline phosphatase = 5
times upper limit of normal.

- Performance Status Karnofsky or Lansky score = 70%.

- Informed Consent must be obtained prior to initiating conditioning therapy.

- Receipt of viable cord blood product(s), single or dual, must be confirmed with the
stem cell processing laboratory prior to initiating conditioning therapy.

Exclusion Criteria:

- Availability of 10/10 or 9/10 HLA-matched related or unrelated donor within a
reasonable timeframe dictated by the clinical urgency of the transplant

- Autologous HSCT < 6 months prior to proposed UCB transplant

- Pregnant or breast feeding

- Current uncontrolled infection

- Evidence of HIV infection or positive HIV serology



Age minimum: 2 Months
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Congenital Hematological Disorder

Acute Leukemia

Chronic Leukemia

Lymphoma

Metabolism Disorder

Myelodysplastic Syndromes

Immune Deficiency Disorder

Solid Tumor

Multiple Myeloma

Aplastic Anemia

Intervention(s)

Drug: Melphalan

Radiation: Total Body Irradiation 1200 cGy

Radiation: Total Body Irradiation 200 cGy

Drug: Fludarabine

Drug: Busulfan

Procedure: Cord Blood Infusion

Drug: Cyclophosphamide

Drug: Mesna

Primary Outcome(s)

Engraftment of ANC and Platelets [Time Frame: 42 days following the infusion of stem cells for ANC [If engraftment of ANC does not occur within 42 days, a subsequent transplant will be performed if a donor is available.]]

Secondary Outcome(s)

Incidence of acute graft-versus-host disease [Time Frame: At 30 days and 100 days after transplant from the date of transplant until the date of documented acute GvHD.]

Disease-free survival [Time Frame: At 30 days, 100 days, 6 months and yearly after transplant from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.]

Rate of non-engraftment and of secondary graft failure [Time Frame: At 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.]

Incidence of chronic graft-versus-host disease [Time Frame: At 100 days, 6 months and yearly after transplant from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.]

Secondary ID(s)

UBMT 15029

Source(s) of Monetary Support

Please refer to primary and secondary sponsors